By Murielle Gonzalez 26-Sep-2019

Design and Build | Pharmaceuticals

Expansion follows the success of first six cleanrooms built at the large-scale manufacturing centre in Stevenage

The centre has been fully operational since April 2019 and achieved MHRA licensure in August 2019

The UK's Cell and Gene Therapy Catapult (CGT Catapult) has completed six additional modules of cleanroom space at its manufacturing centre in Stevenage. The first collaborators are expected to start benefitting from this new space at the beginning of 2020.

CGT Catapult said the centre has seen increasing demand, which accelerated the planned build-out of the expansion phase with an additional six cleanrooms in the already constructed space on the second floor of the building.

The centre has been fully operational since April 2019 and achieved MHRA licence in August 2019. Depending on the process, each module can accommodate 20 parallel autologous cell processes or a bioreactor process up to 1,000L bioreactors for allogeneic cell or viral vector manufacture.

Dr Ian Campbell, Interim Executive-Chair, Innovate UK, for UK Research and Innovation, commented: In its first year and a half, the Cell and Gene Therapy Catapult manufacturing centre has gone from strength to strength and has established itself as a crucial part of the UKs medicines infrastructure.

Each module can accommodate 20 parallel autologous cell processes or a bioreactor process up to 1,000L bioreactors for allogeneic cell or viral vector manufacture

"This world-leading centre is bringing manufacturing of pioneering treatments closer to patients and will complement the new Advanced Therapy Treatment Centres. By bridging the gap between scientific research and commercialisation of the medicines of the future, this centre, which we are proud to support, will play an ever-greater role in this vital sector.

At the centre, CGT Catapult provides the infrastructure and expertise to enable companies to develop their manufacturing capabilities and systems for large scale, commercial cell and gene therapy supply.

Companies currently collaborating at the centre are Adaptimmune, Autolus, Cell Medica, Freeline Therapeutics and TCR2 Therapeutics.

Located within the rapidly growing European cell and gene therapy cluster in Stevenage, the manufacturing centre, alongside wider CGT Catapult initiatives, reinforces the UK Governments ambition to support the rapidly growing global cell and gene therapy industry in the United Kingdom.

The centre is backed by over 75 million of funding, including investment from the UK Governments Industrial Strategy Challenge Fund; the department of Business Energy and Industrial Strategy, from Innovate UK, the UKs innovation agency, and from the European Regional Development Fund.

CGT Catapult was awarded an additional 3.36m in funding from the European Regional Development Fund (ERDF) and 12m Medicines Manufacturing Industrial Strategy Challenge Fund to further support this expansion.

We are very pleased to be able to double the existing capacity of the CGT Catapult manufacturing centre with help from our partners Innovate UK and the European Regional Development Fund," said Keith Thompson, CEO, Cell and Gene Therapy Catapult.

Thompson pointed out that the growth of this centre is integral to the success of the industry in the UK. "We can now collaborate potentially with up to a further six companies to support them in developing their GMP manufacturing processes, preparing for large scale and commercial supply," he said.

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Cell and Gene Therapy Catapult doubles capacity at UK site - Cleanroom Technology

Recommendation and review posted by Bethany Smith

RESEARCH TRIANGLE PARK Fennec Pharmaceuticals Inc. has appointed Jodi Cook to its board of directors the company announced in a statement Tuesday.

Cook is currently the head of gene therapy for PTC Therapeutics Inc., a global biopharmaceutical company. Before beginning her work at PTC, she helped found Agilis Biotherapeutics, a clinical-stage company focused on gene therapies, where she also served as chief operating officer.

Jodi Cook

On behalf of the board of directors, we welcome theaddition of Jodi Cook, saidKhalid Islam, Fennecs chairman, in a press release. She brings extensive scientific, clinical and executive business experience to the Company. Her background and track record of success will enhance our team as we further advance the commercialization and development strategy of PEDMARK.

PEDMARK is a therapy intended to help children with hearing loss as a result of platinum-based chemotherapy. There is currently no preventative measure for this type of hearing loss. Fennec aims to complete its NDA submission for the treatment in early 2020 with a potential commercial launch of PEDMARK in the second half of 2020.

I am delighted to be joining Fennecs board of directors at this key point of development for PEDMARK, said Cook in the press release. Cisplatin induced hearing loss is a significant unmet medical need, a solution to which has evaded medicine for decades.

This story is from the North Carolina Business News Wire, a service of UNC-Chapel Hills School of Media and Journalism.

Excerpt from:
Fennec Pharma names gene therapy exec to its board - WRAL Tech Wire

Recommendation and review posted by Bethany Smith

WEST CHESTER Nathanael Ogden is your average 13-year-old. He has a younger brother, Jackson. He likes to read and play Dungeons & Dragons, enjoys board games and logic puzzles, and is running for vice president of his eighth-grade class at Peirce Middle School.

He also has something the majority of his peers dont have: a white cane. Nathanael has lost 95 percent of his vision over the last 10 years. But theres one more important thing he has: the spirit of a fighter.

Before he was born, Nathanael was diagnosed with Bardet Biedl Syndrome (BBS,) a rare genetic disorder.

Never heard of Bardet Biedl Syndrome? You're not alone. It is a rare syndrome that affects about 1 in 250,000 people in North America. According to bardetbiedl.org, people with BBS have a defect in the way their cells communicate with each other. It's a complex syndrome with a wide range of symptoms and a lot of variation from person-to-person. However, usually, people with BBS have low muscle tone, impaired kidney function, hampered senses, and vision loss.

Nathanael's parents, Tim and Catherine Ogden, first became aware that something was wrong with their baby during a 26-week ultrasound. The scan revealed that his kidneys were three times larger than they should have been, which Tim says is also indicative of a fatal kidney disease.

"We were told he wasn't going to live," said Tim Ogden.

The couple went to Children's Hospital of Philadelphia for a follow-up and doctors concluded that the baby most likely had BBS.

"They couldn't say definitively, but they were like 'Yeah, we think we know what this is.' Honestly, it was a huge blessing because most people will spend years trying to get a diagnosis for their child. It made a big difference."

Enlarged kidneys weren't the only symptom of BBS for Nathanael.

"Cilia are essentially the radio antennas of our cells. They are responsible for sending and receiving signals from cell to cell," explained Tim.

"When they break down, messages get garbled, or they don't get passed and, his kidneys didn't stop developing. He was also born with 24 digits for the same reason. His body made a pinky but didn't receive the message to stop, so it made another one. He was born with six toes on each foot, and seven fingers on his left hand."

Nathanael also had to deal with poor muscle development, speech problems, and dietary issues. All things he works on with pure grit and determination, and the aid of occupational, physical, and speech therapists. Nathanael has also learned to read braille and how to use a cane to help him navigate while walking.

"You never find him feeling sorry for himself or discouraged. It's always just 'This is what I have to do. This is my next task'" Tim says of Nathanael.

And, preparing for the next task he is. On Sunday, Sept. 29, Nathanael and Tim will tackle the third annual Rocky Ride for Bardet Biedl Syndrome. The trek begins at Uptown Worthington in Malvern, and winds through the Chester Valley Trail to the Schuylkill River Trail concluding at the Philadelphia Museum of Art. And, of course, the journey would not be complete without a triumphant run up the famous art museum steps.

The money raised from the Rocky Ride goes to support the Clinical Registry Investigating Bardet Biedl Syndrome (CRIBBS.) The registry consists of slightly over 500 people in the world diagnosed with BBS and helps researchers to develop effective and targeted treatment for those with the syndrome.

In the first year, the Rocky Ride raised $25,000. In year two, it raised nearly $35,000.

Funds raised from the Rocky Ride also support gene therapy research at the University of Iowa.

"Researchers have identified 23 genes associated with BBS. When research first began, there was only one. Four of them account for roughly 80 percent of people with symptoms of BBS. Those four genes are now part of a standard genetic testing panel which allows for a much earlier diagnosis. The earlier the diagnosis, the earlier services can be secured, and a plan can be put in action," said Tim.

"All of the research that has been done is exciting. When Nathanael was born, it felt like there was nothing and now, what we've been able to do over 13 years ... It feels like there's a lot of hope for people now."

Preparing for the task of riding 30 plus miles is a challenge. Tim and Nathanael spent a good part of the summer in training. They use a tandem bike for each of their training rides, which last anywhere from 15 to 25 miles per trip. Physical activity is a crucial part of Nathanael's care. Another symptom of BBS is the body's inability to recognize when it is full from food, so his parents keep a close eye on his diet and physical activity. Nathanael takes medicine to control his appetite.

"We've ridden a total of 165 miles since August in preparation for the Rocky Ride," said Tim.

For Nathanael, he enjoys the time with his father and feels a strong sense of accomplishment. "I like being able to do the bike ride itself. It's nice to be able to do exercise such as this," said Nathanael.

One hundred sixty-five miles is a lot of ground to cover. How does he find the energy?

"I don't know," he laughed.

All of Nathanael's senses are degenerating to some degree, with vision loss being the most severe.

According to his father, in first grade, he could see. Nathanael is now legally blind. He does the majority of his schoolwork on a computer screen with assistive technology.

"All of it has to be magnified and high contrast. It gets harder and harder, and then the question is - at what point is it more work to have him be able to see the screen as opposed to transitioning him to braille?" said Tim.

The Ogdens are humbled by the community support they have received.

"The biggest thing for anyone in the community to do is just to be there. A big part of having a child with a rare disease is the isolation. The feeling that no one knows about this, that no one understands," said Tim.

Families from across the West Chester Area School District are joining them for the ride.

"We're going to be barreling down the Schuykill River Trail - all 30 of us in red T-shirts to show that Nathanael doesn't have to do this alone. It's a big deal."

One repeat rider is Joe DiAntonio, Nathanael's principal at Peirce Middle School. DiAntonio will be riding with his son, who is a seventh-grader at Stetson Middle School.

"Nathanael is so special to our school," said DiAntonio. "He is everything that is right about Peirce. We want to support him in any way that we can. His attitude is so positive and the way he interacts with people. You just want to be around him."

Despite Nathanael's positive attitude, do Tim and Catherine ever question why this happened to their child?

"Absolutely. You can't get away from that. He lost the lottery. It was this totally bizarre thing that my wife and I had these genes that we didn't know we had. It's hard as a parent. You always want the best for your child."

For Tim, it was important to find something that he could do with his son and for other families with BBS. He helped start and is currently the president of the Bardet Biedl Syndrome Foundation, which provides support for families affected by the syndrome in North America and around the world.

"We're not really raising money for Nathanael. We are raising money that's going to benefit people in the future who have this syndrome and so he gets the feeling that there is something he can do. It's not about what he can't do, but what he can do to make the world a better place and help others," said Tim.

Tim and Nathanael drew inspiration for the Rocky Ride from the Rocky movies. The main character's attitude of determination is ever-present in Nathanael.

"Rocky doesn't win that first fight, but he still does it," said Tim. "That's the message that I want for Nathanael."

Nathanael's moto? "Keep pushing through it and try to find ways to be happy."

For more information on Bardet Biedl Syndrome, or to donate to the Rocky Ride, visit http://www.bardetbiedl.org/rockyride.

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Eighth-grader fights for himself and others with rare genetic syndrome - Daily Local News

Recommendation and review posted by Bethany Smith

RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Synteract, an innovative CRO partnering with biotech and pharma companies to bring new medicines to market, has hired two experienced leaders to further strengthen its Centers of Development in Oncology and Rare/Orphan diseases. Given the enhanced strategic solutions that often result from the intersecting expertise of Synteracts centers of development, clients will derive further benefit from the therapeutic knowledge and clinical development acumen gained from these key additions.

Hassan Aly, M.D., Ph.D., has joined Synteract as senior medical director supporting Synteracts Oncology Center of Development and Derek Ansel has joined as director of Rare and Orphan Disease Development. Both are clinical strategists with multidisciplinary experience in developing and operationalizing global therapeutic strategies.

Rare disease, oncology, and hematology clinical trials pose unique challenges that require the ability to think broadly while applying very specific trial strategies, said Steve Powell, Synteract CEO. Both Hassan and Derek bring a wealth of knowledge, expertise, and creativity, coupled with a passion to help patients and families, that will help us to further advance trials in the most complex and acute areas of need.

Dr. Aly is a certified hematologist with over 20 years experience in oncology/hematology medical research and patient treatment, including nine plus years in the clinical research and pharmaceutical industry. He brings deep therapeutic expertise across all phases of oncology/hematology drug development, having consulted on complex study design, conduct, monitoring, and submission. Prior to joining Synteract, he worked at AstraZeneca, Boehringer-Ingelheim, and leading CROs.

In addition, Dr. Aly has authored or co-authored 105 papers on the treatment and biology of malignant hematological diseases in peer-reviewed medical periodicals and made more than 70 presentations at British and European conferences.

Prior to joining Synteract, Derek Ansel was a clinical strategy lead at the Center for Rare Diseases and a co-founder of the Gene Therapy Working Group at a leading CRO. Ansel has a breadth of clinical research experience spanning from the laboratory through clinical monitoring and project management, across Phases I-IV. He will work in a multidisciplinary, cross therapeutic team, where he will apply extensive gene and cell therapy expertise developed from his work in several liver-targeted gene therapy programs.

Ansel frequently speaks at industry events. He is a member of the ACRP and holds a Certified Clinical Research Associate (CCRA) credential. He is an IRB member at the Thomas Jefferson University in Philadelphia and is also a contributing member of Cochrane, a non-profit organization that promotes evidence-based decision making in healthcare.

Dr. Frank Santoro, chief medical officer for Synteract, commented, Hassan and Derek bring unique skillsets to our centers of development with a combination of medical, therapeutic, and operational know-how related to running complex trials. This expertise, along with their crossover experience in multiple therapeutic areas, offers exceptional insight to our clients looking to partner with Synteract for their clinical development needs.

About Synteract

Bringing Clinical Trials to Life represents Synteracts commitment to engage with drug developers, patients, investigators, and regulatory experts to bring insights to action and make better therapies a reality. Synteract supports biotech and pharma companies across all phases of drug development to help bring new medicines to market. Synteract has conducted nearly 4,000 studies on six continents and in more than 60 countries. It has contributed to more than 240 product approvals. Synteract offers notable depth of therapeutic expertise in oncology, dermatology, general medicine, neuroscience, pediatrics, and rare and orphan diseases. Connect on LinkedIn and Twitter.

Original post:
Synteract Augments Leadership Team for Oncology and Rare Disease Centers of Development - Business Wire

Recommendation and review posted by Bethany Smith

NetworkNewsWire Editorial Coverage

NEW YORK, Sept. 24, 2019 /PRNewswire/ -- Advances in biotechnology look to deliver promising new treatment options against breast cancer. Independent researchers have found that Genprex Inc.'s (NASDAQ: GNPX) (GNPX Profile) TUSC2 prevented tumor growth against triple-negative breast cancer. Eli Lilly and Company (NYSE: LLY) is focusing on those patients with the greatest need and, in the process, has developed a new breast-cancer treatment option. Roche Holding (OTCQX: RHHBY) is expanding the use of its biotech to both identify and treat patients with challenging breast cancers. AbbVie Inc. (NYSE: ABBV) has been rapidly increasing its research, targeting more than 15 different types of cancer. AstraZeneca (NYSE: AZN) has seen hopeful results against metastatic breast cancer as it works in collaboration with a Japanese firm.

