The latest market report published by Reports and Data, titled Global Preimplantation Genetic Testing Market, presents an accurate analysis of the estimated market size, share, revenue, and sales & distribution networks of the global Preimplantation Genetic Testing market over the forecast period. The report offers an exhaustive overview of the market, along with a precise summary of the markets leading regions. Our team of analysts has studied the existing competitive landscape of the market inside out, focusing on the leading companies and their business expansion strategies. The report ends with conclusive data offering useful insights into the market growth on both regional and global levels.

The report covers extensive analysis of the key market players in the market, along with their business overview, expansion plans, and strategies. The key players studied in the report include:

Illumina Inc.; Thermo Fisher Scientific, Inc.; Natera, Inc.; Bioarray S.L.; Good Start Genetics, Inc.; Laboratory Corporation of America Holdings; California Pacific Medical Center; Quest Diagnostics Incorporated; CooperSurgical, Inc.; Genea Limited; IGENOMIX; Reproductive Genetic Innovations, Reproductive Health Science Ltd.

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The report draws the focus of the reader on the grave impact of the ongoing COVID-19 pandemic on the Preimplantation Genetic Testing industry and its vital segments and sub-segments. It elaborates on the adverse effects of the pandemic on the global economic scenario, as well as this particular business sphere. The report takes into account the key influencing factors influencing market performance in the present COVID-19 times. The market has been substantially affected by the pandemic, and significant changes have been observed in the market dynamics and demand trends. The report examines the major financial difficulties brought about by the pandemic and offers a future COVID-19 impact assessment.

The market intelligence study takes the reader through the key parameters of the Preimplantation Genetic Testing market, including the strengths and weaknesses of the leading players, using analytical tools like the SWOT analysis and Porters Five Forces analysis. The report includes broad market segmentation based on the different product types, a wide application spectrum, the key regions, and the existing competition among players.

Test Type (Revenue, USD Million; 20162026)Preimplantation Genetic Diagnosis (PGD)Preimplantation Genetic Screening (PGS)Type (Revenue, USD Million; 20162026)Chromosomal AbnormalitiesX-linked DiseasesEmbryo TestingFresh Embryo (Own Eggs)Frozen Embryo (Own Eggs)Fresh Embryo (Donor Eggs)Frozen Embryo (Donor Eggs)Aneuploidy ScreeningHLA TypingOther PGT TypesApplication (Revenue, USD Million; 20162026)Embryo HLA Typing for Stem Cell TherapyIVF PrognosisLate Onset Genetic DisordersInherited Genetic DiseaseOthersEnd Use (Revenue, USD Million; 20162026)Maternity Centers & Fertility ClinicsHospitals, Diagnostic Labs, and Service ProvidersResearch Laboratories & Academic Institutes

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The investigative study further assesses the market on the basis of market reach and consumer base in the key geographical segments. Alongside reviewing the sales network, distribution channels, pricing analysis, profit margins, cost and demand volatility, import/export dynamics, gross revenue, and various other aspects of the market, the report studies several factors affecting market growth over the forecast period, such as drivers, restraints, limitations, growth prospects, and numerous macro- and micro-economic indicators.

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Preimplantation Genetic Testing Market Outlooks 2020: Industry Analysis, Market Segmentation, Challenges and Opportunities to 2027 - The Think...

Recommendation and review posted by Bethany Smith

SAN DIEGO, Oct. 21, 2020 (GLOBE NEWSWIRE) -- Bionano Genomics (NASDAQ: BNGO) announces the most significant update to its suite of software tools for data analysis since the launch of Saphyr in 2017. This version of Bionano Access contains over 100 new features and revisions designed to simplify and accelerate every step in the data analysis workflow and make Bionano data easier to interpret. The update is available now as a free download and will be included with Saphyr systems going forward. With these enhancements, it is expected that clinical labs that adopt Saphyr can more readily develop assays for a wide range of genetic diseases, including tests for pediatric neuro-developmental disorders, liquid biopsies for blood cancers like leukemias, lymphomas and multiple myeloma and tests for solid tumors as well.

Current clinical standards of care for diagnostic testing in genetic disease and cancer are based on a series of medical guidelines that recommend structural variation (SV) analysis as first-tier testing. For genetic disease, chromosomal microarray (CMA) analysis is first tier and karyotyping (KT) and fluorescence in-situ hybridization (FISH) are used as reflex tests. Medical guidelines for testing in heme malignancies recommend using KT as first tier testing, alongside some rapid FISH assays and FISH panels, and recommend using CMA as reflex tests. In multiple publications and presentations, Saphyr has been shown to provide a single test that is 100% concordant with the testing methods currently recommended by these guidelines, which requires the use of three different technologies. To-date, this capability has not been shown by any other genome analysis platform. Saphyr has been shown to be significantly more sensitive and specific for SVs than next-generation sequencing (NGS) and to outperform long-read sequencing platforms from PacBio and Oxford Nanopore Technologies. This software updates further enhance Saphyrs unique advantages for customers.

Building on the success of Bionano EnFocus FSHD Analysis tools that automate the analysis of variants in a form of muscular dystrophy, this update is expected to be a key step forward in finalizing other EnFocus panels that automate analysis of variants in patients with genetic diseases, including autism spectrum disorder, developmental delay and repeat expansion disorders, patients with various heme malignancies, such as AML and CLL leukemias, and patients with solid tumors. This new version of the software makes it possible for Bionano EnFocus FSHD to run on the PC that ships with the Saphyr instrument without the need for cloud-based analysis or costly compute clusters. However, for analysis run on Bionanos cloud-based Compute On Demand, the updated software is more efficient, expands capacity and reduces the analysis time, in some cases by threefold. For discovery research, like in cancer research where exceptional sensitivity is essential, cloud-based analysis now finishes in hours rather than in days.

Mark Oldakowski, Chief Operating Officer of Bionano Genomics, who led the development of the software commented: With the improvements to our analysis and visualization software announced today, Saphyr continues to increase its lead as the most powerful genome analysis platform for the detection of structural variants and copy number variations in human genomes and complex cancer samples. Saphyrs unique ability to detect structural variants down to 1% allele fraction, genome-wide and unbiased, without enrichment or amplification and with the highest sensitivities and lowest false positives is unmatched by current short or long-read sequencing technologies. The new capabilities and improvements to our software further simplify and speed up the analysis and interpretation of clinical samples and we believe that these updates may help accelerate Saphyrs adoption for Next-Generation Cytogenomics in the estimated 2,500 cytogenetics labs around the world.

Bionano Solve v3.6 and Bionano Access v1.6 are available for download at https://bionanogenomics.com/support/software-downloads/

About Bionano GenomicsBionano is a genome analysis company providing tools and services based on its Saphyr system to scientists and clinicians conducting genetic research and patient testing and providing diagnostic testing for those with autism spectrum disorder (ASD) and other neurodevelopmental disabilities through its Lineagen business. Bionanos Saphyr system is a platform for ultra-sensitive and ultra-specific structural variation detection that enables researchers and clinicians to accelerate the search for new diagnostics and therapeutic targets and to streamline the study of changes in chromosomes, which is known as cytogenetics. The Saphyr system is comprised of an instrument, chip consumables, reagents and a suite of data analysis tools, and genome analysis services to provide access to data generated by the Saphyr system for researchers who prefer not to adopt the Saphyr system in their labs. Lineagen has been providing genetic testing services to families and their healthcare providers for over nine years and has performed over 65,000 tests for those with neurodevelopmental concerns. For more information, visitwww.bionanogenomics.com or http://www.lineagen.com.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as may, will, expect, plan, anticipate, estimate, intend and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) convey uncertainty of future events or outcomes and are intended to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the improved performance of our technology as a result of the recent software update; increased adoption of Saphyr resulting from this update and other recent updates; the ability of our technology to support future development of assays by clinical laboratories; Saphyrs capabilities in comparison to other genome analysis technologies and potential to replace traditional cytogenetic technologies; the accelerated adoption of Saphyr for Next-Generation Cytogenomics in cytogenetics labs; and the advancement of our strategic objectives. Each of these forward-looking statements involves risks and uncertainties. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include the risks and uncertainties associated with: the impact of the COVID-19 pandemic on our business and the global economy; general market conditions; changes in the competitive landscape and the introduction of competitive products; changes in our strategic and commercial plans; our ability to obtain sufficient financing to fund our strategic plans and commercialization efforts; the ability of medical and research institutions to obtain funding to support adoption or continued use of our technologies; the loss of key members of management and our commercial team; and the risks and uncertainties associated withour business and financial condition in general, including the risks and uncertainties described in our filings with the Securities and Exchange Commission, including, without limitation, our Annual Report on Form 10-K for the year ended December 31, 2019 and in other filings subsequently made by us with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise.

CONTACTSCompany Contact:Erik Holmlin, CEOBionano Genomics, Inc.+1 (858) 888-7610eholmlin@bionanogenomics.com

Investor Relations Contact:Ashley R. RobinsonLifeSci Advisors, LLC+1 (617) 430-7577arr@lifesciadvisors.com

Media Contact:Darren Opland, PhDLifeSci Communications+1 (617) 733-7668darren@lifescicomms.com

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Bionano Genomics Releases Major Update to Its Suite of Software Tools That Simplifies Clinical Analysis, Reduces Time to Actionable Results and Makes...

Recommendation and review posted by Bethany Smith

CLEVELAND--Brain cell dysfunction in low oxygen is, surprisingly, caused by the very same responder system that is intended to be protective, according to a new published study by a team of researchers at the Case Western Reserve University School of Medicine.

"These powerful protein responders initially protect brain cells from low oxygen as expected, but we find that their prolonged activity leads to unintended collateral damage that ultimately impairs brain cell function," said the study's principal investigator Paul Tesar, a professor in the Department of Genetics and Genome Sciences at the Case Western Reserve School of Medicine and the Dr. Donald and Ruth Weber Goodman Professor of Innovative Therapeutics.

Defining the mechanism of brain-cell damage in low oxygen conditions provides an opportunity to develop effective therapies, including a class of drugs studied in their research that could inform future clinical approaches for many neurological diseases caused by low oxygen. The work also clarifies how the response to low oxygen causes disease in other tissues outside the brain.

Their research was published online Oct. 21 in the journal Cell Stem Cell.

The body's response to low oxygen

With the dawn of an oxygenated atmosphere, a burst of multicellular life was possible, as oxygen could be used to produce the energy needed to support complex life functions. Given the requirement of oxygen for life, nearly all organisms evolved a mechanism to rapidly respond to low oxygen--a condition called hypoxia. The Noble Prize in Physiology or Medicine was awarded in 2019 for discoveries of how cells in our body sense low oxygen levels and respond to stay alive.

At the core of this ancient response are proteins called hypoxia-inducible factors (HIFs), which instruct the cell to minimize oxygen consumption and maximize their access to oxygen. In this way, HIFs can be thought of as valiant heroes attempting to protect and resuscitate cells in the immediate response to low oxygen.

Prolonged hypoxia causes dysfunction in many tissues. In particular, stem cells in the brain are impaired by hypoxia in many diseases, including stroke, cerebral palsy related to premature birth, respiratory distress syndromes, multiple sclerosis and vascular dementia. Even the significant neurological damage caused by COVID-19 is attributed to hypoxia.

Until now, the precise causes of cell malfunction due to low oxygen were unknown.

