In the years since I was diagnosed with hypothyroidism, each day seems to bring about a new and unexpected symptom. But putting the pieces together and discovering that my hypothyroidism was negatively affecting my sex life felt like a gut-punch, even more so than finding out about the gastrointestinal problems or night sweats. This aspect of my life that had come to feel so wholly and totally mine was just another thing to add to the list of things stolen by this illness.

The thyroid is a butterfly-shaped gland located at the base of the throat. It controls the speed at which every cell of the body operates a process known as metabolism. When the thyroid is off, it isnt able to properly produce T3, T4, and TSH (Thyroid Stimulating Hormone) which are the hormones that communicate to the body how each bodily process should go. This can affect literally everything from digestion to brain function to fertility.

Before having a thyroidectomy, I didnt even know what the thyroid was. I was completely unprepared for how it would affect my life, but even more how it would affect my sexual health as doctors arent always so keen on talking about these things with patients. When I was being prepared for life post-thyroid, not once did any of my doctors mention how it would affect my menstrual or sexual health. And, as it turns out, thyroid disease has totally changed my sex life.

Hormonal disordersincluding an under-active thyroidare responsible for about one third of all sexual problems people experience, says Dr. Vedrana Hgqvist Tabor, PHD, CEO of Boost Thyroid. Low T4 (one of two thyroid produced hormones) or high TSH (thyroid stimulating hormone) are to blame.

The imbalance of these hormones can cause hyperprolactinemia, or elevated levels of prolactin in the blood. Prolactin is made by the pituitary gland and affects the level of estrogen and testosterone in people. When T4 and TSH are out of whack, it can also cause the sex hormone binding globulin (SHBG) levels to change, which can throw off the estrogen, testosterone and progesterone balance. Low thyroid hormone levels can cause changes in blood vessels and nerve sensitivity, resulting in reduced sex drive, breast pain, painful vaginal penetration, and vaginal dryness.

Sexual disorders are typically experienced more by people who have fatigue, muscle pain, and/or feel depressed, says Dr. Hgqvist Tabor. These are all common side-effects of a thyroid condition, so tracking can help patients understand when these symptoms worsen and improve.

Thyroid disease can cause a variety of symptoms that affect a persons sex life from low sex drive, difficulty getting aroused, erectile dysfunction, vaginal dryness, inability or difficult reaching orgasm, delayed ejaculation and pain during or after vaginal intercourse.

Awareness depends on three factors: the patient, the physician, and the country, says Dr. Hgqvist Tabor. It depends whether the patient will share any sexual problems with their physician, whether the physician is aware of the thyroid and sex connection and their countrys guidelines for physicians.

Sexual health is a major part of a persons health, and why these sexual side-effects have been continuously ignored or glossed over by my doctors when hypothyroidism affects approximately 10 million Americans (and as many as 10 percent of women experience some thyroid hormone deficiency) still sort of baffles me. If I had known what to look for, I may have been clued-in earlier when I first had a dip in my hormone levels or when my birth control was interacting weirdly with my thyroid meds. Being sexually well is part of being totally well and ignoring that doesnt do anything but hurt patients.

View original post here:
The Common Health Condition That Could Be Killing Your Sex Life - SheKnows

Recommendation and review posted by Bethany Smith

Lee When Ray Garnett moved to the Berkshires five years ago, finding a primary care physician was at the top of his to-do list. I was looking for a specific model of care, he explained in a recent phone interview. Garnett, who identifies as a transgender individual, was looking for hormone replacement therapy via informed consent, which, in many places in the country, trans people can access at Planned Parenthood. There is no Planned Parenthood in Great Barrington, where Garnett lives and works, and the closest equivalent Tapestry Health did not provide this service. So he went to Springfield to have his medical needs met.

Ray Garnett, founder of Berkshire County Trans Group. Photo: Ashley Shade

Good LGBTQ care is really just good care, is how Dr. Jeremy Stoepker sees it. In creating an open, nonjudgmental, collaborative environment where the standards of care are met no matter who you are, what your background is, or what your disease process or screening requirements may be. Its just good care, he said in a recent phone interview. Stoepkers arrival to the Berkshires, just six months ago as a provider, is changing the landscape of accessible health care for individuals who identify as LGBTQ, despite the fact that ones sexual orientation and gender identity should be moot points when it comes to accessing health care.

From the very beginning, I had role models and inspiration for how it can be done well seeping into my little brain, said Stoepker, who was 7 years old when his father was diagnosed with HIV.I dont remember the journey so much as I remember the players, he added, and they were amazing. Stopeker remembers his fathers doctors as loving and caring, supportive and nonjudgmental. And really up-to-date, he added in a nod to his fathers participation in the first clinical trial for the first HIV medication and survival for 20 years following his diagnosis. This remains Stoepkers inspiration in his day-to-day work: caring for his patients while honoring his fathers legacy.

Stoepker is realistic about the examples of bad care that abound in any discipline of medicine, particularly primary care. As to how this manifests in the LGBTQ community? People just arent up to speed with the type of screening that is recommended, he said.Take the triple swab (or triple-site) testing for gonorrhea and chlamydia in men who have sex with men. A lot of providers do not even know what that is or what that means, said Stoepker, who explained that, potentially, one should be tested in the rectum, urethra and the back of the throat. Not getting the standard of care to which a patient is entitled is how this looks in its most benign form; in more upsetting instances, it is doctors saying they do not feel comfortable treating patients or openly shaming patients and being accusatory. Which, unfortunately, happens to lots of patients, including those in the LGBTQ community, Stoepker.

Alex Reczkowski of Pittsfield. Photo: Hannah Van Sickle

Alex Reczkowski of Pittsfield experienced this with his former primary care physician. It started with a routine conversation at his annual physical, one sparked by a startling statistic Reczkowski had heard: that 50 percent of men of color who have sex with men are estimated to be diagnosed with HIV before they die. This number made him pause. As a single, gay man, he decided to look into PrEP pre-exposure prophylaxis, a one-a-day pill to prevent HIV infection. Reczkowski was looking for peace of mind, even if the medication was not necessary, and wanted to explore his options. His doctor assured him this was not necessary. After a second appointment, for the express purpose of getting a PrEP prescription, Reczkowski was denied.The conversation turned to shaming and the need to include a specialist on infectious disease, which didnt feel great. Needless to say, the appointment deteriorated. If you cant meet my needs, I think we are done here, is how Reczkowski left it.

Reczkowski ultimately made his way to Community Health Programs, where he connected with Dr. Stoepker, who relocated from New York City in June to join the primary care team at CHPs Lee Family Practice. In addition to a prescription for PrEP, Reckowski got the most up-to-date information about the HPV vaccine, previously available only for those under 27 years of age. Stoepker knew that the FDA had just approved the vaccine for anyone under the age of 45, and he listened to me, he didnt lecture me and wasnt interested in giving advice for someone else he was really interested in me, which, Reczkowski feels, really speaks to the vision of CHP: It values an inclusive culture that champions patient-centered care. I feel like that is exactly what I experienced, he said. Exceptional, compassionate health care.

The Williams Institute at the UCLA School of Law says about 4.5 percent of U.S. adults 11 million people identify as LGBT; about 1.4 million people identify as transgender. Massachusetts law explicitly prohibits discrimination based on gender identity, sexual orientation and HIV status. But as Garnett puts it, there is friendly, and there is competent. If [a doctor] is not putting out that they are [trans] friendly, Im not even going to call or show up, he explained. And Stoepker understands this. Not getting up-to-date care is [a persistent problem] in all arenas: If it only affects 10 percent of the population, there are going to be some doctors who are up to speed and some doctors who arent, he said. He calls this one of the big problems with medicine. Once you have that end of residency, the training requirements are incredibly flexible; what you do to educate yourself as a physician is highly variable, he said. While LGBTQ-specific training and separate clinics specifically dedicated to providing quality care to those who identify as LGBTQ are being incorporated into medical schools and residencies, there is an underlying truth: Its going to be a generation of physicians before its truly a frame shift, Stoepker said.

From Reczkowskis perspective, it boils down to education. Cultural competency training [is necessary] so we can understand how other people perceive the world and how we can best interact [and] live together; this is what the medical institutions need to be doing as well, he said. In fact it is imperative at the doctors office. In a separate incident with a different provider, Reczkowski ran into trouble with regard to a prostate exam, one of which could be done in the office, rendering it immediate and not very expensive, while the other had expensive lab costs associated with it. Reczkowskis doctor was so uncomfortable, he would not do the prostate exam. It is [the doctors] job to make me feel comfortable about something that can be awkward; now you are making me feel more awkward because you feel uncomfortable and dont have the cultural competency to understand [my position]?

This is part of the ongoing conversation Garnett is facilitating as founder of the Berkshire County Trans Group. I think any time a person makes an effort to say, I am friendly, I am willing to learn, that is great. Health care is a hot topic at monthly meetings across the county,where informal lists of recommendations abound. Dr. Stoepker currently tops this list as one of the only openly LGBTQ-supportive providers in the area. Its the first thing you do when you move to a new area: Who is your doctor? Who is your dentist? Garnett said. Its difficult to see a new provider when you dont really know [what you are walking into], Garnett added, even when the issue for which you might need support is not trans- or LGBT-related. Garnett addressed the topic of labels, often seen as divisive. The value of labels comes when they are self-chosen, he explained. We choose them so we are not alone; having labels helps us find each other; it helps us to hear about someone elses experience, and it helps us to put our own experiences in context with other people.

This is not a local problem. Stoepker recalls his practice in New York City (he moved to the Berkshires 18 months ago but spent a year communing to the city). Every day I was seeing a new patient who said, This is New York City and it boggles my mind, but I was not getting affirming, supportive care at my PCPs office. So I Googled around and found you, which is why he has worked to create a safe zone in his office by providing informed, compassionate medical care to LGBTQ patients not individuals living with HIV or AIDS, who are under the care of specialists, rather individuals seeking good primary care. Its what keeps him motivated in his work: The privilege of doing what I do is amazing, and Im excited about it every day; the ability to honor my dads memory, and the tradition of amazing doctors he had that I was inspired by. But mostly its just because, when you do something the right way and people appreciate it, it feels really good.

Excerpt from:
CHP's Dr. Stoepker helping to change the landscape of accessible health care for LGBTQ infividuals | - theberkshireedge.com

Recommendation and review posted by Bethany Smith

NEW YORK (PRWEB) January 10, 2020

January is Thyroid Awareness Month, and physicians from the Hilda and J. Lester Gabrilove Division of Endocrinology, Diabetes and Bone Disease and the Department of Otolaryngology Head and Neck Surgery at the Mount Sinai Health System are emphasizing the importance of being aware of symptoms that may be related to thyroid disease.

The thyroid gland is located in the front of the neck and under the voice box. It produces hormones that help the body control the rate of metabolism, and regulate the production and consumption of energy. When thyroid function is accelerated, the condition is called hyperthyroidism; when slowed, it is called hypothyroidism. Imbalances in thyroid function may be a result of environmental, autoimmune, or genetic factors. Additionally, thyroid issues may lead to cancer.

Thyroid disease affects roughly 200 million people worldwide, and thyroid cancer is on the rise, with roughly 52,000 new cases diagnosed in 2019, according to the American Cancer Society. Three out of four thyroid cancer diagnoses are made in women. Data from the American Thyroid Association shows that more than 12 percent of the U.S. population will develop a thyroid condition in their lifetime, and the cause of these problems is largely unknown. An estimated 20 million Americans have some form of thyroid disease and up to 60 percent of them dont know they have it, so they go undiagnosed and untreated. Women are five to eight times more likely than men to have thyroid issues, and one in eight women will develop a thyroid disorder. People with a family history of thyroid disease and/or thyroid cancer, and exposure to high doses of radiation, are also at increased risk, said Terry Davies, MD, Co-Director of the Mount Sinai Thyroid Center at Union Square and Professor of Medicine (Endocrinology, Diabetes and Bone Disease) at the Icahn School of Medicine at Mount Sinai.

How to Perform a Thyroid Neck Self-Exam:

Symptoms and Facts about Thyroid Disease

Thyroid Disease and Pregnancy Pregnant women should be aware of changes to their thyroid gland, which can be affected by different levels of pregnancy hormones. The thyroid hormone greatly contributes to the development of a healthy baby, and it is important that expectant mothers be properly diagnosed with and treated for thyroid disease. Otherwise, they could be at higher risk of miscarriage or preterm delivery, and their children may have developmental delays. For that reason, thyroid function is routinely checked in pregnant women.

When it comes to thyroid cancer, a large number of women develop this during their reproductive age. Since thyroid cancer tends to be mediated by hormones in the body, it tends to grow faster when patients are pregnant. There is no special cancer screening recommendation for pregnant women.

Mount Sinai Is a Leader in Noninvasive Thyroid Treatment

Radiofrequency Ablation for Thyroid Nodules Mount Sinai West is one of only two hospitals in New York State offering a minimally invasive procedure to treat non-cancerous thyroid nodules that are symptomatic and would have otherwise required invasive surgery for removal. The procedure is called radio-frequency ablation (RFA). It offers eligible patients a much quicker recovery, less pain and risk of infection, and no scarring. With RFA, surgeons use guided ultrasound to deliver radio-frequency current to heat up and shrink the thyroid nodule. RFA can be done on patients with large non-cancerous nodules that cause swallowing, voice, breathing, and neck discomfort.

Patients who undergo RFA can return to normal activity the day after the procedure and can exercise within several days. Additionally, they are extremely unlikely to require permanent thyroid hormone medication. Patients who have standard thyroid nodule surgery typically cant resume normal activity for at least a month and 20 to 30 percent of these patients require thyroid medication.

