Genetherapy

Violence against women a. Brunetti explains the role of science in understanding the female world – Hardwood Paroxysm

November 21st, 2021

Publication date: Wednesday, November 17, 2021 news release

ROME (Current Affairs) Interview with Professor Guido Brunetti before November 25, the International Day for the Elimination of Violence against Women

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november 25 isInternational Day for the Elimination of Violence against WomenFounded by the United Nations in 1999. The issue is difficult, complex and sensitive. Mr Guido Brunetti Try to guide us in understanding female minds and behaviors.

To understand the dangerous phenomenon of femicide and violence against women, it is necessary to enter the still mysterious planet of the female world, whose analysis presents the specificity and variety of complex neurobiological, mental and emotional characteristics.

From the search emerges a multifaceted personality, always in perpetual change, sometimes an indefinite picture, intersecting with chiaroscuro influences, and difficult to predict. In fact, very little is said about the woman and little or nothing is known. After the remarkable studies of Freud and other authors, it is todays neuroscience that seeks to shed light on the mysterious aspects of women.

A cultural and scientific gap?

Certainly, ancient theories without any scientific value, stereotypes and prejudices have always characterized the female image, which since ancient times was considered inferior to men on a psychological, mental and social level. It is subjected to violence, humiliation, possession, control, evasion and hatred. Unfortunately, the expression is still About an ancient culture pervasive.

For women, the internal tragedy of the millennium.

Until the 20th century, most scholars believed that women were nothing more than smaller men in neurobiological terms and in any other sense. Therefore, little attention was paid to the personality of women and their physiology.

violence and humiliation

The phenomenon of violence against women and femicide has increased in recent times. There are many women murder victims, one every three days. The great health, economic, social and moral crisis brought about by the pandemic as Brunetti explains has had an alluvial effect on the emotional, mental and social structure of the individual, with serious repercussions in society. A fact that further emphasizes the urgency and importance of the issue we are studying.

violence? It is transgressive, transgressive, transitive. It is strength. A physical or verbal act of one individual towards another that causes biological, psychological and sexual harm. Violence has a neurobiological significance found in the oldest structure of the human brain. It is a destructive and self-destructive innate impulse, influenced by experiences and the social and cultural environment.

For us, the concept of violence transcends these dimensions. As we have already discussed on other sites with Professor Giovanni Puglia, the father of child neuropsychiatry in Italy, a process rape At the planetary level, its understanding is connected not only with the economic crisis, but above all with the social, moral and spiritual crisis.

We are witnessing an unstoppable decline in the values and principles that have underpinned civilization for thousands of years. A fluid world arises, insecure, anxious, opaque, elusive, devoid of certain points of reference.

What about violence against women?

Violence against women is considered a global scourge, a health problem of great proportions (United Nations). There is an emergency with serious delays in both European and Italian politics, lack of modern visions, unprepared personnel, social and political underestimation of the phenomenon and a lot more. From rhetoric and a lot of hypocrisy.

What is the contribution of feminism and the sexual revolution?

Feminist scholars, as well as many feminist experts, have announced the declining role of women, who are increasingly similar to men, in the competition for power. Over time, the feminist movement and the sexual revolution failed, due to the presence of aspects of utopia and discontent and the unresolved process of emancipation of women. Inverted masculine chauvinism emerged. The woman became a guinea pig, a bargaining chip, turned into a womb, a substance, an object of pleasure. His freedom is a false freedom. The fact is that a woman who today occupies a prominent position both professionally and intellectually angers if she is identified as a feminist.

You, Professor Brunetti, have mentioned scientific advances in womens knowledge.

We are entering an era where we are beginning to discover great female abilities. Indeed, in recent years, neuroscience has shown exciting new discoveries about a womans personality in terms of brain structure, mental processes, sexual impulses, and behaviours.

Women are no longer inferior to men, as women enjoy, among other things, genetic and immune system superiority along with sympathetic superiority.

The research then discovered that a womans neurological aspects, such as the menstrual cycle, pregnancy, childbirth, breastfeeding, and childcare, are relevant components of her cognitive, social, and emotional development.

Are you saying that women have special gifts?

They have a unique brain structure.

The first differences in the brain appear as early as the eighth week of fetal development. Other research has indicated that less than 24 hours after birth, a newborn responds to another newborns cry. From a year ago, the girls gained a sense of empathy, became more sensitive to the suffering of others.

What are the other essential features?

And there are a lot of them. Women acquire unique and unusual qualities, such as verbal agility, the ability to decipher feelings and moods, form deep social bonds, the ability to smooth out conflicts, and nurture the people around them. By the age of two, they have a vocabulary three times richer than Those of the boys. In fact, they started talking earlier. They are adept at reading facial expressions and emotional nuances.

It has also been discovered that hormones have tremendous neurological effects and adapt the female brain to the point where they perceive reality and their way of life in a different way. The hormonal influence is important at all stages of female development. Motherhood, for example, causes massive hormonal changes, transforming the female brain, altering her structure and functioning.

sexuality

There are many theories, often conflicting and contradictory to each other. It was Freud who discovered child sexuality, emphasizing its centrality also to the genesis of every form of sexual perversion and neurosis.

Womens sexual circuits are associated with oxytocin, a substance that promotes, among other things, healing and emergence behaviors. Maternal influences contribute to the development of sexual differences.

Neuroscientists have proven that they are essential difference Of feminine nature compared to masculine. It is the gender identity of the brain that determines whether people do it or not they feel female or male. The female brain and the male brain have Different Sex systems also relate to sexual drives and love, a feeling that constitutes one of the most irrational states of the brain that can be assumed.

What conclusions should be drawn?

So there is scientific evidence for the differences between female and male brains. The brains of men and women look different at birth. Our brains are different and so are our minds.

Today, remarkable progress has been made in the fields of genetics, neuroscience, biology, neurohormonal development and thanks to remarkable technologies Mind Fantasies They define the famous author identifies a scientific revolution aimed not only at disrupting the methods of diagnosis and treatment in medicine and psychiatry, but our worldview and our millenarian concepts, starting with philosophical systems.

Therefore, there is a need concludes Professor Brunetti to use and enhance all the talents of the womans brain in order to promote the preservation of the species and the progress of mankind.

Anna Gabriel

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Violence against women a. Brunetti explains the role of science in understanding the female world - Hardwood Paroxysm

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SC21- 21st century cellular medicines specialists – The Thaiger

November 21st, 2021

Male hypogonadism | You and Your Hormones from the Society …

November 21st, 2021

Alternative names for male hypogonadism

Testosterone deficiency syndrome; testosterone deficiency; primary hypogonadism; secondary hypogonadism; hypergonadotrophic hypogonadism; hypogonadotrophic hypogonadism

Male hypogonadism describes a state of low levels of the male hormone testosterone in men. Testosterone is produced in the testes and is important for the formation of male characteristics such as deepening of the voice, development of facial and pubic hair, and growth of the penis and testes during puberty. Gonadotrophin-releasing hormone, made in the hypothalamus, stimulates the pituitary gland to produce luteinising hormone and follicle stimulating hormone (gonadotrophins). The gonadotrophins then act on the testes causing them to produce testosterone.Low levels of testosterone can occur due to disease of the testes or from conditions affecting the hypothalamus or pituitary gland. Men can be affected at any age and present with different symptoms depending on the timing of the disease in relation to the start of puberty. In some cases, it can be difficult to tell if there is a true deficiency of testosterone, particularly when the levels are in the borderline range.

Male hypogonadism can be divided into two groups.Classical hypogonadism is where the low levels of testosterone are caused by an underlying specific medical condition, for example Klinefelter's syndrome, Kallmanns syndrome or a pituitary tumour.Late-onset hypogonadism is where the decline in testosterone levels is linked to general ageing and/or age-related diseases, particularly obesity and type 2 diabetes.It is estimated that late-onset hypogonadism only affects about 2% of men over the age of 40.

There are two types of classical male hypogonadism primary and secondary.Primary hypogonadism occurs when the low level of testosterone is due to conditions affecting the testes.Primary hypogonadism is also referred to as hypergonadotrophic hypogonadism, whereby the pituitary produces too much luteinising hormone (LH) and follicle stimulating hormone (FSH) (gonadotrophins) to try and stimulate the testes to produce more testosterone. However, as the testes are impaired or missing, they are not able to respond to the increased levels of gonadotrophins and little or no testosterone is produced. In some patients with primary hypogonadism, testosterone levels may be within the normal range, but the increased LH and FSH indicates that the pituitary gland is trying to compensate for a deficiency and treatment may still be needed.

Examples of conditions affecting the testes, which lead to primary gonadal failure, include:

Secondary hypogonadism results from conditions affecting the function of the hypothalamus and/or pituitary gland.It is also known as hypogonadotrophic hypogonadism due to low levels of LH and FSH resulting in decreased testosterone production.Secondary hypogonadism often occurs as part of a wider syndrome of hypopituitarism.Examples of causes can include:

The signs and symptoms depend on the stage at which the patient presents with hypogonadism in relation to sexual maturity.If testosterone deficiency occurs before or during puberty, signs and symptoms are likely to include:

Around the time of puberty, boys with too little testosterone may also have less than normal strength and endurance, and their arms and legs may continue to grow out of proportion with the rest of their body.

In men who have already reached sexual maturity, symptoms are likely to include:

As some of these symptoms (e.g. tiredness, mood changes) can have multiple causes, diagnosis of male hypogonadism may sometimes get missed initially.

Male hypogonadism is more common in ageing men. The levels of testosterone in men start to fall after the age of 40. It has been estimated that 8.4% of men aged 5079 years have testosterone deficiency.Male hypogonadism is also linked with type 2 diabetes: approximately 17% of men with type 2 diabetes are estimated to have low testosterone levels.

Male hypogonadism does not run in families.There are genetic causes of hypogonadism, which include Klinefelters syndrome and Kallmanns syndrome; however, these conditions occur sporadically, they are not inherited from the parents.

A detailed medical history should be taken.In particular, it is important to find out if virilisation (development of normal male characteristics) was complete at birth, whether the testes descended and to see if the patient went through puberty at the same time as his peers. The patient should be thoroughly examined and the presence and size of the testes recorded, and whether they are correctly positioned in the scrotum.

Many of the symptoms of male hypogonadism are non-specific and can be caused by a range of conditions. Therefore, when diagnosing hypogonadism, it is important that biochemical tests are performed to assess the levels of testosterone in the blood to confirm diagnosis. Blood tests will be carried out to measure testosterone levels.The blood sample should be collected preferably at 9 a.m. (this is because levels of testosterone change throughout the day) and in the fasting state (because eating can lower testosterone leves). The blood test can can be carried out as an outpatient appointment. If the result of the first test shows a low level of testosterone, the test should be repeated after two or three weeks to confirm the result. Other hormones are also tested along with the second blood sample. These hormones include luteinising hormone, follicle stimulating hormone and prolactin (produced by the pituitary gland).The results of these blood tests will help distinguish between primary (low testosterone and high gonadotrophins) and secondary (low testosterone and normal or low gonadotrophins) hypogonadism.Testosterone is carried around the blood stream by a protein called Sex Hormone Binding Globulin (SHBG). SHBG is often checked at the same time as testosterone as it makes it easier to interpret whether there is a true deficiency. In patients with obesity and type 2 diabetes, SHBG is often low which can make the testosterone level appear lower than it really is.

Depending on the findings of the above tests, other investigations may be carried out. These include: a bone densitometry test to assess the impact of testosterone deficiency on bone; semen analysis; genetic studies; and an ultrasound of the testes to check for nodules or growths.

Treatment of classical hypogonadism involves replacement of testosterone with the aim of raising the level of testosterone in the blood to normal levels.Exact treatment will vary between patients and be tailored to their individual needs.Different preparations of testosterone are available:

All these are outpatient treatments. All of these options should be discussed with a medical professional and the most appropriate treatment option chosen.During treatment with testosterone replacement, regular blood tests should be carried out to monitor testosterone levels and if necessary, the dose adjusted to ensure levels return to the normal range.Tablet forms of testosterone taken by mouth are not recommended due to a link with liver damage, and because it is more difficult to monitor replacement.

Testosterone should not be given if the patient has prostate cancer, because it might make the tumour grow quicker. Before starting treatment with testosterone, a blood test to measure a hormone produced by the prostate called PSA (prostate-specific antigen) is carried out (PSA levels are elevated in prostate cancer).The prostate may also be examined (via the back passage) to rule out prostate cancer.

For patients who have been diagnosed with late-onset hypogonadism, there is currently not enough evidence for us to know whether treatment with testosterone is safe and effective over the long term.While there are some short-term studies that indicate it may benefit these patients over a short period of time, there is a need for longer-term clinical trials in this area, following a large number of patients, to assess the long-term impact of testosterone treatment on patients with late-onset hypogonadism. Areas that particularly require focus are assessing the effects of treatment on the likelihood of developing cardiovascular disease, prostate cancer and secondary polycythaemia (a condition in which there are increased numbers of red blood cells in the blood, which may predispose to increased blood clots).

If patients have any concerns about their health, they should contact their GP in the first instance.

There can be mild side-effects of testosterone replacement depending on the form used: injectable forms can cause pain and bruising at site of injection; the gel form can cause skin irritation.

Treatment with testosterone can cause an increase in red blood cells (known as polycythaemia), which increases the risk of thrombosis.Regular blood tests should be carried out during treatment to check for an increase in red blood cells.Enlargement of the prostate is another serious side-effect that should be monitored.Prostate examination and a blood test for PSA should be performed every three months for the first year and then annually in men over the age of 40 years after starting treatment.If patients have any concerns about these possible side-effects, they should discuss them with their doctor.

Symptoms of male hypogonadism, such as lack of sex drive, inadequate erections (erectile dysfunction) and infertility, can lead to low self-esteem and cause depression. Professional counselling is available to help deal with these side-effects; patients should talk to their doctor for more information.Patients generally see an improvement in their sex drive and self-esteem following testosterone replacement therapy. Erectile dusfunction is a common symptom in patients without hypogonadism and may need treatment in addition to testosterone.

Male hypogonadism has been linked with an increased risk of developing heart disease (low testosterone can cause an increase in cholesterol levels). Studies have shown that testosterone levels can be lower in men with type 2 diabetes and in men with excess body weight. However, it is not clear whether this is an association or a direct cause and effect. Lifestyle changes to reduce weight and increase exercise can raise testosterone levels in men with diabetes.

Testosterone levels in men decline naturally as they age.In the media, this is sometimes referred to as the male menopause (andropause) although this is not a generally accepted medical term.Low testosterone levels can also cause difficulty with concentration, memory loss and sleep difficulties.Current research suggests that this effect occurs in only a small group of ageing men.However, there is a lot of research in progress to find out more about the effects of testosterone in older men and also whether the use of testosterone replacement therapy would have any benefits.

Last reviewed: Mar 2018

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Male hypogonadism | You and Your Hormones from the Society ...

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Low testosterone predicts hypoxemic respiratory insufficiency and mortality in patients with COVID-19 disease: another piece in the COVID puzzle -…

November 21st, 2021

This article was originally published here

J Endocrinol Invest. 2021 Nov 18. doi: 10.1007/s40618-021-01700-7. Online ahead of print.

ABSTRACT

PURPOSE: Hypogonadism was described in high number of male subjects with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection. In this study, we investigated whether low testosterone (T) values may influence the clinical presentation and outcome of SARS-CoV-2-related pneumonia in a large population of adult males with coronavirus disease 19 (COVID-19).

METHODS: Two hundred twenty one adult males hospitalized for COVID-19 at the IRCCS Humanitas Research Hospital, Rozzano-Milan (Italy) were consecutively evaluated for arterial partial pressure oxygen (PaO2)/fraction of inspired oxygen (FiO2) ratio, serum T and inflammatory parameters at study entry, need of ventilation during hospital stay and in-hospital mortality.

RESULTS: Subjects low T values (< 8 nmol/L; 176 cases) were significantly older (P = 0.001) and had higher serum interleukin-6 (P = 0.001), C-reactive protein (P < 0.001), lactate dehydrogenase (P < 0.001), ferritin (P = 0.012), lower P/F ratio (P = 0.001), increased prevalence of low T3 syndrome (P = 0.041), acute respiratory insufficiency (P < 0.001), more frequently need of ventilation (P < 0.001) and higher mortality rate (P = 0.009) compared to subjects with higher T values. In the multivariable regression analyses, T values maintained significant associations with acute respiratory insufficiency (odds ratio [OR] 0.85, 95% confidence interval [CI] 0.79-0.94; P < 0.001 and in-hospital mortality (OR 0.80, 95% CI 0.69-0.95; P = 0.009), independently of age, comorbidities, thyroid function and inflammation.

CONCLUSION: Low T levels values are associated with unfavorable outcome of COVID-19. Prospective studies are needed to evaluate the long-term outcomes of hypogonadism related to COVID-19 and the clinical impact of T replacement during and after acute illness.

PMID:34792796 | DOI:10.1007/s40618-021-01700-7

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Low testosterone predicts hypoxemic respiratory insufficiency and mortality in patients with COVID-19 disease: another piece in the COVID puzzle -...