To view an infographic of this editorial, click here.

Battling Breast Cancer

Breast cancer is one of the most widely prevalent cancers in the world. Accounting for a quarter of all cancers in women worldwide, as well as a number of cases in men, it kills over half a million people every year.

Treatment for breast cancer varies, based on both the patient's circumstances and type of cancer. The development of treatments to suppress or even eliminate cancer has led to a high survival rate in wealthy countries, with around 85% of patients in the United States and United Kingdom surviving for at least five years from diagnosis. But even in these countries, survival depends upon the exact form of the cancer, how far it has progressed and whether a treatment has been developed for that particular form. Cancer is a difficult disease to defeat, and survival can depend upon the ability of scientists to counter a specific genetic defect in a specific set of cells.

Good News for Triple-Negative Breast Cancer Patients

Because cancers are so varied and challenging to tackle, every individual win is worth celebrating. That's why recent news relating to triple-negative breast cancer (TNBC) has brought excitement both for independent researchers and for Genprex Inc. (NASDAQ:GNPX), a clinical-stage, gene-therapy company whose tumor suppressor candidate 2 (TUSC2) was found to prevent tumor growth in TNBC.

TNBC covers a variety of cancers that do not express three specific types of receptor proteins. Most hormone therapies for breast cancer target one of these receptors, so tackling a cancer that doesn't feature any of those receptors is more difficult. TNBC is an extremely aggressive subtype of breast cancer associated with poor prognosis and high mortality rates. The lack of targeted treatment for triple-negative breast cancer makes it a particularly feared diagnosis. Because up to 20% of breast-cancer patients are fighting TNBC, finding therapies that effectively fight these forms of cancer is essential.

Recently TNBC patients have heard good news, thanks to research published in Nature. This research shows that TUSC2 prevented the growth of tumors in TNBC. And with Genprex already working on cancer treatments using TUSC2, including its lead drug candidate, Oncoprex(TM) immunogene therapy, the new research appears to validate the company's focus and direction.

Currently Genprex is conducting clinical and preclinical research to evaluate the effectiveness of TUSC2 when combined with targeted therapies and immunotherapies for non-small cell lung cancer. Existing preclinical data also suggest that TUSC2 may be effective against glioblastoma, head and neck cancer, kidney cancer and soft-tissue sarcomas. This new independent study raises the possibility that TUSC2 expression may also be used to treat this most aggressive subset of breast cancer.

"The results of the study evaluating TUSC2 for the treatment of triple-negative breast cancer are encouraging," said Genprex chairman and CEO Rodney Varner. "We believe that the data reported in this Nature article by independent researchers supports our belief that TUSC2 may be effective to treat a variety of cancers, including some of the most-deadly types of cancer."

This new report is a particularly encouraging moment for Genprex, as the study doesn't come from the company's own research work but instead verifies its value through an independent source. With multiple teams and researchers in multiple locations all evaluating and studying TUSC2 as a potential source for cancer treatment, the evidence is mounting that this approach may provide something new and valuable for doctors and patients.

Gene Therapy to Tackle Breast Cancer

Like many of the most promising cancer treatments, Genprex's TUSC2 treatment is a form of gene therapy.

A technology less than 50 years old, gene therapy remains on the cutting edge of modern medicine. The approach involves delivering new genetic material into the patient's body, where it is absorbed by cells. This rewrites the code of those cells, changing how they grow, reproduce, spread and die.

The growth and death of cells is fundamental to the challenge of cancer. Damaged genetic material leads cancer cells to develop in harmful ways and spread through the body, sometimes at a rapid rate. Treatments such as Genprex's Oncoprex can be used to write over the harmful DNA code and introduce a new gene. The result is that cancer itself is rewritten to reduce its harm.

Genetic treatments vary in the way they affect the body's cells. Some directly destroy cancerous cells. Others slow their reproduction, thereby reducing the spread of unhealthy tissue throughout the body. Other treatments make cells more susceptible to forms of treatment that might otherwise be ineffective.

TUSC2 treatment falls into both categories, helping cancer dells to die and preventing the growth of cancerous tumors. If successfully used, this approach could halt the growth of existing cancers and their spread through the body. TUSC2 is a vital element of treatment if cancer is to be prevented from running rampant through patients.

Genprex's Oncoprex therapy delivers cancer-fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles. The nanovesicles are then administered intravenously. They flow through the bloodstream until they locate and are absorbed by the cancer cells. The TUSC2 gene then expresses proteins that are missing or in short supply, bringing the damaged cells closer to normal behavior. By rewriting the very fundamentals of the body, such treatments may save lives.

Disruptive Technology for the Medical Sector

"Disruptive" isn't a term that's often applied to medicine, where the aim is to avoid disrupting the health of the human body. But technology such Genprex's Oncoprex immunogene therapy is disruptive precisely because it could improve doctors' ability to preserve health. It may also change the course of cancer, as new technology often opens up new possibilities.

Since its first successful use in the 1980s, gene therapy has rightly been presented as a disruptive technology. It deals with health on a basic level, altering the building blocks of life. This is why Genprex was among the companies presenting at the 4th Annual Disruptive Growth Conference in New York this September. Oncoprex's multimodal mechanism allows it to tackle cancer in a number of different ways, reducing the cancer's spread, encouraging the death of cancer cells and modulating the response of the immune system to fight cancer. It can block mechanisms that cause resistance to other anti-cancer drugs and so increase the effectiveness of a broader course of treatment.

As both treatments and the business models of companies behind them develop, the battle against cancer is being transformed.

Founded more than 140 years ago, Eli Lilly and Company (NYSE:LLY) is one of the oldest players in the field of cancer treatment, a company that has spent more than a century making medicines to help people around the world. Lilly uses predictive and prognostic biomarkers to work out how a tumor is likely to behave and how it will respond to potential therapies. This approach allows more targeted treatment designed to tackle the patient's specific cancer, thereby increasing the likelihood of success in any given case. LLY's Verzenio treatment has been shown to be effective in cancer treatment, encouraging cell death and reducing the spread of tumors. The company's strategy is a targeted one, concentrating on those cancer patients with the greatest need, and so providing the greatest possible impact from its medicines.

Another long-established company, Roche Holding (OTC:RHHBY) was one of the first to provide targeted treatments such as those offered by gene therapy. The world's largest biotech company, Roche has poured a huge amount of resources into biopharmaceuticals and is a global leader in cancer treatments. The company recently announced the expanded use of its technology to identify TNBC patients and so to provide them with the targeted treatment they need to fight their cancer. The company's support for cancer patients extends beyond better medicine for those in wealthy countries. Roche is a supporter of a health-care approach in South Africa that takes treatment to the rails, providing mobile health-care facilities and services that include breast cancer screening for people who might otherwise lack access.

A research-driven biopharmaceutical company, AbbVie Inc. (NYSE:ABBV) is working on new treatments for more than 15 different cancers, including forms of breast cancer. The company's wide-ranging work means that insights from one area can affect work in another, allowing greater progress in the overall understanding of cancer and the development of specific, targeted treatments. AbbVie's oncology research has increased dramatically over the past six years, often through partnerships with other companies, allowing a rich cross-fertilization of ideas.

Based in the United Kingdom, AstraZeneca (NYSE:AZN) benefits from its proximity to some of the finest minds in the world and an elite recruitment pool. Like AbbVie, AstraZeneca has been making breakthroughs in a wide range of cancer types. Research in collaboration with a Japanese company has recently led to promising results for the treatment of metastatic breast cancer, as the company works to improve survival rates and quality of life for breast cancer patients.

With the attention of so many big players in the biopharmaceutical industry, promising treatments for cancer are developing fast.

For more information about Genprex, please visit Genprex Inc. (NASDAQ:GNPX).

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Gene Therapies with Potential to Conquer Tough-to-Tackle Breast Cancers content= - PR Newswire UK

Recommendation and review posted by Bethany Smith

-- Webcast conference call to be held on Friday, Oct. 4, 2019 at 8:00 a.m. Eastern Time --

CAMBRIDGE, Mass., Sept. 25, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Friday, Oct. 4, 2019 at 8:00 a.m. Eastern Time (ET), it will host a webcast and conference call to present the 9-month functional results from the SRP-9003 gene therapy trial to treat Limb-girdle muscular dystrophy Type 2E, or beta-sarcoglyanopathy.

The presentation will include slides and will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-and-presentations and will be archived there following the call for one year. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers. The passcode for the call is 4986359. Please specify to the operator that you would like to join the "Sarepta hosted LGMD results call."

AboutSarepta TherapeuticsSarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for Limb-girdle muscular dystrophy diseases (LGMD), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit http://www.sarepta.com.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics to Announce 9-Month Functional Results from the SRP-9003 Gene Therapy Trial to Treat Limb-girdle Muscular Dystrophy Type 2E, or...

Recommendation and review posted by Bethany Smith

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Tai Chi is a relaxing exercise that can loosen joints, improve balance and teach graceful movements to music. Fun and no pressure classes are held at 9 a.m. Thursdays in the auditorium at the First Congregational Church Auditorium on Sound Beach Avenue in Old Greenwich. The cost is $10 per one-hour session. Newcomers welcome. For info, call Joe at 203-504-4678.

Special Education parent forum

The Stamford JCC will present a Special Education Parent Forum: Pursuing a Specialized School Placement at 7 p.m. Thursday at 1035 Newfield Ave., Stamford. Free and open to the public. Registration is recommended with Liza Fahey at lfahey@stamfordjcc.org or 203-487-0946. Workshop speakers will include Lawrence Berliner, special education law attorney in Westport; and Clay Kaufman, head of school, and Sue Rappaport, assistant director of admissions and placement, at Eagle Hill School in Greenwich.

Farm to Table Dinner

Chef Geoff Lazlo brings his Farm to Table Dinner series to his hometown of Greenwich at the Greenwich Botanical Center, 130 Bible St., Cos Cob. The elegant four-course dinner kicks off with a signature cocktail and passed hors doeuvres as guests are treated to highlights of the propertys magnificent history, followed by a seated dinner featuring key ingredients of herbs and vegetables locally grown. Dinners will be from 6 to 9 p.m. on Sept. 26 and Oct. 3. For more information and tickets, visit greenwichbotanicalcenter.org.

Qigong at the GBC

Qigong expert Donna Bunte teaches classes at 10 a.m. Fridays at the Greenwich Botanical Center, 130 Bible St., Cos Cob. With roots in Chinese medicine, philosophy and martial arts, qigong is viewed as a practice to cultivate and balance qi (chi), translated as life energy. Qigong practice involves moving meditation, coordinating slow flowing movement, deep rhythmic breathing, and a calm meditative state of mind. For information, visit greenwichbotanicalcenter.org.

Abilis Has Talent Show

The third annual Abilis Has Talent Show is a star-studded evening fun for all ages. The event is held at the First Presbyterian Church at 1 W. Putnam Ave., Greenwich, on Friday. Tickets are $25 with advance reservation or $30 at the door. Register at abilis.us/calendar. Abilis is a nonprofit organization that supports more than 700 individuals with special needs and their families annually from birth through adulthood.

Enjoy Oktoberfest

Enjoy beer and brats for Oktoberfest on Friday at the St. Lawrence Club. Its one of the favorite Family Night Dinners as Chef Tony prepares an authentic German-style feast with beer & cheese soup, schnitzel, sauerkraut, potato salad and strudel. Cost is $25 for adults, $10 for kids. No tax, no tip. Society members get a free drink. The St. Lawrence Club is at 86 Valley Road, Cos Cob. To RSVP, visit http://www.stlawrencesociety.com/events or call 203-618-9036.

Bruce closes temporarily

The Bruce Museum will then be closed through Friday to facilitate work in the main galleries. When it reopens Saturday, admission will be free to all visitors through Jan. 31 while the main gallery spaces are renovated. During the renovation phase, the Permanent Science Gallery will remain open, as will the Bantle Lecture Gallery, Education Workshop, and Museum Store. The galleries will reopen Feb. 1, with the installation of major new art and science exhibitions. Visit BruceMuseum.org for more information.

Farmers Market

The Greenwich Farmers Market runs every Saturday from 9:30 a.m. to 1 p.m. into the fall in the Arch Street commuter lot. Enjoy fresh Connecticut-grown produce all season. Over a dozen farm vendors will be in attendance. The parking lot at Arch Street and Horseneck Lane is off Exit 3 of I-95. For more information, visit http://www.greenwichfarmersmarketct.com/.

Cocktails & Comedy

There will be a cocktails and comedy fundraiser to venefit The Undies Project from 7 to 10 p.m. Saturday at St. Catherine of Siena in Riverside. To purchase tickets and for more info, visit e.givesmart.com/events/dWy/.

Greenwich Symphony Orchestra opens season

The Greenwich Symphony Orchestra will feature five concerts during its 62nd season, beginning Saturday and Sunday. The opening concerts will feature Bartok, Divertimento for Strings; Dittersdorf, Concerto for Harp, with Barbara Allen on harp; Wieniawski, Violin Concerto No. 2, with Edita Orlinyte on violin; and Debussy, Iberia. Concerts are at the Greenwich High School Performing Arts Center, 10 Hillside Road, at 8 p.m. on Saturdays and 4 p.m. on Sundays. Tickets are $40 per person, $10 students. For more information, call 203-869-2664 or visit http://www.greenwichsymphony.org.

Puttin on the Dog

Puttin On The Dog, the popular annual fundraising event for Armonk-based Adopt-A-Dog, will be held from 10 a.m. to 4:30 p.m. Sunday in Roger Sherman Baldwin Park in Greenwich. Dogs and cats will be available adoption that day. The event will also feature a silent auction, childrens activities, pet demonstrations, vendors and food trucks as well as canine competitions. Dogs are welcome, as well as people of all ages. For more information about the organization, visit adopt-a-dog.org, call 914-273-1674 or email adoptions@adoptadog.org.

Springsteen at Seventy

The C. Parker Gallery presents Growin Up Bruce Springsteen at 70, a 70th birthday celebration featuring rare photographs, artwork, gold records and signed memorabilia showcasing The Boss. The exhibit will run through Sept. 29 at the gallery at 409 Greenwich Ave. Highlights include rare unpublished 1972 to 1975 photographs from Bruces earliest days performing in the Philadelphia area from the late Phil Ceccola. Debra Rothenberg, author of Bruce Springsteen In Focus 1980 2012, will showcase her photographs. Free. All works are available for purchase. To learn more, visit http://www.cparkergallery.com.

Archery open shoot

The Cos Cob Archers will hold an Open Shoot from 8 a.m. to 1 p..m. Sept. 29 as they open their club to the public and invite all to attend. Open Shoots are held once a month, generally on the last Sunday of each month; bad weather cancels. The club provides loaner compound bows, arrows and instruction for beginners. The cost $20 for shooting adults, $5 for kids under 16, and $10 for non-shooting adults includes lunch of grilled hamburgers, hot dogs and soft drinks. The Clubs trails cover 23 wooded acres and there are more than 40 shooting stations, with both paper and 3D targets. Cos Cob Archers is located at 205 Bible St. For more information, visit http://www.CosCobArchers.com.