The dark side of the hypoxia response

In this study, researchers developed a new approach to closely study how the hypoxia responder proteins function. By comparing how they work in brain-stem cells with other tissues, such as heart and skin, the scientists confirmed that the hypoxia responder proteins perform a beneficial function to promote cell survival in low oxygen in all tissues. However, these same hypoxia responder proteins had a previously unappreciated dark side, as they also switched on other cellular processes outside of the core beneficial response.

The team then demonstrated that this additional--and previously unknown--response is what impaired brain-stem cell function. This suggests that, while hypoxia responder proteins evolved to promote cell survival in all tissues of the body in low-oxygen conditions, their powerful effects can also have unintended consequences to disrupt cell function.

New opportunities for treating hypoxia damage

The authors tested thousands of drugs to try to restore brain-stem cell function to overcome the damaging effects of the hypoxia responder proteins. They discovered a group of drugs that specifically overcome the damage-inducing response, while leaving the beneficial response intact.

"One of the exciting avenues that stems from this work is identifying drugs that specifically target the damaging side of the hypoxia response while sparing the beneficial side," said first author Kevin Allan, a graduate student in Case Western's Medical Scientist Training Program. "This offers a new perspective on combating tissue damage due to hypoxia."

"Whether the damaging side of the hypoxia response is solely an unintended pathological effect or potentially a previously undiscovered normal process that goes awry in disease remains unknown," Tesar said. "Our work opens the door to a new way of thinking about how cells respond to low oxygen in health and disease."

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New research reveals why low oxygen damages the brain - Science Codex

Recommendation and review posted by Bethany Smith

Vanderbilt University researchers have reported the counterintuitive discovery that certain chemotherapeutic agents used to treat tumors can have the opposite effect of tissue overgrowth in normal, intact mammary glands, epidermis and hair follicles. The researchers also are the first to report the discovery of an innate immune signaling pathway in fibroblaststhe spindle-shaped cells responsible for wound healing and collagen productionthat causes cells to proliferate. Such signaling pathways previously were attributed only to immune cells.

The article describing the research, DNA Damage Promotes Epithelial Hyperplasia and Fate Mis-specification via Fibroblast Inflammasome Activation, was published in the journal Developmental Cell on Oct. 13.

The findings of this work, led by postdoctoral fellow Lindsey Seldin and Professor and Chair of the Department of Cell and Developmental Biology Ian Macara, have broad implications for diseases associated with the immune system like psoriasis, as well as cancer and stem cell research.

Understanding the functionality of stem cells and the way that their behavior is regulated has been a longstanding research interest for Seldin. Normal stem cells have an amazing ability to continuously divide to maintain tissue function without forming tumors, she explained. We wanted to understand what happens to these cells in their native environment when subjected to damage, and if the response was connected to a specific tissue.

By testing perturbations to the epidermis, mammary gland and hair follicles vis--vis mechanical damage or DNA damage through chemotherapeutic agents, the researchers saw a paradoxical response: Stem cells, which otherwise would divide slowly, instead divided rapidly, promoting tissue overgrowth.

When the tissues were subjected to DNA damage, their stem cells overly proliferated, giving rise to different cells than they normally would. This was a very perplexing result, said Seldin, the papers lead author. We were determined to figure out if this was a direct response by the stem cells themselves or by inductive signals within their environment. The key clue was that stem cells isolated from the body did not behave the same way as in intact tissuean indication that the response must be provoked from signals being sent to the stem cells from other surrounding cell types.

The investigators turned their attention to fibroblasts, the predominant component of the tissue microenvironment. When fibroblasts in the epidermis were removed, the stem cell responsiveness to DNA damage was diminished, indicating that they played an important role. RNA sequencing revealed that fibroblasts can signal by way of inflammasomescomplexes within cells that help tissues respond to stress by clearing damaged cells or pathogens, which also in this case caused stem cells to divide. This is an astounding discovery, said Macara. Inflammasome signaling has previously been attributed only to immune cells, but now it seems that fibroblasts can assume an immune-like nature.

Seldin intends to replicate this work in the mammary gland to determine whether fibroblasts initiate the same innate immune response as in the epidermis, and more broadly how fibroblasts contribute to the development of cancer and other diseases associated with the immune system.

This work was supported by NCI/NIH grants R35CA132898, F32CA213794 and T32CA119925, as well as American Cancer Society grant PF-18-007-01-CCG.

Original post:
Vanderbilt researchers make counterintuitive discoveries about immune-like characteristics of cells, chemotherapys impact on tissue growth -...

Recommendation and review posted by Bethany Smith

Global cosmetic skin care market is set to witness a substantial CAGR of 5.5% in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. Increasing self-consciousness among population and rising demand for anti- aging skin care products are the factor for the market growth.

Global Cosmetic Skin Care Market By Product (Anti-Aging Cosmetic Products, Skin Whitening Cosmetic Products, Sensitive Skin Care Products, Anti-Acne Products, Dry Skin Care Products, Warts Removal Products, Infant Skin Care Products, Anti-Scars Solution Products, Mole Removal Products, Multi Utility Products), Application (Flakiness Reduction, Stem Cells Protection against UV, Rehydrate the skins surface, Minimize wrinkles, Increase the viscosity of Aqueous, Others), Gender (Men, Women), Distribution Channel (Online, Departmental Stores and Convenience Stores, Pharmacies, Supermarket, Others), Geography (North America, Europe, Asia-Pacific, South America, Middle East and Africa) Industry Trends and Forecast to 2026

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Market Definition: Global Cosmetic Skin Care Market

Cosmetic skin care is a variety of products which are used to improve the skins appearance and alleviate skin conditions. It consists different products such as anti- aging cosmetic products, sensitive skin care products, anti- scar solution products, warts removal products, infant skin care products and other. They contain various ingredients which are beneficial for the skin such as phytochemicals, vitamins, essential oils, and other. Their main function is to make the skin healthy and repair the skin damages.

Market Drivers:

Market Restraints:

Key Benefits:

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Geographically, this report is segmented into several key regions, with sales, revenue, market share and growth Rate of industry in these regions, from 2020 to 2027, covering

Global cosmetic skin care market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of cosmetic skin care market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Few of the major competitors currently working in the global cosmetic skin care market are LOral, Unilever, New Avon Company, Este Lauder Companies, Espa, Kao Corporation, Johnson & Johnson Services, Inc., Procter & Gamble, Beiersdorf, THE BODY SHOP INTERNATIONAL LIMITED, Shiseido Co.,Ltd., Coty Inc., Bo International, A One Cosmetics Products, Lancme, Clinique Laboratories, llc., Galderma Laboratories, L.P., AVON Beauty Products India Pvt Ltd, Nutriglow Cosmetics Pvt. Ltd, Shree Cosmetics Ltd among others.

Cosmetic Skin Care Market: Key Questions Answered in Report

The research study on the Cosmetic Skin Care market offers inclusive insights about the growth of the market in the most comprehensible manner for a better understanding of users. Insights offered in the Cosmetic Skin Care market report answer some of the most prominent questions that assist the stakeholders in measuring all the emerging possibilities.

Thank you for reading our report. For further queries, kindly get in touch with us and our team will provide excellent assistance in customization of the report according to your requirements

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Cosmetic Skin Care Market (Covid 19 Impact Analysis) Data Highlighting Major Vendors, Promising Regions, Anticipated Growth Forecast To 2027 -...

Recommendation and review posted by Bethany Smith

As research on longevity has become a fixation of biotech startup investors, newly launched skin-care startup OneSkin is bringing it to the world of beauty.

Launching today via DTC e-commerce, the company is bridging the worlds of biotech and beauty after four years in development by a team of scientists. Coming out of the Bay Area-based biotech startup incubator MBC BioLabs, the brand has secured $7.5 million in VC and angel funding from sources including science accelerator IndieBio, SOSV and 2FutureHolding. The companys board includes prominent investor Peter Diamandis.

The brand is launching with one moisturizer it calls a topical supplement, which is available online for $100 for a 50-milliliter bottle via a subscription or $120 with one-time purchase. Some of the promised results are typical of beauty products: firmness, smoothness, hydration and collagen production, based on the results of a clinical trial. Its main differentiation point, however, is that it can reduce the biological age of the skin through a patented peptide it created called OS-01. The brand states it does this by preventing the accumulation of senescent cells that cause aging. The brand ran a 3-month clinical trial ending in March 2020 that was found to improve aesthetic features such as elasticity and wrinkles. To address the biological age question, the next planned trial will run for between six months and a year, testing skin biopsied from behind study participants ears.

In a tweet promoting the brand, Diamandis called it a longevity company, referencing a popular biotech concept that is attracting massive startup investment from billionaires.

We are seeing that the longevity space is growing; people will listen and learn more about what longevity is and expanding your health span and the importance of being healthier, said OneSkin co-founder and chief scientific officer Alessandra Zonari. She referenced the popularity of David Sinclair, the longevity-focused biologist focused on the concept of biological age. Gwyneth Paltrow is also interested in the concept, having her biological age calculated on the Netflix show Goop Lab.

OneSkin, meanwhile, has its own biological age calculator for skin called the MolClock (meaning molecular clock), which it developed in 2016. The concept was developed after three of the brands four co-founders worked together in a Brazil-based lab on 3D skin-tissue models, meant to serve as an accurate alternative to animal testing. That eventually developed into the creation of their first skin-care product.

We decided to focus on the anti-aging market that we saw was growing a lot, and its only going to grow. People are only getting older, but no one really knows which products work, said co-founder and CEO Carolina Reis Oliveira. Oliveira holds a PhD in immunology and worked in Brazil with fellow co-founders Mariana Boroni and Juliana Carvalho, who also each have PhDs.

The three co-founders relocated to the Bay Area in 2016 to create their startup through IndieBio. Zonari, who holds a PhD in skin regeneration and tissue engineering, joined the company in 2017 as the fourth co-founder. In 2018, they began the process of screening over 1,000 peptides in search of anti-aging properties and discovered OS-01.

The brand is betting that this biotech concept will catch on in the age-obsessed beauty world. We get overwhelmed about how many products that are out there, and you really dont know because there are all the claims saying that they rejuvenate, or that you will look younger, said Zonari.

The key for marketing to U.S. consumers is explaining the science in an accessible way. The brand will be focusing on content and education via social media promotions, especially through Instagram, as well as videos, webinars and Reddit groups dedicated to topics like longevity and age reversal. Reddits longevity group features an active and enthusiastic audience of over 64,000 members, who head to the message board to discuss topics related to biomedical rejuvenation, such as stem cell therapies, anti-cancer viruses, gene therapy, senolytics and whatever is coming next, according to the groups description.

We want to simplify this science in a way that people can connect with and can be easier to understand, said Oliveira. Basically, the way that we want to explain it is, OK, this peptide, its able to act in the molecular level of your skin in the deeper layers of your skin. How we differentiate is that most products only treat superficially.

IndieBio, SOSV Ventures, and 2Future Holding were the brands first investors for its seed round, when it raised just over $2 million in 2017. The brands most recent funding round occurred in July 2020, with an angel investment of $1.5 million, following a $3.5 million post-seed round in July 2019 from several angel investors.

Continue reading here:
Biotechs longevity obsession reaches the beauty industry - Glossy

Recommendation and review posted by Bethany Smith

In 2009, studies on telomeres and telomerase (its corresponding enzyme) won three American researchers the Nobel Prize in Physiology or Medicine. Since then there has been a flurry of work surrounding this seemingly redundant protein, that has now been touted as the secret to biohacking the ageing process.