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CLARUS THERAPEUTICS HOLDINGS, INC. Management’s Discussion and Analysis of Financial Condition and Results of Operations. (form 10-Q) -…

November 21st, 2021

The following discussion and analysis of our financial condition and results ofoperations should be read in conjunction with our unaudited condensedconsolidated financial statements and notes thereto appearing elsewhere in thisQuarterly Report on Form10-Qand Old Clarus's audited financial statements and notes thereto for the yearended December 31, 2020 included in the Prospectus as filed with the Securitiesand Exchange Commission pursuant to Rule 424(b)(3) on October 7, 2021. Some ofthe information contained in this discussion and analysis or set forth elsewherein this Quarterly Report on Form10-Q,including information with respect to our plans and strategy for our businessand related financing, includes forward-looking statements that involve risksand uncertainties. As a result of many factors, our actual results could differmaterially from the results described in or implied by the forward-lookingstatements contained in the following discussion and analysis.Unless otherwise indicated or the context otherwise requires, references in thisManagement's Discussion & Analysis of Financial Condition and Results ofOperations section to "Clarus," "we," "us," "our" and other similar terms referto Old Clarus (as defined below) prior to the Business Combination (as definedbelow) and to the Company and its consolidated subsidiary after giving effect tothe Business Combination.OverviewWe are a pharmaceutical company focused on the commercialization of JATENZO, thefirst and only oralT-replacement,orT-replacementtherapy ("TRT") of its kind that has received final approval by the U.S. Foodand Drug Administration ("FDA"). We believe that current users of TRT are not satisfied with their currentoptions and desire a therapeutic that is safe, effective and more convenient.Our primary goal for JATENZO is for it to become the preferred choice for TRTamong men with hypogonadism - T deficiency accompanied by an associated medicalcondition. In parallel, our broader vision is for Clarus to become a profitablepharmaceutical company dedicated to providing solutions to unmet medical needsby advancing androgen and metabolic therapies for men and women.Our corporate objectives include maximizing the commercial success of JATENZO inthe United States and internationally by making it the preferred choice for TRTfor men with hypogonadism, expanding its research and development portfolio withadditional metabolic therapies for men and women and sourcing new technologiesthrough its business development efforts.We believe JATENZO offers hypogonadal men and prescribing physicians a safe andeffective oral replacement option and has a number of advantages over thecurrently approved replacement therapies, including:CONVENIENT Easy-to-swallow softgel taken BID with food (twice daily) Dose adjustableEFFECTIVE 87% of men achieved T levels in normal range Restored T levels to mid-normal rangeSAFE Safety profile consistent with TRT class

No liver toxicity - JATENZO bypasses first-pass hepatic metabolism; liver

toxicity not observed in clinical studies of up to 2 years duration.

continue to commercialize JATENZO in the United States for the treatment

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Table of Contents

incur sales and marketing costs to support the commercialization of JATENZO;

or acquire additional product candidates for other medical conditions;

adapt our regulatory compliance efforts to incorporate requirements

experience delays or encounters issues with additional outbreaks of the

pandemic in addition to any of the above.

salaries, benefits and other related costs, including stock-based

compensation expense, for personnel engaged in research and development

post-marketing requirements of the FDA for JATENZO and pharmaceutical

A $1.7 million decrease in outsourced advertising and promotion costs due

a $0.4 million increase in commercial analytic and market research costs,

A $1.0 million increase in personnel costs, including stock-based

a $0.6 million decrease in consulting and professional fees, primarily

A $1.0 million decrease in costs related to research and development

A $0.9 million increase in license fees related to the License Agreements

with HavaH and McGill.

A $2.6 million increase in marketing costs, primarily related to the

a $0.2 million increase in patient assistance costs and other sales and

a $1.3 million decrease in commercial analytics and market research costs.

A $2.6 million increase in personnel costs, including stock-based

compensation expense, primarily due to an increase headcount and external

a $0.8 million increase in consulting and professional fees, primarily

a $0.5 million increase in insurance fees, related to directors' and

a $0.2 million increase in other general and administrative expenses.

A $0.9 million increase in license fees related to the HavaH Agreement

a $1.0 million increase in clinical costs related to Phase 4 studies

related to the development of JATENZO, our lead commercial product;

a $1.6 million decrease in costs related to research and development

consulting services.

Net cash used in operating activities (34,452 ) (35,661 )Net cash used in investing activities

Net cash provided by financing activities 49,192 47,220

Net increase in cash and cash equivalents $ 14,720 $ 11,497

the costs of future activities, including product sales, marketing,

the costs of manufacturing commercial-grade product and necessary

the costs of preparing, filing and prosecuting patent applications,

obtaining, maintaining, expanding and enforcing its intellectual property

our ability to establish and maintain collaborations on favorable terms,

if at all.

(1) We have $43.1 million outstanding aggregate principal on our senior secured

notes that bear interest at 12.5% and mature on March 1, 2025.

(2) We have an operating lease agreement for our office space.

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CLARUS THERAPEUTICS HOLDINGS, INC. Management's Discussion and Analysis of Financial Condition and Results of Operations. (form 10-Q) -...

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Bio-Identical Hormone Replacement Therapy Market to Rise Due to the Increasing Applications and Wider Reach, Players Pfizer Bayer. Energy Siren -…

November 21st, 2021

New York, United States: The newly published business Bio-Identical Hormone Replacement Therapy Market research report by the team of industrial researchers and market analysts at DECISIVE MARKETS INSIGHTS contains qualitative data on market items like the top leading market corporations, their implemented methods and methodologies, recent developments, market strategies, financials, and many more. Partnerships, successful company methodologies, profit production procedures, construction initiatives, and other procedures are some of the primary strategies and approaches used by top corporations to increase their profits percentages. This market research report also encourages and assists clients in gaining a better understanding of crucial market features that are projected to increase at a faster rate in the near future.

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Bio-Identical Hormone Replacement Therapy Market Segmentation-By Type : Estrogen Hormone Growth Hormone Thyroid Hormone Testosterone HormoneBy Application : Menopause Hypothyroidism Growth Hormone Deficiency Male Hypogonadism Other Diseases By Key Players : Eli Lilly Teva Novo Nordisk Pfizer Bayer

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Bio-Identical Hormone Replacement Therapy Market to Rise Due to the Increasing Applications and Wider Reach, Players Pfizer Bayer. Energy Siren -...

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What is CRISPR technology and can it improve poor eyesight? | World Economic Forum – World Economic Forum

November 21st, 2021

More evidence for the efficacy of a groundbreaking new gene-editing medical procedure has emerged, deepening hope it will provide one-shot treatments or even cures for cancer, sickle cell anaemia and other conditions.

Some people suffering from a rare severe visual disorder were able to see more clearly after being treated using the gene-editing technology known as CRISPR, according to reports.

For the first time, CRISPR gene-editing tools were injected directly into the human body, in this case to tackle the leber congenital amaurosis (LCA) condition that made it difficult for the volunteers to navigate their surroundings or see colours.

A volunteer in the experiment can now see colours and regained more peripheral vision.

Image: Mass Eye and Ear

In 2012, CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, was first engineered as a biological tool capable of precisely altering DNA. Over the last decade, scientists around the world have continued to improve the safety and efficacy of CRISPR technologies, opening the door to potentially treating a range of genetic diseases.

For years, scientists have been trying to devise treatments that directly address the root cause of disease on the genetic level. Many commonly-used drugs act like a sledgehammer and non-specifically treat disease, which can lead to negative side effects and have varying impact across patients.

CRISPR falls into a category called precision medicine, which can precisely address the specific genetic defect causing a particular disease.

CRISPR works by combining scissor-like proteins with other molecules to locate troublesome parts of a persons DNA blueprint the genome.

CRISPR allows scientists to remove these disease-causing regions of the genome or replace them with DNA that stops or reverses the illness.

CRISPR is already used for a range of applications, from simple diagnostic tools to basic research purposes. During the pandemic, scientists created a CRISPR-based test for COVID-19.

But its wider rollout as a therapy depends on more trials and examination of possible side effects. Over a year following the CRISPR-based treatment of a patient with sickle cell disease, positive health improvements suggest that gene editing may offer a viable cure for many genetic diseases, but broad testing and long-term monitoring remain vital.

The application of precision medicine to save and improve lives relies on good-quality, easily-accessible data on everything from our DNA to lifestyle and environmental factors. The opposite to a one-size-fits-all healthcare system, it has vast, untapped potential to transform the treatment and prediction of rare diseasesand disease in general.

But there is no global governance framework for such data and no common data portal. This is a problem that contributes to the premature deaths of hundreds of millions of rare-disease patients worldwide.

The World Economic Forums Breaking Barriers to Health Data Governance initiative is focused on creating, testing and growing a framework to support effective and responsible access across borders to sensitive health data for the treatment and diagnosis of rare diseases.

The data will be shared via a federated data system: a decentralized approach that allows different institutions to access each others data without that data ever leaving the organization it originated from. This is done via an application programming interface and strikes a balance between simply pooling data (posing security concerns) and limiting access completely.

The project is a collaboration between entities in the UK (Genomics England), Australia (Australian Genomics Health Alliance), Canada (Genomics4RD), and the US (Intermountain Healthcare).

The application to LCA volunteers at Oregon Health & Science Universitys Casey Eye Institute in the US is a first of its kind.

To date, CRISPR treatments involved taking cells from a patient and changing parts of the subjects DNA before reinserting the edited cells back into the patient. Once back inside the body, the cells could multiply and hopefully eliminate the disease.

In the Oregon trials, however, the CRISPR tools were injected directly into the seven volunteers without removing any of their cells. In their case, it was inserted into the retina of the eye.

This new technique holds out hope for treatment of conditions in parts of the body from which cells cant be safely removed, such as the brain.

Its a really amazing technology and very powerful, Dr Mark Pennesi, Professor of Ophthalmology at the Institute told NPR.

One of the Oregon volunteers, Carlene Knight, said she was able to safely navigate her surroundings following the procedure, while another, Michael Kalberer, found he could see colours for the first time.

At his cousins wedding, Kalberer discovered that he could see the DJs strobe lights change colour and identify them to my cousins who were dancing with me, he told NPR. That was a very, very fun, joyous moment.

The trial is still ongoing and other volunteers didnt experience the same improvement. So its too soon to say when CRISPR will be able to treat sufferers of other genetic illnesses as effectively.

The views expressed in this article are those of the author alone and not the World Economic Forum.

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What is CRISPR technology and can it improve poor eyesight? | World Economic Forum - World Economic Forum

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CRISPR and the Climate – Foreign Affairs Magazine

November 21st, 2021

Earlier this month, the much-anticipated UN Climate Change Conference came to an end. As part of the flurry of activity, the United States and the United Arab Emirates launched the Agriculture Innovation Mission for Climate, one of the first major international initiatives wholly dedicated to cutting back on farming emissions. More than 30 countries joined. Many states also signed on to two other agriculture-related pledges, one to reduce methane emissions 30 percent by 2030 and the other to reverse deforestation.

The Agriculture Innovation Mission for Climate, the methane goal, and the deforestation pledge all indicate that increasingly, states recognize the powerful relationship between farming and climate change. Food systems are responsible for one-third of global greenhouse gas emissions. Nitrous oxide from soil and fertilizers can warm the earth, as can methane from livestock digestion and manure. Deforestation motivated by agricultural expansion releases the carbon dioxide stored by plants and is itself responsible for more than ten percent of all emissions. The connection between farming and climate change runs both ways, and deforestation in particular creates a vicious cycle of warming. Climate change has already reduced the growth of agricultural productivity by 21 percent since 1961, and as time goes by, that penalty will worsen. To compensate, countries could wind up converting more forests into farms, which will then further release greenhouse gases.

In order to make agriculture more environmentally friendly, many states are trying to encourage organic farming. But improving agriculture to address the twin challenges of climate change and deforestation will require every tool available. That means natural solutions alone will not be enough. Instead, states will need to embrace modern science, including CRISPR (clustered regularly interspaced short palindromic repeats) technology.

CRISPR is a recent gene-editing invention that can help countries decarbonize their food systems by making crops that can still thrive in bad weatherreducing the need for more farmland. Scientists in Belgium, for example, are using CRISPR to develop a new kind of corn that can withstand heat and drought. U.S. scientists, meanwhile, are designing drought- and salt-tolerant soybeans and drought-resistant corn. They are also using CRISPR to create cereal plants that can better absorb nitrogen from the soil, which could decrease emissions and pollution from fertilizers.

To solve climate change, states must embrace modern science.

But CRISPR will realize its full potential only if many countries embrace the technology. And unfortunately, plenty of governments are letting CRISPR fall victim to the same regulatory and public opinion pitfalls that have hobbled genetically modified organisms, or GMOs. CRISPR technology is not the same as GMO technology; it does not introduce DNA from other species into plants. Yet many governments remain largely opposed to using either GMO or CRISPR technology for crops, shrinking the toolbox for addressing climate change.

Europe may pride itself on its climate change measures, but in agriculture, it is an example of what states shouldnt do. In 2018, the European Unions top court ruled that gene-edited crops were subject to the same stifling regulation that has largely kept GMOs out of European fields since the late 1990s. That means that instead of relying on modern technology, the EUs sustainable farming plan runs through its new Farm to Fork strategy, which will increase organic farming from nine percent to at least 25 percent of cropland in Europe. This may seem eco-friendly in theory, but in reality it is a counterproductive approach that will lower crop yields, requiring more land use for farming. The increase in organic farming under Farm to Fork, for example, would shrink cereal crop production in the EU by an estimated 21 percent. To compensate, Europe would have to convert approximately 3.7 million acres of its forests into farms, and the rest of the world would have to convert an additional 12.4 million acres. This would increase the amount of carbon released from the soil and destroy natural habits.

The EU is the main example of anti-GMO and anti-CRISPR government policies. But its not alone. New Zealand has explicitly stated that gene-edited plants must be regulated in the same prohibitive manner as GMOs. Mexico has not established any unique rules for gene-edited crops, so they are still covered under restrictive GMO regulations. India, the country with the worlds second-largest amount of cultivatable land (after the United States), has proposed deregulating only some types of gene-edited crops, and it is still relatively unclear as to which plants would actually be excluded.

Europes farming strategy will lower crop yields, breeding deforestation.

Thankfully, many other states are exempting CRISPR crops from GMO-style rules. Following the 2018 court ruling in Europe, a coalition of ten countriesArgentina, Australia, Brazil, Canada, the Dominican Republic, Guatemala, Honduras, Paraguay, the United States, and Uruguaysent a signed statement to the World Trade Organization arguing that gene-edited plants should be regulated the same way as conventional ones. The United Kingdom is now freeing itself from the EU ruling and pushing forward with research on CRISPR crops. Japan has signaled that it intends not to classify gene-edited plants as modified organisms under the Cartagena Protocol. China has yet to publicly speak up, but the country has invested heavily in genome editing, so it will also probably defend CRISPR. These governments clearly understand that addressing the challenges climate change poses for agriculture requires all the tools the world has, including gene editing.

But even if CRISPR were simply a new way to create GMOs, that wouldnt make it inherently dangerous. GMOs can accomplish tremendous goodincluding by making emission-friendly products that CRISPR cant. Genetic modification is better than gene editing at producing crops resistant to pests and diseases, which increases yields and allows for the production of more food on less land, decreasing deforestation. The use of insect-resistant, genetically modified Bt (Bacillus thuringiensis) crops, for example, has increased yields by an average of 25 percent globally. Genetic modification is also more effective than gene editing at making herbicide-resistant crops, which improves weed control and increases yields. And insect-resistant and herbicide-tolerant GMO plants have reduced tractor use for insecticide spraying and tillage, dramatically cutting yearly greenhouse gas emissions. Indeed, the yearly reduced usage is equivalent to taking 1.6 million cars off the road. States clearly shouldnt limit modern crop improvement to CRISPR.

At least in the United States, the public isnt reflexively opposed to genetic engineering. Instead, polls suggest that acceptance of the practice varies widely based on the type of application. In a Pew Research Center survey, for example, only 21 percent of respondents said genetic engineering was acceptable if used to create glowing aquarium fish. But 70 percent said that limiting mosquito reproduction to reduce disease is an appropriate use of technology, and 57 percent said the same of breeding animals with tissue and organs that could be given to humans. Some environmentalist groups have also signaled that they are open to genetic engineering, provided it advances their causes. The preservationist Sierra Clubwhich has historically opposed all genetically modified organismsrecently indicated it is receptive to planting genetically modified American chestnut trees, which could help restore a species that dominated eastern U.S. forests until it was nearly wiped out by blight in the late 1800s. That means gene editing has an opening. The vast majority of the world is concerned about climate change, as are most environmentalist organizations. It is possible that they will come to endorse, or at least accept, using CRISPR and GMOs to cut agricultural emissions.

But unlocking the potential of genetic engineering requires more than just favorable public opinion and environmentalist acquiescence. In order to get a wide range of CRISPR products that address climate change and appeal to consumers, developers and countries need better access to the technology. Various iterations of CRISPR gene editing are covered by over 6,000 patents in the United States alone, with 200 more filed every month. This complicated structure means that a developer may have to license many different patents in order to commercialize a single product. Without reform, this messy system may inhibit development.