Meters for a Cure ERG Challenge

Swim Across America Fairfield County and Greenwich Crew are teaming for the second annual charity rowing event Meters for a Cure ERG Challenge with proceeds supporting Swim Across America Fairfield County and its local beneficiary, the Alliance for Cancer Gene Therapy, the nations only foundation dedicated exclusively to funding cancer gene therapy research. On Sept. 29, hundreds of crew members, their families and friends, will come together to help raise money for cancer research at the Greenwich Water Club at 49 River Road, Cos Cob, and relay against each other on ERG machines (a rowing machine also known as an ergometer). Teams are divided by age group and each participant is asked to raise at least $100. Open to all. Register at swimacrossamerica.org/gwc2019 or on-site that day starting at 7:30 a.m.

Choral Evensong

Join Christ Church Greenwich in celebrating Saint Michael and All Angels with music by Rose, Sumsion, and Bainton sung by the Choir of Men & Boys with members of the St. Cecilia Choir of Girls. The concert will be at 5 p.m. Sept. 29 at the church at 254 E. Putnam Ave. Admission is free. Visit christchurchgreenwich.org/music/ for more information.

Duplicate Bridge Games

Weekly open duplicate Bridge games are held at 12:15 p.m. Mondays at the Greenwich YWCA. The games are sanctioned by the American Contract Bridge League, with masterpoint awards to top finishers. The card fee to play one session is $12. For more information, contact Steve Becker at 203-637-8927.

Exhibit of Bird Mobiles

The Greenwich Art Society Gallery will present a show called Bird Mobiles: Suspension Of Time through Sept. 30 at the Arts Council Building at 299 Greenwich Ave. The mobiles were created by local artist Susan McHale, who creates her art with discarded branches found on the beach. The process of making my Bird Mobiles is like catching an ephemeral moment suspended in time, the hanging flock fly by and seem to continue their journey, she says. Call 203-629-1533 for more information.

Error art installation

The Brant Foundation Art Study Center is presenting Urs Fischer: Error, a solo exhibition of works by Fischer from the last two decades, through Oct. 1 at 941 North St. Featuring some of the artists most notable large-scale sculptures and paintings from the Brant Collections, Error celebrates The Brant Foundations 10th anniversary at its Greenwich space with Urs Fischer, the first artist to present a solo exhibition at there in 2010. To book a tour, visit brantfoundation.org/exhibitions/error/.

ASOG Fall Art Show

As summer fades and fall beckons, the Art Society of Old Greenwich is preparing for its 2019 ASOG Fall Art Show. The exhibit takes place at the Greenwich Botanical Center in Cos Cob from Oct. 1 to Oct. 28, with an artists reception on Oct. 6 from 3 to 5 p.m. Visitors can view the artwork, mingle with the artists, listen to live music and sample refreshments. The Greenwich Botanical Center is open 9 a.m. to 4 p.m. weekdays.Free parking on premises. For more information, contact artsocietyoldgreenwich@gmail.com or visit http://www.asogct.com/fall-show.

Abilis Gardens & Gifts

Starting in the fall, Abilis Gardens & Gifts will be selling holiday gifts and its greenhouse will be full of holiday wreaths and greenery. It is must-shop store offering handcrafted candles, bath products, childrens gifts, jewelry, housewares and gifts, with fresh flowers, plants and micro greens in the greenhouse. Prices range from $5 to $40 and proceeds support Abilis programs. Abilis Gardens & Gifts provides job training for adults with special needs. Hours are 10 a.m. to 5 p.m. Mondays, Wednesdays and Fridays; 10 a.m. to 3p.m. Tuesdays; 10 a.m. to 7 p.m. Thursdays; and 10 a.m. to 2 p.m. Saturdays for October, November and December. Abilis Gardens & Gifts is at 50 Glenville St. and can be reached at 203-531-GIFT (4438). For more info, visit http://www.abilis.us.

An American Story

The Greenwich Historical Society will present a new exhibition on the immigrant experience called An American Story: Finding Home in Fairfield County from Oct. 2 to Jan. 6. The exhibit will include poignant stories of the grit and resilience of immigrants and refugees, including 12 who found home in Greenwich. An American Story is a celebration of the determination, resilience and courage of the human spirit. The stories illuminated in this timely exhibition reach across the world from five continents, shining a light on the ways that refugees and asylum seekers find hope and persevere in the face of daunting challenges for creating new lives in Fairfield County. The landmark exhibition is presented in partnership with the Connecticut Institute for Refugees and Immigrants. The Greenwich Historical Society is located at 47 Strickland Road, Cos Cob. For more information, visit greenwichhistory.org.

Talk on The Bottom of the World

The Bruce Museum Presents, a new series of monthly public programs featuring thought leaders in the fields of art and science, showcases experts on compelling subjects of relevance and interest to members and visitors to the Bruce Museum. The series continues with an event presented in cooperation with Columbia Universitys Lamont-Doherty Earth Observatory called From the Bottom of the World: The Art and Science of Antarctica from 6 to 8:30 p.m. Oct. 3. It will convene experts in the art and science of this unique and fragile continent. Two research scientists from Lamont-Doherty, Jonny Kingslake and Kirsty Tinto, will reveal their new findings, while photographer Rick Sammon, who has twice visited and documented the Antarctic landscape, will remind us of its beauty. Leonard Jacobs moderates. Doors open at 6 p.m. for a reception with light bites and beverages, followed by the panel discussion and Q&A from 7 to 8:30 p.m. Seats are $30 for museum members, $45 for nonmembers. To reserve a seat, visit brucemuseum.org or call 203-869-0376.

Breast Cancer Awareness

To kick off Breast Cancer Awareness Month, Greenwich Town Hall will raise the Breast Cancer Alliances flag at 9 a.m. Oct. 3 with First Selectman Peter Tesei; Dr. Barbara Ward, director of breast care services at Greenwich Hospital, and Mary Jeffery, president of the BCA. The event will continue as Richards of Greenwich hosts the BCAs GoForPink breakfast called Love, Loss and Cancer: An Intimate Conversation with authors Lee Woodruff and Allison Gilbert at 10 a.m. Oct 3. The organizations annual luncheon, which takes place at 11 a.m. Oct. 21 at the Hyatt Regency Greenwich, will feature celebrity chef and restaurateur Todd English. For more info, go to http://www.BCAgoforpink.org, contact Info@breastcanceralliance.org or call 203-861-0014.

Dinner With a Cause

The Social Justice Committee of the Parish of St. Catherine of Siena and St. Agnes is sponsoring its Dinner with a Cause from 6:30 to 8:30 p.m. Oct. 3 at the Church Hall. The featured guest is Cathleen Caron, founder of Justice in Motion, a nonprofit dedicated to service the needs of migrant workers. Come for a light dinner at 6:30, presentation at 7:15 followed by Q & A. Free will offering to benefit Justice in Motion. For information on the nonprofit, visit https://justiceinmotion.org/about-us. RSVP to parkerelizabeth@sbcglobal.net or 203-637-8092.

Outdoor Arts Festival

More than 80 new and returning artists will showcase their work on the grounds of the Bruce Museum on Oct. 5 and Oct. 6, as the Greenwich museum celebrates the 38th edition of its annual Outdoor Arts Festival. The event will feature juried works, including paintings, mixed media in 2D and 3D, drawings and graphics such as digital media, sculpture and photography. There will be food-truck offerings and free art activities for children. Free parking offered in all nearby municipal lots. Festival admission is free to Bruce Museum members and children less than 5 years old; entry is $10 for non-members. Festival-goers can also tour the museum. Fore more info, visit BruceMuseum.org.

YWCAs walk against domestic violence

On Oct. 6, YWCA Greenwich is hosting a communitywide walk to raise awareness and funds for YWCA Greenwich Domestic Abuse Services and education. The family-friendly event takes place at Bruce Park. Registration will open at 8:30 a.m. Members of the community are encouraged to form teams, come with friends and family, and even dogs are welcome. To preregister, go to ywcagrn.org/walkthewalk. Proceeds will support the work of YWCA Greenwich Domestic Abuse Services. October is Domestic Violence Awareness and Prevention Month. YWCA is the state-designated provider of domestic abuse services for victims of domestic violence in Greenwich. If you or someone you know is a victim of domestic abuse, call the YWCA 24/7 hotline at 203-622-0003. For more information about the walk, contact Jackie Stam at 203-869-6501 x102 or j.stam@ywcagreenwich.org.

Teddy Bear Clinic

The Teddy Bear Clinic Greenwich Hospitals largest community event will celebrate its 21st anniversary from noon to 3 p.m. Oct. 6 at 49 Lake Ave. in the Greenwich Medical Building parking lot behind the hospital. Geared for children ages 3 through 12, this popular free event will take place rain or shine. Children and their families are invited to experience the friendly face of medicine at this event, staged as a mini-hospital under the tents. Visit with Greenwich Hospital doctors, nurses, technicians and other staff during this fun, interactive day of learning. Peer through a microscope, climb aboard an ambulance, check out the X-ray machine and test your balance by negotiating an obstacle course. Families can learn about nutritious food, strong bodies, sun protection, sports safety and other healthy habits. Children are encouraged to bring a favorite stuffed animal or doll for an exam. Depending on the diagnosis, dolls, bears and other fuzzy patients may receive splints, stitches, surgery or X-rays. Free parking in the hospitals staff lot on Lake Avenue. For information, call 203-863-3627.

First Sunday bird walks

A First Sunday Bird Walk will be held at Greenwich Point Park from 9 to 11:30 a.m. on Oct. 6, Oct. 13 and Oct. 20 to see the migrating hawks and raptors. Bring binoculars and meet near the main concession stand at the south end of the beach. The Bird Walks are a series of free, year-round, monthly bird walks providing friendly and informative birding. Guides are knowledgeable local naturalists and birders. Everyone from beginner to expert, of all ages is welcome. No fees or registration are required. For more information, visit birdwalk.home.blog/. Co-sponsored by Wild Wings and Friends of Greenwich Point.

Lecture on Classical Americana

The Greenwich Decorative Arts Society will present Classical Americana: The Life and Legacy of Richard Hampton Jenrette, a lecture by Peter M. Kenny, co-president of the Classical American Homes Preservation Trust. The event will be from 1:15 to 3 p.m., with refreshments to follow, on Oct. 7 at the Bruce Museum. Insights will be offered into the numerous houses he restored, his approach to their interior decoration and his role as a collector of classical American furniture from the workshops of New Yorks premier cabinetmakers of the early 19th century. Kenny was the Ruth Bigelow Wriston Curator of American Decorative Arts and Administrator of the American Wing at the Metropolitan Museum. Admission for nonmembers is $25. Space is limited. Reservations required for members and guests by Sept. 30. For reservations and information: email greenwichdecorativearts@gmail.com or visit http://www.greenwichdecorativearts.com.

Blood drives

The American Red Cross urges people of all races and ethnicities to give blood or platelets to help increase the diversity of the blood supply. The vast majority of blood types fall into one of the major blood groups. However, for patients with rare blood types or those who receive regular blood transfusions, blood must be matched closely - beyond the primary A, B, O and AB blood types - to reduce the risk of developing complications from transfusion therapy. The best match may be someone of the same racial or ethnic group. That includes patients who need blood transfusions to treat complications from sickle cell disease, a genetic disease that is most common among people of African descent or Latino descent. Local blood drives will be held from 1:30 to 6:30 p.m. Oct. 7 at Christ Church Greenwich, 254 E. Putnam Ave., and from 9 a.m. to 2 p.m. Oct. 8 at the Boys & Girls Club of Greenwich, 4 Horseneck Lane. Appointments can be made by downloading the free Red Cross Blood Donor App, visiting RedCrossBlood.org, or calling 1-800-RED CROSS (1-800-733-2767). As a thank-you, donors who give blood or platelets during October will be entered for a chance to win one of five $500 gift cards.

Outdoor movie night

As part of the Greenwich Reads Together program, there will be an outdoor screening of the movie Fahrenheit 451 at 7:30 p.m. Oct. 12 on the front lawn of the Greenwich Arts Council at 299 Greenwich Ave. Legendary French director Franois Truffaut brings to life a terrifying care-free future in which Guy Montag (Oskar Werner) questions his actions after meeting Clarisse (Julie Christie) and begins to rebel against society. Free and open to all, but registration is encouraged at http://www.greenwichlibrary.org. For more information, call 203-862-6750 or visit greenwichartscouncil.org. Co-sponsored by Alliance Francaise of Greenwich, The Avon Theater, The Commission on Aging, Greenwich Arts Council, Greenwich Library, and Sebass Events & Entertainment.

Honoring Columbus

The St. Lawrence Society will celebrate Columbus Day on Oct. 14 with an event honoring First Selectman Peter Tesei. Festivities begin with a 9 a.m. flag raising at Town Hall, followed by a breakfast at The Club and ending with cocktails, presentation of awards and an elegant Italian dinner back at the Club at 6:30 p.m. The cost for dinner is $100 per person. The St Lawrence Club is 86 Valley Road, Cos Cob. To RSVP, visit http://www.stlawrencesociety.com/events or call 203-618-9036.

Lush Landscapes benefit

Landscape designer James Doyle will present a program on Dream Locations and Special Places at 11:30 a.m. Oct. 17 at the Belle Haven Club as a benefit for Community Centers Inc., a Greenwich-based social service agency. Doyle, founder and principal of Greenwich-based Doyle Herman Design Associates, has developed an award-winning design business with projects in the United States, Europe and the Middle East. His work combines his horticultural expertise with strong design philosophy to bring to life unique and innovative landscapes. Projects range from small courtyards to large country estates. CCI, which has been serving the community for 65 years, is dedicated to building skills that empower clients to overcome educational, social and economic barriers. Tickets may be purchased on the CCI website at ccigreenwich.org.

Walk/Run for Abilis

Abilis will host its 15th annual Walk/Run for Abilis on Oct. 20 at Greenwich Point Park. This fun family-friendly event includes childrens activities, a Bubble Bus, music, arts & crafts, a 1-mile wheelchair and stroller-accessible walk and a 5k run. This event is one of Abilis largest fundraiser events of the year and attracts hundreds of participants for a morning of fun. Registration is $40 for adult runners; $20 for child runners (ages 11 to 17) and free for children under 11. There is no cost for the walk portion of the event. To register in advance, donate and or volunteer, visit abilis.us/walkrun. Abilis is a nonprofit organization that supports more than 700 individuals with special needs and their families annually from birth throughout adulthood in the area.

Model sailboat regatta

The Old Greenwich-Riverside Community Center will hold its popular annual Model Sailboat Regatta from 1 to 4 p.m. Oct. 20 at Binney Park. Model sailboat enthusiasts of all ages are welcome, with categories for homemade boats, motorized and remote-controlled boats spread out throughout the afternoon. Hundreds of families are expected to attend. Many families set up a picnic for the day as kids run around and enjoy Binney. The OGRCC provides arts and crafts activities and music. The regatta raises money for the Scholarship Program that supports over 70 families in the community each year for everything from soccer to childcare to summer camps. To buy a ticket, visit myogrcc.org. Walk-ins are welcome. For more info, call 203-637-3659.

TAGs grand prix fundraiser

The Transportation Association of Greenwich will hold its fall fundraiser, the Greenwich Grand Prix, from 3 to 5 p.m. Oct. 27 at the RPM Raceway in Stamford. The proceeds of the go-kart racing event will be used to purchase a new 20-passenger vehicle with wheelchair lift. The go-kart racing teams are from Greenwichs top car dealers, who will be competing for the Greenwich Grand Prix Championship. The community is invited to cheer on the teams and support TAG to raise these much-needed funds. Tickets are on sale now. For more information on tickets, teams, donations and sponsorship, visit ridetag.org/greenwich-grand-prix.