Radiofrequency ablation for thyroid nodules has been performed in Korea for over a decade and throughout Europe and their outcomes are excellent. The published data shows impressive nodule shrinkage rates of more than 80 percent with RFA that is maintained over years of follow-up, explained Catherine Sinclair, MD, Associate Professor of Otolaryngology at the Icahn School of Medicine at Mount Sinai and Director of Head and Neck Surgery at Mount Sinai West. Thyroid nodules are very common and, although many people will never require any intervention for their nodules, there is a significant minority who will seek treatment due to symptoms. I expect RFA to be a terrific new option for these patients.

Ethanol Ablation for Thyroid NodulesAnother noninvasive procedure, performed at Mount Sinai-Union Square, ethanol ablation is when an alcohol solution is injected into thyroid nodules, killing cells and causing the masses to slowly shrink. The procedure leaves only a small scar and is performed in office with local anesthesia.

The candidates for RFA and ethanol ablation are very similar, says Maria Brito, MD, Co-Director of the Mount Sinai Thyroid Center at Union Square. Dr. Brito and Michael Via, MD, both Associate Professors of Medicine (Endocrinology, Diabetes and Bone Disease) at the Icahn School of Medicine at Mount Sinai, are two of only a handful of physicians in the New York metropolitan region to perform ethanol ablation.

The procedures will not necessarily eliminate the nodule completely, and patients will still need to have ultrasound follow-ups to monitor the nodule, Dr. Brito says. But in appropriate cases, they are a terrific option. They make it very easy for the patient.

About the Mount Sinai Health System

The Mount Sinai Health System is New York City's largest integrated delivery system, encompassing eight hospitals, a leading medical school, and a vast network of ambulatory practices throughout the greater New York region. Mount Sinai's vision is to produce the safest care, the highest quality, the highest satisfaction, the best access and the best value of any health system in the nation. The Health System includes approximately 7,480 primary and specialty care physicians; 11 joint-venture ambulatory surgery centers; more than 410 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. The Icahn School of Medicine is one of three medical schools that have earned distinction by multiple indicators: ranked in the top 20 by U.S. News & World Report's "Best Medical Schools", aligned with a U.S. News & World Report's "Honor Roll" Hospital, No. 12 in the nation for National Institutes of Health funding, and among the top 10 most innovative research institutions as ranked by the journal Nature in its Nature Innovation Index. This reflects a special level of excellence in education, clinical practice, and research. The Mount Sinai Hospital is ranked No. 14 on U.S. News & World Report's "Honor Roll" of top U.S. hospitals; it is one of the nation's top 20 hospitals in Cardiology/Heart Surgery, Diabetes/Endocrinology, Gastroenterology/GI Surgery, Geriatrics, Gynecology, Nephrology, Neurology/Neurosurgery, and Orthopedics in the 2019-2020 "Best Hospitals" issue. Mount Sinai's Kravis Children's Hospital also is ranked nationally in five out of ten pediatric specialties by U.S. News & World Report. The New York Eye and Ear Infirmary of Mount Sinai is ranked 12th nationally for Ophthalmology, Mount Sinai St. Lukes and Mount Sinai West are ranked 23rd nationally for Nephrology and 25th for Diabetes/Endocrinology, and Mount Sinai South Nassau is ranked 35th nationally for Urology. Mount Sinai Beth Israel, Mount Sinai St. Luke's, Mount Sinai West, and Mount Sinai South Nassau are ranked regionally.

For more information, visit https://www.mountsinai.org or find Mount Sinai on Facebook, Twitter and YouTube.

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January Is Thyroid Awareness Month: Mount Sinai Doctors Offer Unique Procedures for Thyroid Nodules and Stress Importance of Early Detection - PR Web

Recommendation and review posted by Bethany Smith

Theres a feeling that sweeps over many people this time of year, and it might be summed up best with a twist on a famed quote from the cult movie classic Office Space.

Looks like somebodys got a case of the Januarys.

January is the time of year when the combination of less daylight, colder weather, less activity, and a letdown from the holiday vibe can lead to feelings of isolation, sadness, and in extreme situations, depression.

Why does it happen?

Experts point to a combination of things.

Its almost a perfect storm of seasonal happenings, according to Marna W. Brickman, LGSW, a psychotherapist with Spectrum Behavioral Health in Annapolis, Maryland.

There are the shorter days, the colder weather that means people are isolating themselves more, the less physical activity that can come with this time of year, and seasonal affective disorder, she told Healthline.

A combination of some or all of these factors, Brickman said, can lead to that January blues vibe and even depression.

Leigh Leader of Michigan knows the January feeling.

The mother of two in her early 30s battles a case of the Januarys each year.

You know what it feels like when you look out your window sometimes and just see that dull, snow, cold, gray world? Thats what it feels like, she told Healthline. Some days I wake up and just want to stay under the covers and not move. But I have two small daughters, so I have to.

Phyllis Perkins, a mother of teens also from Michigan, fights the feeling as well.

Before I realized I could take action, all I wanted to do this time of year was get the kids from school, cook dinner, and then go to bed, she told Healthline.

For a while, Perkins thought she was physically ill. But after a panel of tests by her physician showed no physical issue, she said they began to put the pieces together and find a way to help her improve her January mood.

So whats a person like Leader or Perkins to do?

There are steps you can take now, and proactively in years to come, to better your frame of mind and mood in the winter months.

Brickman points to elevated fatigue, oversleeping, isolating yourself, and a sense of helplessness as symptoms people should be aware of.

She says they should take note if they experience any or all of them.

What could be causing those symptoms and what pushes it over to depression?

There is an underlying neurological basis for major depressive disorder with a seasonal pattern, in which people have difficulty regulating the neurotransmitter, serotonin, which is responsible for balancing mood, Dr. Ash Nadkarni, associate psychiatrist and director of wellness at Brigham and Womens Hospital in Boston, told Healthline.

Nadkarni explained that people may also have difficulty with overproduction of melatonin, a hormone produced by the pineal gland that responds to darkness by causing sleepiness.

In the winter, melatonin production increases, causing sleepiness and lethargy, she said. Both decreased serotonin and increased melatonin impact circadian rhythms, or the bodys internal 24-hour clock, which responds to light-dark changes that occur daily and throughout each of the seasons, she said.

In people with depressive disorders, she said, the circadian signal that indicates a seasonal change in day length has been found to be timed differently, challenging the bodys ability to adjust.

In other words, our bodies crave warmth, sun, and light.

There are steps you can take now if youre feeling blue, said Brickman.

Find natural daylight. Find the sun if you can, she said.

Brickman also suggests investing in a light box, in particular one that treats mood disorders and not skin disorders.

One added suggestion: change your diet.

Even healthy eaters can shift to a looser dietary pattern in the holidays and in cold, dark January eating that way can magnify sad feelings.

High-fat, sugary diets are tough on the mind, Brickman said. People dont think enough about food and how it impacts all of this.

She also suggests looking at using eating and food in a positive way by making plans and meeting up with friends for meals.

Eat socially, she said. Everything is better with company.

Perkins uses light therapy. Her physician suggested replacing her kitchen lights with bright 75 watt bulbs and shes found that helped.

She also has another suggestion: pick up or reclaim a hobby. For her, crafting does wonderings.

Its sounds so silly but something simple like this really can help, Perkins said. For me, crafting keeps my mind occupied and makes me feel useful, inspired and busy.

Its also important to report your symptoms to your physician or therapist and take any medical action they may deem necessary.

Seeking treatment is important, said Nadkarni. Antidepressants, light therapy, vitamin D to replete any deficiency, and psychotherapy have all been found to be evidenced-based options for treatment.

Brickman agrees.

Talk therapy is a great treatment for this, she said. Antidepressants, if needed, can also be a fix for 90 days or until the days extend and weather changes.

Leaders physician does something Brickman suggests ups her dosage of vitamin D for this time of year.

Its funny, she said. I never really believed something like that would make a difference until I tried it. It really helps.

Signs that you may sink into a case of January seasonal affective disorder or actual depression may begin to show months before, Nadkarni said.

When major depressive disorder has a seasonal pattern, it most often tends to be associated with an increase in symptoms in October or November, when light patterns first begin to shift, she said.

What to do?

Nadkarni says pay attention to the signs and be aware of them possibly showing up in your life.

When you see one or more, proactively battle those feelings with these suggestions.

How is one to be motivated to do that?

Both Perkins and Leader say people should simply try to avoid the painful sadness theyve fought in the past.

Leader has one more tip: just do it.

I have two small children, so I really have no choice but to try to stay ahead of this and focus on not having it drag me down, she said. Having two little kids to get out of bed and take care of every day is motivation to be mentally healthy for that.

For people without children, she urges them to find a place and a way you are needed.

That makes it all so much more important, and that makes it get your focus, she said.

View original post here:
How to Keep a Sunny Outlook During the Dreary Days of January - Healthline

Recommendation and review posted by Bethany Smith

In the months after the gene therapy infusion at Boston Childrens, her symptoms disappeared. But doctors had given her blood transfusions while she regrew her own red blood cells, so it was not clear if the absence of symptoms was because of the gene therapy or the transfusions.

As she recovered, Helen returned to her passion: dancing. One day, she came back from her school dance group and told her mother, My legs hurt. It feels funny. Ms. Cintron smiled. Thats soreness, she explained. Helen laughed. She had only known pain from sickle cell.

Helen was scheduled for her six-month checkup on Dec. 16. By then, all the transfused cells were gone, leaving only blood made by stem cells in her own marrow. The doctors would finally tell her whether the therapy was working.

The day before, she and her parents visited the New England Aquarium in Boston. She was able to stay outside on a cold, blustery day, watching one seal bully the others, barking and fighting. When Helen mentioned that her hands were cold, Ms. Cintrons stomach clenched in fear. But it was just a normal thing to feel on a winter day.

The next morning, Dr. Esrick delivered the news. Helens total hemoglobin level was so high it was nearly normal a level she had never before achieved even with blood transfusions. She had no signs of sickle cell disease.

Now you are like me, her father told her. I jump in the pool, I run. Now you can do it, too!

Her family, accustomed to constant vigilance, is only now getting used to normal life.

On Dec. 23, Helen and her mother flew to the familys new home in Arizona.

Helen recently described her transformed outlook on Facebook.

Excerpt from:
At 16, Shes a Pioneer in the Fight to Cure Sickle Cell Disease - The New York Times

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Global Gene Therapy Market Outlook and Projections, 2019-2027" report has been added to ResearchAndMarkets.com's offering.

Incidences of cancer are increasing, and the local governing bodies are taking required initiatives to handle the problem, by development of new healing and preventive gene therapeutics.

According to the World Bank, more than 2,300 clinical trials on gene therapy were carried out in 2017. This led to attainment of higher information about the potential applications and working of gene therapies. Besides, gene therapy is becoming a significant sector in bioscience industry research and can be produced on a commercial-scale, which is projected to further expand its application range. Yescarta - axicabtagene ciloleucel are the most recently approved gene therapeutics available in the global market that treats large B-cell lymphoma.

The global gene therapy market is estimated to grow on the back of rising healthcare expenditure with increasing demand for efficient disease treating practices coupled with growing technological developments and discoveries. The World Bank reported a rise in the global current health expenditure (% GDP) from 9.453% in 2011 to 10.023% in 2016.

Increasing incidences of chronic oncogenic diseases such as cancer with an estimated new cases of 18.1 million in 2018 as per the International Agency for Research on Cancer (IARC), is anticipated to display a rapid growth in application of gene therapy technologies in the upcoming years. Additionally, increasing application of gene therapy products for the treatment of various cardiovascular and blood disorders is also expected to back the rampant growth in the upcoming years.

Moreover, government initiatives to eliminate chronic diseases is anticipated to aid the growth in upcoming years. For instance, the World Health Organization (WHO) launched an initiative to eliminate hepatitis completely by 2023. Furthermore, experiments concerning gene therapy technologies in order to explore the benefits for various medical applications is another factor expected to propel the market growth.

Rising geriatric population across the globe holds immense opportunities for gene therapy products in the upcoming years. According to the World Bank, the population aged 65 and above, increased from 7.64% in 2010 to 8.93% of the global population in 2018. Moreover, change in climatic conditions and increase in sedentary lifestyles has led to drastic demographic changes in developed and developing countries, resulting in growing number of disease cases associated with aging. This aspect is attributed to contribute considerably to the market growth and thereby is expected to increase the adoption of gene therapy products across the globe.

The patient pool suffering from diabetes is expanding with growing aging population. The International Diabetes Federation reported that approximately 425 million adults (20-79 years) were living with diabetes in 2017, which is estimated to increase by 2045 to 629 million. Application of gene therapy in patients suffering with diabetes, wherein replacement of a single gene is expected to restore the function of beta cells, which secrete insulin and regulate the blood glucose level of the body is also envisaged to boost the market significantly, during the forthcoming decade.

Key Topics Covered

Introduction

Market Ecosystem

Global Gene Therapy Market Outlook

Industry Analysis

Market Dynamics

Company Profiles of Top 5 Players

For more information about this report visit https://www.researchandmarkets.com/r/ptfbs8

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Global Gene Therapy Market Outlook and Projections, 2019-2027, Featuring Profiles of the Top 5 Players: Novartis, Amgen, Orchard Therapeutics, Kite...

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--44 week median follow up for patients (n=6)----Zero anti-VEGF rescue injections required following intravitreal ADVM-022; First patient has reached 52-weeks post treatment----Vision remains stable and anatomical improvements maintained--

MENLO PARK, Calif., Jan. 11, 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 clinical data for the first cohort of patients (n=6) in the OPTIC phase 1 clinical trial of ADVM-022, the companys intravitreal injection gene therapy, in treatment-experienced patients with wet age-related macular degeneration (wet AMD). The data are being presented today by Charles C. Wykoff M.D., Ph.D., director of research, Retina Consultants of Houston, at the Atlantic Coast Retina Club Macula 20/20 Annual Meeting inNew York, NY.