States need to change their intellectual property laws. But even if they wont, there are steps that private actors can take to make gene editing more accessible. Wageningen University & Research in the Netherlands recently pledged to license its CRISPR patents for free to nonprofit organizations that are using the technology for noncommercial applications, a step that other patent holders should follow. Holders should also make sure their inventions are affordable for small developers and poor countries that dont have the money for massive licensing fees. They can do so through patent-sharing agreements, patent consolidation, and transparent pricing, which would go a long way toward allowing a variety of companies to commercialize CRISPR gene-edited crops. These measures would also help breed innovation: more developers means a wider variety of products.

Finally, regulatory bodies should increase the transparency of their decision-making processes regarding CRISPR by requiring that agricultural companies provide the same kind of information on gene-edited crops exempt from GMO regulations as they do for crops subject to GMO rules. To allow the technology to earn more consumer trust and to reduce public skepticism, developers should also provide product assessments that go beyond safety and show buyers evidence of the environmental benefits. The European Unions Farm to Fork strategy suggests that in many countries, officials and publics are still generally opposed to using modern biotechnology to make farming more sustainable. Fighting climate change and improving agricultures resilience requires a change of mindset. Companies and regulators need to act quickly to prevent unfounded fears of CRISPR from taking root; as the entrenched opposition to GMOs demonstrates, it can be difficult to win people over to a new technology or method once distrust has set in. The world cannot allow a similar dynamic to prevent CRISPR from helping stave off the worst-case climate scenarios.

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CRISPR and the Climate - Foreign Affairs Magazine

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CRISPR Therapeutics and ViaCyte, Inc. to Start Clinical Trial of the First Gene-Edited Cell Replacement Therapy for Treatment of Type 1 Diabetes -…

November 21st, 2021

-Initiation of patient enrollment expected by year-end-

ZUG, Switzerland and CAMBRIDGE, Mass. and SAN DIEGO, Nov. 16, 2021 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ: CRSP), a biopharmaceutical company focused on developing transformative gene-based medicines for serious diseases, and ViaCyte, Inc., a clinical-stage regenerative medicine company developing novel cell replacement therapies to address diseases with significant unmet needs, today announced that Health Canada has approved the companies Clinical Trial Application (CTA) for VCTX210, an allogeneic, gene-edited, immune-evasive, stem cell-derived therapy for the treatment of type 1 diabetes (T1D). Initiation of patient enrollment is expected by year-end.

With the approval of our CTA, we are excited to bring a first-in-class CRISPR-edited cell therapy for the treatment of type 1 diabetes to the clinic, an important milestone in enabling a whole new class of gene-edited stem cell-derived medicines, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. The combination of ViaCytes leading stem cell capabilities and CRISPR Therapeutics pre-eminent gene-editing platform has the potential to meaningfully impact the lives of patients living with type 1 diabetes.

Being first into the clinic with a gene-edited, immune-evasive cell therapy to treat patients with type 1 diabetes is breaking new ground as it sets a path to potentially broadening the treatable population by eliminating the need for immunosuppression with implanted cell therapies, said Michael Yang, President and Chief Executive Officer of ViaCyte. This approach builds on previous accomplishments by both companies and represents a major step forward for the field as we strive to provide a functional cure for this devastating disease.

The Phase 1 clinical trial of VCTX210 is designed to assess its safety, tolerability, and immune evasion in patients with T1D. This program is being advanced by CRISPR Therapeutics and ViaCyte as part of a strategic collaboration for the discovery, development, and commercialization of gene-edited stem cell therapies for the treatment of diabetes. VCTX210 is an allogeneic, gene-edited, stem cell-derived product developed by applying CRISPR Therapeutics gene-editing technology to ViaCytes proprietary stem cell capabilities and has the potential to enable a beta-cell replacement product that may deliver durable benefit to patients without requiring concurrent immune suppression.

Story continues

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

About ViaCyteViaCyte is a privately held clinical-stage regenerative medicine company developing novel cell replacement therapies based on two major technological advances: cell replacement therapies derived from pluripotent stem cells and medical device systems for cell encapsulation and implantation. ViaCyte has the opportunity to use these technologies to address critical human diseases and disorders that can potentially be treated by replacing lost or malfunctioning cells or proteins. ViaCytes first product candidates are being developed as potential long-term treatments for patients with type 1 diabetes to achieve glucose control targets and reduce the risk of hypoglycemia and diabetes-related complications. To accelerate and expand ViaCytes efforts, it has established collaborative partnerships with leading companies, including CRISPR Therapeutics and W.L. Gore & Associates. ViaCyte is headquartered in San Diego, California. For more information, please visit http://www.viacyte.com and connect with ViaCyte on Twitter, Facebook, and LinkedIn.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni and Mr. Yang in this press release, as well as regarding CRISPR Therapeutics expectations about any or all of the following: (i) the safety, efficacy and clinical progress of our various clinical programs including our VCTX210 program; (ii) the status of clinical trials (including, without limitation, activities at clinical trial sites) and expectations regarding data from clinical trials; (iii) the data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials; and (iv) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies, including as compared to other therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients not to be indicative of final trial results; the potential that clinical trial results may not be favorable; potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR Therapeutics Investor Contact:Susan Kim+1-617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel Eides+1-617-315-4493rachel.eides@crisprtx.com

ViaCyte Investor Contact: David Carey, Lazar-FINN Partners+1-212-867-1768david.carey@finnpartners.com

ViaCyte Media Contact: Glenn Silver, Lazar-FINN Partners+1-973-818-8198glenn.silver@finnpartners.com

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CRISPR Therapeutics and ViaCyte, Inc. to Start Clinical Trial of the First Gene-Edited Cell Replacement Therapy for Treatment of Type 1 Diabetes -...

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Need to streamline research on CRISPR gene-editing technology: Experts – Business Standard

November 21st, 2021

The endogenous manufacturing of CRISPR components, through greater research, would make India a commercially successful country in the field of Deep Science, according to Girish Krishnamurthy, CEO & MD, Tata Medical and Diagnostics Ltd.

Participating in a panel discussion on 'Gene-Editing On Centre Stage' at the 'Bengaluru Tech Summit 2021', Krishnamurthy opined that the therapeutics R&D is slow in India as compared to the West, hence seeking deeper research experiences is significant.

"The country also needs to address associated infrastructural issues like the building of cold storage, expanding supply chains and the sorts," he said.The CEO also highlighted the misconception amongst people that CRISPR is meant for therapeutic and not diagnostic purposes and that it needs to change. With basic technology and market being the most crucial focal points, a large number of its applications are to be looked at, serving both urban and rural India.Though grants are channelized from the Department of Biotechnology, Dr Saravanabhavan Thangavel, Assistant Investigator, Centre for Stem Cell Research, discussed the difficulty in attracting private funds to expand the CRISPR technology that deals with almost all primary deficiencies.Talking about the guidelines existing on Therapeutic diagnostics and products, Dr Shambhavi Nayak, Head of Research, Takshashila Institution expressed the vagueness in the policy.

"The Government should move to a facilitator role, making markets more accessible" she added, referring to the potential for Gene-Editing in itself as a boon.Dr Vaijayanti Gupta, Leas Scientist, CrisprBits Pvt Ltd., emphasized on the importance of understanding the licensing, patent rights, legal and ethical framework and the overall impact on health and well-being.

"As CRISPR is trying to hit single-cell and rapid diagnostics, investments from the private sector are essential to allow this space to develop from market angle", she said.Working on CRISPR in plants, particularly Banana, Dr Siddharth Tiwari, Scientist, National Agri-Food Biotechnology Institute, said the enzymes used to target carotenes development are of prime significance.

" While the releasing of genetically engineered crops in India is in the hands of Government, the non-transgenic approach is being preferred recently," he told and stressed the need for a sustained effort to support the endeavours that can bring it to the common man.

(Only the headline and picture of this report may have been reworked by the Business Standard staff; the rest of the content is auto-generated from a syndicated feed.)

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Need to streamline research on CRISPR gene-editing technology: Experts - Business Standard

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Plant Breeding and CRISPR Plants Market Analysis By Industry Size, Share, Revenue Growth and Demand Forecast To 2027 Energy Siren – Energy Siren

November 21st, 2021

The most recent research will provide you with an overview of the global Plant Breeding and CRISPR Plants Market in general, as well as factors that may influence future growth, or lack thereof, as well as potential opportunities and current trends. This study examines the global markets structure, as well as market segmentation, growth rates, and revenue share comparisons. The market study reports research findings aid in the assessment of a range of essential variables, such as investment in a developing market, product success, and market share expansion.

Request a Free Sample of Report @ https://www.intelligencemarketreport.com/report-sample/143338

Major market player included in this report are:

A revenue market size analysis, as well as market drivers, restraints, and opportunities, are all included in the study. The report also includes a picture of the industrys main competitors competitive landscape, as well as the top businesses percentage market share. This research looks into the Plant Breeding and CRISPR Plants Market in great detail. The research reports market estimates and predictions are based on extensive secondary research, primary interviews, and in-house expert opinions. The impact of different political, social, and economic factors, as well as current market conditions, on market growth, is examined in these market projections and estimates.

The detailed segments and sub-segment of the market are explained below:

By Product Type:

By Application Type:

The segmentation of the global Plant Breeding and CRISPR Plants Market in terms of the various regions and countries involved, as well as a breakdown of revenues, market shares, and possible expansion prospects, are all covered in this section. This research looks at revenue growth at the global, regional, and country levels, as well as recent industry trends in each sub-segment.

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COVID-19 Impact Analysis

The influence of COVID-19 on the Plant Breeding and CRISPR Plants market at the global and country levels is examined in this research report. The demand and supply side effects of the target market are considered in this study. Primary and secondary research, as well as private databases and a paid data source, were used in this study. Market players will benefit from the COVID-19 impact study as they implement pandemic mitigation strategy.

Competitive Outlook

The Plant Breeding and CRISPR Plants market study contains a chapter on major global market participants, which includes a review of the companys business, financial statements, product description, and strategic aims. The companies covered in the report can be customized to meet the needs of a client. This chapter will go through each of the major industry competitors and their present market position in detail.

Furthermore, years considered for the study are as follows:

Target Audience of the Global Plant Breeding and CRISPR Plants Market in Market Study:

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Could We Gene Hack Ourselves to Be Blissed Out Sex Maniacs, Like Our Distant Cousins the Bonobos? – Futurism

November 21st, 2021

This week, were pleased to bring you a different version of Futurism, containing stories from the horizon of hedonism. Welcome toThe Science of Pleasure.In collaboration with our friends over atMEL Magazine, this week, well be bringing you stories from both publications about the pleasures of tomorrow, today.

Bonobos are a species of primate believed to be humankinds second-closest living ancestor. Thats intriguing, because zoologists have long observed that these distant cousins of ours are driven by a syrupy mixture of empathy and hedonism, lounging in simian cuddle puddles, caressing one another, kissing with tongue, and engaging in an awe-inspiring variety of bisexual encounters, sometimes with multiple partners, and often while screaming in pleasure.

Sounds pretty fun, doesnt it? Sure, party drugs like MDMA can temporarily flood your brain with oceans of empathy and euphoria, but they can be terrible for you if you take them too much, and the hangovers can be brutal. In other words, theres no drug thats gonna give you bonobo-brain safely and long term.

Relentless progress in the science of genetic engineering raises an interesting alternative, though: what if scientists gene-hacked humans, who already share around 98 percent of their genetic material with bonobos, so that wed be more affectionate, amorous and generally happy? What if you could take a CRISPR-like brain injection that permanently altered your cognition so that you could experience bonobo-like bliss?

If such a thing is theoretically possible, geneticists told us, its far beyond todays biotech capabilities. But eventually, if research continues to barrel ahead, those types of profound alterations to the human mind may well become a reality and if so, theyre likely to raise profound questions about what it means to be human.

I like to think that everything is in the realm of scientific possibility, said Esmerelda Casas-Silva, a biomedical researcher at the National Institutes of Health who cautioned that her musings on our strange question did not reflect the views of her employer. Its really just a matter of us figuring out how to do it.

Overall, she said, she believes that the idea would be tricky yet certainly possible.

If so, what a life it would be. A greater genetic propensity for peace and promiscuity, like that of the bonobos, doesnt sound so bad.

Famed scientist Carl Sagan and his spouse, Cosmos cocreator Ann Duyan, wrote extensively about bonobos in the 1993 book Shadows of Forgotten Ancestors: A Search for Who We Are.

Bonobos use sexual stimulation in everyday life for many purposes besides mere satisfaction of the erotic impulse, they wrote, including in exchange for food, as a way to resolve conflicts among same sex adults, and as a generic, all-purpose approach to social bonding and community organization.

Were we humans to become more like bonobos, militarism and excess aggression could become obsolete, as less authoritarian social structures characteristic of bonobo societies came to predominate. Socioeconomically, a bonobo way of life could embody the ethic of solidarity and cooperative decision-making over competition, domination or exploitation.

The kind of pleasures that bonobos enjoy are not decadent, said Susan Block, a sex therapist and the author of the 2014 book The Bonobo Way: The Evolution of Peace Through Pleasure. They dont spend any money. Maybe a few bananas, but they share those bananas.

Whether gene hacking ourselves to be as guileless and empathic as bonobos would be ethical, of course, is a different question entirely.

For one thing, the whole hypothetical smacks of eugenics. For another, even though the goal of gene therapy to make humans more like bonobos might nominally be to encourage a more equitable society, the ironic reality is that our actual world is rife with inequality, hierarchies, and unfair institutional arrangements.

In other words, itd probably be the wealthy and powerful who would seize the treatment for themselves or, chillingly, foist it on others in order to create a more obedient workforce, sort of similar to how in the 2018 satire film Sorry to Bother You, a parody of contemporary entrepreneurs named Steve Lift tries to use gene-modifying powder to create Equisapien human-horse hybrids capable of laboring harder and longer than people already do today.

We really shouldnt be in a hurry to make designer babies right now, Casas-Silva said. We have very incomplete information, and even if we do know exactly whats going to happen, whos to say what we should decide and what characteristics are best or not?

That point is larger than just the bonobo thought experiment, of course, and its also something the world is likely going to need to confront soon. A Chinese scientist named He Jiankui sparked a global controversy in 2018 when he announced that he had altered the genetic makeup of twin girls, using CRISPR to attempt to make them immune to HIV while they were still embryos and it later emerged that he had been involved in discussions to open a designer baby clinic as well.

If those types of clinics become a reality, itll likely only be affluent parents who can afford them, deepening existing social and economic divides.

Are we going to create even more disparities in health and different populations that are suffering because they dont have access to the same technologies as, you know, other people who might have enough funds to make superhuman babies? Casas-Silva asked. And we could make very dangerous, in my opinion, very dangerous future humans.

And, on a practical level, our anxious human brains keep the power plants running and the industrial agriculture machinery feeding everybody. If we spent all day lounging around and engaging in bacchanalia, that could easily fall apart, for better or worse.

In fact, Block points out that bonobos are a good case study in why giving yourself over to pleasure entirely might not be a great survival strategy.

Block told Futurism about an incident involving bonobos and common chimpanzees our closest living ancestors, for those keeping score in a German zoo during the bombing of Dresden. The zoo wasnt bombed, but the apes could hear the detonations and possibly felt some of the impact.

All the bonobos died of heart attacks, and all the common chimps didnt care, the sexologist said, adding that the zoo kept the former separated in captivity, meaning they werent together to calm each other down.

When youre empathetic, thats a double edged sword, isnt it? she said. Empathy can feel so good, but it also means that when you hear bombs dropping, even if theyre not dropping on you, you freak out. Its like its happening to you. Or if you see someone being hurt, you freak out.

If we do go wild with CRISPR, or whatever advanced tool it takes, to turn human beings into overly peaceable and amorous creatures, Casas-Silva cautions that we could end up falling in love incessantly, with everyone or everything. It might even preclude the intense and exclusionary monogamous love thats often the social norm.

I mean, that could be pretty devastating, Casas-Silva said. Theres a reason that our bodies and our minds decrease our feelings and our memories. Thats a safety measure for anyone whos fallen in love and had it not work out. You know, its a blessing that we can diminish those feelings of hurt and anxiety and everything that comes with a breakup over time.

But, even if we refrain from hacking away at our genes in the hopes of bonoboizing ourselves, Block maintains we still have a lot to learn from our sex positive comrades, including lessons in revaluing our natural bodies and pursuing mutual pleasure without guilt.

The idea that our natural self is Satan is killing us, she said. [The belief] that we are somehow superior, that we are angels, that we are not part of nature, that we are not part of this earth, that we can fly away, like angels maybe a couple people can, you know? Captain Kirk went up there, right? Ninety years old, okay. But most of us are here, and Im looking to improve the lives of those of us that are here on this beautiful, wild sexual planet Earth.