Nostalgia dining

A Remember Maneros Steak Dinner will be held at 6:30 p.m. Nov. 1 at The St. Lawrence Club, 86 Valley Road, Cos Cob. Join the club and reminisce over the beloved and sorely missed Greenwich landmark restaurant. Diners can linger with family and friends over a mouth-watering meal of Steak Tid-bits, garlic bread, fried onions, gorgonzola salad and more. Theres no tax or tip, and members of the St. Lawrence Society get a free drink. Cost is $40 for members, $45 for guests and $25 for kids. To RSVP, visit http://www.stlawrencesociety.com/events or call 203-618-9036.

Celebrating documentaries on artists

The Bruce Museum Presents, a new series of monthly public programs featuring thought leaders in the fields of art and science, showcases experts on compelling subjects of relevance and interest to members and visitors to the Bruce Museum. The series continues with Rewind/Fast-Forward: Celebrating the Artist Documentaries of Olympia Stone from 6 to 8:30 p.m. Nov. 7. Stone is an independent producer of documentary films about art and artists. Her first documentary, The Collector, explored the five-decade career of her father, famed NYC gallery owner and art collector Allan Stone. For the event, join Stone and two of her artist subjects James Grashow (The Cardboard Bernini) and Elizabeth King (Double Take: The Art of Elizabeth King) for a retrospective of these films, and their maker. Moderated by Leonard Jacobs. Doors open at 6 p.m. for a reception with light bites and beverages, followed by the panel discussion and Q&A from 7 to 8:30 p.m. Seats are $30 for museum members, $45 for nonmembers. To reserve a seat, visit brucemuseum.org or call 203-8690376.

Bacon Brothers perform for charity

The Bacon Brothers, a band started by the award-winning actor Kevin Bacon and his brother Michael, an Emmy-winning composer, will perform a rare intimate concert in Greenwich on Nov. 9 at a fundraiser for Fairfield Countys Inner-City Foundation for Charity & Education. The event, the 28th annual fall gala to benefit The Inner-City Foundation, will be held at the Hyatt Regency in Old Greenwich. It is the largest fundraiser of the year for the Inner-City Foundation, which provides support to local Fairfield County programs and organizations that provide education, food, shelter, help for addiction, domestic violence and much more to the neediest in Fairfield County. The band also includes Paul Guzzle (bass, backing vocals), Joe Mennonna (keyboards, accordion), Tim Quick (lead guitar, mandolin and backing vocals) and Frank Vilardi (drums). For more information and tickets, tables or sponsorships, visit innercityfoundation.org or call 203-416-1363.

Sip & Shop Art Show

Stop by the Holiday Sip & Shop Art Show at Abilis Gardens & Gifts from 5 to 8 p.m. Dec. 4. Enjoy wine, cheese and other refreshments while shopping with a 20 percent discount in the entire store. The Art Show will feature paintings and digital art created by adults who are supported by Abilis. To learn more, visit abilis.us/calendar. Abilis is a nonprofit organization that supports more than 700 individuals with special needs and their families annually from birth throughout adulthood in the area.

Talk on art and climate change

The Bruce Museum Presents is a new series of monthly public programs featuring thought leaders in the fields of art and science. It will showcase experts on compelling subjects of relevance and interest to members and visitors to the Bruce Museum. The program continues with Can Art Drive Change on Climate Change? An Evening with Alexis Rockman from 6 to 8:30 p.m. Dec. 5. Among current American artists profoundly motivated by nature and its future from the specter of climate change to the implications of genetic engineering Rockman holds a high place of honor. Join the Bruce for a night of discussion and debate featuring Rockman and a panel of thought leaders in contemporary art and science. Doors open at 6 p.m. for a reception with light bites and beverages, followed by the panel discussion and Q&A from 7 to 8:30 p.m. Seats are $30 for Museum members, $45 for nonmembers. To reserve a seat, visit brucemuseum.org or call 203-869-0376.

SLS golf tourney

The St. Lawrence Society will hold its 29th annual Charity Golf Tournament on May 11. Enjoy a perfect day of golf, food and fun. Get your foursomes together and join the fun. Cost is $175 for everything: the luncheon, cart, golf, cocktail hour and dinner. For cocktails and dinner only, its $100. Starts at 11:30 a.m. at E. Gaynor Brennan Golf Course, with a 12:45 p.m. shotgun start, scramble format. Golf is followed by cocktail hour, antipasti and a prime rib dinner back at the Club. To RSVP, visit http://www.stlawrencesociety.com/events or call 203-618-9036.

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Greenwich Symphony Orchestra concerts and other things to do - CT Insider

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Newswise The Childrens Cancer Institute at the Joseph M. Sanzari Childrens Hospital at Hackensack Meridian Health Hackensack University Medical Center and the John Theurer Cancer Center have announced they are participating in a multicenter Phase I/II clinical trial of an investigational gene therapy from bluebird bio, Inc. This trial is specifically for adolescents and adults with severe sickle cell disease (SCD) who cannot be effectively treated using standard therapies such as antibiotics, vitamins, blood transfusions or any pain relieving medications. The study is evaluating the safety and effectiveness of LentiGlobin for sickle cell disease, a gene therapy produced using the patients own modified stem cells to treat their sickle cell disease.

By using the patients own cells to produce functional hemoglobin that can prevent sickling of their red blood cells, LentiGlobin for SCD offers patients the opportunity to treat their disease without the need to have a matched bone marrow donor. The John Theurer Cancer Center is one of a limited number of centers internationally, and the Joseph M. Sanzari Childrens Hospital is the only pediatric site in New Jersey, where the study, which is enrolling patients age 12-50, is taking place.

Sickle cell affects 100,000 Americans. It affects one in every 365 African American births and one in every 16,000 Hispanic American births, said Alfred P. Gillio, M.D., director, Childrens Cancer Institute and section chief, Pediatric Stem Cell Transplantation and Cellular Therapy Program, Joseph M. Sanzari Childrens Hospital at Hackensack University Medical Center. This trial is for patients who have severe sickle cell disease and seek advanced treatment options but do not have a well-matched stem cell donor. Only 15% of sickle cell patients have a matched sibling donor and only 25 percent of patients have a matched unrelated volunteer donor.

Sickle cell affects every organ in a patients body, said Stacey Rifkin-Zenenberg, D.O., FAAP, pediatric hematologist/oncologist, Childrens Cancer Institute, and section chief, Pain and Palliative Care, Joseph M. Sanzari Childrens Hospital at Hackensack University Medical Center. This disease really has a tremendous effect not only on the patient, but also the family.

Sickle cell disease is an inherited disease caused by a mutation in the beta-globin gene, resulting in abnormal hemoglobin and sickle-shaped red blood cells. Symptoms and complications of the disease include anemia, infections, stroke, poor quality of life and early death. To date, the only cure for sickle cell disease is receiving a stem cell transplant from a matched donor, but this is not a therapeutic option for many patients. Supportive care including hydroxyurea and blood transfusions can ameliorate symptoms of the disease. To date, without a marrow donor, there has been no alternate curative therapy. Life expectancy of a person with sickle cell disease is 20 to 40 years of age. In some cases, patients using disease modifying medications can live to 50 or 60.

This therapy may be a major advance for sickle cell patients and so far, the results look very promising, said Scott D. Rowley, M.D., FACP, hematologist, medical director, Stem Cell Transplantation and Cellular Therapy and medical director, BMT Cell Lab, John Theurer Cancer Center, Hackensack Meridian Health Hackensack University Medical Center, who is enrolling adult patients. This investigational treatment, which is a one-time therapy, may be an option for our patients who have no other treatment options.

The results from early clinical studies are encouraging, said Dr. Gillio. With this treatment, the patient is their own donor and we are modifying their own cells to add copies of a functional beta globin gene.

In the current study:

About Hackensack Meridian Health Hackensack University Medical Center

Hackensack Meridian Health Hackensack University Medical Center, a 781-bed nonprofit teaching and research hospital located in Bergen County, NJ, is the largest provider of inpatient and outpatient services in the state. Founded in 1888 as the countys first hospital, it is now part of the largest, most comprehensive and truly integrated health care network in New Jersey, offering a complete range of medical services, innovative research and life-enhancing care, which is comprised of 34,100 team members and more than 6,500 physicians. Hackensack University Medical Center is ranked #2 in New Jersey and #59 in the country in U.S. News & World Reports 2019-20 Best Hospital rankings and is ranked high-performing in the U.S. in colon cancer surgery,lung cancersurgery,COPD, heart failure, heart bypass surgery, aortic valve surgery,abdominal aortic aneurysm repair, knee replacement and hip replacement. Out of 4,500 hospitals evaluated, Hackensack is one of only 57 that received a top rating in all nine procedures and conditions. Hackensack University Medical Center is one of only five major academic medical centers in the nation to receive Healthgrades Americas 50 Best Hospitals Award for five or more years in a row. Beckers Hospital Review recognized Hackensack University Medical Center as one of the 100 Great Hospitals in America 2018. The medical center is one of the top 25 green hospitals in the country according to Practice Greenhealth, and received 26 Gold Seals of Approval by The Joint Commission more than any other hospital in the country. It was the first hospital in New Jersey and second in the nation to become a Magnet recognized hospital for nursing excellence; receiving its sixth consecutive designation in 2019. Hackensack University Medical Center has created an entire campus of award-winning care, including: John Theurer Cancer Center, a consortium member of the NCI-designated Georgetown Lombardi Comprehensive Cancer Center; the Heart & Vascular Hospital; and the Sarkis and Siran Gabrellian Womens and Childrens Pavilion, which houses the Joseph M. Sanzari Childrens Hospital and Donna A. Sanzari Womens Hospital, which was designed with The Deirdre Imus Environmental Health Center and listed on the Green Guides list of Top 10 Green Hospitals in the U.S. Hackensack University Medical Center is the Hometown Hospital of the New York Giants and the New York Red Bulls and is Official Medical Services Provider to THE NORTHERN TRUST PGA Golf Tournament. It remains committed to its community through fundraising and community events especially the Tackle Kids Cancer Campaign providing much needed research at the Childrens Cancer Institute housed at the Joseph M. Sanzari Childrens Hospital. To learn more, visit http://www.HackensackUMC.org.

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Joseph M. Sanzari Childrens Hospital and John Theurer Cancer Center Launch Clinical Trial Evaluating Gene Therapy for Severe Sickle Cell Disease in...

Recommendation and review posted by Bethany Smith

A study in Molecular Therapy describes how neuroD1-based gene therapy turns glial cells abundant support cells in the brain into neurons, repairing damage that results from stroke and significantly improving motor function in mice.

Once further developed, this NeuroD1-based gene therapy could potentially be used to treat stroke, the researchers suggest.

The current treatment for stroke has a narrow time window, typically within a few hours after the occurrence of stroke, says lead author Yuchen Chen, a postdoctoral fellow at Penn State, in a media release.

Many patients cannot receive the treatment in time and as a result, often suffer from permanent disability caused by irreversible neuronal loss. There is an urgent need to develop a new therapy to regenerate new neurons and restore lost brain functions among stroke patients.

The human brain has approximately 86 billion neurons. While mini-strokes can be tolerated, moderate stroke involving the loss of billions of neurons leaves detrimental effects that do not spontaneously recover.

So, the critical question that is still unanswered in the neuroregeneration field is how can we regenerate billions of new neurons in a patients brain after stroke? said Gong Chen, professor of biology and Verne M. Willaman Chair in Life Sciences at Penn State and leader of the research team. The biggest obstacle for brain repair is that neurons cannot regenerate themselves. Many clinical trials for stroke have failed over the past several decades, largely because none of them can regenerate enough new neurons to replenish the lost neurons.

Gong Chen and his team pioneered a new approach to regenerate functional neurons using glial cells, a group of cells surrounding every single neuron in the brain that provide essential support to neurons. Unlike neurons, glial cells can divide and regenerate themselves, especially after brain injury.

I believe that turning glial cells that are already present in the brain into new neurons is the best way to replenish the lost neurons, Gong Chen states. These glial cells are the neighbors of the dead neurons in the brain and are likely to share the same ancestral cellular lineage.

Gong Chens team previously reported that a single genetic neural factor, NeuroD1, could directly convert glial cells into functional neurons inside mouse brains with Alzheimers disease, but the total number of neurons generated was limited. The research team believed that this limited regeneration was due to the retroviral system used to deliver NeuroD1 to the brain. In the current study, the research team used the AAV viral system, which is now the first choice for gene therapy in the nervous system, to deliver NeuroD1 into mouse motor cortex that had suffered from stroke.

Many neurons die after stroke but surviving glial cells can proliferate and form a glial scar in the stroke areas. The AAV system was designed to express NeuroD1 preferentially in the glial cells that form these scars, turning them directly into neuronal cells. Such direct glia-to-neuron conversion technology not only increased neuronal density in the stroke areas, but also significantly reduced brain tissue loss caused by the stroke.

Interestingly, the newly converted neurons showed similar neuronal properties to the neurons that were lost after stroke. This suggests a potential impact of the local glial lineage on the converted neuronal identity, the release explains.

The most exciting finding of this study is to see the newly converted neurons being fully functional in firing repetitive action potentials and forming synaptic networks with other preexisting neurons, Gong Chen adds. They also send out long-range axonal projections to the right targets and facilitate motor functional recovery.

A separate collaborative work led by Gregory Quirk, professor at the University of Puerto Rico, further tested the NeuroD1-based gene therapy in a rat stroke model. Quirk and colleagues also found that this direct glia-to-neuron conversion technology can rescue cognitive functional deficits induced by stroke.

Because glial cells are everywhere in the brain and can divide to regenerate themselves, our study provides the proof-of-concept that glial cells in the brain can be tapped as a fountain of youth to regenerate functional new neurons for brain repair not only for stroke but also for many other neurological disorders that result in neuronal loss, Yuchen Chen comments.

Our next step is to further test this technology and ultimately to translate it into clinically effective therapies to benefit millions of patients worldwide.

[Source(s): Penn State, Science Daily]

Read more here:
Gene Therapy Could Help Improve Functional Recovery After Stroke - Physical Therapy Products

Recommendation and review posted by Bethany Smith

WASHINGTON, D.C., Sept. 24, 2019 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE -- The Alliance for Regenerative Medicine (ARM) and the National Association of Managed Care Physicians (NAMCP) Medical Directors Institute announced today the joint release of their recent study of medical director and manufacturer perspectives on value demonstration and reimbursement for cell- and gene-based regenerative and advanced therapies.

The study, entitled Roadmap for Navigating Cell and Gene Therapy Value Demonstration and Reimbursement in U.S. Managed Care, characterizes step-by-step considerations for achieving appropriate patient access to transformative and potentially curative therapies in the U.S. managed care setting. The findings identify key issues relevant to value demonstration and access to potentially curative therapies at a pivotal time for the industry, as several products have reached the market, with dozens more in late-stage clinical trials.