A copy of the presentation is available on the Adverum corporate website under Events and Presentations in the Investors section, available here.

In October 2019, Adverum presented data from the first cohort in OPTIC at a median 34-week time point (28-44 week range). Today, additional data for the first cohort are being presented, including efficacy and safety data, with a median follow up of 44 weeks at a range of 40-52 weeks, and included:

As of December 1, 2019, ADVM-022 continues to be well-tolerated in the first cohort with no drug-related or procedure-related serious adverse events (SAEs), no drug-related systemic adverse events and no adverse events meeting the criteria for dose-limiting toxicities (DLTs). Low-grade inflammation was reported in all six patients and was generally mild to moderate and responsive to steroid eye drops. One ocular SAE, a retinal detachment, that was not related to ADVM-022 or the administration procedure was reported.

OPTIC Phase 1 Clinical Trial Data from Cohort 1 (n=6)

1 Best corrected visual acuity (BCVA) as measured by Early Treatment Diabetic Retinopathy Study (ETDRS) (i.e., sight charts) 2 Central retinal thickness (CRT), also referred to as central subfield thickness (CST) assessed using Optical Coherence Tomography (OCT) imaging and measured by an independent Central Reading Center3 BCVA and CST values for patient with retinal detachment (unrelated to study treatment) used last observations prior to detachment 4 This event was deemed unrelated to ADVM-022 or any study procedure

These longer-term follow-up data demonstrate that patients in this first cohort of OPTIC are achieving sustained benefits from ADVM-022, a one-time intravitreal therapy, and have not required any anti-VEGF rescue injections through a median of 44 weeks while demonstrating impressive anatomic improvements, said Charles C. Wykoff M.D., Ph.D., director of research, Retina Consultants of Houston and associate professor of clinical ophthalmology, Blanton Eye Institute, Houston Methodist Hospital and Weill Cornell Medical College, Houston Texas. With a median follow-up period of 44 weeks, ADVM022 continues to control wet AMD disease activity in all 6 patients and the low-grade intraocular inflammation appears manageable with steroid eyedrops. Based on the data to date, ADVM-022 has the potential to be a meaningful and potentially transformative treatment for patients with wet AMD.

Aaron Osborne, MBBS, chief medical officer of Adverum, added, These new clinical data are promising as they continue to support the safety, efficacy, and durable clinical profile of ADVM-022 and this therapys potential to change the treatment paradigm for patients with wet AMD. Anti-VEGF injections, the current standard of care, carry a significant treatment burden and real-world outcomes data suggest that vision outcomes are suboptimal due to undertreatment. In the first cohort of OPTIC, we continue to see stable vision and anatomical improvements being maintained out to a median of 44 weeks after a single ADVM-022 injection in these difficult-to-treat patients who previously required frequent anti-VEGF injections. We look forward to presenting longer-term data from the first cohort and 24-week data from the second cohort of OPTIC on February 8 at the Angiogenesis, Exudation, and Degeneration 2020 symposium.

About the OPTIC Phase 1 Trial of ADVM-022 in Wet AMDThe multi-center, open-label, Phase 1, dose-escalation trial is designed to assess the safety and tolerability of a single intravitreal (IVT) administration of ADVM-022 in patients with wet AMD who are responsive to anti-vascular endothelial growth factor (VEGF) treatment. In the first cohort, patients (n=6) received ADVM-022 at a dose of 6 x 10^11 vg/eye and in the second cohort, patients (n=6) received ADVM-022 at a dose of 2 x 10^11 vg/eye. In the third cohort (n=9), patients also are receiving a dose of 2 x 10^11 vg/eye and in the fourth cohort (n=9), patients will receive a dose of 6x10^11 vg/eye. Patients in the third and fourth cohorts will receive prophylactic steroid eye drops instead of oral steroids which were used in the first and second cohorts. The primary endpoint of the trial is the safety and tolerability of ADVM-022 after a single IVT administration. Secondary endpoints include changes in best-corrected visual acuity (BCVA), measurement of central retinal thickness (CRT), as well as mean number of anti-VEGF rescue injections and percentage of patients needing anti-VEGF rescue injections. Each patient enrolled will be followed for a total of two years.

Eight leading retinal centers acrossthe United States(U.S.) are participating in the OPTIC Phase 1 trial for ADVM-022. For more information on the OPTIC Phase 1 clinical trial of ADVM-022 in wet AMD, please visithttps://clinicaltrials.gov/ct2/show/NCT03748784.

About ADVM-022 Gene TherapyADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is administered as a one-time intravitreal injection, designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance and improve vision outcomes for wet AMD and diabetic retinopathy patients.

In recognition of the need for new treatment options for wet AMD, the U.S. Food and Drug Administration granted Fast Track designation for ADVM-022 for the treatment of this disease.

Adverum is currently evaluating ADVM-022 in the OPTIC Study, a Phase 1 clinical trial in patients 50 years and older with wet AMD. Additionally, Adverum plans to submit an Investigational New Drug Application for ADVM-022 for the treatment of diabetic retinopathy to the U.S. Food and Drug Administration in the first half of 2020.

About Wet Age-related Macular Degeneration (Wet AMD)Age-related macular degeneration (AMD) is a progressive disease affecting the macula, the region of the retina at the back of the eye responsible for central vision. In patients with wet AMD, an aggressive form of AMD, abnormal blood vessels grow underneath and into the retina. These abnormal blood vessels leak fluid and blood into and beneath the retina, causing vision loss.

Wet AMD is a leading cause of vision loss in patients over 60 years of age, with a prevalence of approximately 1.2 million individuals in the U.S. and 3 million worldwide. The incidence of new cases of wet AMD in the U.S. is approximately 150,000 to 200,000 annually, and this number is expected to grow significantly as the countrys population ages.

The current standard-of-care therapy for wet AMD is anti-VEGF intravitreal injections. These are effective but typically require eye injections every 4-12 weeks in order to maintain vision. Compliance with this regimen can be difficult for patients, caregivers, and healthcare systems, leading to undertreatment and resulting in loss of vision.

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

Forward-looking StatementsStatements contained in this press release regarding events or results that may occur in the future are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to statements regarding: Adverums plans to report additional clinical data for ADVM-022 from the OPTIC trial and to advance ADVM-022, including Adverums plans to submit an Investigational New Drug Application for ADVM-022 for the treatment of diabetic retinopathy to the U.S. Food and Drug Administration in the first half of 2020, and the potential benefits of ADVM-022, all of which are based on certain assumptions made by Adverum on current conditions, expected future developments and other factors Adverum believes are appropriate in the circumstances. Adverum may not achieve any of these in a timely manner, or at all, or otherwise carry out the intentions or meet the expectations disclosed in its forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include risks inherent to, without limitation: Adverums novel technology, which makes it difficult to predict the time and cost of product candidate development and obtaining regulatory approval; the results of early clinical trials not always being predictive of future results; the potential for future complications or side effects in connection with use of ADVM-022; obtaining regulatory approval for gene therapy product candidates; enrolling patients in clinical trials; reliance on third parties for conducting the OPTIC trial and vector production; and ability to fund operations through completion of the OPTIC trial and thereafter. Risks and uncertainties facing Adverum are described more fully in Adverums Form 10-Q filed with the SEC on November 7, 2019 under the heading Risk Factors. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Investor and Media Inquiries:

Investors:Myesha LacyAdverum Biotechnologies, Inc.mlacy@adverum.com1-650-304-3892

Media:Cherilyn Cecchini, M.D.LifeSci Communicationsccecchini@lifescicomms.com1-646-876-5196

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Adverum Biotechnologies Reports Additional Clinical Data from First Cohort of OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD at...

Recommendation and review posted by Bethany Smith

DetailsCategory: DNA RNA and CellsPublished on Sunday, 12 January 2020 11:35Hits: 130

Indapta Developing First-in-class, Proprietary Cancer Therapy Based on a Specific, Potent Variety of Natural Killer Immune Cells for Use in Combination with Multiple Monoclonal Antibodies

Lonza to Provide Process Development, Clinical Manufacturing and Regulatory Support for IND Submission

Indapta Emerges from Stealth Mode, Unveils Founding Management Team and Scientific Advisors

SAN FRANCISCO,CA, USA & BASEL, Switzerland I January 10, 2020 I Indapta Therapeutics, Inc., a biotechnology company focused on developing and commercializing a proprietary, first-in-class, off-the-shelf, non-engineered, allogeneic G-NK (FcR-deficient Natural Killer) cell therapy to treat multiple cancers, and Lonza today announced a strategic partnership. Indapta also announced its founding leadership team and scientific advisors.

Under the terms of the agreement, Lonza will manufacture Indaptas off-the-shelf, allogeneic G-NK cell therapy under current good manufacturing practices (cGMP) for use in clinical studies. Indapta will leverage Lonzas process development capabilities and expertise to ensure a robust, reproducible and scalable cGMP process. Process development and manufacturing will take place in Lonza's state-of-the-art cell and gene therapy manufacturing facility in Houston.

We believe our first-in-class, off-the-shelf, allogeneic G-NK cell therapy will drive the next critical phase in the evolution of cancer therapies following CAR T-cell therapies, said Guy DiPierro, founder and chief executive officer of Indapta Therapeutics. Current autologous CAR T-cell therapies have proven efficacy in various hematologic cancers but have been beset with serious clinical and manufacturing challenges. By providing an off-the-shelf solution with our G-NK cell therapy, we can eliminate the need for a patient-specific therapy. Additionally, because our investigational cell therapies are not engineered, they are likely to be more effective, less costly and have a simpler regulatory pathway.

Lonza, with its demonstrated expertise in cell therapy manufacturing, is the ideal strategic partner to help us advance our clinical program and scale the production of our G-NK cell therapy, said Ronald Martell, co-founder and executive chairman of Indapta Therapeutics. We are currently completing Investigational New Drug-enabling studies and plan to submit an IND application in late 2020 and initiate a first-in-human Phase 1/2 study in early 2021.

Indaptas world-class team of NK cell scientists and clinicians and cell therapy experts has created an innovative off-the-shelf immuno-oncology therapy based on a subset of cancer-killing NK cells that could make a truly meaningful impact in the treatment of hematologic malignancies and solid tumors, said Scott Waldman, chief strategy officer at Lonza.

Alberto Santagostino, senior vice president, head of Cell & Gene Technologies at Lonza, added, with our long-standing experience in cell therapy manufacturing, we are committed to providing Indapta with the expertise, resources and services it needs for cGMP manufacturing to advance its promising program into the clinic and beyond.

About Indaptas G-NK Cell Therapy

Indapta Therapeutics is developing off-the-shelf, allogeneic FcRI-deficient NK cells, known as G-NK cells.i,ii,iii These proprietary cells are a specific and potent subset of NK cells with specialized anti-tumor activity when used in combination with a monoclonal antibody. G-NK cells are NK cells that have undergone an epigenetic change after coming into contact with cytomegalovirus (CMV)-infected cells. As a result, they lack the FcRI signaling adapter and, instead, use a different adapter protein, which predisposes them to a far more activated state of antibody-dependent cell-mediated cytotoxicity (ADCC) in the presence of a monoclonal antibody. When the monoclonal antibody binds to the tumor target and to the Fc receptor on G-NK cells, it initiates the release of dramatically more immune-stimulating cytokines and cell-killing enzymes than conventional NK cells, causing the direct killing of tumor cells and driving tumor cell death. G-NK cells have been demonstrated to be safe; in vivo studies demonstrate they do not cause graft-vs-host disease or cytokine release syndrome, which can occur with CAR-T cell therapies.

Preclinical research, conducted under NIH grants by scientists at the University of California, San Francisco (UCSF), demonstrated the safety and efficacy of G-NK cells administered in combination with a therapeutic monoclonal antibody. Clinical models of multiple myeloma and lymphoma demonstrated improved survival, a statistically significant decrease in tumor growth, and a statistically significant increase in the activity of the monoclonal antibody without causing graft-vs-host disease. When administered in combination with a monoclonal antibody, G-NK cells have been shown to be highly persistent (lasting four to nine months), to have the ability to preferentially bind to a therapeutic monoclonal antibody in the presence of a tumor cell, and to demonstrate superior ADCC function compared with conventional NK cells. Under a second Indapta NIH grant, researchers at Stanford University will be conducting in vivo studies in additional tumor models.

Indaptas off-the-shelf G-NK cell therapy is differentiated from an autologous therapy in that it is not necessary to collect cells from each individual patient and produce a unique therapy for every patient. Rather, it is derived from cells from healthy volunteers, which are highly functional and persistent. Indaptas process for producing G-NK cell therapy for use as an immunotherapy involves taking blood from CMV-seropositive donors, identifying and sorting G-NK cells from these samples, and expanding G-NKs cells using the companys proprietary, patented expansion method, which preferentially expands and activates GNK cells. Indapta has also developed a proprietary method for freezing and storing the G-NK cells in a GMP master cell bank for use as off-the-shelf allogeneic outpatient immunotherapy in cancer patients.

Developing off-the-shelf G-NK cells may sidestep some of the clinical and financial challenges presented by other, more customized and engineered immuno-oncology approaches, which involve time-consuming and costly manufacturing processes and often can only be delivered in specialized centers. The manufacturing COGS for Indaptas program will be relatively inexpensive compared to CAR-T or engineered NK cell therapies. Additionally, the regulatory approval process for Indaptas program may be more straightforward than that for autologous CAR-T cell therapy or engineered NK cells because it does not involve complicated cell engineering.