More on CRISPR:WHO Says Gene-Hacking Superhumans Should Be Illegal

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Could We Gene Hack Ourselves to Be Blissed Out Sex Maniacs, Like Our Distant Cousins the Bonobos? - Futurism

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Years later, a first-of-its-kind treatment shows the power, and limits, of gene therapy – BioPharma Dive

November 21st, 2021

When Misty Lovelace was a baby, her eyes were drawn to the light.

She could not focus on faces, only sources of light. Her grandmother Cynthia Lovelace, who would become her main caretaker, suspected vision problems.

By age three, Misty was diagnosed as legally blind. School systems struggled with how to handle her. She was intelligent and intuitive, but people would treat her as if she had a learning disability.

As she got older, Misty started carrying a lamp with her at school. She would put her lunch under it to see what she was about to eat. She learned Braille and used a cane to navigate. When she visited the doctor for checkups, her prognosis seemed to get worse.

"[The doctor] would take her little face and he'd put his hands on her face and say, 'Misty, I'm so sorry, there's nothing more we can do for you, honey. You're going to wake up in the dark one day,'" Lovelace recalled.

"It'd be like looking through a tunnel. And all of a sudden that tunnel goes out."

Misty has Leber congenital amaurosis, or LCA, a genetic disorder that often manifests at a young age, causing vision loss. In Misty's case, and for approximately 1,000 to 2,000 other people in the U.S., the disease is caused by mutations in a gene called RPE65.

Misty Lovelace, age 4

Courtesy of Misty Lovelace

What Misty didn't know as her vision got darker was that a scientist and doctor duo at the Children's Hospital of Philadelphia had already spent years working on a gene therapy for her disease.

The gene therapy, which would eventually become known as Luxturna, was not an overnight success. Decades of research and setbacks preceded the landmark U.S. approval of Luxturna four years ago, the first the Food and Drug Administration had ever granted to a gene therapy for an inherited disease. While Luxturna is not a cure for blindness, treatment has brought sustained improvements in sight, particularly in lower light, for several patients who spoke with BioPharma Dive. As a result, they've needed less help in educational and social environments, and have more independence.

Their experience with Luxturna is proof of gene therapy's potential as well as its limitations. As the first gene therapy of its kind, Luxturna also holds lessons for a field that's grown dramatically since its December 2017 approval.

Lovelace said she never stopped trying to find a way for Misty to regain her sight. The possibility gave her hope as she watched her granddaughter adjust to a life that, for her, was almost in total darkness.

A call from Jean Bennett was a lifeline.

Bennett and her husband, Albert Maguire, met at Harvard Medical School in the early 1980s. The two began researching gene therapy together, attempting to treat blindness in mice. Soon they were testing their approach on Briard dogs with the same defective RPE65 gene that causes LCA in humans.

By 2007, their gene therapy was ready to be tested in people a high-stakes proposition for a field that had largely been shut down nearly a decade before. After 18-year-old Jesse Gelsinger died during a 1999 gene therapy study, many questioned whether such research was safe. The success Bennett and Maguire had with Luxturna was a large part of gene therapy's journey back to the forefront of biomedical research, aided by improvements in how such treatments are designed and delivered.

Testing began at the Children's Hospital of Philadelphia, where Misty was recruited as a study participant. At age 12, she took her first flight out of Kentucky and received the gene therapy in both eyes, starting with the one with worse vision.

"We didn't know if I was going to get worse, stay the same or get better," she said. "But in my mind, I was going to be completely blind by 18, so what's knocking a couple years off?"

The improvements were almost immediate, however. Lovelace recalls her granddaughter commenting on her wrinkles as soon as the eye patches from the procedure were removed. Misty could make out the fine hairs on the manes of horses, her favorite animal and hobby. Rainbows and stars, though, she found underwhelming.

More than eight years later, Misty says she's grateful she "took the leap," attributing to Luxturna her independence and ability to pursue a career as a horse trainer.

Misty Lovelace

Courtesy of Misty Lovelace

Results from early participants like Misty led to the formation of Spark Therapeutics and a larger clinical trial in Pennsylvania and at the University of Iowa that gave the biotech company the evidence needed to approach the FDA.

On Oct. 12, 2017, a panel of scientists and FDA advisers unanimously endorsed the gene therapy, with Misty one of several individuals who shared their stories. The FDA followed with an approval on Dec. 18, a gene therapy milestone.

"For many of us, this is exactly the type of disease that we hoped that gene therapy would someday treat," Wilson Bryan, director of an FDA office tasked with reviewing Luxturna, said at the time. The next year, Luxturna was also approved in Europe.

It's unclear how many people have received Luxturna since. A Spark spokesperson told BioPharma Dive the company does not disclose that information. In 2019, the company told the Philadelphia Business Journal it had shipped 75 vials of the gene therapy in its first year post-approval. (One vial is used per eye.)

Spark is now owned by the Swiss pharmaceutical company Roche, which does not disclose sales of Luxturna. In February, however, Roche reduced the accounting value of Luxturna, citing "reduced sales expectations."

Luxturna consists of one hundred and fifty billion copies of the corrected RPE65 gene encoded into modified viruses, which are delivered into the eye via about 0.3 milliliters of liquid. Those few drops are injected underneath the retina and, over the course of a week, the viral particles shuttle the functional gene into the patient's eye cells. Once inside, the gene instructs the cells to produce a protein that's otherwise missing, helping restore visual function.

Vials of Luxturna

Spark Therapeutics

"This is not a cure," said Jason Comander, a physician at Massachusetts Eye and Ear in Boston who has administered Luxturna. "It will not make your vision normal," he added, "and there's a small chance that it could hurt your vision." Comander consults with other drugmakers and in 2019 received a nominal amount from Spark.

Luxturna also benefits each patient differently. Comander said the vast majority gain some night vision, while others report improvements in central or side vision. Some see more substantial improvements one of his patients was able to see in up to one thousand times dimmer light than in pre-surgery exams. Many have been able to walk without canes and read without using Braille after surgery.

Their vision isn't perfect, however. Some recipients, Misty included, are still considered legally blind and unable to drive. How long the benefit of gene therapy treatment will last is still unclear, though a recent study co-authored by Maguire and Bennett indicated "improvements were maintained up to 3 to 4 years" after Luxturna.

Comander, who was in his residency while Luxturna was tested, said seeing Maguire administer the therapy affirmed his decision to go into the practice. Now, Comander has done close to a dozen surgeries; his youngest patient was 4 years old at the time of treatment and his oldest was in their 30s. While younger patients saw greater improvements, each patient's eyes functioned better in lower light following treatment.

For Comander, Luxturna was an inspiration, one that he said has helped fuel greater interest in gene therapy. "Many careers have been dedicated to expanding on the success of Luxturna, and it's made a huge difference in the field," he said.

Since Luxturna's clearance, Novartis won FDA approval in May 2019 for a spinal muscular atrophy treatment known as Zolgensma, making it the second gene therapy for an inherited disease available in the U.S. A handful of other gene therapies are in late-stage testing and, behind them, are an expanding pipeline of experimental medicines for a constellation of genetic conditions. In 2020 alone, the FDA received more than 230 applications from cell and gene therapy developers to begin clinical trials, the head of the agency's biologic drugs division said earlier this year.

Gordon "Creed" Pettit was one of the kids who couldn't get into clinical trials for Luxturna. His mother, Sarah St. Pierre-Pettit, brought him from Florida to the University of Iowa a number of times. But he couldn't get through the tests needed to qualify him for treatment.

From there, it was a waiting game until Luxturna's approval. Soon after the FDA's decision, Pierre-Pettit brought Creed to Audina Berrocal at the Bascom Palmer Eye Institute in Miami.

Gordon "Creed" Pettit and Audina Berrocal, the surgeon who administered Luxturna to him.

Photo courtesy of Sarah Pierre-Pettit

Creed was Berrocal's first Luxturna patient. As a pediatric retina specialist, Berrocal said Spark sought her out in the fall of 2017. To date, she's performed a dozen surgeries, all of which have yielded positive results.

"Of all the things I've done in my career, this has been the most amazing and the most rewarding in the sense that we are changing the genetics, the DNA of a person, and we're allowing them to do things that before they couldn't do," Berrocal said. Berrocal consults with other drugmakers and has contributed to published research on Luxturna. In 2018 and 2019, she received nominal payments from Spark.

But treatment, even when positive, can come with adjustments, too. In Creed's case, he was overwhelmed by the sudden change, at first telling his mother he wished he had his old eyes back.

With time, however, Creed has started challenging himself more. "I think most of the gains were at the beginning," Pierre-Pettit said. "Whatever Luxturna did is done. But now that he finally feels confident with himself, he's putting Luxturna to the test now."

For Creed, that means being more social and inquisitive about the world around him. Now 12 years old, he hasn't mentioned wanting his old eyes back for years.

"It's still almost like a new kid every day, like a new baby that sees something new," his mother said.

From a young age, Luke Ward told his mother, Stephanie Joachim, about his dream of playing soccer. But the sport as well as many other daily tasks seemed out of reach.

His vision problems were apparent from birth. While his twin sister could track people with her eyes, Luke stared only at sources of light. When he started walking, he needed to put his hands out to stop himself from running into walls.

Genetic testing revealed Luke had LCA. His doctor said he'd be legally blind by kindergarten. Around the same time, Joachim read an article about Luxturna, but was too late to get Luke enrolled in clinical testing. By the time the FDA approved the therapy, the family had already decided that Luke was getting Luxturna.

Luke Ward with his twin sister, Leia.

Courtesy of Stephanie Joachim

But Joachim was anxious after learning Luxturna's price tag of $425,000 per eye. "I was just flabbergasted and I was like, 'You know what, it's fine. We have the best health insurance,'" she said.

To the family's disappointment, and as other Luxturna patients have experienced, insurance denied the request and cited the therapy's then "newness" as a reason.

At some point in the process, however, Luke's file crossed the desk of an anonymous person who was "so moved from Luke's story and from Luke's pictures, he volunteered to pay for Luke's surgery," Joachim said.

Luxturna's cost was criticized when the therapy was approved and has remained an issue within the patient community since. Shortly after the FDA gave its OK, Spark announced a program with health insurer Harvard Pilgrim and affiliates of Express Scripts, through which the company agreed to pay rebates if the drug doesn't help patients meet certain thresholds.

In a statement to BioPharma Dive, Spark said it offers a "range of patient services and payment models to help navigate and support access" to Luxturna, but did not respond to questions on the number of times rebates have been paid.

Luke Ward

Courtesy of Stephanie Joachim

"Parents shouldn't be paying for this out of pocket," Berrocal, who was also Luke's surgeon, said.

Berrocal told Luke he's the "poster child for Luxturna," Joachim said. He can play sports with his twin sister, including soccer and tee-ball. He started kindergarten this year and has no issues seeing the whiteboard. He still has visual impairments, though, including his peripheral vision. His mother says they keep their shoes tucked out of the way in the house to prevent Luke from tripping.

Four years after its approval, Luxturna continues to be sought out by patients. Joachim says she's received messages from people in Spain, South Africa and the U.K. inquiring about Luke and his progress.

And as Luxturna keeps working, other drugmakers hope to replicate its success. The eye, in particular, is the focus of many gene therapy developers, as it's easy to access and targeting it doesn't carry as many safety risks as other organs. Novartis, which sells Luxturna in Europe, AbbVie, Biogen and Johnson & Johnson are all exploring gene therapies for the eye.

Research into gene editing is advancing as well. In September, Editas Medicine shared preliminary results from the first trial testing a CRISPR gene editing treatment that does its work inside the body. Treatment appeared safe, although the efficacy results were mixed, with several patients experiencing little improvement in vision. The treatment uses CRISPR editing to restore the function of eye cells in people with another form of LCA known as type 10.

Berrocal believes Luxturna represents the beginning of what genetic medicine can offer to patients with many inherited diseases, not only those of the eye.

"20 years from now, we could look back and say, 'Oh my god, that was so rudimentary. Look how much you have advanced,'" she said. "But we have to start somewhere, right? And in 2021, this is what we have, and it's working."

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Years later, a first-of-its-kind treatment shows the power, and limits, of gene therapy - BioPharma Dive

Recommendation and review posted by Bethany Smith

A video that originated on InfoWars is filled with falsehoods about COVID-19 vaccines – PolitiFact

November 21st, 2021

A lengthy video posted on TikTok that makes a host of unfounded claims about COVID-19 vaccines all of which have been repeatedly debunked originated with InfoWars, a website renowned for sowing conspiracy theories.

The viral video claims that COVID-19 vaccines "failed miserably" in animal trials and are "a type of gene therapy that several top scientists warn will kill you." It includes hashtags like #truthcomesout and #firefauci.

The video appeared on Facebook, where it was flagged as part of Facebooks efforts to combat false news and misinformation on its News Feed. (Read more about our partnership with Facebook.)

COVID-19 vaccines did not fail in animal trials or result in the death of the animals tested. The vaccines are not a form of gene therapy, which modifies a persons genes to replace or fix mutations that lead to diseases. The scientists cited in the video have spread misinformation about the vaccines.

The footage is an excerpt from a longer video posted online Oct. 13 called "Kill Shot," by Greg Reese, an editor and producer for the website InfoWars, which has spread other vaccine misinformation. Most recently, InfoWars was in the news because its founder and host, Alex Jones, was found liable for defamation against the families of victims from the 2012 Sandy Hook Elementary School shooting, which Jones has portrayed as a hoax.

In the TikTok video, a narrator says that 22 months into the COVID-19 pandemic, "We have all the information needed to paint a clear picture of whats going on." The narrator then recites a laundry list of false claims about Dr. Anthony Fauci, PCR tests, the ingredients of vaccines and more.

The narrator says, "The COVID vaccines are not vaccines but rather highly controversial mRNA tech that failed miserably on its animal trials, a type of gene therapy that several top scientists warn will kill you," a statement that contains multiple falsehoods.

First, COVID-19 vaccines did not fail animal trials. Fact checkers have debunked this claim, noting that the two vaccines most widely used in the U.S. Pfizer and Moderna produced desirable outcomes in animal testing. Results from Modernas animal testing were published in the New England Journal of Medicine after monkeys had a robust immune response to the vaccine.

Animals also did not die during the vaccine trials. Full Fact reported that had any animals died, human trials that were running concurrently would have been halted, which they were not.

Next, COVID-19 vaccines are not a type of gene therapy; PolitiFact and others have reported that the claim is false. Gene therapy is a process of modifying genes to replace or fix mutations that lead to diseases, according to PolitiFact.

Thats different from mRNA vaccines, which send instructions to the bodys cells to make a piece of spike protein, which is also found on the surface of the virus that causes COVID-19, so that the immune system can respond to it.

Finally, the videos narrator says "several top scientists warn" that the COVID-19 vaccines "will kill you," and names Dr. Ryan Cole and Dr. Nathan Thompson. Cole, who is licensed to practice medicine in several states and is under investigation by the Washington Medical Commission, falsely claimed in the spring that mRNA vaccines cause cancer and autoimmune diseases. He was rebuked by the author of the medical paper he cited as evidence for the claim.

The other doctor identified, Thompson, has claimed that COVID-19 vaccines weaken the immune system, which PolitiFact rated False.

Our ruling

A TikTok video posted on Facebook says COVID-19 vaccines "failed miserably" in animal trials and are "a type of gene therapy that several top scientists warn will kill you." The video originated on InfoWars, known for spreading conspiracy theories.

The vaccines did not fail in animal trials or result in the death of the animals tested.

COVID-19 vaccines are not a type of gene therapy, which involves modifying genes to replace or fix mutations that lead to diseases. The mRNA vaccines do not change a persons genetic makeup and never enter the part of the cell that hosts DNA.

The scientists cited in the video have spread misinformation about the vaccines.

We rate this claim False.

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A video that originated on InfoWars is filled with falsehoods about COVID-19 vaccines - PolitiFact

Recommendation and review posted by Bethany Smith

Coave Therapeutics Strengthens Leadership Team with the Appointments of Thomas Blaettler MD as Chief Medical Officer and Patricia Franon PhD as Chief…

November 21st, 2021

PARIS, Nov. 15, 2021 /PRNewswire/ --Coave Therapeutics ('Coave'), a clinical-stage biotechnology company focused on developing life-changing gene therapies in rare Ocular and CNS (Central Nervous System) diseases, today announced that it has strengthened its leadership team with the appointments of Thomas Blaettler MD, as Chief Medical Officer, and Patricia Franon PhD, to the newly created position of Chief Operating Officer.

"I am very pleased to welcome Thomas and Patricia to the leadership team at Coave. Their collective accomplishments and deep domain expertisein neuroscience, cell and gene therapy, in addition to their extensive clinical drug development and project management experience will be invaluable as we progress our lead candidate through clinical development and advance our pipeline of novel gene therapies into clinical development targeting rare Ocular and CNS diseases," said Rodolphe Clerval, CEO.

Thomas Blaettler, MD

Dr Blaettler is an expert in the neuroscience therapy area, having over 25 years' experience in the field, both in clinical residency and industry. Thomas joins Coave from Orphazyme A/Swhere, since 2016, he served as Chief Medical Officer and was responsible for devising the clinical development strategy and progressing the company's rare neurodegenerative pipeline. In addition to championing the clinical and regulatory strategy, Thomas has contributed to Orphazyme's IPO on both the Copenhagen and Nasdaq stock exchanges. Prior to Orphazyme, Thomas held global leadership roles within the clinical neuroscience divisions at both Roche and Bristol Myers Squibb, with a further neuroscience translational medicine role at Novartis.

