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Archive for the ‘Gene therapy’ Category

The Rise Of Patent Wars In Europe’s Gene Therapy Space – Law360

Wednesday, December 11th, 2019

Law360 (December 11, 2019, 1:28 PM EST) -- The gene therapy industry is in an exciting phase of growth, undergoing significant mergers and acquisitions activity, product sales and new marketing authorizations that are being issued with increasing regularity globally.

Recent reports have estimated that the market is likely to be almost four times its current value by 2025[1], with up to 20 new product approvals expected every year[2].

This rapid growth brings inevitable challenges. Significant issues relating to regulatory standards in manufacturing plants, establishing acceptable reimbursement policies and antitrust investigations are among a few.

The intellectual property landscape has been lower profile, with the exception of the ongoing CRISPR...

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Would you pay $1M to partake in an anti-aging gene therapy trial? – Fox Business

Wednesday, December 11th, 2019

Fox News senior judicial analyst Judge Andrew Napolitano on the potential fallout from reports DNA-testing company Family TreeDNA will share data with the FBI in an effort to solve crimes.

Libella Gene Therapeutics is charging volunteers $1 million to undergo clinical trials of a treatment it is working on that is designed to prevent, delay or even reverse aging.

However, participants will be required to go to a small clinic in Cartagena, Colombia, to participate, which the Kansas-based company said was the easiest site among eight different countries it looked into, calling it the path of least resistance.

In a press release, a company executive said traditional clinical trials in the U.S. take years and millions or even billions of dollars.

The treatment would be delivered intravascularly and participants will be monitored over the course of a year, according to the company's website. Gene therapy treatments are intended to be one-off treatments, attacking the problem at its source.

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The technology focuses on lengthening telomeres, which are structures found at the end of chromosomes. Their main function is to protect DNA during cell division.

Every time a cell divides, a part of the telomere is lost until it becomes too short and the cell dies. Some believe that as cells age, so does the body.

Telomerase is an enzyme that lengthens telomeres and thus prevents the cell from dying.

Libellas technology rebuilds the ends of telomeres, andthereby affects the aging process.

I know what were trying to do sounds like science fiction, but I believe its a science reality, Jeff Mathis, CEO of Libella Gene Therapeutics, said in an interview with OneZero.

The treatment may potentially treat other diseases, like cancer and Alzheimers.

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Not everyone, however, agrees that lengthening telomeres will have any effect on the aging process. For example, researchers at the University of Utah were unable to conclude whether shorter telomeres were simply a sign of aging or actually a contributor to the process.

Dr. Andrew Stern, who is one of the founders of Libella Gene Therapeutics, was also one of the principal discoverers of portions of human telomerase.

In order to be eligible for the trial, individuals must be 45 years or older. So far the company has recruited two people, according to the OneZero interview published on Medium.

The study will look into the change in the length of telomeres, and into the incidence of serious adverse events.

The FDA declined to comment specifically on Libella Gene Therapeutics and its decision to hold its trial outside of the U.S. It does, however, accept foreign clinical data and results so long as certain conditions are met.

A spokesperson for Libella Gene Therapeutics did not return FOX Business request for comment.

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Gene therapy could be a revolutionary new treatment for sickle cell disease – The Hill

Wednesday, December 11th, 2019

An effective andinnovative way to treat people with sickle cell anemia using gene therapy may soon be available thanks to efforts by several pharmaceutical companies, a Bloomberg report says.

Sickle cell anemia, a genetic defect that causes red blood cells to form in theshape ofa sickle, hinders the bodys ability to adequately distribute oxygen. This is due to atypical hemoglobin molecules, which is the protein in blood that transports oxygen. Sickle cell disease can be extremely painful, causing blood cells to get trapped in blood vessels and lead to heart failure, debilitating fatigue, strokes and blood clots.About 100,000 people suffer from sickle cell anemia in the U.S,with African Americansbeing disproportionately affected by this condition.

New developments with gene therapy, however, could work to have a positive impact on these symptoms. One of the innovative manufacturers, Bluebird Bio, stole the show at the annual conference of the American Society of Hematology in Florida. Its product, LentiGlobin, debuted positive results; in 17 patients treated with LentiGlobin,more than 40 percent of the hemoglobin in patients' red blood cells appearedin a healthier form thanks to gene therapy, per the article.

Bluebird isnt the only biotechnology making strides in gene therapies. Another potential treatment being researched is based on the technology called CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), a gene-editing tool that is being used for a wide range of biomedical applications.

Documented in an NPR report, sickle cell patient Victoria Gray recently became the first person in the U.S. to have billions of her own cells genetically edited with CRISPR and reintroduced into her body. These cells will hopefully produce fetal hemoglobin to compensate for the faulty hemoglobin in Grays red blood cells. The trial is being expanded to include more patients and is being conducted by Vertex Pharmaceuticals and CRISPR Therapeutics of the Boston area.

Current treatments for sickle cell include blood and bone marrow transfusions and medication. Studies on gene therapy treatments have been encouraging so far, but there is more testing to be done before either CRISPR or LentiGlobin hits the market.

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Hopkins team invents non-viral system for getting gene therapy into cells – FierceBiotech

Wednesday, December 11th, 2019

One of the most popular methods for inserting therapeutic genes into cells to treat disease is to transport them using a virus that has been stripped of its infectious properties. But those noninfectious viruses can still sometimes touch off dangerous immune responses.

A team from Johns Hopkins Medicine is proposing an alternative method for transporting large therapies into cellsincluding genes and even the gene-editing system CRISPR. Its a nano-container made of a polymer that biodegrades once its inside the cell, unleashing the therapy. The researchers described the invention in the journal Science Advances.

The team, led by biomedical engineer Jordan Green, Ph.D., was inspired by viruses, which have many properties that make them ideal transport vehicles. They have both negative and positive charges, for example, which allows them to get close to cells. So Green and his colleagues developed a polymer containing four molecules with both positive and negative charges. They used it to make a container that interacts with the cell membrane and is eventually engulfed by it.

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The Hopkins researchers performed four experiments to prove the nanocontainers would travel into cells and deliver complex therapies once inside. First, they packaged a small protein into the polymer material and mixed it with mouse kidney cells in a lab dish. Using fluorescent tags, they confirmed that the protein made it into the cells. Then they repeated the experiment with a much larger medicinehuman immunoglobulinand observed that 90% of the kidney cells received the treatment.