Not only are G-NK cells widely available from multiple sources, they have the potential to be used in combination with multiple monoclonal antibodies to treat numerous types of cancer (e.g., multiple myeloma, lymphoma, leukemia, melanoma, ovarian, colorectal, renal, liver, breast and lung).

Indaptas Founding Management Team

Indaptas Scientific Advisors

About Indapta Therapeutics

Indapta Therapeutics, Inc. is a biotechnology company focused on developing and commercializing a proprietary, first-in-class, off-the-shelf allogeneic cell therapy to treat multiple types of difficult-to-treat hematologic cancers and solid tumors. Headquartered in San Francisco, Indapta was founded in 2017 by Guy DiPierro along with Ronald Martell and scientists at the University of California, Davis, and Stanford University. The company has developed allogeneic FcRI-deficient natural killer (NK) cells, known as G-NK cells, and is working to bring this off-the-shelf cell therapy to patients to address the limitations of currently available autologous T-cell therapies.

About Lonza

Lonza is an integrated solutions provider that creates value along the Healthcare Continuum. Through our Pharma Biotech & Nutrition segment and our Specialty Ingredients segment businesses, we harness science and technology to serve markets along this continuum. We focus on creating a healthy environment, promoting a healthier lifestyle and preventing illness through consumers' preventive healthcare, as well as improving patient healthcare by supporting our customers to deliver innovative medicines that help treat or even cure severe diseases. Patients and consumers benefit from our ability to transfer our pharma know-how to the healthcare, hygiene and fast-moving consumer goods environment and to the preservation and protection of the world where we live.

Founded in 1897 in the Swiss Alps, Lonza today is a well-respected global company with more than 100 sites and offices and approximately 15,500 full-time employees worldwide at the end of 2018. The company generated sales of CHF 5.5 billion in 2018 with a CORE EBITDA of CHF 1.5 billion. Further information can be found at http://www.lonza.com.

Additional Information and Disclaimer

Lonza Group Ltd has its headquarters in Basel, Switzerland, and is listed on the SIX Swiss Exchange. It has a secondary listing on the Singapore Exchange Securities Trading Limited (SGX-ST). Lonza Group Ltd is not subject to the SGX-STs continuing listing requirements but remains subject to Rules 217 and 751 of the SGX-ST Listing Manual.

Certain matters discussed in this news release may constitute forward-looking statements. These statements are based on current expectations and estimates of Lonza Group Ltd, although Lonza Group Ltd can give no assurance that these expectations and estimates will be achieved. Investors are cautioned that all forward-looking statements involve risks and uncertainty and are qualified in their entirety. The actual results may differ materially in the future from the forward-looking statements included in this news release due to various factors. Furthermore, except as otherwise required by law, Lonza Group Ltd disclaims any intention or obligation to update the statements contained in this news release.

i Hwang I, Zhang T, Scott JM, et al. Identification of human NK cells that are deficient for signaling adaptor FcR and specialized for antibody-dependent immune functions. Int Immunol. 2012;24(12);793-802.

ii Lee J, Zhang T, Lanier LL, Kim S. Epigenetic modification and antibody-dependent expansion of memory-like NK cells in human cytomegalovirus-infected individuals. Immunity. 2015;42:431-442.

iii Zhang T, Scott JM, Hwang I, Kim S. Antibody-dependent memory-like NK cells distinguished by FcR deficiency, Immunol. 2013;190(4):1402-1406.

SOURCE: Indapta Therapeutics

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Indapta Therapeutics and Lonza Announce Strategic Partnership to Advance a Next-generation, Off-the-shelf, Allogeneic Immuno-oncology Therapy | DNA...

Recommendation and review posted by Bethany Smith

Cellular engineering promises new treatments for cancer and other maladies. But most manufacturing processes propel the cost of these so-called living drugs into the stratosphere, far beyond reach of most people who need them.

A technology patented at the University of California, Riverside, and recently licensed to startup Basilard BioTech could bring these prices back down to earth.

The technology, developed by Masa Rao, an associate professor of mechanical engineering in the Marlon and Rosemary Bourns College of Engineering, minimizes damage to the cell in the manufacturing process. This enables both high gene delivery efficiency and cellular viability, a feat that most other approaches cannot match.

Basilard spun out of Raos laboratory earlier this year. The company has obtained an exclusive license to commercialize the technology, which they have branded SoloPore. Basilard is seeking to develop it as a disruptive new platform for engineering ex vivo cell and gene therapies for cancer specifically, as well as genetic disorders and degenerative diseases more broadly.

Basilards SoloPore technology is a differentiated solution that provides greater scalability, safety, efficiency, and versatility than prevailing gene delivery methods, said Basilard CEO Brynley Lee. This will allow us to reduce manufacturing cost, and therefore, bring these revolutionary therapies to more of those in need.

Basilard is raising seed capital and working to build a commercial prototype. The young company is the first biotech instrumentation company to emerge from UC Riversides EPIC entrepreneurship incubator, which guides innovators through the commercialization and entrepreneurial process and helps connect them with investors.

Within the span of less than a year, weve gone from a purely academic effort to the formation of a startup thats on the cusp securing its first venture capital funding, Rao said. UC Riversides Office of Technology Partnerships has been instrumental in this rapid ascent.

Weve worked hard for the past three years to accelerate technology translation and commercialization with entrepreneurial programs that have mentored more than 220 entrepreneurs and 120 startups in the Inland Empire since October 2016, said Rosibel Ochoa, associate vice chancellor for technology partnerships. Basilards quick rise is a sign that we are building a healthy entrepreneurial ecosystem that supports the growth of startups in our region.

Read more:
Putting gene therapy in reach - University of California

Recommendation and review posted by Bethany Smith

Empirical report on Global Gene Therapy Market states the technological advancements, production rate, technological advancements, and marketing channels from 2014-2024. The rapid expansion in key Gene Therapy sectors and market segmentation based on key players, types, applications are explained in detail. Initially, the report states the definitions, classifications, revenue, growth rate, and production from 2014-2024 is analysed. The Gene Therapy Market concentration ratio, manufacturing base distribution, and key industry trends are explained. The production market share, revenue share, mergers & acquisitions, and expansion plans are stated.

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The following key Gene Therapy Market insights and pointers are covered in this report:

Product Analysis and Development Aspects: Complete product portfolio, upcoming trends, and technologies are covered. New product launch events, development activities, import-export details are stated.

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Gene Therapy Market Segmentation By Type, Applications & Regions

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The Gene Therapy Industry potential, key trends, market statistics, trading, competition, and value chain analysis is explained. The SWOT analysis will provide information on strengths, drivers, opportunities and market risks. The new product developments, emerging Gene Therapy Market sectors, technological advancements, and threats are analysed. The growth strategies and business plans implemented by top industry players are evaluated in detail.

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The report serves as a complete guide in providing complete Gene Therapy manufacturing processes, cost structures, raw materials, investment feasibility, and investment return analysis. The SWOT analysis, market growth, production, profit, and supply-demand statistics are offered.

The current business and progressions, future methodologies, market entrants are explained. The consumers, distributors, manufacturers, traders, and dealers in Gene Therapy Market are covered. A comprehensive research methodology, market size estimation, market breakdown, and data triangulation is covered.

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Gene Therapy Market Structure, Major Players, Share, Industry Size and Product Types and Forecast till 2024 - BulletintheNews

Recommendation and review posted by Bethany Smith

British researchers have just developed a revolutionary gene therapy for people with hemophilia A. According to their initial tests, it has been successfully tested on 13 men suffering from this genetic disorder.

Blood Bag

Hemophilia, which affects about 20000 people in the US and 700,000 worldwide, is a rare disease that prevents blood from clotting, which carries a risk of bleeding. Carried in the X chromosome and mainly affecting men, it can be particularly painful and can even cause death if the bleeding is not treated in time.

Read Also: Doctors Investigate CRISPR Gene-Editing for Treatment of Sickle Cell Disease

Caused by the lack or absence of factor VIII, a clotting factor generally present in our plasma that allows the blood to clot, hemophilia A is the most common form: 80% of patients suffering from this genetic disease are affected with the A variant. Because their blood cannot clot properly, patients are exposed to the risk of excessive bleeding at the slightest cut or injury, but also to the risk of spontaneous internal bleeding. Therefore, they are given at least three intravenous injections per week to control and prevent bleeding.

But new research in gene therapy may allow patients to eliminate these regular injections, which affect their quality of life. In a paper published in the New England Journal of Medicine, researchers from Queen Mary University in London, United Kingdom, announced that a single treatment resulted in 13 patients having normal or near-normal levels of blood clotting protein, factor VIII, even after one year.

Although this preliminary work had already been published in 2017, this time the authors announced that, three years after receiving treatment, all patients in the study still benefited from a substantial reduction in bleeding rates. The 13 patients No longer needed regular infusions of factor VIII to prevent bleeding.

Read Also: University of Connecticut Reverses Prader-Willi Syndrome in Lab by Restoring Silent Genes

Our 2017 paper showed that gene therapy could significantly boost factor VIII levels in men with hemophilia A. Our new data are critical in helping the scientific and medical communities understand this pioneering technology, explained Pasi. This latest study confirms both safety and the long-term beneficial impact of the treatment. A long-term treatment that effectively ends the life-long regime of regular injections can transform care and massively improve the quality of life of hundreds of thousands of people born with this challenging genetic condition.

Herpes Could Finally Be Cured With CRISPR According to Study

First CRISPR Trials on Humans Are Now Being Implemented in the US

Gene Therapy To Prevent And Treat Chlamydia

Scientists Are Now Using Viruses to Treat Cancers And, Other Diseases

CRISPR Prevents Liver Disease In Mice Before Birth

Controversial Startup Offers Young Blood to Fight Aging

UC Berkeley Researchers Restore Vision in Mice Through Gene Insertion

Tampering with genes permanently altered brain function in twins

Genetics Have Less Effect on Longevity, Calico Study Shows

Breakthrough Study Reveals That 38 Genes Are Involved In Hearing Loss In Mice

Atherosclerosis Facts: Symptoms, Causes, Diagnosis, Treatments and Prevention

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Hemophilia Cure: New Gene Therapy Offers Hope to Patients - Gilmore Health News

Recommendation and review posted by Bethany Smith

The global CNS Gene Therapy Market reached ~US$ xx Mn in 2019 and is anticipated grow at a CAGR of xx% over the forecast period 2018 2028. The business intelligence study of the CNS Gene Therapy Market covers the estimation size of the market both in terms of value (Mn/Bn USD) and volume (x units).

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CNS Gene Therapy Market To Boost Demand And Forecast In Upcoming Year 2018 2028 - Citi Blog News

Recommendation and review posted by Bethany Smith

Report Description

A recent market intelligence report that is published by Data Insights Partner onGene Therapy marketmakes an offering of in-depth analysis of segments and sub-segments in the regional and international Gene Therapy market. The research also emphasizes on the impact of restraints, drivers, and macro indicators on the regional and world Gene Therapy market over the short as well as long period of time. A detailed presentation of forecast, trends, and dollar values of international Gene Therapy market is offered. In accordance with the report, the global Gene Therapy market is projected to expand at a CAGR of 30% over the period of forecast.

Market Insight, Drivers, Restraints& Opportunity of the Market:

Gene therapy is a medical procedure which replaces defective genes or introduces new genes n order to prevent or cure genetic disorders. This procedure has become a bench mark in medical industry as there is no requirement of surgery or drugs or other procedure which has side effects on the individuals. Gene therapy was first commercialized in China in 2004 by China based SiBono Gene Tech (product Gendicine).

The global gene therapy market has been expanding due to the rigorous research conducted in the field of genetics. The rising awareness about the capability of cure of several rare genetic diseases by gene therapy is another important driver which leads the global gene therapy market during the forecast period. Gene therapy has capability cure several life threatening diseases such as cancer, cardiac diseases, AIDS, cystic fibrosis, age-related disorders, sickle cell anemia etc. In March 2019, the director of the National Health Institute (NIH), the U.S. announced that the recent clinical trials on the gene therapy for the treatment of sickle cell anemia showed promising result- therefore, increasing prevalence of aforementioned lie threatening diseases would likely to drive the growth of the global gene therapy market during the forecast period.

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On the other hand, treatment cost and stringent regulatory conditions etc. may hamper the growth of the global gene therapy market in the upcoming future. The results of Gendicine clinical trials were published in 2003 and the medicine got approval by the China State Food and Drug Administration in the same year. Although approved in China, Gendicines use is not very promising outside China. There are several concerns among the researchers about the quality of the clinical trials performed and safety and efficacy of the treatment. However, Gendicines equivalent Advexin (company Introgen Therapueitcs) is still waiting for the FDA approval.

Increasing investment to the gene therapy related research (around 10 Bn was invested in 2015 by private and public organizations), new product developments such as (Zolgensma in 2019), strategic alliance among the key players (such as collaboration between Axovant and Yposkesi) would bring the global gene therapy market an opportunity to propel during the forecast period. In May 2019, Avexis (a Novartis company) has got the FDA approval for Zolgensma for treatment of spinal muscular atrophy for the pediatric patients (less than 2 years of age).

Segment Covered:

This market intelligence report on the global gene therapy market encompasses market segments based on product, application, target user and geography. On the basis of product, the sub-markets is segmented into Yescarta, Kymriah, Strimvelis, Gendicine, Zolgensma and others (Advexin). Based on application, the global gene therapy market has been segregated into large B-Cell lymphoma, Car T Cell therapy, ADA-SCID (adenosine deaminase deficiency), muscular atrophy, head and neck squamus cell carcinoma, others (Crigler-Najjar syndrome). By target user, the global gene therapy market is also classified into adult and pediatric. By Geography, the global gene therapy market has been divided into North America (the U.S., Canada), Latin America (Brazil, Mexico, Argentina and other countries), Europe (Germany, France, the U.K., Spain, Italy, Russia, and other countries), Asia Pacific (India, Japan, China, Australia and New Zealand and other countries), Middle East and Africa (GCC, South Africa, Israel and Other countries).