"I am delighted to be joining Coave at such an exciting stage of development," said Dr Blaettler. "I look forward to progressing CTx-PDE6b through the clinic, and to contributing to the advancement of the company's pipeline of next-generation gene therapies, which have the potential to deliver life changing outcomes for rare disease patients."

Thomas completed his Doctor of Medicine at the University of Zurich in 1994 and went on to complete almost 10 years of clinical residency and research in the neurology field. Thomas gained board certification from the Swiss Society of Neurology in 2003.

Patricia Franon PhD

Dr Franon is an experienced biotech professional with over 20 years' experience leading global CMC and regulatory strategies for the accelerated development of innovative biologics, advanced cell & gene therapies. Patricia joins Coave from Skinosive where she served as Chief Operating/Technology Officer, managing operational aspects of the business, proactively driving the company towards achieving its development goals. Patricia has also held various clinical development roles at Sartorius, Neuro-Sys, Enterome, Evry, Cellectis, Anaconda Pharma and Sanofi, managing all aspects of product development, coordinating multiple studies, selecting partners and managing regulatory processes.

"Coave's ALIGATER technology is truly innovative and demonstrates an important ability to provide gene therapies with increased tissue targeting and transduction, designed to enhance their potency and efficacy," added Dr Franon. "I look forward to working with Rodolphe and the team to drive the company forward on its mission of improving the effectiveness of advanced gene therapies for rare diseases."

Patricia obtained her PhD in Molecular and Cellular Biology from Paris VI University and completed postdoctoral research at McGill University.

About Coave Therapeutics

Coave Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies in rare ocular and CNS (Central Nervous System) diseases.

Coave Therapeutics' next-generation AAV-Ligand Conjugate ('ALIGATER') platform enables targeted delivery and enhanced gene transduction to improve the effectiveness of advanced gene therapies for rare diseases.

The Company is advancing a pipeline of novel therapies targeting rare ocular and brain diseases where targeted gene therapy using AAV-Ligand has the potential to be most effective.

Coave Therapeutics, which is headquartered in Paris (France), is backed by leading international life science and strategic investors Seroba Life Sciences, Tha Open Innovation, eureKARE, Fund+, Omnes Capital, V-Bio Ventures, Kurma Partners, Idinvest, GO Capital, Sham Innovation Sant/Turenne.

For more information, please visit http://www.coavetx.com or follow us on LinkedIn http://www.linkedin.com/company/coavetx/

CONTACTS

Coave Therapeutics Rodolphe Clerval, CEO [emailprotected]

MEDiSTRAVA ConsultingSylvie Berrebi, Eleanor Perkin, Mark Swallow PhD[emailprotected] Tel: +44 (0)7714 306525

SOURCE Coave Therapeutics

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Coave Therapeutics Strengthens Leadership Team with the Appointments of Thomas Blaettler MD as Chief Medical Officer and Patricia Franon PhD as Chief...

Recommendation and review posted by Bethany Smith

8 medical advances you may have missed during COVID-19 – AAMC

November 21st, 2021

COVID-19 has been all-consuming. For nearly two years, the world has been focused on the race for vaccines, the pressures on providers, the best testing protocols, and simply staying safe.

COVID-19 also slowed some research efforts, but scientists still managed to seek solutions for many other pressing concerns Alzheimers disease, maternal mortality, and prostate cancer among them that have bedeviled patients for decades.

Below are eight medical advances that may not have grabbed your attention but could ultimately improve the lives of millions.

Assessing a stroke demands a rapid, life-or-death assessment: Is the culprit a clot, which requires a blood thinner, or bleeding in the brain, which requires surgery? Now, a portable MRI device can help make that assessment right at a patients bedside and in much less time than required by a trip to a standard machine.

The Swoop MRI which was created with input from Yale Medicine in New Haven, Connecticut received Food and Drug Administration (FDA) approval in August 2020 and is already at work in several U.S. hospitals.

The new portable machine offers many advantages over its massive cousin, says Yale neurologist Kevin Sheth, MD.

The very strong magnets in regular MRIs bring a lot of challenges, he explains. You need intensive power and cooling, precautions like a shielded room, and a lot of training. If you use a weaker magnet, all those problems go away.

The weaker magnet is effective, according to an August 2021 study, which asked clinicians to identify various cerebral pathologies using Swoop images. The goal is not to be as good as a high-magnet MRI, but to be good enough for clinical decisions, says Sheth, who co-authored the study but has no financial interest in Hyperfine, the Connecticut-based company that produces the machine.

Swoops size its smaller than some refrigerators eliminates the need to move frail patients down hospital hallways. Whats more, its cost around $100,000 compared to $1 million for the bigger machine puts it within reach of hospitals and regions with fewer resources. This could essentially democratize brain imaging, argues Sheth.

Prostate cancer strikes 1 out of 8 U.S. men, and it is expected to take more than 34,000 lives this year alone. When it metastasizes, the disease is almost always incurable, leaving physicians focused only on postponing death and improving patients lives.

A promising new approach has succeeded at both goals and did so among men with an advanced form of the disease whose condition had deteriorated despite receiving standard treatments.

In fact, it more than doubled how long patients lived without their cancer worsening, according to a paper published in September. The study, which followed 831 men in 10 countries for a median of 20 months, compared patients who continued to receive standard care with ones who got the new treatment.

The treatments name is complex: lutetium-177-PSMA-617. But its approach is straightforward: Drive radiation directly into a cancer cell while sparing healthy tissue around it.

The method uses a compound called PSMA-617 to hone in on a protein found almost exclusively in prostate cancer cells, explains Oliver Sartor, MD, study co-lead investigator and medical director of Tulane Cancer Center in New Orleans. Then, a radioactive particle carried by the compound blasts the cancer cells, wherever they are.

Its like a little smart bomb, says Sartor.

In September, the FDA granted the treatment priority review status, according to drug manufacturer Novartis, which funded the study. An answer is expected in the first half of 2022.

Sartor feels hopeful. Ive been working in prostate cancer for more than 30 years, and this is the largest advance Ive ever been associated with.

For more than 5,000 years, sickle cell disease (SCD) has caused untold suffering in people of African descent. In patients with the genetic illness, red blood cells are not round but crescent-shaped like a sickle and can clog blood vessels, depriving the body of oxygen and causing tremendous pain. For a long time, the only cure has been a bone marrow transplant, but new gene-editing techniques now may offer a safe and effective alternative.

In research conducted at Boston Childrens Hospital, scientists used a virus to switch off the gene that triggers cells sickling, according to a January 2021 study. The patients subsequently produced healthy red blood cells and nearly all were able to discontinue the blood transfusions SCD often requires.

One participant used to have transfusions every month but has not needed any in three years, says David Williams, MD, chief of the Division of Hematology/Oncology at Boston Childrens and head of the research team. This has completely changed his life.

The study followed six patients for a median of 18 months and found that the treatment completely halted the diseases more severe symptoms.

Im so happy for my sickle-cell patients. This is a terrible disease, notes Williams.

Next up for Williams is a trial with 25 patients. Meanwhile, SCD researchers elsewhere are studying other gene-editing techniques. All these approaches look promising, and we need a lot more research to determine if one or another is better, Williams says.

This is a very exciting time. In the past, we havent had any particularly good treatments, and now we have several possibilities," he adds.

When a womans uterus fails to contract after childbirth, tremendous blood loss can ensue, possibly leading to an emergency hysterectomy or even death. In fact, postpartum hemorrhage affects 3% to 10% of all childbirths in the United States and causes more than one-third of childbirth-related maternal deaths worldwide.

Treatment options include medications that dont always work and inserting a balloon to put pressure on the uterus much like exerting pressure on a cut that comes with risks and must remain in place for a day.

But providers now have another option.

A new vacuum device aids natural post-birth contractions, putting pressure on leaking blood vessels. The FDA approved the device the Jada vacuum uterine tamponade in September 2020 following a 12-site research study.

The vacuum approach is very logical since its like what the body is supposed to do, says Dena Goffman, MD, the primary investigator at Columbia University Irving Medical Center in Manhattan. Also, the vacuum is used for less time than the balloon roughly two or three hours. For moms, thats a big deal because it makes it easier to breastfeed, get out of bed, and bond with their child, she adds.

The vacuum controlled bleeding in a median of three minutes and successfully treated 94% of participants, according to the study, which was funded by the devices manufacturer, Alydia Health. In comparison, other research puts the balloons effectiveness at 87%.

When a patient has a postpartum hemorrhage and youre the doctor at the bedside, its scary because you know how quickly things can deteriorate, says Goffman. Using this device, when you see the bleeding slowing quickly and you can feel the uterus contracting, its just incredible.

Tearing an anterior cruciate ligament (ACL) the flexible band inside the knee that helps stabilize it can upend a sports career and sideline weekend athletes. Between 100,000 and 200,000 ACL tears occur each year in the United States.

The most effective repair option has been removing the ruptured ACL, harvesting a graft from the shin or elsewhere, sewing that tissue into the knee, and hoping both surgical sites heal well.

In December 2020, the FDA approved a simpler, more natural method: the Bridge-Enhanced ACL Restoration (BEAR).

We basically stimulate the ACL to heal itself, says Martha Murray, MD, orthopedic surgeon-in-chief at Boston Childrens Hospital and BEARs creator.

The approach involves placing a protein-based sponge, prepared with some of the patients own blood, between the torn ACL ends. Murray explains that the blood promotes the connection of the two ACL pieces to the sponge and, ultimately, to each other.

So far, the approach has been tested on more than 100 patients. In a May 2020 study, patients and physicians reported that BEAR performed as well as the standard repair and without the graft surgery that can cause ongoing pain or weakness at the donor site. Miach Orthopaedics, which has the worldwide exclusive license for the BEAR implant, has already begun making it available through orthopedic surgeons in the United States.

For Murray, the experience has highlighted the value of serving as a physician-researcher. When youre faced with a patient with a problem and the current solution is imperfect, its great to be able to say, Were working on a better solution. Its incredibly gratifying.

For the first time since 2014, a new obesity medication has hit the market, offering hope to the 78 million Americans who face the many risks of excess weight: cancer, heart disease, diabetes, and complications from COVID-19, among others.

And the new medication semaglutide, also known as Wegovy is significantly more powerful than its predecessors, according to research that helped it garner approval from the FDA in June.

Weve seen 1 to 2 times the amount of weight loss compared to other medications, says Robert Kushner, MD, a researcher at Northwestern University Feinberg School of Medicine who has led semaglutide studies. That's a leapfrog advance.

In fact, semaglutide recipients lost nearly 15% of their body weight on average compared with 2.4% among controls, according to one study of nearly 2,000 patients.

Semaglutide an injectable medication is not entirely new. A synthetic version of a natural hormone that quells appetite, its already used to treat Type 2 diabetes. But the obesity trials, paid for by pharmaceutical company Novo Nordisk, used a much higher dose.

High doses havent been studied long enough to identify long-term side effects, notes Kushner, a paid consultant to Novo Nordisk. But the recent research reported mild-to-moderate gastrointestinal issues that lessened over time.

Now Kushner hopes semaglutide will help spark interest in obesity medications.

Over 40% of U.S. adults have obesity, and the number who are getting a pharmacologic treatment is under 3%, he says. Part of the challenge is educating primary care providers that providing evidence-based obesity care includes consideration of medication."

Randall Bateman, MD, a Washington University School of Medicine in St. Louis (WUSTL) neurologist, is thrilled to have contributed to the first blood test for Alzheimer's disease a devastating condition that affects as many as 5.8 million Americans.

Back in 2017, though, as Bateman geared up to share the discovery that would enable the test, he worried about his peers reaction. After all, scientists were convinced that the blood marker he studied couldnt predict the disease.

But the WUSTL method was much more sensitive and direct than prior approaches. The resultant test called PrecivityAD effectively detects the amyloid plaques that are a hallmark of Alzheimers disease and has proven as accurate as the previously used tools of a spinal tap or positron emission tomography (PET) scan, which are far more costly and complex.

The test, developed by a company called C2N Diagnostics that Bateman co-founded, has been available to physicians since October 2020, when it received approval through a federal lab certification program. It now awaits additional approval from the FDA.

Weve been hoping for a test to diagnose Alzheimers for more than 20 years, says Bateman, WUSTLs Charles F. and Joanne Knight distinguished professor of neurology. Currently, up to half of people with Alzheimers are misdiagnosed.

The road to success in science is paved with hard work and great uncertainty, he adds. Its a real gamble. Youre investing your life in this work, and you hope it will have a positive impact. And then its like, Wow, it worked!

Anger, fear, recurring nightmares, and intense flashbacks are among the many symptoms that can batter patients with post-traumatic stress disorder (PTSD). The condition, which affects about 15 million U.S. adults in a given year, can be extremely difficult to treat.

A potentially groundbreaking PTSD treatment now lies in a seemingly unlikely source: MDMA, better known as the illegal drugs ecstasy and molly that fueled all-night dance raves and caused potentially fatal side effects.

In June, a study in Nature Medicine reported that patients with severe PTSD combat veterans, first responders, and victims of sexual assault and mass shootings, among others experienced significant relief from MDMA.

In fact, two months after treatment, 67% of subjects who received MDMA together with talk therapy no longer qualified for a diagnosis of PTSD. I saw this amazing transformation in patients, says Jennifer Mitchell, PhD, the studys lead author and a University of California, San Francisco, School of Medicine neurology professor.

The treatment involved three eight-hour sessions a month apart during which patients ingested MDMA and processed painful memories and emotions in talk therapy.

MDMA releases a powerful supply of serotonin and stimulates hormones associated with emotional bonding, Mitchell explains. The idea is that it helps patients be open in a way that enables them to connect well with therapists and work through their problems more quickly.

Before the drug can receive FDA approval for PTSD, researchers need to complete one more clinical trial. Even if it succeeds, Mitchell is aware that MDMA still bears stigma from its party drug image.

I hope people are going to be open-minded and look at the data, which included no abuse potential or other serious side effects from MDMA as used in the study. We are talking about use in a controlled, therapeutic situation, she says. Using drugs recreationally is entirely different. Otherwise, people would come back from [the art and community event] Burning Man cured of their psychological issues.

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8 medical advances you may have missed during COVID-19 - AAMC

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Hiltzik: The battle against unlicensed stem cell clinics – Los Angeles Times

November 7th, 2021

In 2017, the Food and Drug Administration closed a loophole exploited by clinics pitching unproven, ineffective and potentially hazardous stem cell therapies directly to consumers.

Those treatments were illegal, the FDA ruled. That was the good news. The agency, however, suspended its enforcement for three years to give these operators time to get right with its regulations. During the pandemic, the FDA added six months to the deadline, so its period of regulatory forbearance expired on May 31.

What happened in the meantime? Instead of stem cell purveyors reaching out to the FDA to work out how to meet federal regulations, a torrent of shady operations poured into the field so many that the task of protecting the public from them may now exceed the FDAs capabilities.

Dont believe the hype.

The FDAs warnings against unlicensed stem cell clinics

Thats the concern of Leigh Turner, a public health expert at UC Irvine, longtime critic of stem cell treatment claims and author of a new study that tracked the explosion of businesses offering purported stem cell treatments and cures during the FDAs hands-off period.

The paper is essentially a follow-up to a seminal study Turner conducted with Paul Knoepfler of UC Davis in 2016, which identified 351 businesses hawking stem cell treatments directly to consumers through 540 clinics.

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Turners new study, which was published Thursday in the peer-reviewed journal Cell Stem Cell, identified 1,480 businesses operating 2,754 clinics nationwide.

That hardly seems like progress, Turner told me. Now the problem the FDA faces is four times larger than what existed in 2016. The FDA only has so many employees and so many inspectors. They dont really have enough inspectors to send them to 1,480 businesses.

The FDA hasnt been entirely inactive. Over the last 3 years it issued more than 400 warning letters to stem cell marketers, clinics and healthcare providers, notifying them that they may be operating outside the law. But it hasnt done much more than that.

The proliferation of stem cell claims points to a major breakdown in Americas healthcare regulatory system generally.

Given the size of this clinic industry the risk to the public is huge, Knoepfler says. The industry threatens the authority of the FDA itself. It might be one of the largest and most serious noncompliance challenges that the FDA has ever faced in its history. What would the FDA do if pharmaceutical firms were selling unapproved drugs at 2,700 clinics all over the country? It would be considered a national emergency.

State medical boards, which have at least nominal authority to ride herd on the practices of licensed physicians in this field, have done little of note. The Medical Board of California, a state that with 347 clinics is the largest center of facilities in Turners database, created a two-member Stem Cell and Regenerative Therapy Task Force in 2018.

The task force hasnt met since 2019, according to a board spokesman, and hasnt issued any reports. Board records indicate that it received 33 complaints about stem cell treatments from 2018 through this year, and has taken no disciplinary or administrative actions in response.

California hasnt done much to rise to the challenge, Turner says.