The initial wave of cell and gene therapies has launched into an environment that was not built with transformative or curative therapies in mind, said Eric Faulkner, Vice President, Precision and Transformative Medicine at Evidera and lead author for the publication. Its crucial for payers, providers, patients, and other stakeholders to align on expectations on value demonstration to ensure sustainable access.

In the next two to three years, the sector expects the number of marketed cell and gene therapies to more than triple, said Janet Lambert, CEO for the Alliance for Regenerative Medicine. We will improve patient lives if we ensure patients have timely access to these treatments and value assessment and reimbursement processes have been structured with these unique therapies in mind.

Appropriate reimbursement and access to transformative therapies is critical to constantly improve managed care practice, appropriate patient access and outcomes. This effort is one step to achieving that goal by identifying and acknowledging the key acceptance requirements and stakeholder perspectives as these therapies enter the marketplace, said Bill Williams, MD, Executive Vice President of the NAMCP Medical Directors Institute.

The study concludes with several key learnings for therapeutic developers and payers:

The study results are available to download here.

About The Alliance for Regenerative MedicineThe Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 350 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visithttp://www.alliancerm.org.

About the NAMCP Medical Directors InstituteThe NAMCP Medical Directors Institute is a non-profit membership association, which was established to provide tools, education, and resources to medical directors, practicing physicians, and other healthcare professionals. NAMCP Medical Directors Institutes mission is to help Medical Directors from purchasers, health plans, and provider systems make effective and informed decisions, respond to opportunities and challenges in managed care, while helping improve healthcare outcomes, and ultimately the lives of members and patients. To learn more about the NAMCP Medical Directors Institute or to become a member, visit http://www.namcp.org

Lyndsey Scull202-213-7086lscull@alliancerm.org

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ARM and NAMCP Publish Recommendations to Increase Patient Access in Joint Study, "Roadmap for Navigating Cell and Gene Therapy Value...

Recommendation and review posted by Bethany Smith

PARIS--(BUSINESS WIRE)--

Regulatory News:

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190922005072/en/

GenSight Biologics (SIGHT.PA) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported the first set of results from Week 96 of the RESCUE Phase III clinical trial. The trial evaluated the efficacy and safety of a single intravitreal injection of GS010 (rAAV2/2-ND4) in 39 subjects whose visual loss due to 11778-ND4 Leber Hereditary Optic Neuropathy (LHON) commenced up to 6 months prior to study treatment. Week 96, which marks the time when individual patient profiles can be analyzed, is the last of the scheduled readouts for the RESCUE trial and completes the data collection from GS010s pivotal trials in Europe.

The results point to continued efficacy of GS010 two years past injection, with best-corrected visual acuity (BCVA) sustaining a clinically meaningful improvement over nadir. Having been treated early in the course of the disease, RESCUE patients vision initially deteriorated to a worst point, or nadir, before beginning to recover.

Note: A mixed model of analysis of covariance (ANCOVA) was used with change from baseline as the response, and subject, eyes of the subject as random factor, treatment and the baseline LogMAR value as covariates in the model.

When visual acuity is measured from the post-baseline nadir, the visual acuity of GS010-treated eyes in fact recovered significantly from the worst BCVA reading post-baseline.

Table 1: BCVA, Change from Nadir*, RESCUE

Week 48

Week 72

Week 96

N

Mean (SD)

N

Mean (SD)

N

Mean (SD)

All-GS010 Eyes

36

+12.8 (17.9)

34

+20.6 (26.3)

34

+24.9 (3.8)

All-Sham Eyes

36

+11.8 (15.6)

33

+21.7 (25.1)

34

+22.3 (3.8)

Note: *Nadir defined as worst BCVA measured in LogMAR after baseline, up to the week of interest; baseline reading was excluded from consideration. Mean change was calculated using observed values (no data imputation).

GS010-treated eyes regained more than two-thirds of the initial loss occurring in the most acute phase of the disease. This improvement from nadir (-0.498 LogMAR mean improvement, or +24.9 ETDRS letters equivalent) corresponds to 5 lines of Snellen acuity and is far above the 3-line threshold commonly accepted as a clinically meaningful level of visual improvement. Such recovery of vision is unprecedented in any gene therapy trial. Moreover, these results demonstrate the durability of improvement seen in earlier readouts of this trial.

Vision in sham-treated eyes evolved in parallel fashion, continuing the bilateral improvement already observed in earlier Phase III readouts. The picture that has emerged is one of durable, bilateral recovery from the effects of the acute phase in LHON.

The BCVA results from RESCUE show a remarkable correspondence with those from the REVERSE trial, which studied the treatment of subjects at 6 to 12 months after onset of vision loss.

The visual evolution in RESCUE appears to be a phase shift of the REVERSE curves, with an additional impact from the acute phase. This coherence will be more rigorously explored in a meta-analysis of the pooled data from the two trials, which is planned for this year.

In previous analyses of these trials, LHON clinical experts confirmed that the visual recovery seen in these trials are at odds with and much superior to their observations from clinical practice. That natural history of vision in untreated LHON patients stands in strong contrast to acuities seen in both RESCUE and REVERSE.

A natural history study conducted by Santhera1 provides another way of assessing the results in RESCUE. In that study, 28% of subjects who had the 11778A mutation achieved the following definition of spontaneous clinically relevant recovery (CRR) from nadir in at least one eye:

By comparison, 58% of RESCUE subjects achieved this definition of CRR in at least one eye at Week 96, with GS010-treated eyes as likely to achieve this as sham-treated eyes (58% vs. 45%, p = 0.0956).

The results from the RESCUE study are encouraging and convincing, particularly because we are seeing a similar pattern to the REVERSE study results, said Dr. Mark L. Moster, Neuro-Ophthalmology, Wills Eye Hospital, Professor of Neurology and Ophthalmology at Thomas Jefferson University, Philadelphia, PA, and Principal Investigator in the RESCUE and REVERSE trials. Patients in RESCUE were treated before the nadir so, as expected, they continued to worsen early on. But then from week 48 until week 96 they experienced a recovery from the nadir. That is much better than the natural history in any prior studies.

Story continues

Examination of other visual functions and biomarkers, including Contrast Sensitivity, show that these measures stabilized at Week 96. Based on preliminary analysis of the safety data, GS010 was well-tolerated after 96 weeks. There were no ocular serious adverse events or discontinuations that were due to ocular events. The ocular adverse events most frequently reported in the therapy group were mainly related to the injection procedure, except for the occurrence of intraocular inflammation (accompanied by elevation of intraocular pressure in some patients) that is likely related to GS010, and which was responsive to conventional treatment and without sequelae. There were no systemic serious adverse events or discontinuations that were related to study treatment or study procedure.

These results are remarkable, showing the durable difference that GS010 can make for patients who would otherwise go blind due to the onset of LHON, commented Bernard Gilly, Co-founder and Chief Executive Officer of GenSight. These findings, which we will be discussing at the meetings we have planned with regulatory authorities, form a compelling core for the clinical and non-clinical data that support our marketing authorization application in Europe. GenSight is excited to have reached this milestone in GS010s clinical development and energized by the prospect of pulling it all together for our European dossier.

GenSight is planning to schedule a pre-submission meeting with the EMA in early 2020 and expects to submit application for marketing approval in Europe in the third quarter of 2020.

An End of Phase II meeting with the U.S. Food and Drug Administration (FDA) has been requested and is expected for November 2019.

GenSight will host a conference call today, September 23, 2019, at 10:30am CEST in French, and at 2.30pm CEST (8.30am EST) in English, to discuss in greater detail these results and the roadmap to submission.

Webcast & Conference call in French

Dial-in numbers:

United States: +1 212 999 6659 France: +33 (0) 1 7037 7166 United Kingdom: +44 (0) 20 3003 2666 Password: GenSight

Webcast link: https://channel.royalcast.com/webcast/gensightbiologicsfr/20190923_1/

Webcast & Conference call in English

Dial-in numbers:

United States: +1 212 999 6659 France: +33 (0) 1 7037 7166 United Kingdom: +44 (0) 20 3003 2666 Password: GenSight

Webcast link: https://channel.royalcast.com/webcast/gensightbiologicsen/20190923_1/

A replay of the calls and webcasts will be available by using the above links.

Reference:

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, GS010, is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

About GS010

GS010 targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function.

About Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1st eye, with the 2nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of patients have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. The estimated incidence of LHON is approximately 1,400 to 1,500 new patients who lose their sight every year in the United States and Europe.

About RESCUE and REVERSE

RESCUE and REVERSE are two separate randomized, double-masked, sham-controlled Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene.

The primary endpoint will measure the difference in efficacy of GS010 in treated eyes compared to sham-treated eyes based on BestCorrected Visual Acuity (BCVA), as measured with the ETDRS at 48 weeks post-injection. The patients LogMAR (Logarithm of the Minimal Angle of Resolution) scores, which are derived from the number of letters patients read on the ETDRS chart, will be used for statistical purposes. Both trials have been adequately powered to evaluate a clinically relevant difference of at least 15 ETDRS letters between treated and untreated eyes adjusted to baseline.

The secondary endpoints will involve the application of the primary analysis to bestseeing eyes that received GS010 compared to those receiving sham, and to worseseeing eyes that received GS010 compared to those that received sham. Additionally, a categorical evaluation with a responder analysis will be evaluated, including the proportion of patients who maintain vision (< ETDRS 15L loss), the proportion of patients who gain 15 ETDRS letters from baseline and the proportion of patients with Snellen acuity of >20/200. Complementary vision metrics will include automated visual fields, optical coherence tomography, and color and contrast sensitivity, in addition to quality of life scales, biodissemination and the time course of immune response. Readouts for these endpoints are at 48, 72 and 96 weeks after injection.

The trials are conducted in parallel, in 37 subjects for REVERSE and 39 subjects for RESCUE, in 7 centers across the United States, the UK, France, Germany and Italy. Week 96 results were reported in 2019 for both trials, after which patients were transferred to a long-term follow-up study that will last for three years.

ClinicalTrials.gov Identifiers: REVERSE: NCT02652780 RESCUE: NCT02652767

About REFLECT

REFLECT is a multi-center, randomized, double-masked, placebo-controlled study to evaluate the safety and efficacy of bilateral injections of GS010 in subjects with LHON due to the NADH dehydrogenase 4 (ND4) mutation.

The trial planned to enroll 90 patients with vision loss up to 1 year in duration and will be conducted in multiple centers in Europe and in the US.

In the active arm, GS010 will be administered as a single intravitreal injection to both eyes of each subject. In the placebo arm, GS010 will be administered as a single intravitreal injection to the first affected eye, while the fellow eye will receive a placebo injection.

The primary endpoint for the REFLECT trial is the BCVA reported in LogMAR at 1-Year post-treatment in the secondaffected/notyetaffected eye. The change from baseline in secondaffected/notyetaffected eyes receiving GS010 and placebo will be the primary response of interest. The secondary efficacy endpoints include: BCVA reported in LogMAR at 2-Years post-treatment in the secondaffected/notyetaffected eye compared to both placebo and the firstaffected eye receiving GS010, OCT and contrast sensitivity and quality of life scales. The first subject was treated in March 2018, and enrolment was completed in July 2019, ahead of schedule.

ClinicalTrials.gov Identifiers: REFLECT: NCT03293524

View source version on businesswire.com: https://www.businesswire.com/news/home/20190922005072/en/

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GenSight Biologics Reports Sustained Efficacy and Safety at 96 Weeks in RESCUE Phase III Clinical Trial of GS010 for the Treatment of Leber Hereditary...

Recommendation and review posted by Bethany Smith

The Global Gene Therapy Market 2019-2025 research report describes the complete characteristics of the industry and market statistics. The latest advancement in technology, Gene Therapy market business plans, possibilities for growth and risks to the sector are being included. The reports two key sections are defined, namely market revenue and market size. Scope of Gene Therapy industry, market concentration and showcase across various topographies are included in detail.

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Global Gene Therapy Market with Indepth Analysis, Competitive Players, Future Trends and Forecast to 2025 : Novartis, Kite Pharma, Inc.,...

Recommendation and review posted by Bethany Smith

YORBA LINDA, Calif. (PRWEB) September 26, 2019

LabRoots, the leading scientific social networking website offering premier, interactive virtual events and webinars, will host its 2nd Annual CRISPR Virtual Event, on October 3, 2019. Since the first conference was held in 2018, research has continued to advance rapidly, however many questions remain about the science, applications, and the use of biotechnological techniques for editing genomes.

CRISPR 2019 resumes the global dialogue on these issues by bringing together a broad range of participants including biomedical researchers, academia and industry, and renowned scientists to explore topics such as CRISPR Biology, CRISPR in Gene Editing Applications, and Ethical and Regulatory dimensions and concerns of Gene Editing.

CRISPR has evolved into various technologies for basic research with new applications. Now, in its 2nd year, this premier event focused on the discovery and classification of CRISPR systems, gene therapy, animal model development for medical research, agriculture and food security will feature eleven prominent speakers, and a keynote delivery by Dr. Marie-Christine Birling, Head Associate of the Genetic Engineering and Model Validation Department at the Institut Clinique de la Souris on generating rodent model by CRISPR/Cas9 genome editing.

CRISPR generates a high level of interest, said Dimitri Perrin, Senior Lecturer in Data Science, Queensland University of Technology and member of the organizing committee. Research in this area intersects with a number of disciplines, from the basic biology of CRISPR systems to applications in gene editing (for instance in model organisms, health or agriculture) and reflections on how to ethically use and adequately regulate these technologies. It has been a pleasure to work with LabRoots to bring you an exciting program of excellent researchers across these domains, added Perrin.

Our inaugural conference was a huge success, said Greg Cruikshank, Chief Executive Officer of LabRoots. CRISPR 2019 returns highlighting momentum and recent progress made in gene editing and biotechnology and its potential moving forward. LabRoots continues to be dedicated to developments in life sciences using its unique stage for sharing data and research critical to the scientific community.

LabRoots interactive platform allows attendees to watch, learn and connect seamlessly across all desktop and mobile devices. By joining this online event, viewers can earn one Continuing Education credit per presentation for a maximum of 14 credits.

To register for the event and for more information, click here. Participants can follow the conversation online by using #LRcrispr.

About LabRoots LabRoots is the leading scientific social networking website, and primary source for scientific trending news and premier educational virtual events and webinars and more. Contributing to the advancement of science through content sharing capabilities, LabRoots is a powerful advocate in amplifying global networks and communities. Founded in 2008, LabRoots emphasizes digital innovation in scientific collaboration and learning. Offering more than articles and webcasts that go beyond the mundane and explore the latest discoveries in the world of science, LabRoots users can stay atop their field by gaining continuing education credits from a wide range of topics through their participation in the webinars and virtual events.

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LabRoots Announces Second Annual Conference on CRISPR Technologies - PR Web

Recommendation and review posted by Bethany Smith

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NORFOLK, Va. -Kaeli Prices piercing cry is a joyful noise to her mom and dad.

She'll be screaming right in your face and all we can think is that we're just so thankful that we can hear her now, said Kelli Price, Kaelis mother.

When Kaeli was born in March, you could barely hear her cry and more signs signaled something wasnt right.

We started noticing she wasn't holding her head up like some babies would normally do, said her mother. So we started to worry a little bit.

Their pediatrician in Belvidere, North Carolina began to worry, too. He sent them to see Dr. Crystal Proud, a pediatric neurologist at Childrens Hospital of The Kings Daughters. The doctor specializes in Spinal Muscular Atrophy. Its the top genetic killer of infants.