Profiling of Market Players:

This business intelligence report offers profiling of reputed companies that are operating in the market. Companies such as Novartis, Gilead Sciences, Orchard Therapeutics Ltd, SiBiono GeneTech Co, Introgen Therapeutics and among others have been profiled into detail so as to offer a glimpse of the market leaders. Moreover, parameters such as gene therapy market related investment & spending and developments by major players of the market are tracked in this global report.

Report Highlights:

In-depth analysis of the micro and macro indicators, market trends, and forecasts of demand is offered by this business intelligence report. Furthermore, the report offers a vivid picture of the factors that are steering and restraining the growth of this market across all geographical segments. In addition to that, IGR-Growth Matrix analysis is also provided in the report so as to share insight of the investment areas that new or existing market players can take into consideration. Various analytical tools such as DRO analysis, Porters five forces analysis has been used in this report to present a clear picture of the market. The study focuses on the present market trends and provides market forecast from the year 2017-2027. Emerging trends that would shape the market demand in the years to come have been highlighted in this report. A competitive analysis in each of the geographical segments gives an insight into market share of the global players.

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Salient Features:

This study offers comprehensive yet detailed analysis of the Gene Therapy market, size of the market (US$ Mn), and Compound Annual Growth Rate (CAGR (%)) for the period of forecast: 2019 2027, taking into account 2017 as the base year

It explains upcoming revenue opportunities across various market segments and attractive matrix of investment proposition for the said market

This market intelligence report also offers pivotal insights about various market opportunities, restraints, drivers, launch of new products, competitive market strategies of leading market players, emerging market trends, and regional outlook

Profiling of key market players in the world Gene Therapy market is done by taking into account various parameters such as company strategies, distribution strategies, product portfolio, financial performance, key developments, geographical presence, and company overview

Leading market players covered this report comprise names such as. Novartis, Gilead Sciences, Orchard Therapeutics Ltd, SiBiono GeneTech Co, Introgen Therapeutics and among others

The data of this report would allow management authorities and marketers of companies alike to take informed decision when it comes to launch of products, government initiatives, marketing tactics and expansion, and technical up gradation

The world market for Gene Therapy market caters to the needs of various stakeholders pertaining to this industry, namely suppliers, manufacturers, investors, and distributors for Gene Therapy market. The research also caters to the rising needs of consulting and research firms, financial analysts, and new market entrants

Research methodologies that have been adopted for the purpose of this study have been clearly elaborated so as to facilitate better understanding of the reports

Reports have been made based on the guidelines as mandated by General Data Protection Regulation

Ample number of examples and case studies have been taken into consideration before coming to a conclusion

Reasons to buy:

vIdentify opportunities and plan strategies by having a strong understanding of the investment opportunities in the Gene Therapy market

vIdentification of key factors driving investment opportunities in the Gene Therapy market

vFacilitate decision-making based on strong historic and forecast data

vPosition yourself to gain the maximum advantage of the industrys growth potential

vDevelop strategies based on the latest regulatory events

vIdentify key partners and business development avenues

vRespond to your competitors business structure, strategy and prospects

Data Insights Partner is a market intelligence and consulting firm. Our customers are spread across the globe and we are dedicated towards catering to the needs of our plethora of clients. In addition to this, our client base comprises leading players operating across all business verticals. Moreover, our business intelligence and industry research reports offer clients with penetrative insights and actionable market data specially designed and presented to aid strategic decision making. We are a specialist in Information Technology, Semiconductor & Electronics, Healthcare, Chemical, Automotive & Defense and Consumer Goods. Furthermore, Data Insights Partner

See more here:
Gene Therapy Market Industry Analysis and Forecast (2019-2027) | Novartis, Gilead Sciences, Orchard Therapeutics Ltd, SiBiono GeneTech Co -...

Recommendation and review posted by Bethany Smith

The major factors driving the growth are rising need for Cancer Gene Therapy worldwide, increase in prevalence of cancer, increase in geriatric population, increase in funding for R&D activities of cancer gene therapy and favorable government regulations.

Global Cancer Gene Therapy Market industry valued approximately USD 0.2 billion in 2016 is anticipated to grow with a healthy growth rate of more than 35.1% over the forecast period 2019-2025.

To request a sample copy or view summary of this report, click the link below:

https://www.digitsnmarkets.com/sample/6650-global-cancer-gene-therapy-market

The objective of the study is to define market sizes of different segments & countries in previous years and to forecast the values to the next eight years. The report is designed to incorporate both qualitative and quantitative aspects of the industry with respect to each of the regions and countries involved in the study. Furthermore, the report also caters the detailed information about the crucial aspects such as drivers & restraining factors which will define the future growth of the market. Additionally, it will also incorporate the opportunities available in micro markets for stakeholders to invest, detailed analysis of competitive landscape and product offerings of key players. The detailed segments and sub-segment of the market are explained below:

End-User:

Therapy:

Some of the key manufacturers involved in the market are OncoGenex Pharmaceuticals, Adaptimmune,Merck, Bluebird bio, Inc,GlaxoSmithKline, Shanghai Sunway Biotech, BioCancell, Celgene, Shenzhen SiBiono GeneTech and SynerGene Therapeutics.Acquisitions and effective mergers are some of the strategies adopted by the key manufacturers. New product launches and focuses on continuous technology innovations are also strategies adopted by the major players. The companies are also trying to dominate the market by investing in research and development.

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Digits N Markets has a vast repository of latest market research reports on trending topics, niche company profiles, market size and other relevant data released by renowned publishers. We have access to the database related to niche markets and trending topics in various industries. We also update the data regularly to provide recent statistics to the client. Recent data and reports will be featured on our websites and clients will be able to access the same. Our clients will be able to benefit from qualitative & quantitative insights in the report which will support them in taking concrete business decisions.

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Original post:
Global Cancer Gene Therapy Market ,Trends, Analysis, Opportunities, Share and Forecast 2019-2025 - Digits N Markets News

Recommendation and review posted by Bethany Smith

A couple years back, some cardiovascular specialists at Bostons Brigham and Womens Hospital stumbled across a surprising result.

Novartis had tasked them with carrying out the long-range Phase III trial for canakinumab, an experimental anti-inflammatory drug the company was trying to market for cardiovascular disease. The main results were mixed modest reductions in fatal cardiovascular events, clear side effects but an additional, exploratory analysis, turned heads over in oncology: Across 10,061 patients, those who had received canakinumab were less likely to develop lung cancer; 33% less likely for the highest dosage.

The hazard ratio was striking, Kwok-Kin Wong, a lung cancer specialist at NYUs Grossman School of Medicine told Endpoints News, referring to the33% figure and an even higher number for a different subgroup.

The question: Why?

New research published today inCancer Research,from one of the earliest RAS-mutant pancreatic cancer researchers, may give a peak into that question, while offering the potential to open up one of the most intractable cancers to the latest wave of targeted therapies.

A team led by NYUs Dafna Bar-Sagi discovered that RAS-positive pancreatic cancer cells emit an immune-signaling protein called interleukin-1 also the protein that canakinumab, the Novartis drug, inhibits. That protein turns down the immune system near the tumor in a variety of ways, they found, most notably by helping build more collagen fibers that essentially guard the cancer from T cells that would ordinarily try to invade and kill it.

Its a wall the tumor build around itself, and does not allow the immune system in, Bar-Sagi told Endpoints.

These walls, she said, accounted not only for the tumors ability to elude the bodys natural immune system, but also the all-but-complete failure of the immune-unleashing PD-1 checkpoint therapies in pancreatic cancer.

The PD-1 checkpoint therapy wont have an effect if the [immune] cell cant get into the tumor, she said.Her paper tested if an anti-interleukin drug like canakinumab could be used in combination with a PD-1 drug, the former clearing the way for the latter, finding a 32% reduction in mice. (Novartis did not fund the study but did provide the interleukin and checkpoint drugs).

Gregory Beatty, an assistant professor of immunology at the University of Pennsylvania School of Medicine who was not involved with the study, said the study was exciting and applicable to not only pancreas cancer but potentially other malignancies.

Approaches to overcome immune resistance in pancreas cancer are of utmost importance and IL-1b is a novel target, Beatty said via email.

Kwok-Kin Wong who was not involved with the study but works at the same institution said the findings were important for a range of cancers, including by offering a mechanism for the 2017 findings in lung cancer. Lung cancer specialists could look for the same process.

This paper will spur a lot of interest, Wong said, noting many companies already have an interleukin antibody and a PD-1 on hand. Theres a lot of therapeutic implications.

Bar-Sagi was one of the earliest researchers into RAS mutations. She joined James Feramiscos Cold Spring Harbor Lab as a postdoc in 1986, shortly after the oncogene was first discovered, and quickly co-authored in Scienceone of the firstpapers suggesting how the RAS gives rise to tumors.

Over the years, though, no one fully identified interleukin-1 in the tumors, even as understanding of RAS progressed to a point at which drug developers could bring targeted therapies into the clinic.

The reason for this was simple, Bar-Sagi said, and it has major implications for any future research: The protein doesnt show up in tumor cell cultures.

Building off of research at her lab, Washington University of St. Louis and elsewhere, Bar-Sagi devised a way of testing it in vivo. They discovered that the tumor relies on a previously undiscovered ligand binding to its TLR4 receptors. By giving antibiotics to clear bacteria in some mice, they found that a warped microbiome may promote the binding, contributing to the development of cancer.

Only once they inserted the ligand into cell cultures, the cells began producing interleukin-1 . And when they gave RAS-positive mice a combination of a PD-1 and an interleukin-1 antibody, they found a 32% reduction in tumors.

Bar-Sagi said its a breakthrough that continues to point to the importance of not just the tumor but also the tumors effect on the micro-environment around it and the immune system there. But to explore that, she said, researchers will have to turn away from Petri dishes and find new methods of studying tumors pancreatic, lung or otherwise as they exist within the body.

Many of these effects are not going to be recognized or will be very hard to study using the conventional study methods, Bar-Sagi said. And thats definitely a shift.

Read the original:
How pancreatic RAS tumors protect themselves. Researchers point to a new protein and maybe a new treatment - Endpoints News

Recommendation and review posted by Bethany Smith

We're not going to dive into all of these, but let's cover a couple that have been getting some extra attention lately: senescent cells, for example. Turns out, there's a limit to the number of times a cell can reproduce, called the Hayflick limit. When cells reach this limit, they're called senescent. "We used to think, well, they're old and in the way, but they're harmless," says Rountree. "But it turns out, they're releasing harmful signals and inflammation to the body."

Another topic you've likely read about right here on mbg is mitochondrial dysfunction. Think back to high school biology classmitochondria are the powerhouses of our cells, which produce ATP or energy. "When we get older, we tend to lose mitochondria because mitochondria don't have the same kind of repair mechanisms as our DNA," says Rountree. "So over time, we get more tired and we don't have the energy to fuel our cellular mechanisms."

Butand here's where things get a little crazySinclair believes there could be one common driver of all nine of these processes, which he sums up with his information theory of aging. "The theory proposes that all of the causes of aging that people are working onfrom loss of mitochondria to senescent cells to telomere shorteningare manifestations of a very simple principle, which is a loss of epigenetic information in the cell rather than genetic information.Meaning that cells lose their ability to read the right genes at the right time, in the same ways that scratches on a CD would mess up the ability to play a beautiful album," he says. (The epigenome, if you're unfamiliar, essentially tells the genome what to do.)

"What I'm proposing," he says, "is that if we can stop the epigenome from degrading, all of these other things go away." But if Sinclair's new theory is true, it raises the question: How the heck do you prevent these scratches (aka epigenome degradation) so cells continue to read the right genes at the right time, and so you can avoid things like fatigue, frailty, and cancer? And can we "clean up" damage that's already there and essentially turn back the clock?

According to Sinclair, there are sort of two levels to that answer. One, there seems to be quite a bit we can do that may effectively slow the aging process (i.e., prevent these scratches) via targeted dietary and lifestyle changes and a few promising supplements, which we'll dive into in the section below.* As for the second part of the question, to truly turn back the clock, lifestyle changes won't cut it. But future therapies and drugs could make that possible.

"We think we've figured out how to reset the age of cells," says Sinclair. "We've figured out that there's essentially a backup hard drive with this epigenetic information that we can access and tell the cells to be young again and reset their clock." Sinclair admits that this work is still quite preliminary, but they've just had some very promising results in animal studies with gene therapy treatments (right now they're injections, but they could eventually be pills). In a study from July 2019, they were able to reprogram damaged optic nerve cells in mice with glaucoma and restore vision.

"That's another level of science that's coming, and we're still in the early stages, but if we can restore vision, what else might we be able to reset?" he asks. Which, to be honest, is equal parts freaky and fascinating.

Read the original:
The New Science Of Aging: How To Biohack Your Way To A Longer, Healthier Life - mindbodygreen.com

Recommendation and review posted by Bethany Smith

LONDON--(BUSINESS WIRE)--Technavio has been monitoring the global head and neck cancer diagnostics market since 2015 and the market is poised to grow by USD 3.41 billion during 2020-2024 at a CAGR of almost 12% during the forecast period. Request Free Sample Pages

Read the 161-page research report with TOC on "Head and Neck Cancer Diagnostics Market Analysis Report by Geography (Asia, Europe, North America, and ROW), by Diagnostic Methods (Biopsy and blood tests, Imaging, Endoscopy, and Dental diagnostics), and Segment Forecasts, 2020-2024".

https://www.technavio.com/report/head-and-neck-cancer-diagnostics-market-industry-analysis

The increasing number of cancer-related initiatives and rising popularity of immunotherapy in head and neck cancer treatment are anticipated to boost the growth of the market.