A few words may be useful about the regulatory environment. The chief targets of the FDAs enforcement program, such as it is, have been clinics that extract fat cells from customers through liposuction and then supposedly extract stem cells from the fat and inject them back into the customers bodies as treatments. Some purportedly extract stem cells from customers bone marrow.

The conditions for which these treatments are commonly offered include pain, sports injuries, heart and lung disease, multiple sclerosis, Parkinsons, Alzheimers, autism, diabetes, vision loss and erectile dysfunction.

No scientifically validated evidence exists for any of these claims, the FDA notes. The only stem cell products approved by the FDA are a few derived from umbilical cord blood, and then only for a very limited category of blood system diseases.

Beyond that, the agency advises consumers, Dont believe the hype.

The purveyors of unproven and unlicensed treatments identified by Turner charge as much as $28,000 for their services, with an average of more than $5,000 often ponied up by unwary customers seduced by advertising and irresponsibly credulous reports in the press.

The treatments typically are not covered by insurance, so customers are paying out of pocket.

Under FDA regulations, most stem cell treatments being sold to customers are illegal. Thats because the products are deemed to be unlicensed drugs. Exceptions exist for some surgical procedures and in cases in which almost identical cells are reinjected into patients, but the FDA says few of the targeted clinics qualify.

The FDAs position was endorsed by a federal judge in Miami in 2019, when she shut down a clinic that the FDA had sued for offering unapproved stem cell procedures. The clinic lost an appeal of her ruling in June. A separate lawsuit the FDA filed against California Stem Cell Treatment Center and associated businesses is awaiting a verdict from U.S. Judge Jesus G. Bernal in Riverside following a trial he conducted in May.

The offered treatments are not only unproven, but potentially hazardous. Reports of adverse outcomes from unlicensed treatments have proliferated, some of them gruesome. In a report issued in June, researchers at the Pew Charitable Trusts documented reports of adverse outcomes from 360 patients between 2004 and mid-2020.

These figures are almost certainly conservative, as clinics operating outside the law are highly unlikely to follow rules mandating that they report adverse reactions among their customers. Indeed, the FDA in its lawsuit against the California defendants asserted that numerous adverse outcomes experienced by their patients were not reported to the agency.

A note cited by the FDA from the file of one patient who was unable to walk for six months after receiving a stem cell injection in her knee from a clinic associated with the defendants, read, Not all treatments are successful. Not really adverse event due to the treatment. At trial, defendant Mark Berman said the clinics product has had very rare adverse events.

FDA officials have said that their expectations that stem cell treatment purveyors would engage with the agency during the forbearance period to work out how to come into compliance with its regulations proved wildly optimistic. We have been very disappointed in the number of clinics that have come in, Wilson Bryan, a top FDA official, told a law conference in June.

The FDA may not have recognized that the clinics they were targeting never had any intention of meeting its regulations.

Were talking about a huge number of businesses that are failing to comply with federal law, but have no reasonable prospects of coming into compliance, Turner says. They dont have clinical research programs. They dont have qualified stem cell researchers. Theyre just peddlers putting out a shingle on the internet.

Instead of using that three-year period to change their practices and comply with the law, lots of businesses stayed in the marketplace and did nothing to change what they were doing, and a huge number of other operators poured into the marketplace.

Over the last five years or so, or since Turner and Knoepfler published their original report, stem cell treatment claims have become just one more offering by practitioners advertising other services of dubious effectiveness.

Stem cell treatment has become just a routine claim, Turner says, from all kinds of orthopedic clinics, sports medicine clinics, podiatrists, chiropractors, naturopaths, wellness clinics. They market an array of services and stem cells are just something else they can sell, like cryotherapy or acupuncture.

Its unclear whether some of these operations really even use stem cells some may be offering just costly placebos, Turner conjectures.

The threat to public health from unlicensed and unproven stem cell claims will only get worse if the FDA fails to act forcefully.

People make the mistake of thinking that these are businesses that will go under if they get a bit of a push from the FDA or FTC [Federal Trade Commission] Turner says. The truth is that some of them are quite well capitalized and have the resources to go out and join these battles.

The FDA may be waiting for Judge Bernals ruling before taking the next step but thats dependent on his finding in the FDAs favor. It could seek mass injunctions, admittedly a big practical challenge, Knoepfler says. He adds, Looking ahead, the agency should take quick, large-scale, and even creative actions if it has any hope to make a dent in this clinic industry.

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Hiltzik: The battle against unlicensed stem cell clinics - Los Angeles Times

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Cell therapy biotech PlateletBio reels in $75M as it looks ahead to first clinical test – MedCity News

November 7th, 2021

PlateletBio, a company developing a new class of cell therapies based on the biology of platelets, has raised $75.5 million to advance its drug pipeline, including a lead candidate for a rare bleeding disorder on track to reach the clinic next year.

Platelets are components of blood best known for their role forming clots that stop bleeding. But Watertown, Massachusetts-based PlateletBio notes that platelets have other properties, including a role delivering growth factors and proteins throughout the body. PlateletBio is developing therapies that take advantage of these properties, but rather than using platelets from a patient or healthy donors, the startup makes them.

In the body, platelets are formed in bone marrow. PlateletBio produces its platelet-like cells, or PLCs, inside a bioreactor that mimics bone marrow conditions. The source material for its PLCs are stem cells, which have the ability to become almost any cell or tissue in the body.

Platelets are technically not cells. They dont have a nucleus, but thats an advantage for therapeutic applications. Since a PlateletBio therapy wont introduce DNA into a patients body, the potential risks that come from introducing foreign genetic material are avoided. PlateletBio says it can produce PLCs with new features and therapeutic payloads that include antibodies, signaling proteins, therapeutic proteins, and nucleic acids.

PlateletBios lead cell therapy candidate is being developed to treat immune thrombocytopenia, a blood disorder in which the immune system mistakenly sees a patients platelets as foreign and destroys them. Immune thrombocytopenia patients have dangerously low platelet counts that make them susceptible to bleeding.

There is no FDA-approved treatment for the underlying cause of immune thrombocytopenia, but corticosteroids are used to try to dampen the immune systems attack on platelets. Platelet transfusions are another option, but the National Organization for Rare Disorders notes that these treatments are usually reserved for emergencies because the platelets are likely to be destroyed by antibodies produced by the patient.

Patients who have not responded to earlier treatments have two FDA-approved small molecule options: Tavalisse, from Rigel Pharmaceuticals, and the Swedish Orphan Biovitrum drug Doptelet. Sanofi aims to treat the disease with a small molecule called rilzabrutinib. That drug is designed to block Brutons tyrosine kinase, a protein that plays a role in the development of a B cells, a type of immune cell. Sanofi acquired the molecule last year via its $3.7 billion acquisition of Principia Biopharma.

The lead disease target for the Principia drug was multiple sclerosis. In September, Sanofi reported that rilzabrutinib failed that Phase 3 study. A separate Phase 3 test in immune thrombocytopenia is ongoing, as is a mid-stage clinical trial in another autoimmune condition called IgG4-related disease.

PlateletBio isnt the only company trying to turn a component of the blood into a new type of cell therapy. Cambridge, Massachusetts-based Rubius Therapeutics is developing cell therapies based on red blood cells. After disappointing early clinical trial results in the rare disease phenylketonuria last year, Rubius shifted its focus to cancer and immune system disorders. PlateletBios PLCs would represent an entirely new approach to treating immune thrombocytopenia. According to PlateletBios website, the company plans to file an investigational new drug application for its therapeutic candidate in the first half of next year.

PlateletBio is based on the research of Harvard University scientist Joseph Italiano, who co-founded the company under the name Platelet BioGenesis. When the startup emerged in 2017, it was developing platelets that could address the platelet shortage problem facing blood donation centers. Two years ago, the startup expanded its Series A round with $26 million in additional financing and plans to make its platelets into cell therapies. Besides immune thrombocytopenia, other diseases the biotech aims to treat include osteoarthritis and liver fibrosis.

PlateletBios latest financing, a Series B round, adds new investors SymBiosis, K2 HealthVentures, and Oxford Finance. Earlier investors Ziff Capital Partners and Qiming Venture Partners also participated in the new round.

This is a major milestone for PlateletBio, adding capital and resources needed to advance our innovative platelet-like cell therapy science and manufacturing platform and support key corporate initiatives over the next 18 to 24 months, Sam Rasty, the startups president and CEO, said in a prepared statement.

Photo by Flickr user Marco Verch via a Creative Commons license

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Cell therapy biotech PlateletBio reels in $75M as it looks ahead to first clinical test - MedCity News

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BioLineRx Announces an Oral Presentation and Three Poster Presentations at the 63rd American Society of Hematology (ASH) Annual Meeting &…

November 7th, 2021

TEL AVIV, Israel, Nov. 4, 2021 /PRNewswire/ --BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical Company focused on oncology, today announced an oral presentation and three poster presentations at the 63rd American Society of Hematology (ASH) Annual Meeting & Exposition, which is being held December 11-14, 2021 in Atlanta, GA, and virtually.

The oral presentation will elaborate on the successful results of the Company's GENESIS Phase 3 pivotal trial. The study showed highly significant and clinically meaningful results supporting the use of Motixafortide on top of G-CSF for mobilization of stem cells for subsequent collection and transplantation in patients with multiple myeloma. In addition, the poster presentations will show that extended inhibition of the CXCR4 receptor by Motixafortide results in the mobilization of high numbers of stem cells, including specific sub-populations, which were correlated with reduced time to engraftment when infused in high numbers.

The Company is also presenting findings from in-vivo and in-vitro pre-clinical studies demonstrating that Motixafortide acts as an immunomodulator by affecting the biology of regulatory T cells (Tregs), supporting biomarker findings from the Company's COMBAT Phase 2 study in pancreatic cancer patients.

"We are very pleased with the breadth of our oral and poster presentations at this year's ASH meeting, which reflect the versatility of Motixafortide as the potential backbone of promising new treatments for both hematological and solid tumor cancers," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Of particular note is the oral presentation on the outstanding results from our GENESIS Phase 3 pivotal study in stem cell mobilization demonstrating that Motixafortide effectively mobilizes a high number of cells enabling ~90% of patients to undergo transplantation following a single administration of Motixafortide and a single apheresis session. In addition, the high number of cells mobilized by Motixafortide enables infusion of an optimal number of cells, which could result in faster time to engraftment, and also allows for cryopreservation for future transplantation(s). These results, together with our recently completed successful pharmacoeconomic study, strongly support our view that Motixafortide on top of G-CSF can become the new standard of care in SCM, if approved, to the benefit of patients and payers alike. We look forward to submitting an NDA in the first half of next year, as previously communicated."

Further details of the presentations are provided below.

Oral Presentation

Title: Motixafortide (BL-8040) and G-CSF Versus Placebo and G-CSF to Mobilize Hematopoietic Stem Cells for Autologous Stem Cell Transplantation in Patients with Multiple Myeloma: The GENESIS Trial

Date: Sunday, December 12, 2021

Time: 12:00 PM

Location: Georgia World Congress Center, Hall A1

This oral presentation describes the GENESIS Phase 3 pivotal trial design, endpoints and results. The GENESIS study was a double blind, placebo controlled, multicenter trial, in which 122 patients were randomized (2:1) to receive either Motixafortide + G-CSF or placebo + G-CSF for stem cell mobilization prior to stem cell transplant in multiple myeloma patients. Total CD34+ cells/kg were analyzed on site to determine if patients mobilized to the goal and all samples were subsequently sent for assessment by a central laboratory. The number of CD34+ cells infused was determined independently by each investigator according to local practice.

The study concluded that a single administration of Motixafortide on top of G-CSF significantly increased the proportion of patients mobilizing 6x106 CD34+ cells/kg for stem cell transplantation (92.5%) vs G-CSF alone (26.2%) in up to two apheresis days (p<0.0001), while enabling 88.8% to collect 6x106 CD34+ cells/kg in just one apheresis day (vs 9.5% with G-CSF alone; p<0.0001). In addition, the median number of hematopoietic stem cells mobilized in one apheresis day with Motixafortide + G-CSF was 10.8x106 CD34+cells/kg vs 2.1x106 CD34+ cells/kg with G-CSF alone.

Poster Presentations

Title:Autologous Hematopoietic Cell Transplantation with Higher Doses of CD34+ Cells and Specific CD34+ Subsets Mobilized with Motixafortide and/or G-CSF is Associated with Rapid Engraftment A Post-hoc Analysis of the GENESIS Trial

Date: Sunday, December 12, 2021

Time: 6:00 PM - 8:00 PM

The CD34+ hematopoietic stem and progenitor cell (HSPC) dose infused during stem cell transplantation remains one of the most reliable clinical parameters to predict quality of engraftment. A minimum stem cell dose of 2-2.5x106 CD34+ cells/kg is considered necessary for reliable engraftment, while optimal doses of 5-6x106 CD34+ cells/kg are associated with faster engraftment, as well as fewer transfusions, infections, and antibiotic days.

An analysis was performed using pooled data from all patients in the GENESIS trial to evaluate time to engraftment based on the total number of CD34+ cells/kg infused, as well as specific numbers of CD34+ cell sub-populations infused.

The addition of Motixafortide to G-CSF enabled significantly more CD34+ cells to be collected in one apheresis (median 10.8x106 CD34+ cells/kg) compared to G-CSF alone (2.1x106 CD34+ cells/kg), as well as 3.5-5.6 fold higher numbers of hematopoietic stem cells (HSCs), multipotent progenitors (MPPs), common myeloid progenitors (CMPs) and granulocyte and macrophage progenitors (GMPs) (all p-values <0.0004). A dose response was observed with a significant correlation between faster time to engraftment and infusion of higher number of total CD34+ HSPC doses (6x106 CD34+ cells/kg) and combined HSC, MPP, CMP and GMP subsets. The high number of CD34+ cells/kg mobilized with Motixafortide on top of G-CSF enables the potential infusion of 6x106 CD34+ cells/kg, as well as cryopreservation of cells for later use.

Title: Immunophenotypic and Single-Cell Transcriptional Profiling of CD34+ Hematopoietic Stem and Progenitor Cells Mobilized with Motixafortide (BL-8040) and G-CSF Versus Plerixafor and GCSF Versus Placebo and G-CSF: A Correlative Study of the GENESIS Trial

Date: Monday, December 13, 2021

Time: 6:00 PM - 8:00 PM

CD34 expression remains the most common immunophenotypic cell surface marker defining human hematopoietic stem and progenitor cells (HSPCs). The addition of CXCR4 inhibitors to G-CSF has increased mobilization of CD34+ HSPCs for stem cell transplantation; yet the effect of CXCR4 inhibition, with or without G-CSF, on mobilization of specific immunophenotypic and transcriptional CD34+ HSPC subsets is not well-characterized.

Motixafortide is a novel cyclic peptide CXCR4 inhibitor with a low receptor-off rate and extended in vivo action when compared to plerixafor. GENESIS Phase 3 trial patients were prospectively randomized (2:1) to receive either Motixafortide + G-CSF or placebo + G-CSF for HSPC mobilization. Demographically similar multiple myeloma patients undergoing mobilization with plerixafor + G-CSF prior to stem cell transplant were prospectively enrolled in a separate tissue banking protocol.

Extended CXCR4 inhibition with Motixafortide + G-CSF mobilized significantly higher numbers of combined CD34+ HSCs, MPPs and CMPs compared to plerixafor + G-CSF or G-CSF alone (p<0.05). Additionally, Motixafortide + G-CSF mobilized a 10.5 fold higher number of immunophenotypically primitive CD34+ HSCs capable of broad multilineage hematopoietic reconstitution compared to G-CSF alone (p<0.0001) and similar numbers compared to plerixafor + G-CSF. Furthermore, lack of CXCR4 inhibition resulted in mobilization of more-differentiated HCSs, whereas extended CXCR4 inhibition with Motixafortide + G-CSF (but not plerixafor + G-CSF) mobilized a unique MPP-III subset expressing genes specifically related to leukocyte differentiation.

Title: The High Affinity CXCR4 Inhibitor, BL-8040, Impairs the Infiltration, Migration, Viability and Differentiation of Regulatory T Cells

Date: Sunday, December 12, 2021

Time: 6:00 PM - 8:00 PM

This poster describes results of pre-clinical in-vivo and in-vitro studies demonstrating that Motixafortide potentially acts as an immunomodulator by affecting the biology of regulatory T cells. Motixafortide reduced the amount of infiltrating Tregs into the tumors, impaired the migration of Tregs toward CXCL12 and induced Tregs cell death. Furthermore, Motixafortide was found to inhibit the differentiation of nave CD4 T cells toward Tregs.

About BioLineRx

BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a late clinical-stage biopharmaceutical company focused on oncology. The Company's business model is to in-license novel compounds, develop them through clinical stages, and then partner with pharmaceutical companies for further clinical development and/or commercialization.