We sit down with Functional Medicine Specialist, Miles Price, to figure out why this little protein is so important to our longevity and our health.

Telomeres are proteins called nucleotides which attach to the ends of DNA strandsto protect them from damage every time the cells divide. They play a central role in the cell fate and ageing. Each time the cell divides, some of the telomere sequence gets lost so the telomere cap shrinks. Eventually, when the telomere becomes too short, the cell will either self-destruct through apoptosis or go into senescence. Senescence is an arrested cell state meaning the cell is no longer functioning at100 percent. This contributes to ageing and puts the body at greater risk of developing abnormal cells like cancer.

Getting older is a fact of life. However, the rate of how fast we age is controlled, amongst other things, by our telomere length. These bits of proteins protect our DNA from damage every time the cells divide. Telomere length generally shortens with age however we can slow down the shortening largely with lifestyle interventions. Things which accelerate the shortening include smoking, pollution exposure, lack of physical activity, obesity, stress and inflammation. Skin ageing is also affected by shortened telomeres, however the efficacy for maintaining skin health with telomerase interventions is still being investigated.

Telomere length is greatly influenced by our environment and lifestyle. Through epigenetic influences we can help protect our telomeres by doing the following:

1. Caloric restriction time-restricted eating or fasting up to a minimum of three days(with supervision).2. Evidence suggests that dietary alterations can affect our telomeres. An example ofthis is restricting protein intake.3. Take regular exercise, preferably high intensity intermittent training.4. Increase antioxidants in the diet, typically from plant foods.

A recent gene therapy study on mice consisted of inducing telomerase, the enzyme which slows down the biological clock by extending telomere length, was successful in increasing the lifespan by a further 24 percent. Whilst human trials are still a ways off, what we can do is consider some supplemental interventions together with lifestyle measures discussed earlier. NAD+ (Nicotinamide adenine dinucleotide) is a supplement which is a derivative of Niacin (vitamin B3), and it directly influences the lifespan of the cells and the way the cell ages. This is done by interacting with proteins inside the cell called sirtuins. Sirtuins stimulate various enzymatic processes inside the mitochondria and nucleus which help to protect the DNA and RNA from damage as the cell divides. As we age, our NAD+ levels decline and thus the enzymatic activity of sirtuins declines also, therefore it is becoming increasingly important to consider supplementing with NAD+ as we age.

As you age your immune system becomes weaker. How this affects you is dependent on many factors, like your diet, exercise levels, sleep patterns, and stress levels, for example. Typically our immune systems become slower to respond, the body heals more slowly from infections, and the ability of the immune system to detect and correct dysfunctional cells also declines. Key immune system organs play a role in this decline. The thymus gland located in the middle of the chest plays a central rolein activating certain white blood cells to identify invaders or rogue cells. It shrinks with age due to free radical damage.

This can be minimised by taking extra zinc and other antioxidants which protect against its shrinkage. Other influences on your immunity include caloric restriction. Research has demonstrated that if you fast for more than three days your immune system is given a real boost, by stimulating autophagy. This is the process whereby the body seeks out and removes old and senescent cells, thereby reversing ageing in that short period of time. Caloric restriction for longer than 24 hours is best supervised by a doctor or functional medicine specialist who can monitor any adverse reactions you may have.

You can test your telomere length by conducting a blood test which isolates our DNA and measures telomere length using the qPCR method. This is the most popular method of assessment. One such test is the ImmniGen Test. ImmuniGen is an at-home telomere test that assesses your immune strength and resilience. By measuring your telomere length (a biomarker of ageing), it can measure how yourimmune system is ageing, known as immunosenescence, as well as key genes that affect ageing and immune health. As telomere length is heavily influenced by our lifestyle, it is recommended to repeat the test at regular intervals to track your ageing progress.

Visit LifeHub to learn more about biohacking your telomeres for a healthier and longer life. LifeHub is currently offering ImmuniGen Testing at HK$1,280, which can be ordered online and delivered straight to your door.

LifeHub is a Modern Wellness Lab in Hong Kong offering the antidote to your busy city lifestyle. As leaders in detox and anti-ageing, we cleanse you of everyday toxins and recharge your body for better performance in work and play.

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Biohack the ageing process by taking care of this little protein in your body - Lifestyle Asia

Recommendation and review posted by Bethany Smith

Yulonda Wilbur remembers precisely the sting of when she was told she had breast cancer.

The news came after a routine physical where her general practitioner did a manual exam, and Wilbur flinched when the doctor touched a tender spot on her left breast. She was already scheduled for her annual mammogram, and her physician added an ultrasound. Wilbur remembers one of the technicians when she had the tests being initially cheerful, and then solemn, and later, the radiologist telling her he didnt like what he was seeing and shed need to undergo a biopsy.

But still, when the call came on Feb. 16, 2019, with news that she had triple-negative breast cancer a cancer that tests negative for estrogen receptors, progesterone receptors and excess HER2 protein and therefore does not respond to normal hormonal therapy medicines or those that target HER2 protein receptors her world metaphorically fell apart.

It was just that moment that changes your life forever, said Wilbur, now 53 and back at work since early this year as a patient care assistant at Lawrence + Memorial Hospital, where she has been employed for two decades.

That journey from her initial physical in December 2018 would include a lumpectomy, followed by chemotherapy, then an additional diagnosis of DCIS, or cancer in her milk ducts, and finally a mastectomy in October 2019. This year has been rough with COVID, but for Wilbur, a mother of four grown children, 2019 was the year she battled breast cancer and shes grateful she didnt wage that war during a pandemic.

While shes worked at L+M for 20 years, shes been a patient care assistant for more than three decades and said working in the medical field doesnt make a breast cancer diagnosis any easier.

What shed learn along the way is that theres a higher prevalence of triple-negative breast cancer in African-American women and that friends and family are essential to get you through.

My advice is dont go it alone. Ask for help. You need support, said Wilbur, who lives in Waterford. And ask questions if you dont understand.

After the diagnosis, when one physician came into the room where Wilbur was waiting and started talking at her, Wilbur reminded her that she hadnt introduced herself and asked her to slow down and explain things more thoroughly.

You need to understand what you are going through, and it is a lot to take in, she said.

The lumpectomy was painful, the chemotherapy made her nauseous and left a distasteful film on her tongue, and she developed neuropathy, anemia and fatigue.

I heard a sermon once, said Wilbur, who is active in her church. And what they said was, When you say why me, what you are really saying is why not someone else? So I just accepted it for the most part, and I wasnt happy about it, but I accepted it.

Shes an advocate of regular checkups and mammograms, of all preventive medical care, and marvels that someone who comes from strong stock, like she does, ended up so ill.

Im one of those people who dont get colds, who dont get sick, but I did, she said. There was no history of breast cancer in her family, and shed test negative for the BRCA gene.

When Wilbur was invited to attend a breast cancer support group at Smilow Cancer Hospital Care Center in Waterford, she initially resisted.

I didnt want to sit around with people who were angry, or be depressed, she said. But it wasnt that way, everybody takes a different path, everyone has their own journey.

Not so long ago, when Wilbur found a lump on her right breast, she panicked, but the diagnosis was a benign cyst.

Its not that I cant, but I dont want to go through it again, she said. Later, I thought maybe I should have had both breasts removed.

Shes back at work at L+M and doing the things she enjoys walking, reading and keeping active in her church.

I have faith, and I prayed a lot when I was going through it," she said. "I asked Jesus to get me through it, and He did."

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'I have faith and I prayed a lot' - News from southeastern Connecticut - theday.com

Recommendation and review posted by Bethany Smith

This article was reviewed by Russell N. Van Gelder, MD, PhD

Neuronal cell replacement therapies remain a challenge in retinal diseases. Some fish and salamanders have the innate ability to regenerate retinal tissue after injuries and, as Russell N. Van Gelder, MD, PhD, pointed out, if researchers could harness this ability in humans, the possibilities would be great for repairing or replacing damaged tissue in a wide variety of retinal diseases. Stem cells are the key to cell replacement therapies.

Stem cells are cells that have not terminally differentiated and still have the potential to become many types of terminal cells, said Van Gelder, from the Department of Ophthalmology at the University of Washington in Seattle. We all started as embryonic stem cells in the earliest phases of development.

Related: Retinal pathologies challenging to image with current technologies

Van Gelder went on to explain that there are now methods to create equivalently totipotent stem cells from individual induced progenitor stem cells derived from an individuals blood or epithelial cells.

The overarching goal is to create a cell type that needs replacement from a stem cell precursor, he said.

A major achievement in this quest for regenerative ability occurred in 2014 when an entire eye cup was grown from progenitor stem cells.

Van Gelder also described a study1 in which green fluorescent proteinlabeled retinal precursors derived from embryonic stem cells were transplanted into the subretinal space of macaques. Three months after the procedure, the researchers demonstrated that the bolus of cells persisted and had outgrowth of axons that were seen going to the optic nerve and on to the brain.

This result establishes the validity of a stem cell-based approach for doing regenerative medicine in primates, he said.

Related: Persistent retinal detachment associated with retinoblastoma

Replacement therapy hurdlesAs of now, however, no stem cell-based replacement treatment has received FDA approval. The problems preventing establishment of a treatment have been technical in nature and include correct cellular differentiation as well as generating adequate numbers of cells for large transplantation experiments, establishing correct cell polarity and connectivity, and ensuring the safety of these approaches regarding tumor or hamartoma formation, Van Gelder explained.

Managing inflammatory responses is a problem after cell transplantation. He cited a Japanese study2 of individual progenitor cell-derived retinal progenitor cells transplanted subretinally in monkey models.

Even with an immune HLA-matched donor, there was still a marked inflammatory response at the site of the transplantation, Van Gelder said. This and other inflammatory responses will have to be managed for cell transplantation to be successful.Related: Intravitreally injected hRPCs improve vision in retinitis pigmentosa cases

There are regulatory hurdles to clear. The FDA Center for Biologics Evaluation and Research regulates cellular therapy products, human gene therapy products, and certain devices related to cell and gene therapy.

Van Gelder recalled the well-publicized case of transplantation of fat-derived mesenchymal cells into patients eyes, resulting in loss of vision bilaterally. He pointed out that it is important to temper patient expectations regarding these therapies and to ensure that the work is being done with the highest degree of ethical integrity.

While great progress has been made in this field, significant barriers remain to the successful adoption in the clinical setting in the coming years, Van Gelder concluded. The barriers to cell replacement should be overcome.

Read more by Lynda Charters

--

Russell N. Van Gelder, MD, PhDe: russvg@uw.edu Van Gelder has no financial interests in this subject matter. He serves on the advisory committee for the National Eye Institute Audacious Goals Initiative.

--

References

1. Chao JR, Lamba DA, Kiesert TR, et al. Transl Vis Sci Technol. 2017;6:4; doi:10.1167/tvst/6/3/4

2. Fujii S, Sugita S, Futatsugi Y, et al. A strategy for personalized treatment of iPS-retinal immune rejections assessed in cynomolgus monkey models. Int J Mol Sci. 2020;21(9):3077. doi:10.3390/ijms21093077

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Harnessing regeneration of retinal tissues: An option almost within reach - Ophthalmology Times

Recommendation and review posted by Bethany Smith

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Oct. 21, 2020 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company focused on developing adeno-associated virus (AAV) based gene therapies for the treatment of rare inherited diseases, today announced that Sue Washer, President & Chief Executive Officer, and Dave Knop, PhD, Vice President, Process Development and Manufacturing, will participate in the Cantor Fitzgerald Key Considerations for Gene Therapy Manufacturing and Platforms virtual panel at 10:00 a.m. ET on Wednesday, October 28, 2020.