From there, they made the payload even bulkier, packaging the nanocontainers with the gene-editing system CRISPR. With the help of fluorescent signals, they were able to confirm that CRISPR went to work once inside the cells, disabling a gene 77% of the time.

"That's pretty effective considering, with other gene-editing systems, you might get the correct gene-cutting result less than 10 percent of the time," said graduate student Yuan Rui in a statement.

Finally, the Hopkins researchers injected CRISPR components into mouse models of brain cancer using the polymer nanocontainers. Again they saw evidence that successful gene editing had occurred.

Developing improved methods for gene therapy is a priority in the field. In October, for example, scientists at Scripps Research described a way to use a small molecule called caraphenol A to lower levels of interferon-induced transmembrane (IFITM) proteins, which could, in turn, allow viral vectors to pass more easily into cells. And earlier this year, an Italian team described a method for including the protein CD47 in lentiviral vectors to improve the transferring of therapeutic genes into liver cells.

The next step for Hopkins researchers Rui and Green is to improve the stability of the nanocontainers so they can be injected into the bloodstream. They hope to be able to target them to cells that have certain genetic markers, they reported.

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Pfizer to bring gene therapy production in-house – BioPharma-Reporter.com

Wednesday, December 11th, 2019

Pfizer moved into gene therapies earlier than some of its peers, partnering with Spark Therapeutics in 2014 and paying close to $200m (180m) upfront to acquire Bamboo Therapeutics two years later. The Bamboo takeover gave Pfizer ownership of a manufacturing facility in North Carolina, US.

Earlier this year, Pfizer doubled down on in-house production of gene therapies, committing $500m to expand its footprint in North Carolina.

Talking at a recent investor conference, Mikael Dolsten, chief scientific officer at Pfizer, said the spending commitment is, in part, a reflection of a belief that keeping production in-house will deliver better results than relying on third parties.

Dolsten said, When we compare that with what we get from other companies, we think we can really improve the yield, the purity and the characterization of the product.

Across the industry, poor yields have exacerbated capacity constraints created by the rapid expansion of the gene therapy pipeline, turning quality manufacturing capacity into a sought after resource.

A desire to possess in-house manufacturing capacity was a factor in many of the recent acquisitions of gene therapy companies, such as Astellas $3bn takeover of Audentes Therapeutics.

Gene therapy startups, such as Audentes and Bamboo, bypassed the limitations of contract capacity by establishing internal capabilities. Those capabilities enabled the companies to advance their gene therapies and, ultimately, to attract takeover offers, but their creation required the sort of upfront investments in infrastructure that many venture-backed startups typically try to avoid.

Through its $500m gene therapy investment, Pfizer thinks it can provide an alternative for startups that are struggling to access high-quality contract capacity but are unable or unwilling to build their own facilities.

Dolsten said, We think it's a competitive advantage, not just for our product, but for companies that want to partner with Pfizer that may allow them to have an easier and more high-end dialogue with regulators across the globe about this new field and a new type of product.

If Dolsten is right, the North Carolina manufacturing capacity could give Pfizer an edge when it tries to partner with gene therapy startups that have other options open to them, such as alliances with rival drugmakers and contract manufacturing organizations.

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Research targets gene therapy for exudative AMD patients – Modern Retina

Wednesday, December 11th, 2019

Abstract / Synopsis:

Two anti-VEGF gene therapies are being investigated in clinical trials of patients with exudative age-related macular degeneration. Initial efficacy and safety results are encouraging.

Anti-VEGF gene therapy for exudative age-related macular degeneration (AMD) has transformative potential for reducing treatment burden and improving patient outcomes, according to Szilrd Kiss, MD.

Two investigational anti-VEGF gene therapies are currently being investigated in clinical trialsRGX-314 (Regenxbio) and ADVM-022 (Adverum). Dr. Kiss described the two technologies and reviewed some preliminary clinical trial results that support their promise for providing sustained benefit with a single injection.

Considering the treatment burden of anti-VEGF therapy for other ocular diseases, we can imagine that exudative AMD is just the first indication that will be targeted for anti-VEGF gene therapy, said Dr. Kiss, chief, Retina Service, associate professor of ophthalmology, and associate dean at Weill Cornell Medical College, New York, NY.

RGX-314 delivers a gene for an anti-VEGF fab protein that is similar to ranibizumab. It uses adeno-associated virus-8 (AAV8) as a vector and is administered in the operating room as a subretinal injection.

AAV is the most common viral vector carrier used for gene therapy. Different AAV serotypes have different tissue selectivity, Dr. Kiss explained. AAV8 is a wild type AAV that has the propensity for greater transfection of retinal cells compared with AAV2 following subretinal gene therapy delivery.

RELATED:AAO 2019: Encouraging results revealed from early trial of subretinal gene therapy for wet AMD

Disclosures:

Szilrd Kiss, MDe: [emailprotected]This article was adapted from Dr. Kiss presentation at the 2019 meeting of the American Academy of Ophthalmology. Dr. Kiss is a consultant to RegenxBio and Spark Therapeutics and is a consultant and equity owner in Adverum.

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Dr. James Wilson, a scientific pioneer, on the future of gene therapy – STAT – STAT

Wednesday, December 11th, 2019

Dr. James Wilson is a pioneer in gene therapy. That does not mean he is necessarily impressed with the current state of affairs.

In five years, when we look back on the way were executing on gene therapy now, were going to realize that things are going to be very different, Wilson said at the STAT Summit in Cambridge, Mass., recently. The way in which were going to treat Duchenne muscular dystrophy, potentially cure it, is not the way in which its being evaluated in the clinic now.

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Buyer beware of this $1 million gene therapy for aging – MIT Technology Review

Wednesday, December 11th, 2019

Its said that nothing is certain except death and taxes. But doubt has been cast over the former since the 1970s, when scientists picked at the seams of one of the fundamental mysteries of biology: the molecular reasons we get old and die.

The loose thread they pulled had to do with telomeresmolecular timepieces on the ends of chromosomes that shorten each time a cell divides, in effect giving it a fixed life span. Some tissues (such as the gut lining) renew almost constantly, and it was found that these have high levels of an enzyme called telomerase, which works to rebuild and extend the telomeres so cells can keep dividing.