Many private and government organizations are undertaking several initiatives to prevent and control cancer by promoting cancer awareness, cancer treatment, and early cancer diagnosis. For instance, the American Cancer Society, established in 1913, has introduced global health programs and global cancer-control initiatives to facilitate early diagnosis of cancers such as head and neck cancer to save lives and minimize suffering. This is driving the demand for head and neck cancer diagnostic equipment such as computed tomography, magnetic resonance imaging scanners, and emission tomography. Thus, the increasing number of cancer-awareness initiatives are expected to drive market growth during the forecast period.

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Major Five Head and Neck Cancer Diagnostics Market Companies:

Accuray Inc.

Accuray Inc. is headquartered in the US and operates the business under the Oncology systems group segment. The company offers Radixact, which is an intensity modulated radiation therapy (IG-IMRT) system. The company also offers TomoTherapy with an integrated 3D image guidance and unique binary MLC.

Apteryx Imaging Inc.

Apteryx Imaging Inc. is headquartered in Canada and offers products through the following business units: Hardware and Software. The company offers VELscope Vx System, which is a powerful device for enhanced visualization of mucosal abnormalities.

Canon Inc.

Canon Inc. is headquartered in Japan and operates under various business segments, namely Office Business Unit, Imaging System Business Unit, Industry and Others Business Unit, and Medical System Business Unit. The company offers an MR system, Vantage Titan 1.5T.

General Electric Co.

General Electric Co. is headquartered in the US and offers products through the following business segments: Aviation, Power, Oil and gas, Healthcare, Renewable energy, Transportation, Lighting, and Capital. The company offers SIGNA Pioneer - 70cm, Discovery MR750w GEM - 70cm, SIGNA PET/MR with QuantWorks, and others.

KARL STORZ SE & Co. KG

KARL STORZ SE & Co. KG is headquartered in Germany and offers products through the following business segments: Human Medicine, Industrial, and Veterinary Medicine. The company offers C-MAC FIVE S and C-MAC S USB.

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Head and Neck Cancer Diagnostics Diagnostic Methods Outlook (Revenue, USD Million, 2020-2024)

Head and Neck Cancer Diagnostics Regional Outlook (Revenue, USD Million, 2020-2024)

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Global Head and Neck Cancer Diagnostics Market 2020-2024 | Evolving Opportunities With Accuray Inc. and Canon Inc. | Technavio - Business Wire

Recommendation and review posted by Bethany Smith

LA SALLE On Tuesday morning, the barn at Coyote Ridge Ranch in Weld County served as the bovine equivalent of a hair salon.

Some of the ranchs top Hereford cattle were brought in for a bath and blow-dry. Outside, workers gave the rust and white-colored animals a final clipper trim, preparations for their impending closeups.

The National Western Stock Show is back for its 114th year in Denver and Coyote Ridge Ranch Herefords are right in the thick of it, as theyve been for three decades.

The Cornelius family, lead by Jane Evans and her son Hampton, founded the ranch in Boulder County, but for the past 25 years, its operated on a 1,000-acre spread south of La Salle. The barn/cattle hair salon there is 130 years old and may be the oldest structure in the Beebe Draw valley, according to the family.

Over the decades, the Corneliuses have established a reputation as one of the countrys if not the worlds preeminent producers of top quality Herefords and Hereford genetics. And the stock show is their biggest marketing opportunity of the year.

Denver is like a trade show for us. Were not there to win a ribbon. Its cool when you win one but were there to promote our genetics and make contacts, Jane said of the National Western show, where Coyote Ridge will be showing a pen of three heifers and a pen of three bulls this year.

Were showing our spring-born 2019 cattle, Hampton said. Everything is for sale. The idea is to drum up interest to get people to come back here and take a look at our other ones.

The business-first approach doesnt mean they arent proud of how Coyote Ridge Ranch has performed at National Western. Hampton rattles off the stack of honors the operation has come away with in years past. They include three grand champion pens, two bulls that won individual championships in competitions on the hill at National Western and countless individual class champions.

Its a validation of what youre doing, Jane said.

Coyote Ridge Ranch is what is known in the cattle business as a seedstock producer. The means its herd of 160 or so Herefords is being raised to further the genetics of the breed. It sells bulls, heifers, semen and embryos to commercial Hereford ranching operations that in turn produce steers for slaughter and sale to consumers.

The ranch dates back to when Hampton and his sisters Katie and Coleman, a former Denver Post staff writer, were kids raising cows and calves as part of 4-H and Future Farmers of America programs. The hobby blossomed into a passion and the family herd grew large enough to become a viable business.

The Corneliuses were drawn to Herefords because of the breeds disposition, its hardiness and the animals deep connection to ranching culture in the American West. Nowadays, Coyote Ridge is considered an elite Hereford seedstock producer with genetics from their animals spread across ranching operations in the U.S. and all over the world.

I would describe that family as just being committed to making really good cattle and breeding Herford cattle the way they need to be bred for the commercial industry, said Jack Ward, executive vice president of the American Hereford Association.

Ward and many other staffers from his association are in Denver this week for the stock show. Among the events Ward is organizing is the national Hereford junior heifer show on Wednesday morning and the Mile High Night Hereford Sale, which is expected to bring more than 1,000 people to the National Western Stadium Arena at 6:30 p.m. Friday, Ward said. Coyote Ridge will be represented at both events, of course, with Hampton Cornelius son, John, showing a heifer in the junior show.

Beyond their work furthering the Hereford breed, the Corneliuses have become ambassadors for the cattle industry in Colorado. With a ranch thats within an hours drive of the downtown Denver and a willingness to open their operation to visitors, theyve hosted school groups, chefs, and delegations from countries including China, Japan and South Korea.

(Hamptons wife) Kay and Jane Evans are both so very well-spoken on so many segments of their industry, Colorado Beef Council marketing director Tami Arnold said. Just the typical consumer, we know we could take them out to their place and they would be able to really represent the beef industry well.

For Jane Evans, the most exciting visit came last summer when a Taiwanese trade delegation stopped at Coyote Ridge Ranch. It wasnt just because the visitors were so impressed by seeing a cattle operation where riders on horseback drive a herd across a pasture. She was pleased because the delegates were in Colorado to sign a letter of intent with Gov. Jared Polis to expand access in Taiwan for Colorado agricultural goods including beef.

It gets its moment in the sun in Denver every January during the National Western, but Jane is quick to point out the livestock industry is a major force in Colorados economy. Cattle operations alone generated $3.4 billion in cash receipts in the state last year, according to the University of Colorados 2020 business economic outlook.

Jane Evans, 78, has established a reputation of her own over the years. Cattle ranching has been a male-dominated industry, but she hasnt shied away from being at the center of it. In the mid-1990s, she became the first woman elected to the American Hereford Association board of directors.

She paved the way for women in the beef industry, specifically for leadership and we love her for that, said Arnold, who in addition to working with the Cornelius family through the Colorado Beef Council also ranches nearby and has known them most of her life.

Janes love for agriculture goes back to her childhood in Alabama, when her grandfather would let her tag along when he would assess farms and ranches as part of his work as a banker.

I was very lucky. In those days girls did one thing and boys did another, she said. My mother used to say, When Jane Evans grows up shes going to own a large cattle ranch in the West like Dale Evans, and I do. (Dale Evans was married to singing cowboy Roy Rogers and they had a popular TV show in the 1950s.)

The next week will be a busy one for the Cornelius family. But now that theyve settled into their pen in the National Western Centers evolving stock yards, they do expect to have a little fun.

These guys work alone a lot. When they get together they definitely will kick up their heels a little bit, she said of her family and her fellow ranchers at the stock show. You see people that you have a lot in common with, that you work with, that you swap genetics with. Even though there is stiff competition, there is an awful lot of camaraderie.

Visit link:
National Western Stock Show: Reputation of Weld County familys Herefords spans the globe - The Denver Post

Recommendation and review posted by Bethany Smith

(Photo : moviehole.net)Hybrid sharks were found in Australia and they were able to swim in cold or tropical waters. This is a fact, not fiction ane researchers are now convinced that it will impact the local environment. All bets are on if there will be more found too.

Sharks are the perfect alpha predator according to nature's design, it is also the most adapted for the aquatic environments. Next question is whether they can evolve any further than they have. Like fiction turning into reality, that might soon be the scenario in the open ocean. It seems evolution, natural selection has found a way to improve an already fearsome alpha predator.

Is this disturbing or just another movie theme, for shark-buffs? No, it has gotten real and not a hoax at all. As stated, evolution and natural selection chooses the survival of the fittest. Several Australian shark scientists did find hybrid sharks, because two different species interbred and produce offspring. It was found out by accident when a survey was underway in Aussie waters.

The sharks catalogued had genetic traits that were not bound to a specific kind of species. So, the best guess it that they have mated and produced young with hybrid traits. After more sifting through the genetic data, the analysis revealed a mix of common blacktip shark and Australian blacktip shark. They must have mated in the wild, since mating is automatic when in spawning season.

Scary as it sounds, these sharks are not producing genetically infertile offspring, but normal individuals capable of reproduction. One thing to consider is they are close sister species, still distinct between each other. Researcher conclude because of the relative closeness of these shark species made the change to a mixed breed possible too.

Another tidbit might be somewhat on the negative side, climate change caused by human activitieshave influenced natural selection very unnaturally. Places where the sharks breed are changing, indirectly and this is why hybrids of mixed species are born. If the environment is steady and no changes, then both species will not stray into each other.

Though they are cartilaginous fish, reproduction is via male and female of the same kind. One big revelation is that sharks will not reproduce with other sharks because of differences in attraction the other sex. Discovery of hybrid sharks have forced shark specialists to rethink their assumptions, in regard to this. Abnormal mating behavior and breeding hybrids is just the tip of the iceberg for researchers, studying sharks.

Will these hybrids be better than their predecessors, or be eliminated by natural selectionas nature deems fit. If these hybrids do find individuals of the same hybrid kind, it will be the birthing of whole new shark species. Most animals alive today and those extinct were part of the process of natural selection, or survival of the fittest.

For sharks that have remained the same for millions of years, introduction of a new hybrid is important. The common blacktip shark and Australian blacktip sharkwill have specific gene set which is evolved to keep the species ready for better adaptations. The hybrid can adjust to different temperatures so they can enter other zones in the ocean. Unlike their parent species, who are located in different waters. This is what makes sharks such adaptable species and ancient species.

Yes, hybrid sharks might be common with the shift in the conditions of the eco-system. all because mankind is responsible for all climatic changes happening now. Will a Great White, Bull Shark, or Tiger Shark hybridcome about? It might and the common blacktip shark, and Australian blacktip shark is proof of that. An ocean with these fish will not be so exciting to go into either.

Related Article: Hybrid Sharks Ready to Take Over the Oceans: Fact and/or Fiction

Visit link:
Fact or Fiction: Hybrid Sharks To Dominate the World's Oceans Very Soon - Science Times

Recommendation and review posted by Bethany Smith

In recent years, new gene editing tools have been used for everything from genetic modification of plants to increase crop yields to, far more controversially, genetic tampering with human embryos. Could a form of gene therapy also be useful in helping treat cocaine addiction, a form of addiction that proves highly resistant to alternative approaches, such as conventional medical treatment and psychotherapy? Thats what researchers from the world-famous Mayo Clinic are hoping to prove.

They are seeking approval for the first-in-human studies of an innovative new single-dose gene therapy. Their approach involves the delivery of a gene coding for an enzyme, called AAV8-hCocH, which metabolizes cocaine in the body into harmless byproducts. In order to progress to this next step in their work, they first have to gain permission from the U.S. Food and Drug Administration (FDA) in the form of an Investigational New Drug Application.

The researchers have already demonstrated the safety of their approach in mice. In a prior experiment, they showed a complete lack of adverse effects in mice which had both been previously exposed to cocaine and those which had not.

Mice given one injection of AAV8-hCocH and regular daily injections of cocaine had far less tissue pathology than cocaine-injected mice with no vector treatment, the researchers wrote in the abstract for their paper describing the work. Biodistribution analysis showed the vector located almost exclusively in the liver. These results indicate that a liver-directed AAV8-hCocH gene transfer at reasonable dosage is safe, well-tolerated, and effective. Thus, gene transfer therapy emerges as a radically new approach to treat compulsive cocaine abuse.

This is not the first time similar work has been carried out. In February 2017, scientists at the University of British Columbia genetically engineered a mouse so as to be incapable of becoming addicted to cocaine. However, one of the researchers on the project told Digital Trends that transferring this work across to humans for possible treatment for addiction was not straightforward. Instead, that work was more focused on exploring the link between drug use and genetics and biochemistry.

Theres still a whole lot more research that needs to be done in this area. Even if the FDA grants the Mayo Clinic researchers permission for their human trials, well most likely be waiting a few years at least before this treatment could be rolled out to the general public. Its an exciting leap forward, nonetheless.

Link:
Scientists want human trials for gene therapy that could help battle addiction - Digital Trends

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass.--(BUSINESS WIRE)--ReadCoor, Inc., a company reinventing omics and pathology with its 3-dimensional (3D) multi-omic spatial sequencing platform, today announced a $27 million Series B financing to accelerate the commercialization of its proprietary FISSEQ technology for use in research, drug discovery, and diagnostics. The Series B financing includes new investor Pavilion Capital and other investors, as well as existing investors Decheng Capital and Hansjrg Wyss.