The panel will focus on the critical components and capabilities to gene therapy manufacturing, including a discussion on AGTCs proprietary manufacturing processes associated with AAV, and key company catalysts expected over the next 12 months. The call will be moderated by Kristen Kluska, Biotechnology Research Analyst of Cantor Fitzgerald.

Presentation Information:Panel: Key Considerations for Gene Therapy Manufacturing and PlatformsDate: Wednesday, October 28, 2020Time: 10:00 a.m. 11:30 a.m. ET

To register for the event, please click here.

About AGTCAGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Companys most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its pre-clinical programs build on the Companys industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

IR/PR CONTACTS: David Carey (IR) or Glenn Silver (PR)Lazar FINN PartnersT: (212) 867-1768 or (646) 871-8485david.carey@finnpartners.com or glenn.silver@finnpartners.com

Corporate Contact:Bill SullivanChief Financial OfficerApplied Genetic Technologies CorporationT: (617) 843-5728bsullivan@agtc.com

Stephen PotterChief Business OfficerApplied Genetic Technologies CorporationT: (617) 413-2754spotter@agtc.com

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AGTC to Participate in Cantor Fitzgerald Virtual Panel: Key Considerations for Gene Therapy Manufacturing and Platforms - BioSpace

Recommendation and review posted by Bethany Smith

A breakthrough study, led by researchers from the University of California, Irvine, results in the restoration of retinal and visual functions of mice models suffering from inherited retinal disease.

Published today in Nature Biomedical Engineering, the paper, titled, Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing, illustrates the use of a new generation CRISPR technology and lays the foundation for the development of a new therapeutic modality for a wide range of inherited ocular diseases caused by different gene mutations.

"In this proof-of-concept study, we provide evidence of the clinical potential of base editors for the correction of mutations causing inherited retinal diseases and for restoring visual function," said Krzysztof Palczewski, PhD, the Irving H. Leopold chair and a distinguished professor in the Gavin Herbert Eye Institute, Department of Ophthalmology at the UCI School of Medicine. Our results demonstrate the most successful rescue of blindness to date using genome editing.

Inherited retinal diseases (IRDs) are a group of blinding conditions caused by mutations in more than 250 different genes. Previously, there was no avenue available for treating these devastating blinding diseases. Recently, the FDA approved the first gene augmentation therapy for Leber congenital amaurosis (LCA), a common form of IRD which originates during childhood.

As an alternative to gene augmentation therapy, we applied a new generation of CRISPR technology, referred to as base editing as a treatment for inherited retinal diseases, said first author Susie Suh, assistant specialist in the UCI School of Medicine Department of Ophthalmology. We overcame some of the barriers to the CRISPR-Cas9 system, such as unpredictable off-target mutations and low editing efficiency, by utilizing cytosine and adenine base editors (CBE and ABE). Use of these editors enabled us to correct point mutations in a precise and predictable manner while minimizing unintended mutations that could potentially cause undesirable side effects, said co-first author Elliot Choi, also an assistant specialist in the UCI Department of Ophthalmology.

Using an LCA mouse model harboring a clinically relevant pathogenic mutation in the Rpe65 gene, the UCI team successfully demonstrated the therapeutic potential of base editing for the treatment of LCA and by extension other inherited blinding diseases. Among other results, the base editing treatment restored retinal and visual function in LCA mice to near-normal levels. Base editing was developed at the Broad Institute of MIT and Harvard in the lab of David Liu, PhD.

After receiving treatment, the mice in our study could discriminate visual changes in terms of direction, size, contrast and spatial and temporal frequency, said Palczewski. These results are extremely encouraging and represent a major advance towards the development of treatments for inherited retinal diseases."

Gene therapy approaches to treating inherited retinal diseases are of special interest given the accessibility of the eye, its immune-privileged status and the successful clinical trials of RPE65 gene augmentation therapy that led to the first US Food and Drug Administration-approved gene therapy. Now, as demonstrated in this study, base-editing technology can provide an alternative treatment model of gene augmentation therapy to permanently rescue the function of a key vision-related protein disabled by mutations.

This research was supported in part by grants from the National Institutes of Health, the Research to Prevent Blindness Stein Innovation Award, Fight for Sight, the Eye and Tissue Bank Foundation (Finland), The Finnish Cultural Foundation, the Orion Research Foundation, the Helen Hay Whitney Foundation, US Department of Veterans Affairs, and a Research to Prevent Blindness unrestricted grant to the Department of Ophthalmology, University of California, Irvine.

Reference: Suh S, Choi EH, Leinonen H, et al.Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing.Nat. Biomed. Eng. 2020. doi:10.1038/s41551-020-00632-6

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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CRISPR Therapy Restores Retinal and Visual Function in Mice - Technology Networks

Recommendation and review posted by Bethany Smith

MELBOURNE, Australia and KING OF PRUSSIA, Pa., Oct. 20, 2020 /PRNewswire/ -- In its annual R&D briefing to investors today, CSL Limited (ASX:CSL; USOTC:CSLLY) demonstrated how the company is advancing a novel research portfolio across four strategic scientific platforms (Plasma Fractionation, Recombinant Technology, Cell and Gene Therapy, Adjuvanted Cell and Egg-based Vaccines) across six therapeutic areas (Immunology, Hematology, Respiratory, Cardiovascular and Metabolic, Transplant, Influenza Vaccines) and two businesses (CSL Behring and Seqirus) to help patients lead full lives, protect public health and drive future business growth. It's this scientific capability and diverse portfolio that positioned the company to quickly respond to the COVID-19 public health crisis.

"CSL's acumen in vaccines, monoclonal antibodies, recombinant technologies, manufacturing capabilities and external partnerships, along with a therapeutic area focus and insight that includes Immunology and Respiratory, has supported the growth and progress of our pipeline and has also enabled us to respond quickly to the need for potential solutions in the world's fight against COVID-19," said Bill Mezzanotte, MD, MPH,Executive Vice President, Head of Research and Development and Chief Medical Officer. "Right across the spectrum of infection and disease, CSL has taken on projects we think make sense both scientifically and that fit our capabilities."

The company's efforts in the fight against COVID-19 include:

"The same collaborative mindset with which CSL has responded to the COVID-19 pandemic is also reflected in how we are advancing our pipeline," said Andrew Nash, PhD, Senior Vice President, Head of Research and Chief Scientific Officer. "Across therapeutic areas, strategic platforms and geographies we continue to grow our footprint and capabilities so that we can deliver a significant benefit to patients around the world."

Other advancements in CSL's R&D pipeline in the last year include:

Setting the pace in Plasma Product development:CSL is continuously improving current plasma fractionation processes and developing new therapeutic options, which has shown major relevance in the fight against COVID-19 and is also evident in other therapeutic areas. For example:

Advancing its Cell and Gene Therapy research by:

Building momentum in Recombinant Technology:CSL has established a highly differentiated preclinical and clinical stage pipeline of recombinant proteins and monoclonal antibodies from its proficiency in protein biology and innate cell immunity. In addition to using this technology against COVID-19, the company is studying:

About CSLCSL (ASX:CSL) is a leading global biotechnology company with a dynamic portfolio of life-saving medicines, including those that treat hemophilia and immune deficiencies, as well as vaccines to prevent influenza. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL including our two businesses, CSL Behring and Seqirus - provides life-saving products to more than 70 countries and employs 27,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For more information about CSL Limited, visitwww.csl.com.

SOURCE CSL

http://www.csl.com

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CSL's Broad Response to COVID-19 and Advancements across the Company's Strategic Scientific Platforms are the Focus of Annual R&D Day - PRNewswire

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ReportsWeb delivers well-researched industry-wide information on the Gene Therapy market. It studies the markets essential aspects such as top participants, expansion strategies, business models, and other market features to gain improved market insights. Additionally, it focuses on the latest advancements in the sector and technological development, executive tools, and tactics that can enhance the performance of the sectors.

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Geographically, this report focuses on product sales, value, market share, and growth opportunity in key regions such asUnited States, Europe, China, Japan, Southeast Asia, and India.

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GENE THERAPY MARKET KEY TRENDS, GROWTH, SHARE, SIZE, ANALYSIS AND FORECAST TO 2026 - PRnews Leader

Recommendation and review posted by Bethany Smith

SAN DIEGO and WALTHAM, Mass., Oct. 21, 2020 /PRNewswire/ --Poseida Therapeutics, Inc. (NASDAQ:PSTX), a clinical-stage biopharmaceutical company utilizing proprietary gene engineering platform technologies to create cell and gene therapeutics with the capacity to cure, and TScan Therapeutics, Inc., a biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell therapies in oncology, today announced a research collaboration and license agreement to explore developing TCR-T cell therapies for the treatment of COVID-19. Poseida's allogeneic T cell platform will be used in combination with TScan's discovered immunodominant epitopes and TCR sequences for the development and commercialization of allogeneic TCR-T cell therapies.

"The TScan relationship will allow us to expand our platform and T cell experience, utilizing one or more TCRs in allogeneic cell therapy applications," said Devon Shedlock, Ph.D., Senior Vice President of Research and Development for Poseida. "We are impressed by TScan's novel platform for the discovery of TCRs. This, coupled with the opportunity to explore our allogeneic T cell platform's potential for also treating diseases outside of oncology, including COVID-19 and eventually other infectious diseases, represents an exciting and differentiated approach."

"Therapeutic strategies to treat COVID-19 have largely focused on neutralizing antibodies that block viral infection or small molecule drugs that inhibit viral replication," said Gavin MacBeath, Ph.D., Chief Scientific Officer at TScan. "We are excited about the possibility of combining our SARS-CoV-2 TCR findings with Poseida's allogeneic T Cell approach to explore a potential cellular therapy for patients with COVID-19. We look forward to collaborating with the Poseida team on this approach."

Emerging studies suggest that patients who fail to mount effective T cell responses often end up with more severe and life-threatening disease. Administering virus-specific engineered T cells may provide a rapid and effective way to address this deficiency. Results from TScan demonstrate that generating a memory T cell response targeting specific proteins may be important for fighting the virus and for long-term immunity. Combining these findings with Poseida's off-the-shelf allogeneic T cell product approach, which has generated a very high percentage of stem cell memory T cells (Tscm) and has the potential to yield hundreds of doses from a single manufacturing run using proprietary booster molecule technology, may provide both an immediate therapeutic effect, as well as longer-term immunity.

TScan recently announced data generated by their TCR/Target Discovery platform identifying the precise targets of T cells isolated from COVID-19 convalescent patients, along with the TCRs that recognize them. A core set of targets were shared among patients with the same human leukocyte antigen (HLA) type and were primarily located outside the spike protein. A paper describing this work is currently in press at Immunity, a Cell Press journal. TScan worked with clinical partner Atlantic Health Systems to recruit recovering COVID-19 patients for this work. Combining these findings with Poseida's off-the-shelf allogeneic T cell product platform may provide an accessible cell-based therapy for patients with insufficient T cell responses.