The babies that were diagnosed with this disorder passed away from losing the ability to breathe because those muscles became so weak, explained Dr. Proud.

The doctor said the source of the disorder is a missing or mutated gene which keeps the babies muscles from developing. Most diagnosed with SMA die by the age of two. Ten babies a year are diagnosed with the deadly condition in Virginia. At just eight weeks old, testing confirmed Kaeli Price was one of them.

We were overwhelmed, said Kaelis mother.

We felt powerless, said Brandon Price, Kaelis father.

Dr. Proud started Kaeli on a drug called Spinraza, which has shown promise in babies with SMA since it was approved by the FDA in 2016. It requires an injection into their tiny bodies every four months for the rest of their lives.

Not long after Kaelis initial treatment on Spinraza, a newly approved gene therapy drug called Zolgensma hit the market. It would essentially give Kaelis body the missing gene, and it would only require one dose. However, the drug came with a price tag of $2.1 million. The Price familys insurance covered it, allowing Kaeli to be the first baby in Virginia to be treated with Zolgensma under FDA approval.

I can't even describe how thankful we are, said Mrs. Price.

I hope and I expect that she will sit, stand, and walk. She will go through school and just excel, said Dr. Proud. And at some point well down the road she will have her own family that she can tell the story to.

Zolgensma has come under scrutiny because of its multi-million dollar price tag.

Dr. Proud, who was part of the clinical trials for the gene therapy drug, said it is actually cost effective when you weigh the billions of dollars it takes for clinical trials and the medical bills a child would incur without the drug.

Dr. Proud said beginning this year, all newborns in Virginia will be tested for SMA. The earlier doctors can intervene, preferably within the first few weeks of life, the better their chances for treatment.

Link:
Gene therapy drug priced at $2 million saves North Carolina babys life - WTKR News 3

Recommendation and review posted by Bethany Smith

Here's What Happened to Dr. Sharpe on 'New Amsterdam' Details!AcceptBrowsers may block some cookies by default. Click accept to allow advertising partners to use cookies and serve more relevant ads. Visit our privacy policy page for more information.Source: Karolina Wojtasik/NBCBy Amber Garrett

1 day ago

The good news for New Amsterdam fans who love Helen is that the oncologist, portrayed by Freema Agyeman, survived her injuries in the horrific ambulance accident that ended Season 1. However, there seems to be a significant obstacle jeopardizing her future at the hospital, in the form of a former rival.

The three-month time jump revealed medical director Max Goodwin has been placed under a new doctor's care, Dr. Valentina Castro. When we saw her occupying Helen's office, we felt a pit in our stomachs.

While showrunner David Schulner says it was always going to be Georgia who would not make it, the fervor of "Sharpwin" shippers almost changed that outcome.

"We have a huge fan base who all they want is to see Max and Sharpe together," he said in an interview with TVLine. He says that's "not in the cards now," and since Georgia's death would seem to open that possibility up, he had reservations.

"We don't want them to be together," he continued. "They're the best of friends, and that's the relationship that we're really excited to explore: two adult friends who love and care for each other."

While it's unlikely they would have killed off Helen instead, a change of plans may have affected Lauren Bloom's fate or lessened the impact of the finale by having none of the three women die.

While Helen's still around, she seems to be back in her role as ambassador for the hospital, promoting the revolutionary gene therapy treatment that seems to have Max on a path to being completely cured of his cancer. While that outcome obviously is fantastic news for everyone, including Helen, it does reveal a troublesome rivalry.

Doctors Castro and Sharpe clearly have a history, as it's revealed Helen once fired Valentina. Now that the latter's promising gene therapy research seems vindicated, she's using it to bargain for essentially co-running Helen's department. While Dr. Sharpe ultimately agrees to that arrangement, I doubt that will clean up all the bad blood between these two.

There also seems to be a clear rift between Max and Helen after the accident, and not just because they've removed the complicated doctor-patient dynamic of their relationship. It's too soon to tell whether Max's coldness is a product of his grief or something larger.

However, it's clear from the showrunner's comments about the importance of Max and Helen's friendship that there's no danger of the latter being written out of the story anytime soon even if the nature of that relationship won't satisfy Sharpwin shippers!

New Amsterdam airs Tuesdays at 10 p.m. ET on NBC.

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Here's What Happened to Dr. Sharpe on 'New Amsterdam' Details! - Distractify

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When Lizzie Acevedos fraternal twin sons, now 15, were newly diagnosed with autism, she was hopeful about diets and vitamin injections that were being touted as miracle treatments or even cures.

Around age 4, the boys were on a gluten-free, casein-free diet, and for a couple months they mainly ate a special organic brand of chicken nuggets. Wheat products or anything else with gluten were out, as were dairy foods containing the milk protein casein. Acevedo also started giving vitamin B12 injections, prescribed by a Defeat Autism Now! doctor, to her son Omar, who is nonverbal and has more significant intellectual disabilities than his brother, Jorge, who speaks and is more independent.

Back then, I just needed something to make things better, Acevedo said. She tried the treatments for a few months but stopped when she didnt see a noticeable effect.

Now, a decade later, Acevedo has heard about lots of hyped alternative approaches for autism, most recently worrisome reports of parents giving their kids bleach drinks or enemas. However, shes also learned there arent any quick remedies for autism, which affects brain development and is characterized by difficulties with communication and social skills and by restricted interests and repetitive behaviors.

Theres no cure for autism and anybody who tries to sell you a cure is lying, Acevedo, a single parent and a fifth grade teacher in Los Angeles, said.

But she understands why parents of autistic children can fall prey to scams. Ive been where they are now, and I know how desperate it feels to want to get your child to be better, she said.

When autism research started to really accelerate a couple decades ago, many scientists thought finding a cure might be easier. Today, the latest science points away from a single cure, but there are ways to help autistic people lead healthier, happier lives and more that can be done to help.

I think that given the complexity and the variability of the causes and the manifestations of autism, trying to come up with a cure is probably not the right approach, said autism researcher and psychologist Len Abbeduto, director of the University of California, Davis, MIND Institute in Sacramento.

An estimated 80 percent of autism cases involve genetic factors, and it tends to run in families, but there is no single autism gene, Abbeduto explained. In fact, research has shown that more than 100 genes, and maybe upwards of 1,000, may play a role. Researchers also suspect that environmental factors such as exposures to infectious agents, pesticides or other toxins in pregnancy may play a role.

Scientists are investing a lot of work into understanding the genes but were also realizing its a lot more complicated than anybody ever thought when they started out, psychologist Ann Wagner, national autism coordinator for the U.S. Department of Health and Human Services, said.

Its highly likely that there are different causes for different kinds of ASD.

We do know that its highly genetic, we just havent identified how particular kinds of genes might interact with each other or with other factors to cause autism spectrum disorder, Wagner said. Autism is such a heterogenous disorder, so its highly likely that there are different causes for different kinds of ASD.

These research developments come amid growing controversy over whether autism even needs a cure. Autism Speaks, an advocacy and research group founded in 2005, removed the word cure from its mission statement in 2016.

In the beginning, [researchers] were looking more for the magic bullet, the magic pill. We were looking for the autism gene, and we thought that would ultimately lead to some kind of cure of autism, psychologist Thomas Frazier, chief science officer at Autism Speaks in New York, said. Then we recognized that we were way off base.

Now, researchers have turned much of their attention to identifying autism in children as early as possible in hopes of intervening sooner with therapies to try to alter the developmental trajectory of their young brains. While skilled practitioners can diagnose autism in toddlers at 18 to 24 months of age with some research indicating there are detectable signs in babies as young as 6 months most kids arent diagnosed until age 4.

Katarzyna Chawarska, a professor of child psychiatry who leads Yale Universitys Autism Center of Excellence in New Haven, Connecticut, is studying signs of autism in babies. The reason why we are focusing so much on early diagnosis is that it is our hope that by intervening early, we can capitalize on still tremendous brain plasticity that is present in the first, second, third year of life, she said.

The goal, Chawarska said, is to help alleviate the symptoms and make sure that every child with autism reaches their full potential.

If youre trying to get rid of autism, youre trying to get rid of us.

Doctors, for instance, would like to minimize any intellectual disabilities and help patients communicate better and improve socials skills. They also want to quickly identify and address any medical conditions that often accompany autism, such as seizures, gastrointestinal problems, sleep disorders, Attention Deficit Hyperactivity Disorder and anxiety.

Researchers already are seeing positive results with interventions such as behavioral treatments and speech therapy in toddlers.

One of the things that we do know is that intensive early intervention improves outcomes for kids, so the earlier we can intervene the better, Abbeduto said.

The idea of curing autism also has become highly controversial with the growth of the neurodiversity movement, which emphasizes respecting and valuing all people for who they are, regardless of whether they are neurotypical.

The C word raises a lot of attention in the community at large, said Michael Maloney, executive director of the Organization for Autism Research, a group in Arlington, Virginia, that funds research to improve the daily lives of autistic people. The largest objection is from people with autism who see themselves as independent and competent and dont see themselves as broken and needing to be fixed.

Among the critics is Julia Bascom, executive director of the Autistic Self Advocacy Network, a Washington, D.C.-based group that is run by autistic people, including herself.

Self-advocates have been largely successful at saying this concept of a cure is really offensive, she said. Who we are is OK, we just need support.

Bascom isnt opposed to research and therapies that help autistic people as long as they arent trying to strip them of their autistic traits.

If youre trying to get rid of autism, youre trying to get rid of us, and thats something our community takes really personally, she said. There are certainly a lot of co-occurring conditions like epilepsy that a lot of us have that wed like to not have. But we dont tend to feel that way about autism and we get really concerned when we see all this money going into risk factors and causation and genetics as opposed to finding out why autistic people tend to have shorter lifespans, or why our suicide rate is nine times higher than average, or what autism really looks like in adults.

Some of her other questions include why girls and people of color are diagnosed later in life, why autism has so many co-occurring conditions, why people with autism tend to react differently to medications, and why they engage in self-injurious behaviors, such as head banging and skin scratching.

Like Acevedos boys, a growing number of teens and adults are living on the autism spectrum, but Bascom and others say there is far too little research on understanding how autistic people are affected across their lifespan and how to help them live life to the fullest. Most autism research dollars in the United States go toward understanding the biological underpinnings of autism in order to diagnose and treat young children.

Autism research spending in the U.S. totaled more than $364.4 million in 2016, the latest year for which figures are available, with 80 percent of that money coming from federal agencies and 20 percent from private organizations. Of the spending, just 2 percent went toward autism lifespan issues and 5 percent toward services, according to the governments Interagency Autism Coordinating Committee. An additional 35 percent went to biology, 24 percent to risk factors, 16 percent to treatment and interventions, 10 percent to infrastructure and surveillance, and 8 percent to screening and diagnosis.

Paul Shattuck, director of the Life Course Outcomes Research Program at the A.J. Drexel Autism Institute in Philadelphia, and a member of the scientific council of the Organization for Autism Research, agrees that not enough attention is paid to adults with autism.

Were expending a lot of effort for very young children with autism, but as a society we kind of drop the ball once these young people become young adults, he said. Theres really not much there for autistic adults or their families in terms of services or even thinking how to support autistic people across the lifespan.

There arent exact figures on the total number of Americans with autism but by one estimation 3.5 million people are on the spectrum, and diagnoses have been increasing. About 1 in 59 children are on the autism spectrum, according to the latest Centers for Disease Control and Prevention figures from 2014, up from 1 in 150 kids in 2000. While some of the increased prevalence may be a true increase in autism cases, the CDC says that a broader definition of the autism spectrum and improved diagnosis efforts likely contributed to the higher numbers.

By Shattucks latest research estimates, 70,000 to 80,000 or more autistic youths per year will turn 18. Thats close to a million people over the next decade, he said, highlighting an urgent need for research to address the health and well-being of autistic adults.

Autistic kids are eligible for special education services while they are in school, and services can last until age 21, but help is harder to come by after that. When teens exit high school, they fall off what is called the services cliff, Shattuck said. It becomes much more difficult to find help and services once kids age out of eligibility for special education. And the young adult outcomes and the adult outcomes are pretty dismal frankly.

After high school, most young autistic adults do not have jobs, career training or additional educational opportunities. Autistic adults also struggle to find independent living arrangements, maintain friendships, get involved with community activities or have enough money to pay for their needs, said Shattuck, whose center helps autistic people and their families with paperwork for Medicaid, Social Security, group housing and more. Many autistic adults continue to live with their parents, raising concerns about what happens when the parents pass away.

Wagner, the national autism coordinator, agrees there needs to be more research on autism across the lifespan and said the government is trying to attract and fund more research in this area.

Just like parents everywhere, Acevedo wants the best for her kids. But after Omar and Jorge finish high school and special education services end, she wonders and worries about what the future holds.

I would love to see some more money put into the transition of young adults with autism into the most independent living situation they can get, Acevedo said. I would love to see money put into job training, taking the skills that these children have because everybody has skills, something that they can do and just really refining it and making these kids marketable to where they can earn some sort of income. Theres something about getting a paycheck and having your name on it as an adult that means so much, and Im sure its going to mean a lot to my kids.

Shattuck says helping autistic adults or those with disabilities ultimately helps everyone.

Our organizations and our communities function better when we make space for everyone of all abilities, he said. Its about helping ourselves and helping our communities be better, higher quality places for all of us.

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Why the focus of autism research is shifting away from searching for a 'cure' - NBCNews.com

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Cancer is a devastating illness. Mostly, because of how rapidly it spreads to other areas in the body. Because of this danger, researchers are developing treatments that have the ability to specifically target abnormal cells and then slow or stop those cells from growing and spreading. This method is known as targeted therapy. As scientists discover more about gene and protein changes, their focus is to develop new drugs that hone in and target those changes.

Targeted therapies offer an alternative treatment option to chemotherapy and radiationwith less severe side effects. The reason why there are fewer side effects is because targeted treatment specifically targets abnormal cells, versus chemotherapy and radiation, which cant distinguish between abnormal and healthy cells.

Malignant mesothelioma is an aggressive, fatal disease that has seen rising cases over the past 60 years. A person with mesothelioma cancer is generally experiencing tumor growth in the lining (mesothelium) of the lungs, abdomen, or heart (pleura, peritoneum, or pericardium). Within the past few years, researchers have been testing the use of targeted therapies on mesothelioma patients in clinical trials.

There are two primary types of targeted therapies: monoclonal antibodies and molecule medicines. Molecule medicines are small enough to dive into cancer cells and obliterate them, and monoclonal antibodies fight cancer cells on the surface, or surrounding areas, and are too big to slip into cancer cells.

Sometimes, oncologists use monoclonal antibodies to launch chemotherapy and radiation treatments straight into tumors. This can occur through an IV in a vein, or as a shot. Monoclonal antibody medication treatments usually end with the stem -mab. Three monoclonal antibody types are:

One of the most common targeted therapies, signal transduction inhibitors block signals that tell cancer cells to divide too much and too fast.

This form of treatment blocks the growth of blood vessels that cancer cells form in order to retrieve nutrients and oxygen. In the case of mesothelioma and some other cancers, the target is a substance called vascular endothelial growth factor (VEGF) and utilizes the drug bevacizumab. Its usually administered in conjunction with chemotherapy medications pemetrexed and cisplatin.