That was enough to win Elizabeth Blackburn, Carol Greider, and Jack Szostak a Nobel Prize in 2009. The obvious question, then, was whether telomerase could protect any cell from agingand maybe extend the life of entire organisms, too.

While telomere-extending treatments in mice have yielded intriguing results, nobody has demonstrated that tweaking the molecular clocks has benefits for humans. That isnt stopping one US startup from advertising a telomere-boosting genetic therapyat a price.

Libella Gene Therapeutics, based in Manhattan, Kansas, claims it is now offering a gene therapy to repair telomeres at a clinic in Colombia for $1 million a dose. The company announced on November 21 that it was recruiting patients into what it termed a pay-to-play clinical trial.

Buyer beware, though: this trial is for an unproven, untested treatment that might even be harmful to your health.

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The company proposes to inject patients with viruses carrying the genetic instructions cells need to manufacture telomerase reverse transcriptase, a molecule involved in extending the length of telomeres.

The dangers are enormous, says Jerry Shay, a world expert on aging and cancer at the University of Texas Southwestern Medical Center. Theres a risk of activating a pre-cancerous cell thats got all the alterations except telomerase, especially in people 65 and over.

For years now, people involved in the company have made shifting claims about the study, raising uncertainty about who is involved, when it might start, and even where it would occur. Trial listings posted in October to clinicaltrials.gov currently show plans for three linked experiments, each with five patients, targeting critical limb ischemia, Alzheimers, and aging, respectively.

Jeff Mathis, president of Libella, told MIT Technology Review that two patients have already paid the enormous fee to take part in the study: a 90-year-old-woman and a 79-year-old man, both US citizens. He said they could receive the gene therapy by the second week of January 2020.

The decision to charge patients a fortune to participate in the study of an experimental treatment is a red flag, say ethics experts. Whats the moral justification for charging individuals with Alzheimers? asks Leigh Turner, at the University of Minnesotas Center for Bioethics. Why charge those bearing all the risk?

The telomere study is occurring outside the US because it has not been approved by the Food and Drug Administration. Details posted to clincaltrials.gov indicate that the injections would be carried out at the IPS Arcasalud SAS medical clinic in Zipaquir, Colombia, 40 kilometers (25 miles) north of Bogot.

It takes a lot longer, is a lot more expensive, to get anything done in the US in a timely fashion, Mathis says of Libellas choice to go offshore.

To some promoters of telomerase gene therapy, urgency is justified. Heres the ethical dilemma: Do you run fast and run the risk of low credibility, or move slowly and have more credibility and global acceptancebut meanwhile people have died? says Mike Fossel, the president of Telocyte, a company planning to run a study of telomerase gene therapy for Alzhheimer's in the US if it can win FDA signoff.

Our reporting revealed a number of unanswered questions about the trial. According to the listings, the principal investigatorwhich is to say the doctor in charge--is Jorge Ulloa, a vascular surgeon rather than an expert in gene transfer. I dont see someone with relevant scientific expertise, says Turner.

Furthermore, Bill Andrews, who is listed as Libellas chief scientific officer, says he does not know who Ulloa is, even though on Libellas website, the mens photos appear together on the list of team members. He said he believed that different doctors were leading the trial.

Turner also expressed concerns about the proposed 10-day observation period described in the posting for the overseas study: If someone pays, shows up, has treatment, and doesnt stick around very long, how are follow-up questions taking place? Where are they taking place?

Companies seeking to try the telomere approach often point to the work of Maria Blasco, a Spanish scientist who reported that telomere-lengthening gene therapy benefited mice and did not cause cancer. Blasco, director of the Spanish National Centre for Cancer Research, says she believes many more studies should be done before trying such a gene experiment on a person.

This isnt the first time Libella has announced that its trial would begin imminently. It claimed in late 2017 that human trials of the telomerase therapy would begin in the next few weeks. In 2016, Andrews (then partnered with biotech startup BioViva) claimed that construction of an age reversal clinic on the island nation of Fiji would be complete before the end of the year. Neither came to pass.

Similar questions surround Libellas most recent claims that it has two paying clients. Pedro Fabian Davalos Berdugo, manager of Arcasalud, said three patients were awaiting treatment in December. But Bioaccess, a Colombian contract research organization facilitating the Libella trial, said that no patients had yet been enrolled.

Also unclear is where Libella is obtaining the viruses needed for the treatment. Virovek, a California biotech company identified by several sources as Libellas manufacturer, did not answer questions about whether any treatment had been produced.

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New Anti-Aging Clinical Trial Begins. For $1 Million, You Can Be a Participant. – Livescience.com

Wednesday, December 11th, 2019

An American biotech company has launched clinical trials in Colombia to test a new therapy designed to reverse the aging process, and in turn, treat age-related diseases, according to news reports.

But to steal a sip from this purported fountain of youth, participants in the trial must first fork over $1 million a fee that seems even more astronomical when you consider that most clinical trials are either free or provide participants with financial compensation, according to a report by OneZero, a Medium publication about tech and science.

The pricey trial is being run by Libella Gene Therapeutics, a Kansas-based company whose website proclaims that "the future is here." The company announced its intention to test its anti-aging remedies in Cartagena, Colombia, in 2018, and began recruiting for the trials in October of this year. Using a single-gene therapy, Libella aims to "prevent, delay, or even reverse" the general effects of aging, as well as treat diseases that emerge in old age, such as Alzheimer's, according to ClinicalTrials.gov.

In fact, in its own press release, the company boasted, without evidence, that its gene therapy "may be the world's first cure for Alzheimer's disease." The bold claim raises an obvious question: Will the treatment actually work?

Short answer: No one really knows, but the fact that Libella shipped its operation beyond the reach of the U.S. Food and Drug Administration (FDA) doesn't inspire confidence, experts told OneZero.

Related: 5 Reasons Not to Fear Getting Older

Unlike anti-aging face creams that soften the superficial signs of aging, the Libella therapy aims to reverse aging from the ground up, so to speak, starting at the level of our genes. Specifically, the gene therapy is intended to lengthen patients' telomeres structures that cap the tips of chromosomes and prevent the genetic material inside from fraying. Telomeres grow shorter each time a cell divides, and when the structures reach a critical length, cells either stop dividing or perish, according to Stanford Medicine.