The Company's lead program, Motixafortide (BL-8040), is a cancer therapy platform that was successfully evaluated in a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation, has reported positive results from a pre-planned pharmacoeconomic study, and is currently in preparations for an NDA submission. Motixafortide was also successfully evaluated in a Phase 2a study for the treatment of pancreatic cancer in combination with KEYTRUDA and chemotherapy under a clinical trial collaboration agreement with MSD (BioLineRx owns all rights to Motixafortide), and is currently being studied in combination with LIBTAYO and chemotherapy as a first-line PDAC therapy.

BioLineRx is also developing a second oncology program, AGI-134, an immunotherapy treatment for multiple solid tumors that is currently being investigated in a Phase 1/2a study.

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Factors that could cause BioLineRx's actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: the initiation, timing, progress and results of BioLineRx's preclinical studies, clinical trials and other therapeutic candidate development efforts; BioLineRx's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; BioLineRx's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of BioLineRx's therapeutic candidates; BioLineRx's ability to establish and maintain corporate collaborations; BioLineRx's ability to integrate new therapeutic candidates and new personnel; the interpretation of the properties and characteristics of BioLineRx's therapeutic candidates and of the results obtained with its therapeutic candidates in preclinical studies or clinical trials; the implementation of BioLineRx's business model and strategic plans for its business and therapeutic candidates; the scope of protection BioLineRx is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; estimates of BioLineRx's expenses, future revenues, capital requirements and its needs for additional financing; risks related to changes in healthcare laws, rules and regulations in the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; risks related to the COVID-19 pandemic; and statements as to the impact of the political and security situation in Israel on BioLineRx's business. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on February 23, 2021. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contact:

Tim McCarthyLifeSci Advisors, LLC+1-212-915-2564[emailprotected]

or

Moran MeirLifeSci Advisors, LLC+972-54-476-4945[emailprotected]

SOURCE BioLineRx Ltd.

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BioLineRx Announces an Oral Presentation and Three Poster Presentations at the 63rd American Society of Hematology (ASH) Annual Meeting &...

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Magenta Therapeutics Announces Data Presentations Related to its Mobilization and Conditioning Programs at the 2021 American Society of Hematology…

November 7th, 2021

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines designed to bring the curative power of stem cell transplants to more patients, today announced positive top-line results from an investigator-initiated Phase 2 clinical trial of MGTA-145 stem cell mobilization in multiple myeloma. The data were accepted for a poster presentation at the 2021 American Society of Hematology (ASH) Annual Meeting, to be held in Atlanta and virtually from December 11-14, 2021. Oral and poster presentations of preclinical data related to the companys CD117 targeted conditioning program will also be made at the ASH Annual Meeting.

We have made significant progress with our mobilization and targeted conditioning programs and we look forward to the presentation of the data that have been generated to support both programs, said Jason Gardner, D.Phil., President and Chief Executive Officer, Magenta Therapeutics.

Stem Cell Mobilization and Collection Program (MGTA-145)

Poster Presentation Highlighting Investigator-Initiated Phase 2 Clinical Data of MGTA-145 Stem Cell Mobilization in Multiple Myeloma:

Title: MGTA-145 + Plerixafor Provides G-CSF-Free Rapid and Reliable Hematopoietic Stem Cell Mobilization for Autologous Stem Cell Transplant in Patients with Multiple Myeloma: A Phase 2 Study (Poster #3888)

Date and Time to View Poster Presentation: Monday, December 13, 2021, 6:00pm 8:00pm ET

Trial Design

Surbhi Sidana, M.D., Assistant Professor of Medicine in the Division of Blood and Marrow Transplantation and Cellular Therapy at Stanford University School of Medicine led this investigator-initiated, Phase 2 open-label clinical trial. The trial evaluated the ability of MGTA-145, in combination with plerixafor, to mobilize stem cells for autologous stem cell transplantation in patients with multiple myeloma. This trial had broad inclusion criteria and included the transplant-eligible population of patients with multiple myeloma who may have a variety of risk factors for mobilization.

Topline Clinical Data

Next Steps in Multiple Myeloma

As described in the companys third quarter earnings release, the results from this investigator-initiated trial represent a positive step forward in the development of MGTA-145, in combination with plerixafor, as a potential first line stem cell mobilization regimen. Based on the encouraging collection and engraftment data, the company intends to explore further development of MGTA-145 in a Phase 2b clinical setting. This approach would enable a comprehensive evaluation of the multiple myeloma patient population and allow for adjustments of dosing and administration which the company, in both cases, has identified as opportunities for optimization as a result of this investigator-initiated study and the companys other MGTA-145 development efforts.

While Dr. Sidana and her team are collecting and analyzing additional patient-level data, these topline results are encouraging and support further development of MGTA-145. commented Dr. Jeffrey Humphrey, M.D., the companys Chief Medical Officer. We believe this novel mobilization regimen has the potential to replace G-CSF regimens and to enable reliable, predictable, rapid and well-tolerated mobilization of stem cells for both transplant and gene therapies.

MGTA-145 is also being evaluated for its ability to mobilize stem cells for collection from donors for allogenic transplantation in patients with acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) in a Phase 2 clinical trial. The company is planning to open an additional Phase 2 clinical trial for mobilization and collection of stem cells for patients with sickle cell disease in December 2021.

Antibody-Drug Conjugate (ADC) Targeted Conditioning Program

Oral Presentation Showcasing Non-human Primate Data of Targeted ADC Conditioning for Gene Therapy

Title: CD117 Antibody Drug Conjugate-Based Conditioning Allows for Efficient Engraftment of Gene-Modified CD34+ Cells in a Rhesus Gene Therapy Model (Oral Abstract #560)

Presenting Author: Naoya Uchida, M.D., National Institutes of Health

Date: Sunday, December 12, 2021, 4:45pm ET

This preclinical study evaluated escalating doses of a tool CD117-ADC. As monotherapy conditioning, a single dose of the CD117-ADC allowed for efficient engraftment of gene-modified autologous stem cells in a rhesus model of gene therapy, without chemotherapy or radiation conditioning. Engraftment of gene-modified stem cells achieved with monotherapy CD117-ADC was robust and durable, equivalent to that achieved with four doses of myeloablative busulfan conditioning. Sustained gene expression of hemoglobin F was confirmed at the protein level in this CD117-ADC-conditioned rhesus transplant model of gene therapy for sickle cell disease. Compared to chemotherapy or radiation-based conditioning regimens, conditioning with monotherapy CD117-ADC could be both sufficiently potent and well tolerated to improve the safety and risk benefit profile for gene therapies that require stem cell transplantation.

Poster Presentation Highlighting Preclinical Data of Targeted ADC Conditioning Program:

Title: CD117-Targeted ADC, in Combination with Lymphodepleting Antibodies, Enables Allogeneic Hematopoietic Stem Cell Transplantation in Mice without Chemotherapy or Radiation (Poster #1682)

Presenting Author: Leanne Lanieri, M.S., Magenta Therapeutics, Inc.

Date to View Poster Presentation: Saturday, December 11, 2021, 5:30pm 7:30pm ET

This study evaluated the combination of a tool CD117-ADC with lymphodepleting antibodies as the conditioning regimen in a murine model of allogeneic HSC transplantation. The targeted conditioning regimen enabled complete donor chimerism in a fully mismatched allogeneic HSC transplant murine model, without use of chemotherapy or radiation. Antibody-based targeted conditioning regimens could offer a more favorable risk-benefit profile over chemotherapy and radiation-based conditioning regimens. An improved risk benefit profile, in turn, could extend the curative potential of allogeneic HSC transplantation to more patients with malignant and non-malignant diseases who otherwise would not be eligible for HSC transplantation.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines designed to bring the curative power of stem cell transplants to more patients with blood cancers, genetic diseases and autoimmune diseases. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise and broad networks in the stem cell transplant community to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Massachusetts. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statements

This press release may contain forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Magentas future expectations, plans and prospects, including, without limitation, statements regarding expectations and plans for presenting pre-clinical and clinical data, the initiation of clinical trials or the results of ongoing and planned clinical trials, the development of product candidates and advancement of preclinical programs, projections regarding future revenues and financing performance, long-term growth, cash, cash equivalents and marketable securities, the anticipated timing of clinical trials and regulatory filings, the potential benefits of product candidates, the timing, progress and success of collaborations, as well as other statements containing the words anticipate, believe, continue, could, endeavor, estimate, expect, intend, may, might, plan, potential, predict, project, seek, should, target, will or would and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical studies and in the availability and timing of data from ongoing clinical studies; whether interim results from a clinical trial will be predictive of the final results of the trial; whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials; the expected timing of submissions for regulatory approval or review by governmental authorities; discussions with governmental agencies such as the FDA; regulatory approvals to conduct trials or to market products; whether Magenta's cash resources will be sufficient to fund Magenta's foreseeable and unforeseeable operating expenses and capital expenditure requirements; risks, uncertainties and assumptions regarding the impact of the continuing COVID-19 pandemic on Magentas business, operations, strategy, goals and anticipated timelines, Magentas ongoing and planned preclinical activities, Magentas ability to initiate, enroll, conduct or complete ongoing and planned clinical trials, Magentas timelines for regulatory submissions and Magentas financial position; and other risks concerning Magenta's programs and operations are described in additional detail in its Annual Report on Form 10-K filed on March 3, 2021, as updated by Magentas most recent Quarterly Report on Form 10-Q, and its other filings made with the Securities and Exchange Commission from time to time. Although Magenta's forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Magenta. As a result, you are cautioned not to rely on these forward-looking statements. Any forward-looking statement made in this press release speaks only as of the date on which it is made. Magenta undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise.

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Magenta Therapeutics Announces Data Presentations Related to its Mobilization and Conditioning Programs at the 2021 American Society of Hematology...

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City of Hope Doctors Present New Research on Cancer Immunotherapies – Business Wire

November 7th, 2021

DUARTE, Calif.--(BUSINESS WIRE)--City of Hope announced today that it will present new research on bispecific antibodies at a press briefing during the ASH 63rd Annual Meeting and Exposition on Dec. 11 to 14 in Atlanta.

Other innovative City of Hope research on stem cell transplants and blood cancer treatments will also be presented during the conference organized by ASH. ASH is the world's largest professional society for clinicians and scientists around the world who are working on blood cancers and other hematological diseases.

City of Hope is finding new treatments for some of the hardest to treat cancers by accelerating innovative clinical research and therapies. The comprehensive cancer centers bone marrow transplant program is one of the largest and most successful in the nation and its chimeric antigen receptor (CAR) T cell therapy program is also focused on finding new therapies. City of Hope is leading other innovative immunotherapy treatments for blood cancers.

Elizabeth Budde, M.D, Ph.D., associate professor, City of Hope Division of Lymphoma, Department of Hematology & Hematopoietic Cell Transplantation, will present research on a bispecific antibody mosunetuzumab for relapsed/refractory follicular lymphoma at an ASH press briefing titled Immune System 1, Cancer 0: Advances in Immunotherapy on Saturday, Dec. 11 at 8:30 a.m. ET Follicular lymphoma is associated with frequent relapses and decreasing progression-free intervals with successive lines of conventional therapy. Later-line treatments may be less effective due to refractory disease. Mosunetuzumab is a CD20xCD3 bispecific antibody that redirects T cells to eliminate malignant B cells. In the dose-escalation phase of an ongoing Phase I/II study (NCT02500407), Mosunetuzumab was highly active and well tolerated in R/R FL patients (pts).

Dr. Budde will also present the research at a plenary session.

Title: Mosunetuzumab Monotherapy Is an Effective and Well-Tolerated Treatment Option for Patients with Relapsed/Refractory (R/R) Follicular Lymphoma (FL) Who Have Received 2 Prior Lines of Therapy: Pivotal Results from a Phase I/II Study Publication Number: 127Type: OralSession Name: 623. Mantle Cell, Follicular and Other B Cell Lymphomas: Clinical and Epidemiological: Evolution of Immunotherapeutic Regimens in B Cell LymphomasSession Date and Time: Saturday, Dec. 11, 2021, Noon to 1:30 p.m. ETPresentation Time: Saturday, Dec. 11, 2021, Noon ET

During the ASH conference, additional City of Hope researchers will also make presentations onsite or virtually:

Title: Pembrolizumab Plus Vorinostat Induces Responses in Patients with Hodgkin Lymphoma Who Are Refractory to Prior PD-1 Blockade Publication Number: 234Type: Oral.Session Name: 624. Hodgkin Lymphomas and T/NK cell Lymphomas: Hodgkin Lymphoma Clinical TrialsSession Date and Time: Saturday, Dec. 11, 2021, 2 to 3:30 p.m. ET Presentation Time: Saturday, Dec. 11, 2021, 3:15 p.m. ETPresenter: Alex Herrera, M.D., associate professor, City of Hope Division of Lymphoma, Department of Hematology & Hematopoietic Cell Transplantation

Herrera will also introduce an abstract at a plenary scientific session on Sunday, Dec. 12, 2 to 4 p.m. ET

Title: A Randomized Open Label Pilot Study of Clostridium Butyricum Miyairi 588 (CBM588) in Recipients of Allogeneic Hematopoietic Cell Transplantation Publication Number: 334Type: OralSession Name: 722. Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Infection and Immune ReconstitutionSession Date and Time: Saturday, Dec. 11, 2021, 4 to 5:30 p.m. ET Presentation Time: Saturday, Dec. 11, 2021, 4:45 p.m. ETPresenter: Karamjeet S. Sandhu, M.D., assistant professor, City of Hope Division of Leukemia, Department of Hematology & Hematopoietic Cell Transplantation

Title: The Impact of Somatic Mutations on Allogeneic Hematopoietic Cell Transplantation in Chronic Myelomonocytic Leukemia: A Center for International Blood and Marrow Transplant Research (CIBMTR) Analysis Publication Number: 417Type: OralSession Name: 732. Allogeneic Transplantation: Disease Response and Comparative Treatment Studies: Prognostic Biomarkers for Donor Selection and Recipient OutcomesSession Date and Time: Sunday, Dec. 12, 2021, 9:30 a.m. to 11 a.m. ET Presentation Time: Sunday, Dec. 12, 2021, 10 a.m. ETPresenter: Matthew G. Mei, M.D., associate professor, City of Hope Division of Lymphoma, Department of Hematology & Hematopoietic Cell Transplantation

In addition, Andrew Artz, M.D., M.S., professor, City of Hope Division of Leukemia, Department of Hematology & Hematopoietic Cell Transplantation, will speak at an education session titled How Can We Ensure That Everyone Who Needs a Transplant Can Get One? about allogeneic hematopoietic cell transplantation for older adults and will also give opening remarks a scientific workshop on hematology and aging.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin, monoclonal antibodies and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope is ranked among the nations Best Hospitals in cancer by U.S. News & World Report. Its main campus is located near Los Angeles, with additional locations throughout Southern California and in Arizona. Translational Genomics Research Institute (TGen) became a part of City of Hope in 2016. AccessHope, a subsidiary launched in 2019, serves employers and their health care partners by providing access to NCI-designated cancer center expertise. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

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City of Hope Doctors Present New Research on Cancer Immunotherapies - Business Wire

Recommendation and review posted by Bethany Smith

Talaris therapy ends need for immune drugs in transplant patients – – pharmaphorum

November 7th, 2021

Two kidney transplant patients who received a stem cell therapy developed by Talaris Therapeutics were able to come off all immunosuppressant drugs within a year, without any evidence of graft rejection.

The first findings from Talaris phase 3 trial of the cell therapy called FCR001 suggest it may be possible to eliminate the need entirely for patients to take what may be dozens of tablets daily after organ transplants, according to the US biotech.

While still preliminary, the experience with the two patients back up Talaris hope that giving a one-shot dose of FCR001 the day after an organ transplant could stimulate immune tolerance in the recipient, and avoid the side effects of current drug treatments such as infections, heart disease, and some forms of cancer.

The companys approach relies on administering haematopoietic stem cells from the individual who donated the organ, in order to generate what Talaris refers to as chimerism, with both donor and recipient cells present in the bone marrow. That allows the immune system to see the transplanted organ as self rather than foreign.

The first two recipients in Talaris FREEDOM-1 phase 3 trial had received FCR001 at least 12 months earlier, and showed stable kidney function, according to Talaris.

A larger group of five patients who were at least three months from the cell therapy maintained more than 50% chimerism in their T cells, which the biotech said was a sign of long-term, immunosuppression-free tolerance to the donated kidney in its phase 2 trials.

The FREEDOM-1 results reported at the American Society of Nephrology (ASN) meeting this week were accompanied by updated results from Talaris phase 2 study, in which all 26 patients originally weaned off immunosuppressants have continued to remain off them without rejecting their donated kidney.

Some transplant patients treated with Talaris therapy in earlier trials have now been off all immunosuppression for more than 12 years without signs of kidney rejection.

Talaris intends to enrol 120 subjects into the phase 3 trial, which is scheduled to generate results in 2023.

Earlier this year, Talaris raised $150 million via a Nasdaq listing that will be used to take FCR001 through the phase 3 programme in organ transplantation and as a treatment for rare autoimmune disease scleroderma.

It also recently started a phase 2 trial of the cell therapy to see if it is able to induce immune tolerance to a transplanted kidney in patients who received the transplant from a living donor up to a year prior to administration of FCR001.