This treatment utilizes the patients immune system to locate and destroy cancer cells. An oncologist can administer immunotherapies that boost the immune system to better attack cancer, or immunotherapies that highlight tumor cells so theyre quicker and easier for the immune system to find and attack.

This treatment is helpful in that it can penetrate cell membranes and interact with targets from the inside of a cell. Molecule medicines are designed to interrupt the enzymatic activity of a specified protein and generally end with the stem -ib. Three molecule medicine variations include:

Another form of targeted therapy, gene expression modulators modify the proteins that control the abnormal instruction or expression of genes in cancer cells.

When cells die after growing old or becoming damaged, this is called apoptosis. Cancer cells tend to avoid this natural process, and apoptosis inducers cause abnormal cells to go through normal cell death.

As with most cancer treatments, targeted treatments come with side effects. A patients experience with these side-effects varies, as each individuals case is unique. More common side effects include:

Rarer, but possible side effects of targeted treatments can include:

Your doctor may have some medications available to help relieve side effects, and its recommended you call or stop by their office if youre experiencing them.

A doctor will need to test your tumors to find targets the treatments can focus on. They may use a biopsy to take these tests, which consists of sampling the tumor and then checking it in a lab. Two patients with the same type of mesothelioma may not have the same targets, and some medications may be ineffective if you do not have certain gene mutations. An oncologist may have to administer other mesothelioma medications or treatments before targeted therapy can be applied.

You dont have to go through this alone. Your oncologist will have resources available if you have any questions or issues during any of your mesothelioma treatment. If targeted therapy sounds like an option for you, definitely run that by your cancer-care team, so they can fully assess whether thats the best option based on your health, mesothelioma stage, and other factors.

A mesothelioma diagnosis is a trying time for patients. Download our free guide to learn more about the disease.

Jennifer Verta thrives as a digital content writer and SEO specialist at Mesothelioma Hub. She has been producing content for clients since before she graduated from the University of Colorado at Denver with a Bachelor of Arts in Communication and a Minor in English Writing. Jens mission is to help promote awareness of mesothelioma to as many people as possible by providing only the most up-to-date and accurate content available. When she isnt cranking the gears at work, Jen can be found snowboarding, hiking, catching live music or socializing with friends.

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Targeted Therapy | Treating Mesothelioma - Mesothelioma Hub

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Heart cells derived from patients stem cells and grown in a lab dish can reveal important clues about the development of heart ailments associated with Fabry disease.

The study, A Human Stem Cell Model of Fabry Disease Implicates LIMP-2 Accumulation in Cardiomyocyte Pathology, was published in Stem Cell Reports.

Fabry is a rare genetic disorder caused by mutations in the GLA gene. Located on the X chromosome, the gene provides instructions for the production of an enzyme called alpha-galactosidase A (alpha-GAL A).

These mutations typically affect the activity of alpha-GAL A, leading to the accumulation of a type of fat called globotriaosylceramide (GL-3) in different tissues and organs, including the heart, kidneys and nervous system, gradually compromising their normal function.

For this reason, most Fabry patients develop heart disease over the course of their lives, which may progress to heart failure, the most common cause of death among people living with the disorder.

A major obstacle for advancing therapy for patients with [Fabry disease] is the knowledge gap between the direct molecular consequences of alpha-GAL A deficiency in CMs [cardiomyocytes, or heart cells] and the cascade of events driving disease in the heart; the inaccessibility of CMs from patients precludes adequate investigation of these events, especially at early stages, the investigators wrote.

In a previous study, researchers describe the generation of induced pluripotent stem cells (iPSCs) from Fabry patients carrying nonsense mutations in the GLA gene. This gave them the possibility, for the first time, to study the impact of alpha-GAL A deficiency on heart cells derived from patients iPSCs grown in a lab dish.

(iPSCs are fully matured cells that are reprogrammed back to a stem cell state, where they are able to grow into any type of cell. A nonsense mutation is a mutation in which the alteration of a single nucleotide (the building blocks of DNA) makes proteins shorter.)

Investigators from Sanofi, in collaboration with researchers at the University of Manchester, further investigated the properties of heart cells derived from patients iPSCs. Their aim was to discover more clues about the molecular mechanisms involved in the development of heart disease linked to Fabry.

Functional and structural characterization experiments revealed that heart cells from Fabry patients had higher levels of GL-3, and showed a series of abnormalities in the way they responded to electrical stimuli and in how they regulated their calcium usage, compared to heart cells from healthy people serving as controls. Calcium is essential to coordinate the hearts function by contributing to the electrical signals involved in heart muscle contraction.

When researchers analyzed the protein contents of heart cells grown in a lab dish, they found these cells produced more than 5,500 different proteins. This analysis also showed that compared to controls, heart cells from Fabry patients produced large amounts of lysosomal membrane protein 2 (LIMP-2) and heat shock-related 70 kDa protein 2 (HSPA2/HSP70-2).

(LIMP-2 is a protein normally found on the membrane of lysosomes small structures within cells that accumulate, digest, and recycle materials that regulates their transport within cells; HSPA2/HSP70-2 is a protein involved in cellular quality control, participating in the folding of other proteins and targeting abnormal proteins for degradation.)

Heart cells from Fabry patients released high amounts of cathepsin F, a protein that helps breakdown materials being transported inside lysosomes, as well as HSPA2/HSP70-2. As expected, when researchers corrected the genetic mutation associated with Fabry in heart cells derived from patients iPSCs, all these defects were reversed.

To confirm the validity of these proteins as Fabry biomarkers, researchers then forced healthy heart cells to produce high amounts of LIMP-2. They discovered this also triggered the release of large amounts of cathepsin F and HSPA2/HSP70-2, resulting in a massive accumulation of vacuoles (enclosed compartments filled with water and other substances) inside cells.

In summary, our study has shown the power of the iPSC model to reveal early functional changes and the development of a distinctive biomarker expression profile in [Fabry disease] CMs. These biomarkers may be of utility in drug screening and in elucidating the earliest pathological events and cascades in [Fabry disease] cells. Quantification in patient plasma and urine samples will be an important next step toward validating their relevance in patients, the researchers wrote.

A better understanding of these mechanisms will no doubt accelerate the development of more effective and increasingly personalized therapies for patients, they added.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

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Fabry Heart Cells Grown in Lab Dish Give Hints to Cardiac Complications - Fabry Disease News

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Artificial intelligence has been embraced for its ability to offer insight from big data. By applying the technology to genetics, a research team led by Queen Mary University of London has found clues that they say could aid the development of new drugs for heart failure and identify people at risk of the disease.

Based on an AI analysis of heart MRI images from 17,000 volunteers in UK Biobank, the researchers linked genetic factors to 22% to 39% of abnormalities in the size and function of the hearts left ventricle, which pumps blood into the aorta. They published the findings in the journal Circulation.

The team identified or confirmed 14 regions in the human genome that play a part in determining the size and function of the left ventricle, becausethey contain genes that regulate the early development of heart chambers and the contraction of heart muscle. Enlargement of left ventricle is a condition that can hamper the heart muscles ability to contract and pump blood, putting the patient at high risk of heart attack.

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This study has shown that several genes known to be important in heart failure also appear to regulate the heart size and function in healthy people, said study co-author Steffen Petersen of Queen Mary in a statement. That understanding of the genetic basis of heart structure and function in the general population improves our knowledge of how heart failure evolves.

RELATED:Bayer teams up with AI firm Sensyne Health to mine NHS data for its heart disease pipeline

There is a growing interest in using AI to gain insights into cardiovascular disease. Bayer recently partnered up with Sensyne Health, which uses AI to mine patient data from the U.K. National Health Service, including genomic sequencing data and real-world evidence, to help design clinical studies and accelerate drug discovery.

Many research teams having been looking at different ways to treat heart disease, including using immune therapies and regenerative approaches. Scientists at the University of Pennsylvania, for example,developed genetically modified T cells to attack and remove cardiac fibroblasts, which can lead to cardiac fibrosis. Vanderbilt University researchers identified Roches SYN0012, originally designed to treat rheumatoid arthritis, as a promising candidate that could dampen inflammation of heart tissue after a heart attack. Such inflammation can progress to acute episodes andchronic heart failure.

To help repair damaged cardiac tissue after a heart attack, scientists at the University of Cambridge in the United Kingdom and the University of Washington combined two types of cells derived from human stem cellsheart muscle cells and supportive epicardial cells that help the muscle cells live longer. A team at the the Morgridge Institute for Research previously added a drug called RepSox to stem cells to build better smooth muscle cells that can grow into functional arterial cells.

The Queen Mary researchers believe the 14 regions of the genome they fingered in their new study could be just the beginning of a larger story about genes and heart disease. Our academic and commercial partners are further developing these AI algorithms to analyze other aspects of cardiac structure and function,lead researcher Nay Aung said in the statement.

Aung and colleagues argue the genetic markers theyve already uncovered could help identify those at high risk of developing heart disease or open up new avenues for targeted treatments. The genetic risk scores established from this study could be tested in future studies to create an integrated and personalized risk assessment tool for heart failure, Aung said.

Link:
AI uncovers genes linked to heart failure - FierceBiotech

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All living organisms function under specific conditions, the so-called laws of nature. But with the expansion of scientific knowledge in the past centuries, humans havent always just been helplessly swayed by the powers of nature.

While far from controlling nature, we have invented substances and came up with strategies that allow us to stretch the boundaries of science, from altering cell processes to becoming fully immune to diseases.

One of the most promising innovations of our times nanotechnology is taking it even further. Sometimes nicknamed the next industrial revolution, it pervades virtually every field from manufacturing to the food and drink industry.

By operating on the atomic and molecular scale, nanotechnology carries out the most precise interventions. What new possibilities does it bring to industries dealing with biology?

In 2018, global healthcare nanotechnology reached $160 billion and this number is likely to grow to over $300 billion by the end of 2025. Theres no wonder why healthcare is the true harvest field of nanotechnology with diverse opportunities, mostly in diagnostics, treatment, and prevention of diseases itself.

Experts have developed nanosized diagnostic devices that can be deployed throughout the human body to monitor levels of toxins or other substances. This allows for constant and real-time monitoring of an individual on a very detailed level something that was hardly imaginable just a few years ago.

Read More:Challenges in achieving precision medicine, personalized healthcare

And whats more! due to their size, these tiny sensors can enter spaces that are normally difficult to examine, including the brain.

Regarding treatment, there have been experiments with nanosized robots that can travel through bodily fluids. They can work to deliver active substances in a highly effective way.

Likewise, they could bring implants that destroy old cells and inject healing substances to promote the growth of new ones or the recovery of existing ones.

In this respect. In fact, researchers at North Carolina State University are developing a method to deliver cardiac stem cells to damaged heart tissue.

This highly-targeted approach also means a revolution in cancer treatment as we know it: nanotechnology could eliminate the adversary side effects of the conventional methods that affect the whole system.

Read More:Doctors will navigate this passive pill cam like they were playing Xbox

And with closer oversight and enhanced possibilities for direct intervention, cancerous cells can be destroyed even before a major breakout occurs.

Scientists at the Worcester Polytechnic Institute are working on such non-invasive preventive strategies. They have developed a chip made of carbon nanotubes that can capture circulating tumor cells of all sizes.

These can be analyzed easily to help identify any early-stage tumors and monitor treatment progress.

Nanotechnology has also seen a big boom in the cosmetics industry. In recent years, we have seen a rise in the usage of various nano-substances, including peptides, proteomics, stem cells, and epigenetics. These could directly intervene against the sources of any dermatological phenomenon, be it wrinkles, pigment spots, or acne.

The potential is immense, which is demonstrated by the industrys rising investment and the fact that major cosmetic producers, including LOral, P&G, Dior, and Johnson & Johnson, publish several nanotechnology-related patents every year.

LOral specifically designated a web page to nanoparticles to educate their consumers about the power of these substances in many aspects; from intensifying the shade of mascara to providing a matte finish effect on the skin.

The uses are truly diverse. For example, we can find the adoption of nanoemulsions that encapsulate active ingredients to be carried deeper into hair shafts, or nanosilvers and nanogolds that are known for their antibacterial effects and are used in deodorant or toothpaste.

Nanotechnology is practically used in all everyday products, including moisturizers, haircare, or sunscreens.

In fact, the usage of nanoparticles in sunscreens has perhaps earned the most attention. These SPF creams contain zinc oxide and titanium dioxide as their main compounds. Such products can reflect UV rays, in contrast to the traditional chemical sunscreens that absorb the rays.

Thats why nano-powered sunscreens appear transparent, instead of leaving a white layer on the skin. Yet, this method has been associated with safety concerns, arising from the risk of the particles penetrating tissue and entering the human organism.

While there is still research to be done, an Australian study from last year disproved this notion and asserted that nano-powered sunscreens are unlikely to be harmful.

But its not just healthcare and beauty. Nanotechnology also brings opportunities to conservation and preservation. By being able to disrupt biological processes at the most detailed level, scientists are working to delay wilting and enhance desired processes, such as fostering an environment unfriendly to bacteria.

Specifically, there has been a lot of progress done in the field of food storage and preservation. For example, the encapsulation of nutraceuticals through nanotechnology is a step towards greater food safety and bioavailability, allowing us to benefit from food to its full nutritional potential.

However, similar applications could boost a plethora of other industries, including design and art, education, and science. Laboratories could find easier ways to preserve biological samples, while impressive natural artworks could be exhibited in museums for decades, bringing awe to many generations.

One of the innovations already being put to practice is the NanoFreeze technology, which can directly battle the sources of flower decay. This preservation strategy relies on a uniquely set up freezing process that can halt decomposition.

It kills present microorganisms, stops enzymatic reactions, and establishes a protected environment that prevents the occurrence of parasites. This way, NanoFreeze technology succeeds in maintaining the bloom looking fresh even years after it was cut off, bringing unprecedented possibilities to the floriculture industry and beyond.

From live-saving innovations to more mundane consumer upgrades, nanotechnology presents many opportunities for the future.

While predicted to grow significantly in the upcoming years, its crucial to understand that the innovation still hasnt reached its peak and is yet to experience its full bloom.

Disclosure: This article is brought to you by a client of an Espacio portfolio company

Originally posted here:
How nanotechnology is sizing up healthcare, beauty and conservation - The Sociable

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ORLANDO, Fla.--(BUSINESS WIRE)--

- Grant is part of the Helping to End Addiction Long-Term Initiative (NIH HEAL Initiative) launched in 2018 -

Hesperos, Inc., (www.hesperosinc.com) announced today the University of Central Florida (UCF) and Hesperos have received the first phase of a $3.8 million milestone-based National Institutes of Health (NIH) grant for research involving the companys human-on-a-chip system. The goal of the research is to better understand how overdosing on opiates works, their impact on multiple organs and the effect of drugs used to treat those overdose episodes including their potential toxicity to organs. James Hickman, Ph.D., Chief Scientist at Hesperos and Professor at UCF, is the principal investigator for the research.

Hesperos, Inc., (www.hesperosinc.com) announced today the University of Central Florida (UCF) received the first phase of a $3.8 million milestone-based National Institutes of Health (NIH) grant for research involving the companys human-on-a-chip system.