The theory goes, if you rebuild the body's shortened telomeres, the process of aging might be thrown in reverse. This is not a new idea. Several studies in mice suggest that using gene therapy to lengthen telomeres can reverse certain signs of aging in the animals. A 2015 study from Stanford prompted similar effects in isolated human cells; the treatment lengthened cells' telomeres by fiddling with a close cousin of DNA, called RNA, which helps cells build proteins.

The Libella therapy aims to help cells rebuild telomeres by activating a gene in their DNA that would normally be switched "off." The gene, called TERT, contains instructions to build a protein called "telomerase," an enzyme that adds molecules to the end of telomeres and prevents the structures from shortening during cell replication, according to a 2010 report in the journal Biochemistry.

Libella's lead scientific officer, molecular biologist William Andrews, originally helped identify the human telomerase enzyme at the biotech firm Geron. Later, he licensed a gene therapy based on the finding to Libella, according to OneZero. "I can't say [telomere shortening is] the only cause of aging, but it plays a role in humans," Andrews told the publication.

Related: 8 Tips for Healthy Aging

Andrews' therapies will soon be put to the test in Colombia, where one 79-year-old will receive the anti-aging treatment in next month, according to OneZero. The anti-aging trial will include four more participants over age 45 and focus on verifying that the treatment is "safe and tolerable," meaning it does not harm patients or cause unacceptable side effects.

Two more trials will use the same therapy but aim to "prevent, delay, or even reverse the development" of Alzheimer's disease and critical limb ischemia, an age-related condition in which a person's arteries become severely obstructed. Participants in these trials must already be diagnosed with the disorders.

After treatment, participants in all three trials will remain in the clinic for 10 days for further monitoring, and then return at regular intervals for checkups over the following year.

Libella's gene therapy involves a one-time injection delivered through an IV; the Alzheimer's therapy uses the same formula but doctors inject the product into the patient's spinal fluid. Within the product, a modified virus carries the TERT gene into cells and injects the genetic material into their DNA. The modified viruses cannot transmit diseases to people, but in high enough doses, the germs could provoke a harmful immune response in the patient, according to a 2018 animal study. Libella representatives declined to say how high a dose their clinical trial participants will receive.

"All I can say is, it's a lot," Andrews told OneZero.

Potential side effects aside, the fact that the Libella treatment will be administered beyond the purview of the FDA is telling, according to one expert. Leigh Turner, a bioethicist at the University of Minnesota, told OneZero that "even though the company is based in the United States, they've managed to find a way to evade U.S. federal law by going to a jurisdiction where it's easier to engage in this activity."

The $1 million entry fee is also alarming, Turner said, given that most clinical trials don't charge patients anything to enter. Andrews told OneZero that the fee is justified because it costs the company hundreds of thousands of dollars to make enough product to treat just one person.

The appearance of the trials on ClinicalTrials.gov, an official registry maintained by the National Institutes of Health, does not boost their credibility, she added. The automated database can be easily manipulated and "can basically be used as a marketing platform," she said.

Other stakeholders in the telomere-lengthening business are concerned, too. Michael Fossel, founder and president of the biotech startup Telocyte, told OneZero that his company's own therapy is similar to the Libella treatment the difference is that Telocyte is seeking approval through the FDA. "We're afraid that something will go wrong [with the Libella trials], whether it's from a safety or efficacy standpoint," he said.

Related: Extending Life: 7 Ways to Live Past 100

But even in a best case scenario, wherein no patients come to harm, the Libella therapy still might not deliver any notable health benefits. Some research suggests that no link exists between telomere length and aging.

For instance, a study published this year examined more than 261,000 people between age 60 and 70, and found no correlation between participants' telomere lengths and their age-related health outcomes, including their overall cognitive function, muscular integrity and the age of their parents. Long telomeres were associated with a lowered risk of coronary heart disease as compared with short telomeres, but longer telomere length was also linked to a heightened risk of cancer.

"Telomere lengthening may offer little gain in laterlife health status" and lead to an increased risk of cancer, the authors noted.

It remains to be seen whether Libella has truly tapped the fountain of youth, but given the dubious nature of their clinical trials, potential participants may want to exercise caution before relocating to Colombia and shelling out $1 million for a chance to live longer.

Originally published on Live Science.

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GenEdit and Editas Medicine Enter into Exclusive License and Collaboration Agreement for Nanoparticle Gene Therapy Delivery – BioSpace

Wednesday, December 11th, 2019

Dec. 11, 2019 14:00 UTC

BERKELEY, Calif.--(BUSINESS WIRE)-- GenEdit Inc., a developer of a novel polymer nanoparticle technology platform for non-viral- and non-lipid-based delivery of gene therapies, today announced that it has entered into a worldwide, exclusive license and collaboration agreement with Editas Medicine, Inc., a leading genome editing company. GenEdit has developed a comprehensive delivery system for CRISPR-based therapeutics, including gene knockout and gene repair therapies, to enable safer delivery options with improved efficiency.

"This license and collaboration agreement further validates the strength of our intellectual property portfolio and the potential of GenEdits technology," said Kunwoo Lee, Ph.D., co-founder and chief executive officer of GenEdit. "We are pleased to establish our relationship with Editas Medicine as they leverage our technology to develop potential genomic medicines."

Under the terms of the agreement, GenEdit has granted Editas Medicine an exclusive worldwide license, with rights to sublicense, to GenEdits Cpf1-based technologies. In return for these rights, GenEdit will receive undisclosed upfront and development milestone payments, including royalties on net sales of products incorporating the licensed intellectual property. In addition, GenEdit and Editas Medicine will collaborate on evaluating delivery of Cpf1-based technologies with GenEdits nanoparticle platform. Editas Medicine will provide research funding and have an option to continue development after the initial collaboration period.

GenEdits nanoparticle platform consists of a proprietary non-viral, non-lipid library of polymers that efficiently encapsulate and deliver cargo [RNA, DNA, protein and/or ribonucleic acid-protein complexes (RNP)] to specific tissues. The company screens the library to identify initial hits and then uses computational analysis and medicinal chemistry for iterative lead optimization. The company has used this platform to identify multiple candidate polymers for efficient and specific delivery of gene editing to a range of tissues.

"Compared to viral vectors and lipid-based nanoparticles, our approach has the potential for better targeting, more cargo, and lower manufacturing cost," said Timothy Fong, Ph.D., chief scientific officer of GenEdit. "In particular, our approach has the potential to enable in vivo gene editing of multiple tissues with CRISPR and expand the potential of gene therapies to treat more diverse sets of diseases."