Under the terms of the Agreement, Poseida will gain access to TScan's data and IP related to TCR sequences and targets potentially valuable in treating COVID-19, and TScan will provide TCR expertise and counsel to the research collaboration. Detailed financial terms of the collaboration were not disclosed.

About Poseida Therapeutics, Inc.Poseida Therapeutics is a clinical-stage biopharmaceutical company dedicated to utilizing our proprietary gene engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure. We have discovered and are developing a broad portfolio of product candidates in a variety of indications based on our core proprietary platforms, including our non-viral piggyBac DNA Modification System, Cas-CLOVER site-specific gene editing system and nanoparticle- and AAV-based gene delivery technologies. Our core platform technologies have utility, either alone or in combination, across many cell and gene therapeutic modalities and enable us to engineer our wholly-owned portfolio of product candidates that are designed to overcome the primary limitations of current generation cell and gene therapeutics.

About TScan TherapeuticsTScan discovers and develops transformative T cell therapies (TCR-T) to treat liquid cancers, solid tumors, and other serious diseases. Our proprietary, high-throughput platform identifies previously uncharacterized, clinically-derived shared T cell antigens and all off-target TCR interactions, to enable the development of highly efficacious TCR-Ts with minimal off-target effects. Lead program TSC-100 is expected to enter clinical development for liquid cancers in 2021, and the Company is advancing additional TCR-Ts for solid cancers. TScan was co-founded by Chair Christoph Westphal (Partner, Longwood Fund) based on pioneering research from the Elledge Lab at Brigham and Women's Hospital. The Company has raised over $80 million to date from leading strategic collaborators and investors including Longwood Fund, Novartis Institutes for Biomedical Research, Astellas Venture Management, Novartis Venture Fund, Bessemer Venture Partners, GV, 6 Dimensions Capital, and Pitango Venture Capital.

Forward-Looking StatementsStatements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon Poseida's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with development and regulatory approval of novel product candidates in the biopharmaceutical industry and the other risks described in Poseida's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Poseida undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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Poseida Therapeutics and TScan Therapeutics Announce Research Collaboration and License Agreement to Explore Developing Allogeneic T Cell Receptor...

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Facts & Factors (FnF),a leading market research company recently published a research report onResearch Report on Cancer Gene Therapy Market Size & Share 2020 Covering COVID-19 Impact Analysis and Forecast to 2026to its research database. This[190+] PDF pages Cancer Gene Therapy Marketreport analyzes the comprehensive overview of the market comprising an executive summary that covers core trends evolving in the market.

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Cancer Gene Therapy Market Size & Share Insights on Growing Applications by 2026 - re:Jerusalem

Recommendation and review posted by Bethany Smith

Report is a detailed study of the Cell and Gene Therapy market, which covers all the essential information required by a new market entrant as well as the existing players to gain a deeper understanding of the market. The primary objective of this research report named Cell and Gene Therapy market is to help making reliable strategic decisions regarding the opportunities in Cell and Gene Therapy market.

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Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST.

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The Global Cell and Gene Therapy Market segments and Market Data Break Down are illuminated below:

By Therapy Type:Cell TherapyStem CellsT CellsDendritic CellsNK CellsTumor CellsGene TherapyGlobal Cell and Gene Therapy Market, By Indication:Cardiovascular DiseaseCancerGenetic DisorderInfectious DiseaseNeurological DisordersOthers

The Cell and Gene Therapy market report offers the current state of the market around the world. The report starts with the market outline and key of the Cell and Gene Therapy market which assumes a significant job for clients to settle on the business choice. It additionally offers the key focuses to upgrade the development in the Cell and Gene Therapy market. Some fundamental ideas are likewise secured by reports, for example, item definition, its application, industry esteem chain structure and division which help the client to break down the market without any problem. Also, the report covers different factors, for example, arrangements, efficient and innovative which are affecting the Cell and Gene Therapy business and market elements.

Download Sample Report of Cell and Gene Therapy Market Report 2020 (Coronavirus Impact Analysis on Cell and Gene Therapy Market)

Competitive Analysis has been done to understand overall market which will be helpful to take decisions. Major players involved in the manufacture of Cell and Gene Therapy product has been completely profiled along with their SWOT. Some of the key players include Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST. It helps in understanding their strategy and activities. Business strategy described for every company helps to get idea about the current trends of company. The industry intelligence study of the Cell and Gene Therapy market covers the estimation size of the market each in phrases of value (Mn/Bn USD) and volume (tons). Report involves detailed chapter on COVID 19 and its impact on this market. Additionally, it involves changing consumer behavior due to outbreak of COVID 19.

Further, report consists of Porters Five Forces and BCG matrix as well as product life cycle to help you in taking wise decisions. Additionally, this report covers the inside and out factual examination and the market elements and requests which give an entire situation of the business.

Regional Analysis for Cell and Gene Therapy

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Chapters Define in TOC (Table of Content) of the Report:

Chapter 1: Market Overview, Drivers, Restraints and Opportunities, Segmentation

Overview

Chapter 2: COVID Impact

Chapter 3: Market Competition by Manufacturers

Chapter 4: Production by Regions

Chapter 5: Consumption by Regions

Chapter 6: Production, By Types, Revenue and Market share by Types

Chapter 7: Consumption, By Applications, Market share (%) and Growth Rate by

Applications

Chapter 8: Complete profiling and analysis of Manufacturers

Chapter 9: Manufacturing cost analysis, Raw materials analysis, Region-wise

Manufacturing expenses

Chapter 10: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 11: Marketing Strategy Analysis, Distributors/Traders

Chapter 12: Market Effect Factors Analysis

Chapter 13: Market Forecast

Chapter 14: Cell and Gene Therapy Research Findings and Conclusion, Appendix, methodology and data source

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The qualitative contents for geographical analysis will cover market trends in each region and country which includes highlights of the key players operating in the respective region/country, PEST analysis of each region which includes political, economic, social and technological factors influencing the growth of the market. The research report includes specific segments by Type and by Application. This study provides information about the sales and revenue during the historic and forecasted period of 2020 to 2027.

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Covid-19 Impact On Cell and Gene Therapy Market Rising Trends Forecast to 2027 | Leading Players Amgen, Biogen, BioMarin Pharmaceuticals,...

Recommendation and review posted by Bethany Smith

INCOG BioPharma Services, a biopharmaceutical manufacturing services startup, announced on Oct. 15 that it will be establishing its first manufacturing facility and global headquarters in Fishers, Indiana. In addition, it will develop 150 new positions by the end of 2024.

The new facility will be approximately 60,000 square feet in size, and the company is investing $60 million in the construction of the building. With this facility in place, INCOG BioPharma will be able to launch its manufacturing operations and support biopharmaceutical businesses seeking to advance and accelerate early phase clinical projects.

Indiana is globally recognized as a leader in life sciences, ranking second in the nation for total exports," said Governor Eric J. Holcomb. "As a state, were committed to continuing to fuel this sector by providing the business-friendly environment and skilled workforce that companies like INCOG BioPharma need to launch operations and create quality careers for Hoosiers. We're grateful INCOG BioPharma chose to establish its global headquarters in the Hoosier state and look forward to supporting their growth in central Indiana."

More than 65 team members will be added to the company over the course of the next two years. Hiring will begin in early 2021 for positions in engineering, manufacturing sciences, quality assurance and other sectors.

Over the past several months, we evaluated several states and cities to identify the optimal location for our new venture," said Cory Lewis, founder and CEO of INCOG BioPharma. "It became clear that the state of Indiana and the city of Fishers was the ideal location, given the vast amount of life science industry experience that exists in the state. By locating our business in Fishers, we will have access to a large talent pool of skilled and trained resources. INCOG BioPharma is excited to partner with the state of Indiana and the city of Fishers as we build a life science company with a passion for customer service and a long-term vision focused on sustainability for both our employees and customers."

INCOG BioPharma is not the only company expanding in the biopharmaceutical industry. Pfizer, for example, announced back in August that it would be making a half billion dollar investment in the construction of its new gene therapy manufacturing facility. The structure, which is currently located in Sanford, N.C., will continue to support Pfizers investment in gene therapy research and development, similar to its Chapel Hill and Kit Creek, N.C., facilities.

This investment will further strengthen Pfizers leadership in gene therapy manufacturing technology, said Mike McDermott, President, Pfizer Global Supply. The expansion of the Sanford site is expected to create hundreds of highly skilled jobs, which would increase Sanfords high-tech manufacturing environment and is part of our overall plan to invest approximately $5 billion in U.S.-based capital projects over the next several years.

Through this new investment, the company will expand its end-to-end capabilities in gene therapy by investing in all stages of research, development and manufacturing. Pfizer will also become better suited to produce and supply both clinical- and commercial-scale quantities of potentially life-altering gene therapy medicines to patients with rare diseases.

At Pfizer, our purpose is breakthroughs that change patients lives, said Angela Hwang, Group President, Pfizer Biopharmaceuticals Group. Were excited to build this new state-of-the-art facility in Sanford because it will have the potential to help us develop novel methods to deliver transformative treatments to patients.

Originally posted here:
INCOG BioPharma Paves the Way for New Jobs with Expansion in Indiana - BioSpace

Recommendation and review posted by Bethany Smith

Global Gene Therapy for Inherited Genetic Disorders Market: Overview

Rapid advances in mammalian DNA sequencing technologies over the past several years have enabled the identification of the aberrant genes responsible for a vast spectrum of genetic disorders. Gene therapy as a novel approach inarguably holds profound potential in finding universal therapeutic alternatives to treating inherited genetic disorders. Gene therapy for inherited genetic disorders entails introducing a functional copy of the defective gene to make up for the missing function, and can be accomplished using in vivo or ex vivo gene transfer.

Gene therapy for inherited genetic disorders has generated groundswell of interest in the research fraternity in finding cure for or in treatment of Mendelian genetic error causing rare diseases. Particularly, gene therapy in recent years has held promising potential in the treatment of a range of recessive gene disorders most notably sickle cell anemia, hemophilia, muscular dystrophy, cystic fibrosis, and other monogenic disorders. The axes of developments in the gene therapy for inherited genetic disorders market have been in the U.S., Europe, China, and Australia.

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Global Gene Therapy for Inherited Genetic Disorders Market: Notable Developments

Growing body of clinical studies done on mice models have unrivalled troves of preclinical data, which bodes well for the effectiveness of gene therapy for inherited genetic disorders. New approaches in the gene therapy for inherited genetic disorders market are being adopted to bring progress in this direction. In this regard, Salmeterol, a medicine approved for asthma, has shone a new light. The vasodilator to be used along with gene therapy has shown potential in increasing the effectiveness of the therapy for Glycogen storage disease type II (Pompe disease).

A team of investigator led by the researcher at Duke University Medical School discussed the preclinical data recently at 2019 annual meeting of the American Society of Gene & Cell Therapy. The preclinical data showed that the Asthma medicine reduces the accumulation of toxic glycogen accumulated in lysosome. The researchers concluded that it holds potential as an adjunctive therapy, and building on that may pave way for novel approaches on gene therapy for inherited genetic disorders.