"We are grateful to have funding to support research in an area that represents such a large and growing need," said Dr. Hickman. "Our interconnected human-on-a-chip system provides a non-invasive way to emulate the response of compounds among all 'organ' compartments, and to concurrently predict potential toxicity and efficacy of drugs, including opioids and opioid antagonists such as Narcan."

The funding comes from the NIHs Helping to End Addiction Long-term Initiative, or the NIH HEAL Initiative. The initiative aims to improve treatments for chronic pain, curb the rates of opioid use disorder and overdose and achieve long-term recovery from opioid addiction. The National Institutes of Health launched the Helping to End Addiction Long-term Initiative, or NIH HEAL Initiative, in April 2018 to improve prevention and treatment strategies for opioid misuse and addiction and enhance pain management. More information about the grants awarded by the NIH HEAL Initiative can be found here.

Its clear that a multi-pronged scientific approach is needed to reduce the risks of opioids, accelerate development of effective non-opioid therapies for pain and provide more flexible and effective options for treating addiction to opioids, said NIH Director Francis S. Collins, M.D., Ph.D., who launched the initiative in early 2018. This unprecedented investment in the NIH HEAL Initiative demonstrates the commitment to reversing this devastating crisis.

Under this program, Hesperos will build overdose models in a multi-organ system and evaluate the acute and chronic effects of overdose treatments, such as Narcan, on overdose recovery and efficacy. The research will provide insight into the impact of both opiate overdoses and treatment drugs on the kidneys, heart, muscles and liver, as well as explore how these drugs impact the part of the brain that controls breathing to reproduce overdose conditions.

Hesperos seeks to radically change established practice in drug discovery and testing by bypassing animal experiments and extensive clinical trials to provide treatments for diseases and clinical conditions such as overdose. Dr. Hickman developed the human-on-a-chip system at UCF in collaboration with Michael Shuler, President and CEO at Hesperos and Professor Emeritus, Cornell University. UCF licensed the technology to Hesperos, which was co-founded by Dr. Hickman and Dr. Shuler.

Over the past few years we have formed multiple collaborations with companies and nonprofit organizations seeking more efficient and effective alternatives to preclinical evaluation of drugs or toxicity tests on chemicals without lengthy, expensive animal studies, Dr. Shuler said. We recently published results in Nature Scientific Reports and Science Translational Medicine supporting the ability of our system to truly revolutionize the drug discovery process.

About Hesperos:

Hesperos, Inc. is a leader in efforts to characterize an individuals biology with human-on-a-chip microfluidic systems. Founders Michael L. Shuler and James J. Hickman have been at the forefront of every major scientific discovery in this realm, from individual organ-on-a-chip constructs to fully functional, interconnected multi-organ systems. With a mission to revolutionize toxicology testing as well as efficacy evaluation for drug discovery, the company has created pumpless platforms with serum-free cellular mediums that allow multi-organ system communication and integrated computational PKPD modeling of live physiological responses utilizing functional readouts from neurons, cardiac, muscle, barrier tissues and neuromuscular junctions as well as responses from liver, pancreas and barrier tissues. Created from human stem cells, the fully human systems are the first in vitro solutions that accurately utilize in vitro systems to predict in vivo functions without the use of animal models. More information is available at http://www.hesperosinc.com

View source version on businesswire.com: https://www.businesswire.com/news/home/20190926005748/en/

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Hesperos Human-on-a-Chip Technology Awarded First Phase of $3.8 Million Milestone-based NIH Grant to Study Opiate Overdoses in Collaboration With UCF...

Recommendation and review posted by Bethany Smith

CAIRO 22 September 2019: Renowned Professor of Cardiothoracic surgery Magdi Yacoub said that the foundation stone of a heart center in Cairo will be laid soon. The center will provide cardiac care.

In an interview with Egypt Today, the Egyptian-British cardiothoracic surgeonsaid that an inauguration ceremony of the Cairo center will beheldin January 2020, and will beattended by a large number of parliamentarians, senior doctors and statesmen to support the center and urge Egyptians to donate.

The MagdiYacoub Global Heart Foundation launched a campaign in May to raise fund for the new center.

A set of remarkable scientists and public figures took part in the campaign such as Professor MagdyIshak, and Egyptian Ambassador to the United States Yasser Reda, among others.

The MagdiYacoub Global Heart Foundation supports Aswan heart centre in Upper Egypt and is raising funds for the future MagdiYacoubglobal heart centre in Cairo.

Besides providing urgently needed cardiac care, the centers impact the region and continent by advancing scientific understanding through research and building human health capacities with training programs.

The new center will cost an estimate of $150 million and will include 300 beds, hence expected to upgrade network care capacity from 33,000 to 140,000 outpatients and from 4,000 to 17,000 inpatients annually.

Moreover, the training capacity will grow from 550 to over 2300, dramatically increasing the sectors workforce.

Yacoub was among the first three surgeons to perform an open heart surgery in Nigeria in 1974. In 1986, he was part of the team that developed the techniques of the heart-lung transplantation at the National Heart and Lung Institute.

He also led a British research team at Harefield hospital in 2007, aiming to grow a part of the human heart using stem cells. These efforts were all exerted in order to overcome the shortage of heart transplant donations.

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Cairo heart center to be inaugurated January: Magdi Yacoub - Egypttoday

Recommendation and review posted by Bethany Smith

A new research study from HTF MI with titleGlobal Autologous Stem Cell Based Therapies Market Size, Status and Forecast 2019-2025provides an in-depth assessment of the Autologous Stem Cell Based Therapies including key market trends, upcoming technologies, industry drivers, challenges, regulatory policies, key players company profiles and strategies. The research study provides forecasts forAutologous Stem Cell Based Therapiesinvestments till 2025.

If you are involved in the Autologous Stem Cell Based Therapies industry or intend to be, then this study will provide you comprehensive outlook. Its vital you keep your market knowledge up to date segmented by Neurodegenerative Disorders, Autoimmune Diseases & Cardiovascular Diseases, , Embryonic Stem Cell, Resident Cardiac Stem Cells & Umbilical Cord Blood Stem Cells and major players. If you have a different set of players/manufacturers according to geography or needs regional or country segmented reports we can provide customization according to your requirement.

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This study primarily helps understand which market segments or Region or Country they should focus in coming years to channelize their efforts and investments to maximize growth and profitability. These stakeholders include Autologous Stem Cell Based Therapies manufacturers such asRegeneus, Mesoblast, Pluristem Therapeutics Inc, U.S. STEM CELL, INC., Brainstorm Cell Therapeutics, Tigenix & Med cell Europe, etc.Primary sources are mainly industry experts from core and related industries, and manufacturers related to all segments of the industry supply chain. The bottom-up approach is being utilized to project themarket size of the Autologous Stem Cell Based Therapiesbased on end-user industry and region, in terms of value. With the help of data triangulation procedure and validation of data through primary interviews and expert calls helps cover primary market, along with individual market share & sizes are determined and confirmed with this study.

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Global Autologous Stem Cell Based Therapies (Thousands Units) and Revenue (Million USD) Market Split by Product Type such as , Embryonic Stem Cell, Resident Cardiac Stem Cells & Umbilical Cord Blood Stem Cells

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Global Autologous Stem Cell Based Therapies Sales (Thousands Units) by Application (2017-2022)

(2017-2022)

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What will be the market size and the growth rate in 2025?What are the key factors driving the Global Autologous Stem Cell Based Therapies market?Who are the key market players and what are their strategies in the Global Autologous Stem Cell Based Therapies market?What are the key market trends impacting the growth of the Global Autologous Stem Cell Based Therapies market?What trends, challenges and barriers are influencing its growth?What are the market opportunities and threats faced by the vendors in the Global Autologous Stem Cell Based Therapies market?What are the key outcomes of the five forces analysis of the Autologous Stem Cell Based Therapies market?

Geographically, this report is segmented into several key Regions such as United States, Europe, China, Japan, Southeast Asia, India & Central & South America, with production, consumption, revenue (million USD), and market share and growth rate of Global Autologous Stem Cell Based Therapies in these regions, from 2014 to 2025 (forecast), covering

The report provides a basic overview of the Autologous Stem Cell Based Therapies industry including definitions, classifications, applications and industry chain structure. And development policies and plans are discussed as well as manufacturing processes and capital expenditures.Further it focuses on global major leading industry players with information such as company profiles, product picture and specifications, sales, market share and contact information. Whats more, the Autologous Stem Cell Based Therapies industry development trends and marketing channels are analyzed.The study is organized with the help of primary and secondary data collection including valuable information from key vendors and participants in the industry. It includes historical data from 2012 to 2017 and projected forecasts till 2022 which makes the research study a valuable resource for industry executives, marketing, sales and product managers, consultants, analysts, and other people looking for key industry related data in readily accessible documents with easy to analyze visuals, graphs and tables. The report answers future development trend of Autologous Stem Cell Based Therapies on the basis of stating current situation of the industry in 2017 to assist manufacturers and investment organization to better analyze the development course of Autologous Stem Cell Based Therapies Market.

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About Author:HTF Market Report is a wholly owned brand of HTF market Intelligence Consulting Private Limited. HTF Market Report global research and market intelligence consulting organization is uniquely positioned to not only identify growth opportunities but to also empower and inspire you to create visionary growth strategies for futures, enabled by our extraordinary depth and breadth of thought leadership, research, tools, events and experience that assist you for making goals into a reality. Our understanding of the interplay between industry convergence, Mega Trends, technologies and market trends provides our clients with new business models and expansion opportunities. We are focused on identifying the Accurate Forecast in every industry we cover so our clients can reap the benefits of being early market entrants and can accomplish their Goals & Objectives.

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Autologous Stem Cell Based Therapies Market Report with Depth Analysis 2019 | Regeneus, Mesoblast - Tech Platform

Recommendation and review posted by Bethany Smith

DURHAM, N.C., Sept. 25, 2019 (GLOBE NEWSWIRE) -- Dance Biopharm, a clinical-stage company reimagining the treatment of chronic diseases with inhaled therapies, today announced it is rebranding as Aerami Therapeutics and moving its headquarters to Durham, North Carolina. The new brand, which will be implemented immediately, comes as the company pursues strategic partners for its lead asset, Dance 501 inhaled human insulin, to progress into pivotal registration studies, and expands its pipeline to include inhaled therapeutics for endocrinology diseases designed to provide patients an alternative to routine injectable treatments.

The new brand better reflects the Companys broadened focus beyond diabetes to include additional chronic conditions that could potentially achieve better treatment efficiency from the patient friendly administration afforded by Aeramis gentle mist, smart inhaler. Aerami plans to advance new pipeline programs in hypoparathyroidism and human growth hormone (HGH) deficiency in addition to its existing inhalable GLP-1 receptor agonist candidate. Each of these programs could enter clinical development in 2020, subject to our ability to obtain additional funding.

We are proud to launch Aerami, which is derived from a combination of Greek and Latin words. Aero means air/wind and ami means friend/friendly. The new brand reflects our commitment to provide patients living with chronic diseases, a patient-friendly alternative to routine injections, with our gentle mist inhaler, said Anne Whitaker, chief executive officer of Aerami Therapeutics. Our initial focus going forward is on rare and severe diseases in the endocrinology, respiratory and cardiovascular space. We have launched new pipeline programs in hypoparathyroidism and human growth hormone deficiency, which could benefit from the frequent pulsatile administration of inhaled parathyroid hormone and HGH, respectively, afforded by our smart inhaler. In addition, we are targeting advancing our inhalable pre-meal GLP-1 receptor agonist into the clinic in 2020, subject to additional funding, for treatment of type 2 diabetes patients who are not meeting their HbA1c goals with their current oral anti-diabetic treatments.

About Aerami Therapeutics

Aerami Therapeutics is a clinical-stage company reimagining the treatment of chronic diseases with inhaled therapies. The Companys gentle mist inhaler is designed with smart technology to optimize the precise delivery of biologic therapies through the lungs. In addition to its Phase 3-ready inhaled insulin product Dance-501, Aerami is building a diverse pipeline of inhaled therapies with an initial focus on rare and severe diseases in the endocrinology, respiratory and cardiovascular space, where its platform and expertise present a unique opportunity to achieve better treatment efficiency and patient experience. Aerami has initiated development programs for inhaled glucagon-like peptide-1 (GLP-1) for the treatment of type 2 diabetes, inhaled parathyroid hormone (PTH) for the treatment of hypoparathyroidism and inhaled human growth hormone (HGH) for the treatment of HGH deficiency.

For more information, visit: https://www.aerami.com/.

Investor Contact:Jeremy FefferLifeSci Advisors, LLC(212) 915-2568jeremy@lifesciadvisors.com

Media Contact:Gloria GasaaturaLifeSci Public Relations(646) 627-8387ggasaatura@lifescipublicrelations.com

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Dance Biopharm Announces Rebranding to Aerami Therapeutics Reflecting a Broadened Strategic Focus on Inhaled Therapies for Chronic Diseases - Yahoo...

Recommendation and review posted by Bethany Smith

Fountain Hills Internal Medicine and Pediatrics has expanded to include two naturopathic physicians and the services of a cardiologist/vascular specialist.

Joining medical director Dr. Kaveh Karandish are licensed naturopathic physicians Dr. Tess Price and Dr. Chad Patterson.

Dr. Moshen Sharifi, director of Arizona Cardiovascular Consultants & Vein Clinic, also is a staff member.

An open house is scheduled from 6 to 9 p.m. on Thursday, Oct. 10, to meet the new health care providers. Light refreshments will be served.

Since Karandish acquired ownership of the practice last January, he has been assessing the communitys medical needs. He said his office combines preventative and integrative medicine.

He created a six-day-a-week urgent walk-in clinic and established home visits within a five-mile radius of town for patients who are physically unable to travel to his office at 13620 N. Saguaro Blvd., suite 100.

Cosmetic procedures, including services and skin products, are provided. Laser therapy hair removal is a service.

Bio-Identical hormone pellets designed to optimize hormones that a body naturally produces are a new procedure for men and women. Karandish said they have the ability to improve the quality of life for some patients.

A weight loss program to meet individual needs is provided. Under the category of regenerative medicine, the practice can customize platelet-rich plasma and stem cell treatments. Acupuncture is another new service.

Dr. Patterson specializes in pediatric patients between birth and 18 years of age. His primary method of treatment includes nutritional supplements and herbal remedies.

He was born in Denmark and grew up in Carlisle, Penn. A Scottsdale resident, he completed his undergraduate degree majoring in biology from Pennsylvania State University. Post-baccalaureate education took place at Lake Erie College of Osteopathic Medicine. He earned his doctorate in naturopathic medicine from Southwest College of Naturopathic Medicine in Tempe.

Dr. Prices bachelor degree in veterinary science prepared her to enter the work force. She taught in the public and private education sector and counseled college students before returning to school. She also received her doctoral degree in medicine at Southwest College of Naturopathic Medicine.

She has been involved in domestic violence shelters as well as an in-patient treatment facility for addictions, pain and mental health.

She also specializes in gastrointestinal disorders, thyroid dysfunction, mental health issues and preventative medicine.

Dr. Sharifi is an adjunct professor at A.T. Still University in Mesa and is board certified in interventional cardiology, cardiovascular diseases, vascular medicine, internal medicine and nuclear cardiology.

The practices telephone number is 480-837-6800.

Originally posted here:
Medical practice adds providers, services | Business - FH Times

Recommendation and review posted by Bethany Smith