About GenEdit

GenEdit was founded to transform the delivery of gene and gene editing therapies. We have synthesized the NanoGalaxy library of polymers that can encapsulate RNA, DNA, protein and/or RNP. Through advanced screening methods, computational analysis and iterative medicinal chemistry, we have demonstrated efficient delivery of gene editing cargo to specific tissues. We seek development partnerships for specific tissues and/or gene targets while advancing our internal pipeline of gene editing therapies.

For more information, please visit http://www.genedit.com.

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Cancer gene therapy backed by Blackstone gets trial win – BioPharma Dive

Wednesday, December 11th, 2019

A gene therapy for bladder cancer that recently received $400 million in support from the private equity company Blackstone Group helped more than half of treated patients with resistant disease achieve remission.

The therapy, called nadofaragene firadenovec, was discovered by a Finnish-based research institute and first entered clinical study in 2012. The data revealed today at the Society of Urologic Oncology meeting came from a Phase 3 trial that is part of the agent's Biologics License Application now before the FDA.

Licensed by its original owner, FKD Therapies Oy, to Switzerland-based Ferring Pharmaceuticals, nadofaragene firadenovec is now in the hands of the U.S. subsidiary FerGene. That company was created with the Blackstone investment and an additonal $170 million from Ferring. FerGene will commercialize the gene therapy in the U.S., with Ferring holding rights elsewhere.

Nadofaragene firadenovec is an an adenovirus-based gene therapy encoding production of the immunity-stimulating protein interferon alfa-2b. Viral vectors containing the gene are administered by catheter once every three months into the bladder, where they are absorbed into cells in the organ's walls and begin stimulating interferon.

Delivery through a catheter, called intravesical administration, limits systemic exposure to both the viral vectors and to inteferon, said Neal Shore, medical director for the Carolina Urologic Research Center and an investigator in the trial.The side-effects of interferon include flu-like symptoms in patients who inject it for other conditions like multiple sclerosis.

The clinical trial enrolled 157 patients with bladder cancer that has not spread to muscle walls and has stopped responding to treatment with Bacillus Calmette-Gurin vaccine.

Alternative treatments for these patients include chemotherapy or a procedure called "complete cystectomy." This surgery entails complete removal of the bladder, which in men means removal of the prostate and seminal vesicles and in women the uterus, ovaries, fallopian tube and part of the vagina.

"Radical cystectomy is one of the most invasive surgeries we do not just in urology but in all of surgery," Shore said, requiring a lengthy hospital stay and having a high rate of post-procedural complications.

Out of a group of 103 patients with superficial tumors in the bladder wall, just over half were in complete remission at three months, 41% at six months, and 24% at one year. In a group of 48 patients whose cancer had spread to the connective tissue outside the bladder, 73% had no recurrence of serious disease at three months, which fell to 44% at 12 months.

In this type of bladder cancer, the FDA has said a single-arm trial, without a placebo control, using complete remission is sufficient to be considered for approval, and the study does not need to pre-specify a rate that would define success. "The natural history of [disease]is well understood, and the complete response rate is negligible in the absence of therapy," the agency said in guidelines published in February 2018.

One chemotherapy agent, called Valstar (valrubicin), is approved for this patient group. It won FDA approval on a complete response rate of 18%.

In seeking FDA approval, nadofaragene firadenovec is in a race with Merck & Co.'s Keytruda (pembrolizumab) to achieve approval first. That immuno-oncology agent tested Keytruda in a similar population in the Keynote-057 trial, in which it achieved a 39% complete response rate.

Keytruda will be the subject of a meeting of the FDA's Oncologic Drugs Advisory Committee on Dec. 17.

Aside from the remission rates,Shore said nadofaragene firadenovec would differentiate itself from Keytruda in practice because its intravesical delivery means it could be administered by community-based urologists at outpatient clinics.

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A #ReUp of 2019: The year when gene therapy, DNA modifications came of age & saved lives – Economic Times

Wednesday, December 11th, 2019

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

"I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

"But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection.

"You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

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Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

"Not everyone is a biologist or scientist," she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

"I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

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A #ReUp of 2019: The year when gene therapy, DNA modifications came of age & saved lives - Economic Times

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Watch out, Keytruda. Ferring’s bladder cancer gene therapy rival has new dataand they look competitive – FiercePharma

Tuesday, December 10th, 2019

On the heels of an FDA speedy review for Keytrudas potential use in non-muscle invasive bladder cancer (NMIBC), its close rival, a gene therapy by Ferring Pharmaceuticals spinout FerGene, has posted late-stage data. By the looks of it, the two drugs are up for a fight.

Among patients with high-risk NMIBC superficial disease thats unresponsive to standard-of-care Bacillus Calmette-Gurin (BCG), nadofaragene firadenovec eliminated tumors in 53%, or 55 of 103 patients,at month three in a phase 3 study, FerGene unveiled Thursday at the Society of Urologic Oncology meeting.

By comparison, in Keytrudas own registrational trial on the same target patient population, the Merck & Co. PD-1 completely cleared tumors in 41.2%, or 42 of 102 patients, after three months, according to an update at the European Society for Medical Oncology annual meeting in September.

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The length of time responses lasted appeared similar between the two drugs in their separate studies. For Keytruda, 24 patients (23.5%) continued to show no signs of disease after a year. As for nadofaragene firadenovec, 24.3%, or 25 patients, were still tumor-free at month 12.

In terms of safety, Keytruda recorded Grade 3/4 side effects in12.7% of patients, while FerGene said there were no Grade 4/5 events in its study.

We are pleased with these Phase 3 data results, including the complete response rates and favorable safety profile seen with nadofaragene firadenovec, Nigel Parker, scientific founder of FKD Therapies, said in a statement. The data have also helped FKD'snew drug application earn an FDA priority review.

RELATED:Merck's Keytruda is bound for new bladder cancer territory. But can it hold up against gene therapy?

Ferring recently gained commercial rights to the gene therapy from FKD, and, with $400 million in help from Blackstone Life Sciences, spun it into FerGene. Interestingly, it was Merck that licensed the drugout to FKD in the first place in 2011 in return for an equity stake in the then-newly formed Finnish company.