ReadCoor has made rapid progress in bringing multi-omic spatial sequencing from development to the market, said Victor E. Tong, Jr., Partner at Decheng Capital and Chairman of the ReadCoor board of directors. The platform that ReadCoor has developed features a leading-edge technology to sequence and characterize tissue in 3D that offers the potential for new insights in clinical research and drug discovery.

Proceeds from the Series B financing will be used to accelerate commercialization of ReadCoor's platform, powered by its proprietary FISSEQ (fluorescent in situ sequencing) technology, which simultaneously reads the sequences of thousands of molecules and visualizes high resolution 3D spatial information within whole cells and tissues. With first-of-a-kind capabilities to sequence and characterize data in three dimensions, ReadCoors platform gives researchers access to previously undiscovered data to guide evaluation and validation of new drug targets, pathogen identification, disease diagnosis, and the advancement of regenerative medicine. The platform is comprised of an instrument, kits, and data analysis and visualization cloudware, so that it can be incorporated into the workflow of researchers in research and drug discovery.

ReadCoor's platform and product portfolio have been validated across a range of applications over the past year, including non-small cell lung cancer, breast cancer, and a brain atlas. The company's initial product suite includes research and discovery applications in oncology, infectious disease, and neuroscience. The Select Release Program and platform will be unveiled at the 20th Advances in Genome Biology and Technology (AGBT) in February 2020.

The development true spatial sequencing is currently burdened by technical challenges, including the need for specialized imaging hardware, laborious protocols, and unsupported analysis software. To overcome these challenges and integrate these methods, we created an all-in-one solution with unparalleled capacity for true multi-omic spatial sequencing with high-resolution 3D imaging, robust data collection and analysis, said Richard Terry, Founder, President, and Chief Executive Officer of ReadCoor. It is gratifying that our investors support our technology and approach as we move rapidly towards commercialization, and we are excited to expand the availability of our platform to customers.

Leading up to this Series B financing, ReadCoor's progress has been marked by collaborative agreements and research grants with leading research institutions, including the Chan Zuckerberg Initiative, Bill & Melinda Gates Foundation, Oregon Health State University, and Harvard University.

About Fluorescent in situ Sequencing (FISSEQ)ReadCoors platform is powered by proprietary Fluorescent in situ Sequencing (FISSEQ) technology, which offers researchers the ability to sequence across multiple omic formats (genomics, transcriptomics, proteomics, and metabolomics) without disruption to cell structure or loss of spatial data. This platform delivers the precise locations of molecular targets and a corresponding 3D image plotting the coordinates within the sequenced sample. Currently, available sequencing technologies can only provide sequencing information, not true spatial information. FISSEQ provides both, representing the first true merging of imaging and sequencing. This 3D molecular atlas can enable meaningful scientific insights into therapeutic areas, including cancer, immuno-oncology, gene therapy, and neurodegenerative diseases, enabling researchers to gain more robust diagnostic insights to deliver precise therapeutics to patients. FISSEQ was developed in the Church Lab at Harvard University and the Wyss Institute for Biologically Inspired Engineering, and first published in Science in 2014 (Highly multiplexed three-dimensional subcellular transcriptome sequencing in situ). The platform has broad applications in the areas of drug development, diagnostics, and machine learning.

About ReadCoor, Inc.ReadCoor is leading the next generation of omics by delivering the first true multi-omic spatial sequencing platform to the global audience of researchers, clinicians, pharma and diagnostics companies, and ultimately patients. The company is accomplishing this with Fluorescent in situ Sequencing (FISSEQ), a fundamental new technology that simultaneously integrates high-throughput sequencing and three-dimensional morphometric analysis. This uniquely powerful platform is expected to revolutionize the next phase in understanding the multi-omic landscape and introduce vast new opportunities for important and meaningful clinical insight.

Read this article:
ReadCoor, Inc. Announces $27 Million Series B Financing for Commercial Launch of 3D RNA, DNA, and Protein Spatial Sequencing Platform - Business Wire

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass., Jan. 10, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics Inc. (Nasdaq:LOGC), a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients today announced a research collaboration with Takeda Pharmaceutical Company Limited (Takeda) to further develop LB-301, an investigational therapy using LogicBios proprietary, promoterless, nuclease-free genome editing technology, GeneRide, for the treatment of Crigler-Najjar syndrome. LB-301 is a recombinant adeno-associated viral (AAV) vector with a uridine disphosphate-glucuronosyltransferase-1 (UGT1A1) gene. The collaboration will bring together LogicBios propriety platform for genome editing and Takedas expertise in researching and developing gene therapies.

LogicBios innovative, site-specific, genome editing platform has the potential to overcome the limitations that make it challenging to apply conventional gene editing and gene transfer in pediatric patients, said Dan Curran, Head, Rare Diseases Therapeutic Area Unit at Takeda. We see GeneRide as a promising approach to explore as part of our aspiration to develop transformative or even potentially curative therapies to patients living with rare diseases.

We are thrilled to be working with Takeda to advance our GeneRide platform in a second indication, said Fred Chereau, CEO of LogicBio. Their insights and expertise in rare diseases drug development is expected to significantly accelerate the development of a much-needed therapy for this devastating pediatric disease. This collaboration recognizes GeneRide as a promising approach for bringing the transformational power of genome editing to children with an array of relentless, progressive pediatric diseases.

Under the agreement, LogicBio and Takeda will collaborate to further research and develop LB-301. Takeda will provide funding for the research program under the collaboration agreement and will have an exclusive option to negotiate an exclusive, worldwide license to LogicBios LB-301 program.

CriglerNajjar syndrome is a rare monogenic pediatric disease caused by a deficiency in the liverspecific UGT1A1 gene, resulting in severely high levels of unconjugated bilirubin in the blood starting at birth, with lifelong risk of permanent neurological damage and death. Current clinical practice consists of daily, intense phototherapy treatment for approximately 12 hours, but this treatment becomes less effective with age, ultimately leaving liver transplantation as the only therapeutic option for survival.

LogicBio has demonstrated that a murine GeneRide construct of LB-301 can correct the gene deficiency in an animal model of Crigler-Najjar syndrome. The introduction of a UGT1A1 gene into the albumin locus in mouse liver cells resulted in normalization of bilirubin levels and long-term survival of mice deficient in UGT1A1 from fewer than 20 days to at least one year. The results from this research were published in EMBO Molecular Medicine (Porro et al., 2017).

About LB-301LB-301 is an investigational pediatric genome editing therapy based on LogicBios GeneRide technology. GeneRide enables site-specific integration and lifelong expression of therapeutic transgenes, without the use of exogenous promoters or nucleases. LB-301 is designed to incorporate a functioning version of the faulty uridine disphosphate-glucuronosyltransferase-1 (UGT1A1) gene into the genome of Crigler-Najjar patients. LogicBio has demonstrated preclinical proof-of-concept of GeneRide in multiple animal models of the disease, improving survival and reversing disease pathology.

About LogicBio TherapeuticsLogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients with significant unmet medical needs using GeneRide, its proprietary technology platform. GeneRide enables the site-specific integration of a therapeutic transgene in a nuclease-free andpromoterlessapproach by relying on the native process of homologous recombination to drive potential lifelong expression. Headquartered in Cambridge, Mass., LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families.

For more information, please visitwww.logicbio.com.

Forward Looking Statements This press release contains forward-looking statements within the meaning of the federal securities laws. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. These risks are discussed in the Companys filings with theU.S. Securities and Exchange Commission(SEC), including, without limitation, the Companys Annual Report on Form 10-K filed onApril 1, 2019with theSEC, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with theSEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

LogicBio Contacts:

Brian LuqueAssociate Director, Investor Relationsbluque@logicbio.com951-206-1200

Stephanie SimonTen Bridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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LogicBio Therapeutics Announces Collaboration with Takeda to Develop New Genome Editing Candidate LB-301 for the Treatment of Crigler-Najjar Syndrome...

Recommendation and review posted by Bethany Smith

SAN FRANCISCO & BASEL, Switzerland--(BUSINESS WIRE)--Indapta Therapeutics, Inc., a biotechnology company focused on developing and commercializing a proprietary, first-in-class, off-the-shelf, non-engineered, allogeneic G-NK (FcR-deficient Natural Killer) cell therapy to treat multiple cancers, and Lonza today announced a strategic partnership. Indapta also announced its founding leadership team and scientific advisors.

Under the terms of the agreement, Lonza will manufacture Indaptas off-the-shelf, allogeneic G-NK cell therapy under current good manufacturing practices (cGMP) for use in clinical studies. Indapta will leverage Lonzas process development capabilities and expertise to ensure a robust, reproducible and scalable cGMP process. Process development and manufacturing will take place in Lonza's state-of-the-art cell and gene therapy manufacturing facility in Houston.

We believe our first-in-class, off-the-shelf, allogeneic G-NK cell therapy will drive the next critical phase in the evolution of cancer therapies following CAR T-cell therapies, said Guy DiPierro, founder and chief executive officer of Indapta Therapeutics. Current autologous CAR T-cell therapies have proven efficacy in various hematologic cancers but have been beset with serious clinical and manufacturing challenges. By providing an off-the-shelf solution with our G-NK cell therapy, we can eliminate the need for a patient-specific therapy. Additionally, because our investigational cell therapies are not engineered, they are likely to be more effective, less costly and have a simpler regulatory pathway.

Lonza, with its demonstrated expertise in cell therapy manufacturing, is the ideal strategic partner to help us advance our clinical program and scale the production of our G-NK cell therapy, said Ronald Martell, co-founder and executive chairman of Indapta Therapeutics. We are currently completing Investigational New Drug-enabling studies and plan to submit an IND application in late 2020 and initiate a first-in-human Phase 1/2 study in early 2021.

Indaptas world-class team of NK cell scientists and clinicians and cell therapy experts has created an innovative off-the-shelf immuno-oncology therapy based on a subset of cancer-killing NK cells that could make a truly meaningful impact in the treatment of hematologic malignancies and solid tumors, said Scott Waldman, chief strategy officer at Lonza.

Alberto Santagostino, senior vice president, head of Cell & Gene Technologies at Lonza, added, with our long-standing experience in cell therapy manufacturing, we are committed to providing Indapta with the expertise, resources and services it needs for cGMP manufacturing to advance its promising program into the clinic and beyond.

About Indaptas G-NK Cell Therapy

Indapta Therapeutics is developing off-the-shelf, allogeneic FcRI-deficient NK cells, known as G-NK cells.i,ii,iii These proprietary cells are a specific and potent subset of NK cells with specialized anti-tumor activity when used in combination with a monoclonal antibody. G-NK cells are NK cells that have undergone an epigenetic change after coming into contact with cytomegalovirus (CMV)-infected cells. As a result, they lack the FcRI signaling adapter and, instead, use a different adapter protein, which predisposes them to a far more activated state of antibody-dependent cell-mediated cytotoxicity (ADCC) in the presence of a monoclonal antibody. When the monoclonal antibody binds to the tumor target and to the Fc receptor on G-NK cells, it initiates the release of dramatically more immune-stimulating cytokines and cell-killing enzymes than conventional NK cells, causing the direct killing of tumor cells and driving tumor cell death. G-NK cells have been demonstrated to be safe; in vivo studies demonstrate they do not cause graft-vs-host disease or cytokine release syndrome, which can occur with CAR-T cell therapies.

Preclinical research, conducted under NIH grants by scientists at the University of California, San Francisco (UCSF), demonstrated the safety and efficacy of G-NK cells administered in combination with a therapeutic monoclonal antibody. Clinical models of multiple myeloma and lymphoma demonstrated improved survival, a statistically significant decrease in tumor growth, and a statistically significant increase in the activity of the monoclonal antibody without causing graft-vs-host disease. When administered in combination with a monoclonal antibody, G-NK cells have been shown to be highly persistent (lasting four to nine months), to have the ability to preferentially bind to a therapeutic monoclonal antibody in the presence of a tumor cell, and to demonstrate superior ADCC function compared with conventional NK cells. Under a second Indapta NIH grant, researchers at Stanford University will be conducting in vivo studies in additional tumor models.

Indaptas off-the-shelf G-NK cell therapy is differentiated from an autologous therapy in that it is not necessary to collect cells from each individual patient and produce a unique therapy for every patient. Rather, it is derived from cells from healthy volunteers, which are highly functional and persistent. Indaptas process for producing G-NK cell therapy for use as an immunotherapy involves taking blood from CMV-seropositive donors, identifying and sorting G-NK cells from these samples, and expanding G-NKs cells using the companys proprietary, patented expansion method, which preferentially expands and activates GNK cells. Indapta has also developed a proprietary method for freezing and storing the G-NK cells in a GMP master cell bank for use as off-the-shelf allogeneic outpatient immunotherapy in cancer patients.

Developing off-the-shelf G-NK cells may sidestep some of the clinical and financial challenges presented by other, more customized and engineered immuno-oncology approaches, which involve time-consuming and costly manufacturing processes and often can only be delivered in specialized centers. The manufacturing COGS for Indaptas program will be relatively inexpensive compared to CAR-T or engineered NK cell therapies. Additionally, the regulatory approval process for Indaptas program may be more straightforward than that for autologous CAR-T cell therapy or engineered NK cells because it does not involve complicated cell engineering.

Not only are G-NK cells widely available from multiple sources, they have the potential to be used in combination with multiple monoclonal antibodies to treat numerous types of cancer (e.g., multiple myeloma, lymphoma, leukemia, melanoma, ovarian, colorectal, renal, liver, breast and lung).