Efforts to translate the findings of clinical research on gene therapy for inherited disorders to make the therapy a part of standard treatment has caught momentum in recent times. In this regard, vectors containing non-viral vectors have attracted the attention of scientists. A team of researchers at Fred Hutchinson Cancer Research Center in 2019 found that gold nanoparticles enable them to deliver gene-editing tools to blood stem cells in lab models. This might, they opined, pave way for more practicaland accessiblegene therapies for inherited disorders, notably for treating life-threatening blood disorders. Gene therapies were mediated by CRISPR. In the coming years they hope to collaborate with companies with commercial interest to develop the therapy for patient populations.

Some of the bigplayerseyeing promising stakes in the gene therapy for inherited genetic disorders market areSpark Therapeutics Inc., Orchard Therapeutics, Novartis AG, bluebird bio Inc., and BioMarin Pharmaceutical.

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Global Gene Therapy for Inherited Genetic Disorders Market: Key Drivers

Since 2000, scores of clinical trials involving patients with inherited genetic disorders have raised hopes of the medical fraternity of the potential of gene therapies. Thus far, more than 5000 clinical trials on gene therapy have been conducted, especially for hard-to-treat diseases. Diseases such as inherited blindness and leukemia have seen the efficacy and safety of gene therapies. Advances in bioengineering are expected to invigorate pre-clinical pipelines. In the not-so-distant future, success of more protocols will catalyze the prospects of the gene therapy for inherited genetic disorders market.

Further, advances have been made in viral and non-viral vectors with the purpose of making gene transfer more efficient, thereby boosting the gene therapy for inherited genetic disorders market. Particularly, new approaches emerged with the aim of making vectors more powerful.

Global Gene Therapy for Inherited Genetic Disorders Market: Regional Assessment

On the regional front, Asia Pacific bears considerable potential in the gene therapy for inherited disorders market. Of note, numerous strategic alliances have shifted their focus on the region, particularly China. The North America market has also been rising at a promising pace, driven by several gene-therapy tools and related drugs in the final stages of clinical trials. Favorable reimbursement models has also encouraged research into the gene therapy for inherited disorders.

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Gene Therapy for Inherited Genetic Disorders Market Robust pace of Industry during 2018-2028 - The Think Curiouser

Recommendation and review posted by Bethany Smith

NEW YORK, Oct. 21, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or the "Company") today announced that the National Institutes of Health has awarded Actinium a Small Business Technology Transfer grant to support a clinical collaboration with Memorial Sloan Kettering Cancer Center ("MSK") to study Iomab-ACT, Actinium's CD45-targeting Antibody Radio-Conjugate, for targeted conditioning to achieve lymphodepletion prior to administration of a CD19-targeted CAR T-cell therapy developed at MSK. The CD19 CAR-T has been previously studied by MSK in a Phase 2 trial with chemotherapy conditioning in patients with relapsed or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL) or diffuse large B-cell lymphoma (DLBCL). MSK will lead this first of its kind study to utilize targeted radiopharmaceutical ARC-based lymphodepletion to replace chemotherapy-based conditioning prior to CAR T-cell therapy. The study will assess the feasibility of using Iomab-ACT targeted lymphodepletion prior to MSK's 19-28z CAR-T and assess safety and efficacy outcomes relative to results with MSK's CAR-T 19-28z in patients who had received chemotherapy-based lymphodepletion prior to CAR-T administration.

Results published in the New England Journal of Medicine with MSK's 19-28z CD19 CAR-T in 53 patients with R/R B-ALL reported complete remissions in 83% (44/53) of patients. Median event-free survival (EFS) was 6.1 months and median overall survival (OS) was 12.9 months at a median follow up period of 29 months (range 1 65 months) for all patients. Patients with low disease burden, defined as less than 5% blasts in the bone marrow, had markedly enhanced outcomes with increased median EFS of 10.6 months and median OS of 20.1 months. There was a 26% (14/53) rate of Grade 3 of greater cytokine release syndrome (CRS), with 1 patient death as a result, and 42% of patients experienced Grade 3-4 immune effector cell-associated neurotoxicity syndrome (ICANS). In addition to improved duration of response and survival, patients with low disease burden prior to receiving CAR T-cell therapy had lower rates of CRS and neurotoxicity.1

"We are excited to be collaborating with MSK on this trial as they are aleader in the field of cellular therapies. We selected MSK's 19-28z CAR T-cell therapy for this NIH grant funded collaboration because ithas produced high response rates in patients with relapsed or refractory B-ALL who have previously undergone several lines of standard therapy. However, toxicities such as cytokine release syndrome and neurologic toxicity, as well as durability of response, remain a challenge as is the case with many other CAR T-cell therapies" commented Dr. Mark Berger, Actinium's Chief Medical Officer. "Iomab-ACT enables the delivery of targeted radiation that selectively and specifically targets immune cells, including those implicated in the CAR-T-associated toxicities of cytokine release syndrome and neurotoxicity. We are hopeful that this study will demonstrate improvements in safety and outcomes with MSK's CAR 19-28z as a result of Iomab-ACT targeted lymphodepletion and that this will allow clinicians to make important improvements in patients' ability to receive CAR T-cell therapies."

CAR-T is a type of cellular therapy in which a patient's own (autologous) T-cells are genetically engineered outside of the body to target the patient's cancer cells and which are then reinfused back into the patient to seek out and kill cancer cells. Currently there are 2 approved CD19 targeted CAR-T therapies, which both require chemotherapy-based conditioning to deplete the patient's lymphocytes, known as lymphodepletion, and many other CAR-T constructs in development that also use chemotherapy conditioning for lymphodepletion.

Iomab-ACT targets cells that express CD45, an antigen found on immune cells such as lymphocytes and macrophages as well as leukemia and lymphoma cancer cells and delivers the radioisotope warhead iodine-131 to achieve cell depletion. Iomab-ACT is intended to deplete CD45+ immune cells such as macrophages that are implicated in CAR-T related toxicities and may also have an anti-tumor effect on chemo-refractory cancers. Iomab-ACT is a low dose extension of Actinium's lead program, Iomab-B, which is being studied in a pivotal Phase 3 trial for targeted conditioning prior to a bone marrow transplant. Preclinical data supporting Iomab-ACT's application in targeted lymphodepletion prior to ACT such as CAR-T was recently published in the journal Oncotarget (https://www.oncotarget.com/archive/v11/i39/). In addition, clinical data with trace doses of Iomab-B has shown transient, reversible lymphodepletion in patients and drug clearance pharmacokinetics that fit within the vein to vein time of CAR-T manufacturing and administration.

Sandesh Seth, Actinium's Chairman and CEO, said, "This clinical trial collaboration with MSK is a strong step forward for Actinium and our targeted conditioning program. The 19-28z CAR-T has already produced promising data and we look forward to working with MSK to explore Iomab-ACT's potential to reduce toxicities and improve patient outcomes. As we advance towards the SIERRA interim analysis in the fourth quarter, we are focused on the continued expansion of our ARC-based targeted conditioning program for bone marrow transplant and cell and gene therapies with the goal of providing targeted conditioning regimens that are less toxic and more effective than current chemotherapy-based conditioning. With these therapies being administered in a select number of concentrated centers, we see a large and growing market opportunity where our ARC-based targeted conditioning can improve outcomes and increase access to these important curative treatment options."

Iomab-ACT Program Update Call DetailsWebcast link: https://ir.actiniumpharma.com/presentations-webinarsDate: October 22, 2020Time: 8:00 AM EDT

Source:

1) Park et al. Long-Term Follow-up of CD19 CAR Therapy in Acute Lymphoblastic Leukemia. N Engl J Med 2018;378:449-59. DOI: 10.1056/NEJMoa1709919

About Actinium Pharmaceuticals, Inc.

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively kill patient's cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, CAR-T and other cell therapies or gene therapy to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, Iomab-B is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and promising single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. Beyond Iomab-B, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 120 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc.

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update, including but not limited to, statements relating to the Company's expectations regarding the intended use of proceeds of the public offering. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the SEC, including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Investor Contact:

Clayton RobertsonActinium Pharmaceuticals, Inc. [emailprotected]

Hans VitzthumLifeSci Advisors, LLC[emailprotected] P: (617) 430-7578

SOURCE Actinium Pharmaceuticals, Inc.

http://www.actiniumpharma.com/

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Actinium Pharmaceuticals, Inc. Awarded Grant by National Institutes of Health to Study Novel Iomab-ACT Targeted Conditioning with a CD19 CAR T-Cell...

Recommendation and review posted by Bethany Smith

-- Dr. Carl Regillo to present OPTIC data at the Retina Subspecialty Day onFriday, November 13, 2020 at 2:15 pm PT --

-- Company to host webcast with key opinion leaders on Saturday, November 14, 2020 at 7:30 am PT --

REDWOOD CITY, Calif., Oct. 20, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced the presentation of additional clinical data for Cohorts 1-4 in the OPTIC Phase 1 clinical trial of ADVM-022 intravitreal injection gene therapy in wet age-related macular degeneration (wet AMD). This will be in the form of a live broadcast during the American Academy of Ophthalmology (AAO) 2020 Virtual Meeting.

Presentation of New OPTIC Data:Event: Retina Subspecialty Day 2020 Virtual MeetingTitle: Intravitreal Gene Therapy with ADVM-022 for Neovascular AMD: OPTIC Phase 1 StudyDate: November 13, 2020Time: 2:15 pm PTPresenter: Carl Regillo, M.D., F.A.C.S, chief of retina services at Wills Eye HospitalPresentation: At the beginning of Dr. Regillos presentation, Adverum plans to issue a press release and post the new data presentation under Events and Presentations in the Investors section of the companys website.

Presentation of Existing OPTIC Data:Event: AAO 2020 Virtual MeetingTitle: Phase 1 Study of Intravitreal Gene Therapy with ADVM-022 for Neovascular AMD (OPTIC Trial Cohorts 1-4, from the August 10, 2020 data presentation)Date: November 14-17, 2020Presenter: Arshad M. Khanani, M.D., M.A., managing partner and director of clinical research, Sierra Eye Associates, clinical associate professor of ophthalmology, University of NevadaPresentation: On or before November 13, 2020 the virtual presentations will be available on demand to AAO participants, and Adverum plans to post the presentation under Events and Presentations in the Investors section of the companys website.

Adverum Webcast:Date: November 14, 2020Time: 7:30 9:00 am PT (10:30 am 12:00 pm ET)Presenters:

The live video webcast will be accessible under Events and Presentations in the Investors section of the company's website. The archived audio webcast will be available on the Adverum website following the call and will be available for 30 days.

On November 5, 2020 after market, Adverum plans to report recent business progress and financial results for the third quarter of 2020. The Company plans to host the KOL webcast on November 14, 2020 to review new OPTIC data instead of hosting a quarterly conference call this quarter.

About Adverum BiotechnologiesAdverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is advancing the clinical development of its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of patients with wet age-related macular degeneration and diabetic macular edema. For more information, please visit http://www.adverum.com.

More:
Adverum Biotechnologies to Present New Data from the OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD at AAO 2020 Virtual -...

Recommendation and review posted by Bethany Smith

Paris, October 21, 2020 SparingVision (the Company), a genomic medicine company focused on ocular diseases, today announces a 44.5 million financing round. The round was led by 4BIO Capital (4BIO) and UPMC Enterprises, and included Jeito Capital (Jeito)and Ysios Capital (Ysios). In addition, current investors Bpifrance and Foundation Fighting Blindness (FFB) participated in the round.