Priority reviews in hand, the two companies could be looking at FDA approvals soon. The burning question is, how does FerGene plan to price a gene therapy, which belongs to a class of drug thats notoriously costly? In a statement sentto FiercePharma, Ferring said it's too early to discuss pricing, that its top priority is still to get nadofaragene firadenovec approved andinvest into R&Dto study the product in more indications.

Keytruda is meant to be given ata fixed dose every three weeks. Nadofaragene firadenovec, which uses an adenovirus vector to deliver the gene interferon alfa-2b to stimulate an innate immune response to fight cancer, is administered into the bladder every three months.

Merck does have an upper hand against FerGene. The Big Pharma has been the sole supplier of BCG in the U.S. and several other key markets globally for several years now. So, it could offer BCG and Keytruda as a one-two punch for NMIBC, similar to the wayBayer is billing Nexavar and Stivarga as a part of the same continuumin first- and second-line liver cancer.

RELATED:Merck limits orders for bladder cancer drug as demand outstrips supply

There are other players eyeing the same patient population. Sesen Bio has Vicinium, an antibody-drug conjugate that targets epithelial cell adhesion molecule antigens on the surface of tumor cells to deliver a toxin payload. In its own phase 3 trial dubbed Vista also in high-risk, BCG-unresponsive NMIBC, Vicinium eradicated tumors in 40% of 89 patients at month three, according to an update the company provided in August. However, its response seems to wane over time more quickly than its rivals', as only 17% of patients showed no signs of tumor activity after 12 months.

The Cambridge, Massachusetts-based biotech recently held two meetings with the FDA and confirmed a submission process, including the design for a post-marketing confirmatory trial. It would enroll BCG-refractory patients who, because of supply constraints, haven't received an optimal BCG dose, which the company said represents a broader patientpopulation in light of anongoing shortage.

Sesen now expects to submit a biologics license application under rolling review by year-end with potential approval in 2020.

As for its pricing, during a presentation at the H.C. Wainwright investor conference in September, Sesens president and CEO Thomas Cannell pointed out that PD-1/L1s would cost about $150,000 to $200,000 per patient per year in NMIBC.

Weve done two rounds of market research with payers, and they think thats reasonable, he said. They think at those levels, there will probably be minimal prior authorization or step edits in terms of restricting a treatments use.

Assuming an official launch in 2021, Jefferies analysts, in a Nov. 12 note to clients, pegged $167.5 million for Viciniums U.S. sales in 2024. Before the priority designation, SVB Leerinks Daina Graybosch predicted a Keytruda launch in NMIBC in 2022 and forecastU.S. sales of $250 million in the indication for the Merck PD-1 inhibitor in 2025.

Editor's Note: The story has been updated with a statement from Ferring Pharma.

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First 2 Adults with Severe Hemophilia A Respond Well to Gene Therapy BAY 2599023 in Clinical Trial – Hemophilia News Today

Tuesday, December 10th, 2019

Bayersinvestigationalgene therapy BAY 2599023 safely and effectively increased the levels ofclotting factor VIII (FVIII) and prevented or lessened bleeding in the first two people with severe hemophilia A treated ina Phase 1/2 clinical trial, preliminary data show.

The ongoing trial (NCT03588299; 2017-000806-39) is enrolling up to 30 eligible adult patients. More information, including recruiting sites in the U.S. and Europe, is availablehere.

These early results will be presented in the poster, First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A, at the 61st American Society of Hematology (ASH) Annual Meeting & ExpositionrunningDec. 710 in Orlando, Florida.

BAY 2599023 initially by Dimension Therapeutics as DTX201 is being developed by Bayer in collaboration with Ultragenyx Pharmaceuticals. The potential gene therapy aims to promote a sustained production of FVIII and overcome its deficit in hemophilia A patients, reducing or eliminating the need for prophylatic, or preventive, FVIII replacement therapy and the occurrence of bleeding events.

Administered as a single infusion, the therapy uses a modified and harmless version of the adeno-associated virus (AAV), called AAVhu37, to deliver a shorter but functionalcopy of the FVIII gene to liver cells, where clotting factors are produced. This version of the FVIII gene is known as B-domain deleted FVIII gene.

Preclinical studies showed that AAVhu37 effectively delivered the FVIII gene to liver cells, had a favorable distribution, and induced a durable FVIII production.

In addition, preclinical data showed that BAY 2599023 had a good safety profile, and the potential to promote FVIII production to levels considered to be therapeutic over a long period of time.

The ongoing, dose-establishingPhase 1/2 trial (NCT03588299; 2017-000806-39) is evaluating the safety, tolerability and early effectiveness of three ascending doses of BAY 2599023 in adult men with severe hemophilia A who have been previously treated with FVIII products.

It is the first clinical trial to evaluate a gene therapy based on the AAVhu37.

Up to 30 enrolled patients will be given a single intravenous infusion of one of three doses of BAY 2599023. The studys primary goal is to measure safety through reports of adverse events. Secondary goals include measuring FVIII activity and assessing the number of patients who reach more than 5% of FVIII production at six and 12 months after treatment at the different doses.

Data on the first two men treated at BAY 2599023s starting dose (0.5 x 1013 gene copies/kg) will be presented at the meeting. These men had more than 150 days of treatment with FVIII products, no history of FVIII inhibitors, and no detectable immune response against AAVhu37.

No adverse events were reported after more than 15 weeks of safety evaluations (about four months). Blood levels of liver enzymes also remained within a normal range, and either of these patients needed to be treated with corticosteroids.

The first man reached a stable FVIII production of around 5%, and was free of bleeding events or a need for prophylactic treatment for six weeks. The second patient, who had 99 bleeds in the year before receiving the gene therapy, reached a stable FVIII production of around 17%, and has been bleed-free for more than 5.5 months (at the time of data collection).

These preliminary data suggest that BAY 2599023 is safe and effective in promoting the production of FVIII and in reducing or preventing the occurrence of bleeding events and the need for prophylactic treatment, the researchers wrote.

Overall, data generated from this first dose cohort demonstrate that successful translation from pre-clinical to clinical development and proof-of-mechanism for BAY 2599023 was achieved, they concluded.

Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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Orgenesis and Theracell to Launch Point of Care Cell and Gene Therapy Centers within HYGEIA Group’s Hospital Network in Greece – GlobeNewswire

Tuesday, December 10th, 2019

GERMANTOWN, Md., Dec. 06, 2019 (GLOBE NEWSWIRE) -- Orgenesis Inc. (NASDAQ: ORGS)(Orgenesis or the Company), a leading cell and gene therapy enabling company providing centralized CDMO manufacturing and development services through its subsidiary Masthercell Global, Inc., as well as localized point-of-care(POCare) development and processing centers for therapeutic treatments, today announced a strategic partnership agreement (Partnership) between the HYGEIA Group and the TheracellOrgenesis joint venture (JV). Under the Agreement, the JV will implement Orgenesis POCare cell therapy platform for clinical development and commercialization of cell and gene therapies within HYGEIA Groups network of three hospitals in Greece. As previously announced, Orgenesis and TheraCell Advanced Biotechnology formed a JV to advance Orgenesis POCare platform in Greece, Cyprus, the Balkan region and selected Middle Eastern countries.

The POCare platform is designed to collect, process and supply cells within the patient care setting for various therapeutic treatments. The goal of the platform is to reduce the cost and complexity of supplying cell and gene therapies, as well as elevate quality standards by integrating automated processing units and proprietary technologies.

HYGEIA is the first hospital network in this region to implement Orgenesis POCare cell therapy platform. The Partnership is intended to provide HYGEIA Group with resources to advance clinical development and deliver personalized, advanced therapies across its network for a wide range of diseases in oncology, hematology, orthopedics, nephrology, dermatology and diabetes.

This partnership with the HYGEIA Group further validates the significant value proposition of our POCare platform, as it enables the development and delivery of cell and gene therapies onsite at hospitals. We believe this platform has the potential to transform the cell and gene therapy market, by bringing life-saving therapies to market in a much more time and cost-effective manner, said Vered Caplan, CEO of Orgenesis. Theracell has proven to be an ideal partner with extensive experience and capabilities in autologous cell therapy and regenerative medicine, with operations in Greece and strong relationships throughout the region. We are in active discussions to establish PoCare locations and partnerships with hospitals and healthcare networks in other countries and regions across the world.

Andreas Kartapanis, CEO, HYGEIA Group, commented, HYGEIA Group is honored to work with Theracell and Orgenesis to become the first hospital network in Greece to provide advanced cell and gene therapies for both clinical research and patient treatment utilizing the POCare platform. We believe this Partnership will provide us a strong competitive advantage in this rapidly developing field. More importantly, this Partnership will benefit patients that will now have greater access to these important therapies.

About HYGEIA Group

HYGEIA Group operates three hospitals in Greece, with a total capacity of 1,261 beds, 52 operating rooms, 19 delivery rooms and 10 intensive care units. More than 3,100 employees and approximately 3,900 associate physicians offer their services to the HYGEIA Group, which was founded in 1970 by medical doctors, most of which were professors at the University of Athens and have since been active in providing primary and secondary care services. The following hospitals are also part of the HYGEIA Group: MITERA General, Obstetrics - Gynecology & Pediatrics Hospital and LITO Obstetrics, Gynecology & Surgical Center, licensed for 459 and 100 hospital beds, respectively.

About Theracell

TheraCell is a regenerative biotechnology company with operations in Greece, where its laboratories and primary facilities are located. The Company focuses in the areas of autologous cell therapy and regenerative medicine. TheraCell has extensive experience in the isolation, processing and application of adipose derived stem cells (ADSCs), as well as somatic cells and has developed a patented platform for tissue engineering and cell therapies in the areas of Dermatology, Chondral Defects and Chronic Kidney Injury.

About Orgenesis

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

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

Investor contact for Orgenesis:David WaldmanCrescendo Communications, LLCTel: 212-671-1021Orgs@crescendo-ir.com

Media contact for Orgenesis:Image Box CommunicationsNeil Hunter / Michelle BoxallTel +44 20 8943 4685neil@imageboxpr.co.uk/michelle@imageboxpr.co.uk

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Heading into 2020, Sickle Cell Community Welcomes Next Generation of Treatment – Morning Consult

Tuesday, December 10th, 2019

The nascent field of gene therapy, which hit its stride this year, has unlocked an opportunity for tackling sickle cell that was inconceivable just years ago, experts said. While hydroxyurea, the de-facto standard of treatment since its 1998 approval, can meaningfully reduce the frequency of pain crises, potentially curative treatments offer hope of eliminating crises altogether.

Over the weekend, ASH released its highly awaited full set of new clinical guidelines for sickle-cell treatment the most comprehensive slate of recommendations to date with even more nearing completion.

Since the passage of the Orphan Drug Act of 1983 provided financial incentive for pharmaceutical companies to focus on rare diseases, experts have been troubled by a lack of consistency in evaluation of clinical trials. The new guidelines come as drugmakers such as GBT, Novartis and Bluebird establish themselves in this space, offering researchers a uniform, measurable framework for trials to demonstrate the value of their experimental treatments.

All stakeholders agreed that this is only the beginning. Orphan drugs are estimated to comprise one-fifth of global prescription sales by 2024, according to EvaluatePharma, and blood is the leading therapeutic area by sales and market share.

As with any new drugs, uncertainties on cost loom. But experts swiftly rejected the idea that those concerns should dampen the renewed energy around tackling sickle cell, and said attempts to do so may be rooted in prejudice given sickle cells disproportionate impact on black communities.

Therapies for cancers, cystic fibrosis and hemophilia are routinely priced in the hundreds of thousands and even millions, Osunkwo said. The fact that those treatments are widely celebrated as worthwhile endeavors, while the cost of gene therapy for sickle cell is under a microscope before even winning approval, is stigmatizing and rooted in conscious bias, she said.

Both Novartis and GBT said they are actively talking with payers to facilitate coverage for their drugs, both priced around $100,000 per year, and are taking steps to shoulder some of the burden with their own patient support centers.

We should have at least another two drugs, if not more, by next year, Osunkwo said. The sickle cell community is riled up, ready to participate in clinical trials and do what it takes to get more tools in their treatment toolbox. And theyre ready to speak out about how unfair health care and research has been to their cause.