Indaptas Founding Management Team

Indaptas Scientific Advisors

About Indapta Therapeutics

Indapta Therapeutics, Inc. is a biotechnology company focused on developing and commercializing a proprietary, first-in-class, off-the-shelf allogeneic cell therapy to treat multiple types of difficult-to-treat hematologic cancers and solid tumors. Headquartered in San Francisco, Indapta was founded in 2017 by Guy DiPierro along with Ronald Martell and scientists at the University of California, Davis, and Stanford University. The company has developed allogeneic FcRI-deficient natural killer (NK) cells, known as G-NK cells, and is working to bring this off-the-shelf cell therapy to patients to address the limitations of currently available autologous T-cell therapies.

About Lonza

Lonza is an integrated solutions provider that creates value along the Healthcare Continuum. Through our Pharma Biotech & Nutrition segment and our Specialty Ingredients segment businesses, we harness science and technology to serve markets along this continuum. We focus on creating a healthy environment, promoting a healthier lifestyle and preventing illness through consumers' preventive healthcare, as well as improving patient healthcare by supporting our customers to deliver innovative medicines that help treat or even cure severe diseases. Patients and consumers benefit from our ability to transfer our pharma know-how to the healthcare, hygiene and fast-moving consumer goods environment and to the preservation and protection of the world where we live.

Founded in 1897 in the Swiss Alps, Lonza today is a well-respected global company with more than 100 sites and offices and approximately 15,500 full-time employees worldwide at the end of 2018. The company generated sales of CHF 5.5 billion in 2018 with a CORE EBITDA of CHF 1.5 billion. Further information can be found at http://www.lonza.com.

Additional Information and Disclaimer

Lonza Group Ltd has its headquarters in Basel, Switzerland, and is listed on the SIX Swiss Exchange. It has a secondary listing on the Singapore Exchange Securities Trading Limited (SGX-ST). Lonza Group Ltd is not subject to the SGX-STs continuing listing requirements but remains subject to Rules 217 and 751 of the SGX-ST Listing Manual.

Certain matters discussed in this news release may constitute forward-looking statements. These statements are based on current expectations and estimates of Lonza Group Ltd, although Lonza Group Ltd can give no assurance that these expectations and estimates will be achieved. Investors are cautioned that all forward-looking statements involve risks and uncertainty and are qualified in their entirety. The actual results may differ materially in the future from the forward-looking statements included in this news release due to various factors. Furthermore, except as otherwise required by law, Lonza Group Ltd disclaims any intention or obligation to update the statements contained in this news release.

i Hwang I, Zhang T, Scott JM, et al. Identification of human NK cells that are deficient for signaling adaptor FcR and specialized for antibody-dependent immune functions. Int Immunol. 2012;24(12);793-802.

ii Lee J, Zhang T, Lanier LL, Kim S. Epigenetic modification and antibody-dependent expansion of memory-like NK cells in human cytomegalovirus-infected individuals. Immunity. 2015;42:431-442.

iii Zhang T, Scott JM, Hwang I, Kim S. Antibody-dependent memory-like NK cells distinguished by FcR deficiency, Immunol. 2013;190(4):1402-1406.

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Indapta Therapeutics and Lonza Announce Strategic Partnership to Advance a Next-generation, Off-the-shelf, Allogeneic Immuno-oncology Therapy -...

Recommendation and review posted by Bethany Smith

STONY BROOK, N.Y.--(BUSINESS WIRE)--Applied DNA Sciences, Inc. (NASDAQ: APDN) (Applied DNA or the "Company"), a leader in Polymerase Chain Reaction (PCR)-based DNA manufacturing for product authenticity, traceability solutions, nucleic acid-based biotherapeutic development, and liquid biopsies for cancer diagnostics, today announced that CEO Dr. James A. Hayward and leadership of the Companys majority-owned subsidiary, LineaRx, Inc., will be meeting with members of the global investment community from January 13 to 15, 2020 in San Francisco concurrent with the JP Morgan 38th Annual Healthcare Conference, the worlds largest healthcare investment symposium.

In addition, Dr. Hayward will present an overview of the Company, including its supply chain platform, its diagnostic platform for the blood-based capture of metastatic cells, and review the linear DNA platforms in nucleic acid delivery, gene expression, non-integrated DNA replication, and its robust pipeline of more than 15 contract research opportunities with top cancer, gene therapy, vaccine and RNA partners at the Biotech Showcase 2020 at 3:00 pm PT on Tuesday, January 14, 2020. Dr. Haywards presentation will be webcast live at: https://event.webcasts.com/starthere.jsp?ei=1279407&tp_key=6924765b8a.

About LineaRx

LineaRx seeks to commercialize the biotherapeutic value of Applied DNAs deep expertise and experience in the design, manufacture and chemical modification of DNA by large scale polymerase chain reaction (PCR). Linear DNA is a form of DNA distinct from the circular form of DNA most commonly produced in plasmids and grown in bacteria. Plasmids are extrachromosomal DNA found in bacteria and are associated with the genes for antibiotic resistance which are often exchanged between bacteria and consequentially, are seen by many to embody a serious threat to global health. In addition, many nucleic acid-based therapies also rely on viral vectors for efficient transfection and expression of plasmid DNA. These viral vectors carry additional nontrivial risks and are extremely time consuming and expensive to manufacture. LineaRx is also developing a liquid biopsy form for isolating metastatic cells from blood. Go to http://www.adnas.com for more information on LineaRx and to learn more about how Applied DNA makes life real and safe. LineaRx is a majority-owned Applied DNA Sciences, Inc. (Nasdaq: APDN) company.

About Applied DNA Sciences

Applied DNA is a provider of molecular technologies that enable supply chain security, anti-counterfeiting and anti-theft technology, product genotyping and pre-clinical nucleic acid-based therapeutic drug candidates.

Applied DNA makes life real and safe by providing innovative, molecular-based technology solutions and services that can help protect products, brands, entire supply chains, and intellectual property of companies, governments and consumers from theft, counterfeiting, fraud and diversion.

Visit adnas.com for more information. Follow us on Twitter and LinkedIn. Join our mailing list.

Common stock listed on NASDAQ under the symbol APDN.

Forward-Looking Statements

The statements made by Applied DNA in this press release may be forward-looking in nature within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. Forward-looking statements describe Applied DNAs future plans, projections, strategies and expectations, and are based on assumptions and involve a number of risks and uncertainties, many of which are beyond the control of Applied DNA. Actual results could differ materially from those projected due to the possibility of a failure to make timely payment on its outstanding secured convertible notes and resulting enforcement by noteholders of remedies on collateral which includes substantially all of Applied DNAs assets, its history of net losses, limited financial resources, limited market acceptance, the uncertainties inherent in research and development, future clinical data and analysis, including whether any of Applied DNAs or its partners product candidates will advance further in the preclinical research or clinical trial process, including receiving clearance from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies to conduct clinical trials and whether and when, if at all, they will receive final approval from the U.S. FDA or equivalent foreign regulatory agencies, and various other factors detailed from time to time in Applied DNAs SEC reports and filings, including our Annual Report on Form 10-K filed on December 12, 2019 and other reports we file with the SEC, which are available at http://www.sec.gov. Applied DNA undertakes no obligation to update publicly any forward-looking statements to reflect new information, events or circumstances after the date hereof or to reflect the occurrence of unanticipated events, unless otherwise required by law.

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Applied DNA to Hold Investment Community Meetings During the JP Morgan 38th Annual Healthcare Conference and Present at the Biotech Showcase 2020 -...

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GERMANTOWN, Md., Jan. 10, 2020 (GLOBE NEWSWIRE) -- Orgenesis Inc. (NASDAQ: ORGS), a leading cell and gene therapy enabling company providing centralized CDMO manufacturing and development services, as well as localized point-of-care development and processing centers through its subsidiary Orgenesis Maryland, Inc., today announced the addition of the University of California, Davis (UC Davis) to its Point of Care (POCare) Network. Under the collaboration agreement, UC Davis Health will utilize Orgenesis POCare platform to develop, commercialize and supply cell and gene products and therapies. Orgenesis POCare Network enables hospitals to design and manage localized clean rooms, implementing Orgenesis proprietary automated, closed systems and know-how to process select cell therapies at each point-of-care site for the treatment of patients.

The first collaboration under the agreement involves scaling up and integrating UC Davis lentiviral vector process as part of the Orgenesis POCare platform for localized, development and processing of cell and gene therapies for treating patients. The UC Davis GMP facility has developed a small-intermediate scale, high quality vector process that has been successfully utilized to manufacture lentiviral vectors in several clinical trials, including manufacturing of CAR T cell therapies. Orgenesis POCare platform, which combines processing and therapeutic technologies, is designed to allow for the efficient production of high quality, affordable cell and gene based products. Upon successful completion of the collaboration, Orgenesis and UC Davis plan to pursue further commercialization of the technology and expand the processing and supply of their products under development at the UC Davis site. Lentivirus is a family of viruses that insert their DNA into the host cells' genome. Lentiviral vectors are increasingly utilized in cell and gene therapy as a method for inserting, modifying, or deleting specific genes within cells.

Vered Caplan, CEO of Orgenesis, stated, We are delighted to add UC Davis to our POCare Network, which will allow us to collaborate with the university to develop and supply therapeutics within the point-of-care setting in general and specifically in our need for virus supply. Additionally, we look forward to leveraging our POCare platform to assist UC Davis in expanding their ability to address the worldwide shortages of lentiviral vectors. Their new system is designed to address the global need for a more efficient, large scale vector manufacturing processes in an efficient manner. We believe this partnership further validates the significant value proposition of our POCare platform.

Adjunct Professor Gerhard Bauer, Director of the GMP Facility at UC Davis, commented, We, at the UC Davis Health, in the Stem Cell Program and in the GMP Facility, are committed to bringing these novel cell and gene therapy based treatments to patients in need and making them affordable.

Professor Jan A. Nolta, Director of the Stem Cell Program and the Gene Therapy Center at UC Davis Health, added, We look forward to leveraging Orgenesis expertise to accelerate the development and commercialization of our lentiviral vector manufacturing system, which addresses a significant unmet need in the market for an efficient and scalable manufacturing process.

About Orgenesis

Orgenesis is a biopharmaceutical company specializing in the development, manufacturing and processing of technologies and services in the cell and gene therapy industry. The Company operates through two platforms: (i) a point-of-care (POCare) cell therapy platform (PT) and (ii) a Contract Development and Manufacturing Organization (CDMO) platform conducted through its subsidiary, Masthercell Global. Through its PT business, the Companys aim is to further the development of Advanced Therapy Medicinal Products (ATMPs) through collaborations and in-licensing with other pre-clinical and clinical-stage biopharmaceutical companies and research and healthcare institutes to bring such ATMPs to patients. The Company out-licenses these ATMPs through regional partners to whom it also provides regulatory, pre-clinical and training services to support their activity in order to reach patients in a point-of-care hospital setting. Through the Companys CDMO platform, it is focused on providing contract manufacturing and development services for biopharmaceutical companies. Additional information is available at: http://www.orgenesis.com.

About UC Davis Stem Cell Program and Gene Therapy Center

UC Davis Stem Cell Program brings together physicians, research scientists, biomedical engineers and a range of other experts and collaborative partners at its Institute for Regenerative Cures, which is located on the universitys Sacramento campus. The $62 million facility, which was supported by the California Institute for Regenerative Medicine (CIRM), is the hub for collaborative, team-oriented science that is advancing breakthrough discoveries designed to bring stem cell therapies and cures to patients everywhere.

The UC Davis Gene Therapy Center brings together a uniquely comprehensive and established interdisciplinary network of experts and resources to lead the field of gene therapy through research, manufacturing, training and policy. The Gene Therapy Center offers expertise and state-of-the-art facilities and equipment including one of the largest university-based Good Manufacturing Practice (GMP) facilities and a Viral Vector Core.

About UC Davis

UC Davis is one of the top public universities in the United States. Since opening in 1908, it has been known for standout academics, sustainability and Aggie Pride as well as valuing the Northern California lifestyle. These themes are woven into its 100-plus-year history and its reputation for solving problems related to food, health, the environment and society. The universitys health system is based in Sacramento and provides the region's only academic health center. UC Davis Health is focused on discovering and sharing knowledge and providing the highest quality of care. It is a hub of innovation that encompasses UC Davis Medical Center, UC Davis School of Medicine, The Betty Irene Moore School of Nursing at UC Davis and UC Davis Medical Group.

Notice Regarding Forward-Looking Statements

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended. These forward-looking statements involve substantial uncertainties and risks and are based upon our current expectations, estimates and projections and reflect our beliefs and assumptions based upon information available to us at the date of this release. We caution readers that forward-looking statements are predictions based on our current expectations about future events. These forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and assumptions that are difficult to predict. Our actual results, performance or achievements could differ materially from those expressed or implied by the forward-looking statements as a result of a number of factors, including, but not limited to, the success of our reorganized CDMO operations, the success of our partnership with Great Point Partners, our ability to achieve and maintain overall profitability, the sufficiency of working capital to realize our business plans, the development of our POCare strategy, our trans-differentiation technology as therapeutic treatment for diabetes which could, if successful, be a cure for Type 1 Diabetes, the technology behind our in-licensed ATMPs not functioning as expected, our ability to retain key employees, our competitors developing better or cheaper alternatives to our products and the risks and uncertainties discussed under the heading "RISK FACTORS" in Item 1A of our Annual Report on Form 10-K for the fiscal year ended November 30, 2018, and in our other filings with the Securities and Exchange Commission. We undertake no obligation to revise or update any forward-looking statement for any reason.

Contact for Orgenesis:Crescendo Communications, LLCTel: 212-671-1021Orgs@crescendo-ir.com

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Orgenesis Announces Addition of University of California, Davis, to its Point of Care Network; UC Davis Health to Utilize Orgenesis' Point of Care...

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