Proceeds from the financing will be primarily used to advance the development of SparingVisions breakthrough treatment SPVN06 for the mutation-agnostic treatment of retinitis pigmentosa (RP). Most notably the funding will support SparingVisions GMP activities (including the manufacturing of a first clinical batch of the product, currently ongoing), the IND/CTA regulatory activities and the conduct of a first-in-man study, scheduled to start in 2021. The Company also intends to further expand its management team and commence operations in the US.

SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy consisting ofone neurotrophic factor and one oxidative stress reducing enzymewhich, acting synergistically, aim to slow or stop the degeneration of photoreceptors. Loss of photoreceptors leads to blindness in RP, one of the most common inherited retinal diseasesthat affects two million patients worldwide.There is currently no treatment approved for RP patients independently of their genetic background.

Torreya Capital, LLC served as exclusive placement agent for the offering.

SparingVision also announces today that the Companys Chairman of the Board, Stphane Boissel, has been appointed Chief Executive Officer of the Company.

Stphane Boissel, SparingVisions President and Chief Executive Officer, said,We are delighted to have closed this financing round, which demonstrates the excitement around SparingVisions lead compound, SPVN06. With its singular mutation-agnostic approach, SPVN06 could have a much broader commercial potential than most gene therapy products for RP currently in development and will be used as an anchor to build an economically-viable portfolio of therapies in the field of ophthalmology. Our shareholders, both new and existing, are all long-term, strategic and patient-centric investors that share our vision and we are excited to be working with them to achieve our goals.

Before joining SparingVision as President and CEO, Stphane Boissel was Executive Vice President, Corporate Strategy at Sangamo Therapeutics (Sangamo), a Nasdaq-listed gene-editing company based in San Francisco, California. Prior to Sangamo, he was CEO of TxCell, the first ever CAR-TReg company, that was sold to Sangamo in 2018. Prior to TxCell, Stphane served as CEO of Genclis, a molecular diagnostic company, and EVP and CFO of Innate Pharma, a Nasdaq-listed company, and Transgene.

Stphane has also been a Board member of several leading biotechnology companies, including Nasdaq-listed Erytech Pharma and Elsalys Biotech, where he served as Chairman. Earlier in his career, Stphane worked in investment banking for Lazard, where he focused on principal investment in France, Singapore and Hong Kong. He started his career at PWC. Stphane graduated in management and finance from the IAE Lyon, University of Lyon and PSL Paris-Dauphine University (France) and received his MBA from the University of Chicago Booth School of Business.

Dr. Jos-Alain Sahel, Chair of the Department of Ophthalmology at the University of Pittsburgh School of Medicine, Director of the Institut de la Vision (Sorbonne Universite, Inserm, CNRS, Paris) and co-founder ofSparingVision along with Dr. Thierry Lveillard, added,SparingVisions neuroprotective approach, which I started working on some 20 years agoat Institut de la Vision, has all the attributes of a winning gene therapy solution for retinitis pigmentosa patients in desperate need of a universal therapeutic option. I am looking forward to helping Stphane and the team best position SPVN06 for clinical development up to regulatory licensing.

The Board of Directors of SparingVision expresses its deepest gratitude to Florence Allouche, co-founder, who is leaving her position as President of the Company, for her hard work and support over the years which has enabled the Company to pass many milestones and successfully refinance itself.

**ENDS**

Contacts:

SparingVision

Stphane Boissel

President and CEO

Stanislas Piot

CFO

Consilium Strategic Communications

Amber Fennell, Olivia Manser, Lizzie Seeley

+44 (0)20 3709 5700

sparingvision@consilium-comms.com

NOTES TO EDITORS:

About SparingVision:

SparingVision is a biotechnology company focused on the discovery and development of innovative therapies for the treatment of blinding inherited retinal diseases. SparingVision is developing SPVN06, a gene-independent treatment for retinitis pigmentosa, the most common inherited retinal degeneration. There is currently no treatment to treat all genetic forms of this rare retinal disease that leads to blindness and affects nearly 2 million people worldwide. SparingVision is a spin-off of the Paris Vision Institute. SparingVisions investors include4BIO Capital,Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre,UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visitwww.sparingvision.com

About 4BIO Capital:

4BIO Capital isan international venture capital firm focused solely on the advanced therapies sector. 4BIOs objective is to invest in, support, and grow early stage companies developing treatments in areas of high unmet medical need, with the ultimate goal of ensuring access to these potentially curative therapies for all patients. Specifically, it looks for viable, high-quality opportunities in cell and gene therapy, RNA-based therapy, targeted therapies, and the microbiome.

The 4BIO team comprises leading advanced therapy scientists and experienced life science investors who have collectively published over 250 scientific articles in prestigious academic journals includingNature,The Lancet,Cell, and theNew England Journal of Medicine. 4BIO has both an unrivalled network within the advanced therapy sector and a unique understanding of the criteria that define a successful investment opportunity in this space.

For more information, please visitwww.4biocapital.com

About UPMC Enterprises:

UPMC Enterprises is the innovation, commercialization, and venture capital arm of UPMC, a $21 billion health care provider and insurer based in Pittsburgh. With an emphasis on translational science and digital solutions, UPMC Enterprises provides its portfolio companies and partners with capital, connections and resources to develop solutions to health cares most complex problems. Working in close collaboration with innovators from UPMC and the University of Pittsburgh Schools of the Health Sciences, as well as others worldwide, UPMC Enterprises strives to accelerate science from the bench to the bedside and has committed to investing $1 billion in novel drugs, diagnostics and devices by 2024.

For more information, please visitwww.enterprises.upmc.com

About Jeito Capital:

Jeito Capital is an international investment company with a patient benefit driven approach that focuses both on financing ground-breaking medical innovation and promoting positive societal impact. Jeito has a unique, long-term investment strategy, with the aim of providing continuity from clinical development to market access for breakthrough drugs with validated proofs of concept. This continuity is expressed both in the support provided to entrepreneurs by the Jeito Capital team and by the investment of significant capital to ensure the growth of companies, the acceleration towards commercialization and faster access to these major innovations. Jeito Capital is based inPariswith a presence inEuropeand the United States.

About Ysios Capital:

Ysios Capital is a leading Spanish independent venture capital firm that provides private equity financing to early and mid-stage life science companies that develop disruptive therapeutic products and platform technologies. Founded in 2008, Ysios Capital manages c.a. 350 M through three dedicated funds in life sciences and is led by a team of professionals experienced in building biotech companies.For more information, please visit:www.ysioscapital.com

About Bpifrance:

Bpifrance is the French national investment bank. It finances businesses at every stage of their development through loans, guarantees, equity investments and export insurances. Bpifrance also provides extra financial services (training, consultancy) to help entrepreneurs meet their challenges (innovation, export). For more information, please visit:www.bpifrance.comandpresse.bpifrance.fr. Follow us on Twitter: @Bpifrance @BpifrancePresse

About the Foundation Fighting Blindness:

Established in 1971, the Foundation Fighting Blindness is the worlds leading private funding source for retinal degenerative disease research. The Foundation has raised more than $800 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of retinal degenerative diseases including: retinitis pigmentosa, age-related macular degeneration, Usher syndrome, and Stargardt disease. VisitFightingBlindness.orgfor more information.

AboutFondation Voir et Entendre:

Created in May 2007, the Foundation"Voir et Entendre"'s challenges are to defy sensory handicaps linked to vision and hearing, both of which have become public health stakes. As we can see throughout industrialized countries ocular and auditory diseases are gaining ground, mainly due to the ageing populations.

The Foundation therefore brings together the best-known French and international scientific teams, hospital services and industries around common research themes, with the aim to rapidly find new and adapted treatments.

Within the Foundation is the research center Vision Institute managed by Jos-Alain Sahel, the Clinical Investigation Centre of the National Ophthalmologic Hospital Centre (CHNO) of the Quinze-Vingts, reference centers for rare diseases and four clinical hospital departments.

The head office is located at the Vision Institute, itself within the CHNO of the Quinze-Vingts in Paris. The latter has supported this project from the outset along with the Pasteur Institute, National Institute of Health and Medical Research (INSERM), Pierre and Marie Curie University and the French Federation of the Blind and Visually Impaired.

https://www.institut-vision.org/fr/institute/2-uncategorised/8-the-foundation.html

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SparingVision Raises 44.5 Million and Appoints Stphane Boissel as Chief Executive Officer - BioSpace

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Swiss pharma giant Roche has partnered with Cambridge, Massachusetts-based Dyno Therapeutics for the development of gene therapies for central nervous system (CNS) and liver diseases.

The companies will utilise Dynos CapsidMap platform to develop adeno-associated virus (AAV) vectors for gene therapies targeting a range of CNS and liver-directed diseases.

Dynos CapsidMap artificial intelligence platform will be used to identify novel AAV capsids it does this by optimising tissue targeting and immune-evading properties, as well as improving packaging capacity and manufacturability, according to Dyno.

Dynos technology uses artificial intelligence to design novel capsids, the cell-targeting protein shell of viral vectors. Current gene therapies are limited to small number of naturally occurring AAV vectors, which face challenges with delivery, immunity, packaging size and manufacturing.

As part of the collaboration, Dyno will be responsible for the design of the novel AAV capsids, while Roche and its Spark Therapeutics division will develop the capsids in preclinical studies, with the aim of taking them through clinical development and to commercialisation.

In return for access to its capsid identifying technology, Dyno will receive an undisclosed payment as well as further payments related to research, clinical and sales milestone payments.

In addition to potential royalties for any resulting products developed from the collaboration, Dyno could be eligible to receive payments over a total value of $1.8bn.

This new partnership represents Dynos largest collaboration to date, and we are excited to work with Roche and Spark Therapeutics to expand the frontier of gene therapies for the central nervous system and liver, said Eric Kelsic, chief executive officer and co-founder of Dyno.

"The Dyno, Roche and Spark teams share a bold vision for gene therapy and believe that enhancing vectors that deliver these therapies is key to developing new treatments for patients in need, he added.

The Roche deal is Dynos third pharma collaboration, in addition to its existing partnerships with Novartis and Sarepta Therapeutics.

In May, Dyno and Novartis announced that they had signed a deal focused on developing improved gene therapies with AAV vectors for research, development and commercialisation of gene therapies for ocular disease.

The Sarepta deal is focused on the development of new AAV vectors for gene therapies aimed at targeting muscle diseases.

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Roche to collaborate with Dyno on gene therapies for CNS/liver diseases - PMLiVE

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MONTPELLIER, France--(BUSINESS WIRE)--Regulatory News:

Sensorion (Paris:ALSEN) (FR0012596468 ALSEN), a pioneering clinical-stage and gene therapy biotech company which specializes in the development of novel therapies to treat, prevent and restore within the field of hearing loss disorders, today announces its interim annual results as of June 30, 2020 alongside its outlook for 2020.

Sensorions landmark gene therapy agreement with Institut Pasteur was a major inflection point in the Companys development. The positive preliminary preclinical data from the program targeting the OTOF gene reinforces the potential of this partnership, which expands our pipeline and supports our goal to become a leader in the field of hearing loss. The Phase 2 trial of SENS-401 in sudden sensorineural hearing loss is progressing and we expect results in mid-2021. We were delighted to successfully close an oversubscribed private placement in September, which raised 31 million. As part of this financing, we were pleased to see renewed support from existing shareholders such as Invus and Sofinnova Partners and to welcome new high-quality US and European investors. Following this financing, we estimate the cash in hand will carry us through to H2 2022 comments Nawal Ouzren, CEO of Sensorion.