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Talaris therapy ends need for immune drugs in transplant patients - - pharmaphorum

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BioRestorative Therapies Prices $23 Million Public Offering – GlobeNewswire

November 7th, 2021

Common stock will begin trading on The Nasdaq Capital Market under the ticker symbol BRTX November 5, 2021

MELVILLE, N.Y., Nov. 04, 2021 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (the Company") (NASDAQ:BRTX), a life sciences company focused on adult stem cell-based therapies, today announced the pricing of the underwritten public offering of 2,300,000 units, each consisting of one share of its common stock and a warrant to purchase one share of its common stock at a per unit price of $10.00. The warrants have a per share exercise price of $10.00, are exercisable immediately, and expire five years from the date of issuance. The aggregate gross proceeds from the offering are expected to total $23 million, before deducting the underwriting discounts and commissions and estimated offering expenses payable by the Company and without giving effect to proceeds from any subsequent exercise of warrants.

As a result of the offering, the Companys common stock will become listed on the Nasdaq Capital Market and will trade under the ticker symbol BRTX beginning November 5, 2021. The offering is expected to close on or about November 9, 2021, subject to customary closing conditions. In addition, the Company has granted to the underwriters of the offering a 45-day option to purchase up to 345,000 additional shares and/or additional warrants to purchase up to 345,000 shares of common stock to cover over-allotments, if any.

Roth Capital Partners is acting as sole manager for the offering.

BioRestorative Therapies advancement to The Nasdaq Capital Market continues a year of growth and accomplishment for our company during which time we emerged from Chapter 11 reorganization, transformed our business, strengthened our financial position and enhanced our IP position said Lance Alstodt, President and Chief Executive Officer of BioRestorative.

The securities described above are being sold by BioRestorative Therapies pursuant to a registration statement on Form S-1 (Registration No. 333-258611) that was previously filed by BioRestorative Therapies with the Securities and Exchange Commission (the SEC) and declared effective on November 4, 2021 and an additional registration statement filed pursuant to Rule 462(b), which became effective upon filing. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

The offering is being made only by means of the written prospectus forming part of the effective registration statement. Electronic copies of the accompanying prospectus may be obtained, when available, by contacting Roth Capital Partners, 888 San Clemente, Newport Beach, CA 92660, Attn: Prospectus Department, telephone: 800-678-9147, or email at rothecm@roth.com, or by visiting the SECs website at http://www.sec.gov.

About BioRestorative Therapies, Inc.BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain arising from degenerative disc disease.

Metabolic Program (ThermoStem): We are developing a cell-based therapy candidate to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:

Email: ir@biorestorative.com

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BioRestorative Therapies Prices $23 Million Public Offering - GlobeNewswire

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Shockwave therapy brings new healing opportunities for heart attack patients and hope for people with spinal cord injuries – KULR-TV

November 7th, 2021

Success Story of Extracorporeal Shock Wave Therapy (ESWT)

Successful for over 40 years in urology for the disintegration of kidney stones, with high efficiency and hardly any side effects worth mentioning.

How does the shock wave work?

Without causing mechanical damage, shockwaves trigger a biological response in the treated tissue through their compressive, tensile and shear forces (mechanotransduction). Genes are activated in the cell nucleus starting to produce proteins (including growth factors) responsible for the healing process. This also causes increased ingrowth of newly formed blood vessels, which improves local metabolism. The additional modulation of the inflammation necessary for healing enables regeneration of pathological tissue.

Recent studies prove.

Shockwaves also trigger the production of messenger substances to the cell nucleus, which mobilize the body's own stem cells from the bone marrow, stimulating them to migrate to the treated tissue, settle there and develop into the required tissue (e.g. heart muscle cells). Instead of conventional stem cell transplantation shockwaves make it possible to initiate the body's own regeneration without risk of complications.

Therapy for a wide variety of tissues.

Since the underlying pathology can be treated with these methods, shockwave therapy is being used in more and more medical disciplines.

This creates a tool that opens up completely new possibilities in tissue regeneration without triggering significant side effects. Since conventional medicine has not been able to offer any significant therapeutic options to date, the present results of shockwave therapy are of particular importance and are therefore applied in the following areas. It can be assumed that shockwave therapy can be used in practically all medical specialties.

Spinal cord injury/cross-sectional lesion.

What was long considered unthinkable is now one of the major hopes for causal therapy: shockwave has also made great progress in the treatment of paraplegia. Since November 2020, the first patients have been included in an Austria-wide study. Due to the COVID pandemic, the initiation of the individual study centers has been somewhat delayed, but so far eight patients have already been enrolled in the study. In addition, the Unfallkrankenhaus Berlin, one of the most important centers for spinal cord injury in Germany, will participate in the study.

Dr. Wolfgang Schaden, adj. Prof., President of ISMST, Ludwig Boltzmann Institute for Experimental and Clinical Traumatology, Deputy Medical Director of AUVA, Austria

Cardiac Surgery.

Regeneration of heart muscle after myocardial infarction has long remained a dream of modern medicine. Despite extensive efforts to develop stem cell and gene therapies, none of these methods could be brought into clinical routine. Cardiac shockwave therapy brings a scientific breakthrough: Cardiac function is improved, and impressive results show the increase of patients' quality of life. Shockwave therapy in cardiac surgery has a favorable side effect profile and is on the verge of bringing cardiac regeneration into daily clinical practice.

PD Dr. Johannes Holfeld, University Department of Cardiac Surgery, Innsbruck Medical University, Austria.

Sexual Medicine.

Low-energy shockwave therapy has been a fabulous addition to sexual medicine armamentarium for men and women with various forms of sexual dysfunction, e.g. erectile dysfunction, premature ejaculation, persistent genital arousal disorder PGAD/genito-pelvic dysesthesia GPD. Many patients (and their partners) describe these comfortable and quick shockwave treatments as life changing.

Prof. Dr. Irwin Goldstein, Alvarado Hospital, San Diego, CA, USA

Aesthetic-, hand-, burn- and reconstructive surgery.

Shockwave medicine can support these four pillars of surgery noninvasively. Two significant examples: In aesthetic surgery with significant improvement in cellulite with shockwave therapy after six to eight sessions, lasting for a period of about one year. In burns, shockwave therapy can accelerate epithelialization (healing) of superficial burn injuries clinically relevant by three days, with a significant reduction in infections and hospitalization.

Prof. Dr. Karsten Knobloch, SportPraxis Prof. Knobloch, Hanover, Secretary General of the German Shockwave Society DIGEST.

Sports Medicine.

After more than 30 years of experience, shock wave treatment is now a standard in sports medicine and rehabilitation facilities worldwide.

Leprosy.

Shockwaves used in a similar way as for diabetic foot ulcers have also led to the healing of wounds in leprosy patients and significantly improved the quality of life of these patients. This work, carried out in Agua de Dios, Colombia, by the Bosque University group in Bogot, is now being used in several medical centers around the world with very positive results.

Prof. Dr. Carlos Leal, Bosque University, Fenway Medical, Bogot, Colombia.

Wound healing.

Chronic wounds are challenging for patients concerned and practitioners and will have an increasing impact on health care systems. Treatment with shockwaves has a positive conditioning influence on the wounds and in a high proportion for healing, independent of otherwise aggravating factors (e.g. diabetes mellitus, immunosuppression, cortisone therapy, and other exacerbating factors).

With an average treatment frequency of one treatment every second week, in addition to the established wound therapy, healing was observed in more than 70% of the cases of ulcers and other wound healing disorders.The therapy is free of side effects and helps to reduce the burden of the health care system due to the enormous savings potential.

Rainer Mittermayr, MD, MBA, assoc. Prof., Treasurer of the ISMST and Conference Secretary, Senior Surgeon Orthopedic and Traumatology, AUVA (Austrian Workers Compensation Board, Vienna, Austria)

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Shockwave therapy brings new healing opportunities for heart attack patients and hope for people with spinal cord injuries - KULR-TV

Recommendation and review posted by Bethany Smith

Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent … – KULR-TV

November 7th, 2021

Updates from OTL-201 Clinical Proof-of-Concept Study in MPS-IIIA and OTL-204 Preclinical Study for GRN-FTD at ESGCT Showcase Potential for HSC Gene Therapy in Multiple Neurodegenerative Disorders

Launch Activities for Libmeldy Across Key European Countries, including Reimbursement Discussions, Progressing in Anticipation of Treating Commercial Patients

Frank Thomas, President and Chief Operating Officer, to Step Down Following Transition in 2022; Search for a Chief Financial Officer Initiated

Cash and Investments of Approximately $254M Provide Runway into First Half 2023

BOSTONandLONDON, Nov. 04, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reported financial results for the quarter ended September 30, 2021, as well as recent business updates and upcoming milestones.

This quarter, we are pleased by the progress demonstrated by our investigational neurometabolic HSC gene therapy programs with promising preclinical and clinical updates at ESGCT, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With follow-up in OTL-201 for MPS-IIIA patients now ranging between 6 and 12 months, biomarker data remain highly encouraging, showing supraphysiological enzyme activity and corresponding substrate reductions in the CSF and urine. The launch strategy for Libmeldy is also advancing in Europe with momentum building on reimbursement discussions and patient finding activities.

Recent Presentations and Business Updates

Data presentations at ESGCT

Clinical and pre-clinical data from across the companys investigational hematopoietic stem cell (HSC) gene therapy portfolio were featured in two oral and seven poster presentations at the European Society of Gene & Cell Therapy Congress (ESGCT) on October 19-22. Highlights from key presentations are summarized below:

OTL-201 for Mucopolysaccharidosis type IIIA (MPS-IIIA): A poster presentation featured supportive biomarker data from the first four patients with evaluable results, with duration of follow-up ranging from 6 to 12 months. The treatment has been generally well-tolerated in all enrolled patients (n=5) with no treatment-related serious adverse events (SAEs). Supraphysiological N-sulphoglucosamine sulphohydrolase ( SGSH) enzyme activity above the normal range was seen in leukocytes and plasma within one to three months in all evaluable patients (n=4).A greater than 90% reduction in urinary glycosaminoglycans (GAGs) was seen within three months in all evaluable patients (n=4).SGSH activity in the cerebrospinal fluid (CSF) increased from undetectable at baseline to within or above the normal range by six months in all patients with available data (n=3).CSF GAGs decreased from baseline in patients with available data (n=3).OTL-204 for Progranulin-mutated Frontotemporal Dementia (GRN-FTD): Preliminary in vivo data from the preclinical proof-of-concept study showed that murine GRN -/- HSPCs, transduced with an LV expressing progranulin under the control of a novel promoter, are able to engraft and repopulate the brain myeloid compartment of FTD mice and to locally deliver the GRN enzyme.

R&D Investor Event Summary

In September, Orchard hosted an R&D investor event highlighting its discovery and research engine in HSC gene therapy, including an update on the OTL-104 program in development for NOD2 Crohns disease (NOD2-CD) and potential new applications in HSC-generated antigen-specific regulatory T-cells (Tregs) and HSC-vectorization of monoclonal antibodies (mAbs).

The discussion also covered the differentiated profile of Orchards HSC gene therapy approach, which has exhibited favorable safety, long-term durability and broad treatment applicability.

In particular, Orchards lentiviral vector-based HSC gene therapy programs have shown no indication of insertional oncogenesis and no evidence of clonal dominance due to integration into oncogenes. Importantly, the promoters and regulatory elements of Orchard vectors are derived from human (not viral) sequences and are specifically designed to have limited enhancer activity on neighboring genes thereby mitigating the potential for safety concerns.In addition, because of the fundamental biological differences between the HSC and adeno-associated virus (AAV) gene therapy approaches, Orchards programs have not, to date, seen the safety and durability concerns experienced by the AAV gene therapy field.

Libmeldy (atidarsagene autotemcel) launch in Europe

Orchard is providing an update on the following key launch activities for Libmeldy in Europe:

Discussions with health authorities and payors are underway across Europe in key markets including Germany, the UK, France and Italy.Qualification of treatment centers is progressing with The University of Tbingen in Germany ready to treat commercial patients and other centers in the final stages of qualification and treatment readiness.Disease awareness and patient identification activities continue and have supported patient referrals in major European centers. Orchards partnerships in the Middle East and Turkey allow for opportunities to treat eligible patients from these territories at qualified European centers.Orchard is providing sponsorship for an ongoing newborn screening pilot in Germany and is working with laboratories to implement pilots in Italy, the UK, France and Spain.

Executive organizational update

The company also announced that Frank Thomas will step down from his role as president and chief operating officer, following a transition in 2022. A search for a chief financial officer is underway. Mr. Thomas other responsibilities will be assumed by existing members of the leadership team in commercial and corporate affairs. Orchard recently strengthened the executive team with the appointments of Nicoletta Loggia as chief technical officer and Fulvio Mavilio as chief scientific officer and the promotion of Leslie Meltzer to chief medical officer.

I want to extend my gratitude to Frank Thomas for his immense contributions to Orchard, said Gaspar. During his tenure, Frank oversaw the transition of the organization to a publicly traded company and has managed operations with a focus on cross-company innovation, including his role as a key architect in creating and executing the focused business plan we rolled out in 2020. Along with the entire board of directors and leadership team, I appreciate Franks commitment to facilitate a smooth transition during this time.

Gaspar continued, Our search is focused on a CFO to lead the broad strategic planning efforts necessary to capitalize on the full potential of our hematopoietic stem cell gene therapy platform. We have a strong team in place to aid Orchards success in this next phase of growth and are well capitalized through the anticipated completion of several value-creating milestones.

Upcoming Milestones

In June 2021, Orchard announced several portfolio updates following recent regulatory interactions for the companys investigational programs in metachromatic leukodystrophy (MLD), Mucopolysaccharidosis type I Hurler syndrome (MPS-IH) and Wiskott-Aldrich syndrome (WAS).

OTL-200 for MLD in the U.S: Based on feedback received from the U.S. Food and Drug Administration (FDA), the company is preparing for a Biologics License Application (BLA) filing for OTL-200 in pre-symptomatic, early-onset MLD in late 2022 or early 2023, using data from existing OTL-200 patients. This approach and timeline are subject to the successful completion of activities remaining in advance of an expected pre-BLA meeting with FDA, including future CMC regulatory interactions and demonstration of the natural history data as a representative comparator for the treated population.OTL-203 for MPS-IH: Orchard is incorporating feedback from FDA and the European Medicines Agency (EMA) into a revised global registrational study protocol, with study initiation expected to occur in 2022.OTL-201 for MPS-IIIA: Additional interim data from this proof-of-concept study are expected to be presented at medical meetings in 2022, including early clinical outcomes of cognitive function.OTL-103 for WAS: The company expects a MAA submission with EMA for OTL-103 in WAS in 2022, subject to the completion of work remaining on potency assay validation and further dialogue with EMA. The company will provide updated guidance for a BLA submission in the U.S. following additional FDA regulatory interactions.

Third Quarter 2021 Financial Results

Revenue from product sales of Strimvelis were $0.7 million for the third quarter of 2021 compared to $2.0 million in the same period in 2020, and cost of product sales were $0.2 million for the third quarter of 2021 compared to $0.7 million in the same period in 2020. Collaboration revenue was $0.5 million for the third quarter of 2021, resulting from the collaboration with Pharming Group N.V. entered into in July 2021. This revenue represents expected reimbursements for preclinical studies and a portion of the $17.5 million upfront consideration received by Orchard under the collaboration, which will be amortized over the expected duration of the agreement.

Research and development (R&D) expenses were $20.8 million for the third quarter of 2021, compared to $14.7 million in the same period in 2020. The increase was primarily due to higher manufacturing and process development costs for the companys neurometabolic programs and lower R&D tax credits as compared to the same period in 2020. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and development milestone payments under the companys agreements with third parties, and personnel costs to support these activities.

Selling, general and administrative (SG&A) expenses were $13.0 million for the third quarter of 2021, compared to $13.0 million in the same period in 2020. SG&A expenses are expected to increase in future periods as the company builds out its commercial infrastructure globally to support additional product launches following regulatory approvals.

Net loss was $36.4 million for the third quarter of 2021, compared to $20.3 million in the same period in 2020. The increase in net loss as compared to the prior year was primarily due to higher R&D expenses as well as the impact of foreign currency transaction gains and losses. The company had approximately 125.5 million ordinary shares outstanding as of September 30, 2021.

Cash, cash equivalents and investments as of September 30, 2021, were $254.1 million compared to $191.9 million as of December 31, 2020. The increase was primarily driven by net proceeds of $143.6 million from the February 2021 private placement and $17.5 million in upfront payments from the July 2021 collaboration with Pharming Group N.V., offset by cash used for operating activities and capital expenditures. The company expects that its cash, cash equivalents and investments as of September 30, 2021 will support its currently anticipated operating expenses and capital expenditure requirements into the first half of 2023. This cash runway excludes an additional $67 million that could become available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers granted by the FDA following future U.S. approvals.

About Libmeldy / OTL-200 Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability. For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website. Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. About Orchard

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website ( http://www.orchard-tx.com ), the investor relations website ( ir.orchard-tx.com ), and on social media ( Twitter and LinkedIn ), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2023. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2021, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent ... - KULR-TV

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