First-half 2020 financial results

The half-year accounts as of June 30, 2020, drawn up according to IFRS standards and approved by the Board of Directors on October 20, 2020, have been duly reviewed by statutory auditors.

The simplified income statement as of June 30, 2020 is as follows:

In Euros IFRS standards

30.06.2020

30.06.2019

Operating income

902,203

1,042,407

Research and Development expenses

3,661,766

5,226,883

General and Administrative expenses

1,915,400

1,257,185

Total operating expenses

5,577,166

6,484,068

Operating profit/loss

-4,674,963

-5,441,662

Financial profit/loss

-44,031

-22,929

Net profit/loss

-4,718,994

-5,464,591

On June 30, 2020, Sensorions operating income, mainly the research tax credit, amounted to 0.9m, compared to 1.0m on June 30, 2019.

Operating expenses decreased by 14%, down from 6.5m on June 30, 2019 to 5.6m on June 30, 2020, mainly due to a 1.5m reduction in research costs partially offset by a 0.7m increase in G&A expenses.

The research and development expenses decreased by 30%, down from 5.2m on June 30, 2019 to 3.7m on June 30, 2020 following the halting of the SENS-111 program in December 2019 and the slowdown of expenditure on the SENS-401 clinical trial as a result of delays due to the COVID-19 pandemic.

G&A expenses are up 52%; they amounted to 1.9m on June 30, 2020, compared with 1.3m on June 30, 2019 mainly due to the increase in personnel expenses.

Operating losses on June 30, 2020 thus amounted to 4.7m, compared with a loss of 5.4m on June 30, 2019.

Net loss amounted to -4.7m on June 30, 2020, compared with -5.5m on June 30, 2019.

As of June 30, 2020, the company employed 24 people.

Financial structure

On February 10, 2020, Invus Public Equities LP converted all the 12,500,000 convertible bonds (CBs) it had subscribed for in June 2019 into ordinary shares in the Company. The conversion was undertaken on a price basis of 0.76 per share. Following this operation, Invus held 20,591,259 ordinary shares and 42.29% of the share capital and voting rights in Sensorion.

On February 13, 2020, Sofinnova Crossover I SLP converted all the 7,500,000 convertible bonds (CBs) it had subscribed for in June 2019 into ordinary shares in the Company. The conversion was undertaken on a price basis of 0.76 per share. Following this operation, Sofinnova Crossover I SLP held 11,822,258 ordinary shares and 20.19% of the share capital and voting rights in Sensorion.

Equity capital amounted to 28.7m on June 30, 2020, compared with 13.2m on June 30, 2019.

As of June 30, 2020, cash and cash equivalents amounted to 30.7m compared with 30.4m on December 31, 2019.

On September 18, 2020, Sensorion successfully raised 31m of gross proceeds before deducting underwriting commissions and estimated expenses payable by the Company.

Based on its forecasted expenses, the cash position of 30.7m at June 30, 2020 and the net proceeds from the offering, the Company believes it will be able to fund its operations until the second half of 2022.

Key developments: Science and research & development

In the second half of 2019, Sensorion launched two preclinical gene therapy programs targeting Usher Syndrome type 1 and Otoferlin deficiency, two monogenic forms of hereditary deafness. Under the framework agreement signed with Institut Pasteur in May 2019, other projects could also emerge in the area of genetic disorders of the inner ear. During the five years partnership agreement, Sensorion has preferred rights to the genetic disorders of the inner ear research pipeline of Institut Pasteur and the ability to implement collaborations leading to a license. These programs are conducted under the sponsorship of Professor Christine Petit, Director of the French Hearing Institute and Chair of our Scientific Advisory Board.

On June 9, 2020, Sensorion announced positive preliminary preclinical data from its gene therapy program targeting Otoferlin deficiency. In vivo experiments conducted in non-human primates (NHPs) show good safety and promising preliminary data on inner ear tissue tropism and the achievement of a high transduction rate efficiency.

The SENS-401 Phase 2 clinical trial in the treatment of sudden sensorineural hearing loss (SSNHL) in adults is a randomized, double-blind and placebo-controlled study, aiming to recruit ~260 patients. It is being conducted in 11 countries at approximately 30 sites in Europe and Canada.

On February 17, 2020, Sensorion received Ethics Committee approval to include new military sites in the SENS-401 Phase 2 study. The new centers will recruit volunteer military personnel exposed to extreme noise during their professional activities and suffering from hearing loss.

On March 13, 2020, Sensorion provided an update on the SENS-401 SSNHL Phase 2 AUDIBLE-S trial enrollment. Patient recruitment rates from this trial now indicate the data will be available by mid-year 2021, which is later than previously announced. An important factor resulting in delayed recruitment in the trial was the reallocation of emergency room resources due to the COVID-19 situation.

The independent Data Safety Monitoring Board (DSMB) undertook a review of the safety data for the patients included in the Phase 2 clinical trial on June 5, 2020. It confirmed the absence of any concern on the safety of SENS-401 and recommended continuing the trial as scheduled.

Following the agreement signed in December 2017, Sensorion and Cochlear (world leader in cochlear implants) have continued their collaboration. Thanks to its otoprotective properties demonstrated in several preclinical models, SENS-401 could potentially preserve residual hearing in patients with cochlear implants. Since 2018, we have successfully conducted additional safety studies to assess the feasibility of long-term treatment with SENS-401 that may be required in cochlear implant indications. Preclinical data from these studies are expected by the end of 2020.

Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases enabling it to select the best targets and modalities for drug candidates in the field of small molecules and gene therapy. This platform makes it possible to carry out a panel of investigations ranging from histology and cell culture (in vitro) to behavioral and electrophysiological tests (in vivo). The Company is also working on the identification of biomarkers to improve diagnosis and treatment of these illnesses with a high unmet medical need.

On January 30, 2020, Sensorion presented new SENS-401 preclinical data at the ARO (Association for Research in Otolaryngology) Mid-Winter Meeting 2020. A poster and oral presentation highlighted the potential to significantly reduce hearing loss from chronic noise exposure in a rat model. A second poster featured the age-related hearing loss with significant early decline in functional auditory measures in Wistar rats.

Capital breakdown after the September 2020 capital increase

Sensorions financial position strengthened further at the end of September 2020 following a 31m capital increase. The capital raise was achieved at a share price which was a 3.5% discount over the weighted average share price on the day preceding the date on which the issuance price was set (the Reserved Offering).

The completion of this capital increase was, amongst others, supported by existing shareholders, Invus Public Equities LP, Sofinnova Partners and WuXi AppTec.

To the best of the Companys knowledge the capital structure on a non-diluted basis before and after the private placement is as follows:

Shareholders

Number of shares before the Reserved Offering (1)

% of the share capital before the Reserved Offering

% of voting rights before the Reserved Offering

Number of shares after the Reserved Offering (1)

% of share capital after the Reserved Offering

% of voting rights after the Reserved Offering

Subscription

(in )

Inserm Transfert Inititiative

982,911

1.68%

1.68%

982,911

1.28%

1.28%

-

Innobio (Bpifrance)

3,499,874

5.98%

5.98%

3,499,874

4.56%

4.56%

-

Management, employees and directors

160,000

0.27%

0.27%

160,000

0.21%

0.21%

-

Cochlear

533,755

0.91%

0.91%

533,755

0.70 %

0.70%

-

Invus Public Equities LP

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Sensorion reports 2020 first half results - Business Wire

Recommendation and review posted by Bethany Smith

Four years after a Phase I/II setback in spinal muscular atrophy (SMA), Novartis is hoping its drug branaplam will find more success in a new neurological indication: Huntingtons disease.

The decision was announced a year after the head of research, Jay Bradner, said he did not see a big opportunity in SMA, according to Reuters. Novartis says it has preclinical data showing that branaplam reduces levels of mutant huntingtin protein, and SMA data showing patients on the drug had reductions in huntingtin mRNA. The FDA gave branaplam their orphan drug designation, and Novartis plans to move forth with a Phase IIb trial next year.

Novartis intends to start a development program for branaplam to determine if it has the potential to be a transformative treatment for people living with this devastating condition, the company said in a statement.

Novartis isnt dropping the SMA indication altogether according to a company statement, branaplam is still under investigation for SMA. But a 2-year testing hiatus set Novartis behind its rival Roche, which won approval for its SMA therapy Evrysdi last year.

In 2016, Novartis halted enrollment in a Phase I/II study in SMA after signs of nerve injury appeared in some animal tests, according to a letter it released. The trial was resumed about 2 years later in Belgium, Germany, Denmark and Italy, with additional nerve tests added to the protocol.

It is still a long road, but we are pleased to be back on track, the letter read.

Evrysidi is priced by weight, beginning at less than $100,000 for infants under 15 pounds and capping at $340,000 per year. Analysts predicted that the drug would threaten Biogens SMA drug Spinraza, which comes in at $382,500 on the low-end. But Mani Foroohar of SVB Leerink predicted Novartis gene therapy Zolgensma would take the lead in young SMA patients. Given strong data, one-time therapy, and simple IV administration, we see Zolgensma as remaining the treatment of choice for very young SMA patients, he wrote, according to aFierce Pharma report.

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Years after a major trial setback, Novartis switches gears with SMA drug. This time they're trying it for Huntington's - Endpoints News

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COVID-19 impact will also be included and considered for forecast.

Global Gene Therapy Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level.

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By Type of Vectors Viral vectors Non-viral vectors

By Type of Cells Somatic cells Germline cellsBy Region North Americao U.S.o Canadao Mexico Europeo UKo Franceo Germanyo Russiao Rest of Europe Asia-Pacifico Chinao South Koreao Indiao Japano Rest of Asia-Pacific LAMEAo Latin Americao Middle Easto AfricaGene Therapy Market Key Players: Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc. Boehringer Ingelheim International GmbH uniQure N.V. bluebird bio, Inc. Celgene Corporation OthersThis comprehensive report will provide: Enhance your strategic decision making Assist with your research, presentations and business plans Show which emerging market opportunities to focus on Increase your industry knowledge Keep you up-to-date with crucial market developments Allow you to develop informed growth strategies Build your technical insight Illustrate trends to exploit Strengthen your analysis of competitors Provide risk analysis, helping you avoid the pitfalls other companies could make Ultimately, help you to maximize profitability for your company.Our Market Research Solution Provides You Answer to Below Mentioned Question: Which are the driving factors responsible for the growth of market? Which are the roadblock factors of this market? What are the new opportunities, by which market will grow in coming years? What are the trends of this market? Which are main factors responsible for new product launch? How big is the global & regional market in terms of revenue, sales and production? How far will the market grow in forecast period in terms of revenue, sales and production? Which region is dominating the global market and what are the market shares of each region in the overall market in 2017? How will each segment grow over the forecast period and how much revenue will these segment account for in 2025? Which region has more opportunities?

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Which are the driving factors responsible for the growth of market?

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How big is the global & regional market in terms of revenue, sales and production?

How far will the market grow in forecast period in terms of revenue, sales and production?

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Gene Therapy Market: 2020 Opportunities, Key Players, Competitive and Regional Analysis by Forecast 2025 - The Think Curiouser

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