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Abeona Therapeutics Cleared to Initiate Pivotal Phase 3 Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa |…

Tuesday, December 10th, 2019

DetailsCategory: DNA RNA and CellsPublished on Tuesday, 10 December 2019 11:11Hits: 187

FDA removes clinical hold; Company may proceed with VIITALstudy

Company expects to initiate study in the first quarter of 2020

Primary endpoint confirmed as proportion of wounds with greater than 50% healing at 3 months vs control wounds

Majority of potential subjects have been pre-screened for the study

NEW YORK, NY and CLEVELAND, OH, USA I December 09, 2019 I Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has removed its clinical hold and provided clearance to proceed with the VIITALstudy, the Companys pivotal Phase 3 clinical trial evaluating EB-101 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The FDA removed the clinical hold following the Companys submission of additional data points on transport stability of EB-101 to clinical sites. Abeona expects to initiate the VIITALstudy in first quarter of 2020.

The Abeona team has worked diligently to provide a prompt and thorough response to the FDA, enabling us to proceed with our pivotal Phase 3 trial for EB-101, said Joo Siffert, M.D., Chief Executive Officer of Abeona. Recently published long-term follow up data from our Phase 1/2 trial leaves us increasingly confident that EB-101 can provide durable healing for large, chronic wounds that afflict many RDEB patients. We are now focused on initiating the VIITALstudy in the first quarter of 2020. The success in building and qualifying a state-of-the-art GMP manufacturing facility also represents a critical step toward bringing this novel product to patients in dire need of effective treatment.

With two to five years of follow-up, data from a Phase 1/2 clinical trial conducted by Stanford University evaluating EB-101 showed that the gene-corrected cell therapy provided durable wound healing for RDEB patients, including for the largest, most challenging wounds that constitute the majority of wounds in this population.

About The VIITALStudyThe VIITALPhase 3 study will be a multi-center, randomized clinical trial assessing EB-101 in 10 to 15 RDEB patients, with approximately 30 chronic wound sites treated in total. The primary study endpoint will be the proportion of wounds with greater than 50% healing at three months, comparing treated with untreated wound sites on the same patient. Secondary endpoints include the patients global impression of change in pain from baseline as well as other patient reported outcomes assessing pain during dressing changes, pain impact and physical function.

About EB-101 EB-101 is an autologous, gene-corrected cell therapy in late-stage clinical development for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patients own skin cells (keratinocytes) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Rare Pediatric designations for EB-101 and Orphan Drug designation in both the U.S. and EU.

About Recessive Dystrophic Epidermolysis BullosaRecessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

About Abeona TherapeuticsAbeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Companys clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Companys portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses the novel AIM AAV vector platform to address all mutations of cystic fibrosis. Abeona has received twenty regulatory designations from the FDA and EMA for its pipeline candidates. For more information, visit http://www.abeonatherapeutics.com.

SOURCE: Abeona Therapeutics

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Drug Launch of the Year: Zolgensma – BioPharma Dive

Tuesday, December 10th, 2019

Up until three years ago, Vamshi Rao didn't have any treatments to offer the families of babies born with a genetic disease that meant they likely wouldn't live past the age of two.

No medicines existed for the degenerative condition called spinal muscular atrophy, or SMA. So Rao, a neurologist at the Lurie Children's Hospital of Chicago, could only help manage expectations and day-to-day care.

His role, and that of other physicians, changed dramatically in 2016, when Biogen's Spinraza became the first drug to win U.S. approval for the condition, and then again this May, when the Food and Drug Administration cleared a gene therapy called Zolgensma.

Rao can now give families options that represent a realistic shot at a longer, better life for babies with SMA.

"We've never been in this situation before," he said in a September interview.

Between May and October, about 100 families facing the diagnosis opted for Zolgensma, just the second gene therapy for an inherited disease to reach commercial markets in the U.S. Unlike Spinraza, Zolgensma is designed to be a one-time treatment and, possibly, a cure.

Several of those infants were treated at Lurie Children's, where Rao is also helping run clinical studies sponsored by Novartis, the Swiss pharmaceutical company that sells Zolgensma. About 50 other U.S. institutions have administered Zolgensma commercially, outside of clinical testing.

Approval was a major accomplishment in a field that had for decades held the potential to develop curative fixes for devastating genetic disorders like SMA.

But its arrival clouded by a scandal over manipulated animal testing data submitted by Novartis made real once theoretical concerns over how patients will receive and pay for expensive, one-time therapies.

Infused via inactivated, hollowed-out viruses, Zolgensma replaces the missing or defective gene that causes SMA to develop. Infants in the Phase 1 study used to win approval could sit up independently, stand and, in two cases, even walk in the months and years after receiving Zolgensma functional milestones that wouldn't have been reached otherwise.

Whether Zolgensma can actually be a cure won't be known definitively for years. But in theory it could be if given to infants early enough, before their affected neurons start to die, says Jerry Mendell of Nationwide Children's Hospital in Ohio, who led the first study of what would become Zolgensma.

The promise of lifelong benefit was central to Novartis' justification for pricing Zolgensma at $2.1 million per patient, more than any other drug before it.

While insurers initially balked at covering such a pricey, one-time treatment, Novartis says coverage is now in place for 90% of eligible patients on commercial plans and 30% of those on Medicaid.

"It's clear that payers are not happy here, but they have largely relented," said Ronny Gal, an analyst at Bernstein, in an interview.

"It's hard to push against a drug that takes a kid who would die and allows him to live. But for products that are less efficacious, I'd expect pushback."

Hoping to ease insurer concerns, Novartis offered to spread Zolgensma's cost over five years, and is working to set up agreements linking reimbursement to patient outcomes. While the latter option has proved popular, none have yet taken up the drugmaker on its pay-over-time proposal, said Dave Lennon, CEO of AveXis, the biotech developer of Zolgensma that Novartis bought for $8.7 billion last year, in an interview.

Also helping the pharma's case to insurers was an estimate from the Institute for Clinical and Economic Review that judged Zolgensma, if given very early, could be cost-effective at a price between $1.2 million and $2.1 million. (A more conservative finding by ICER, however, put Zolgensma's cost-effective price at just under $900,000.)

"Certainly you see now the methodology that ICER is using and the concept of value-based pricing is an extremely important part of the discussions with payers," said Lennon.

Yet early treatment, before symptoms arise, is dependent on genetic screening for SMA. While the federal government has recommended the condition be tested for in newborns since July 2018, only 15 states are currently doing so. Two more, Michigan and Colorado, are expected to soon begin screening.

Nami Sumida/BioPharma Dive

"When states puts these programs in place, we do see much higher utilization in newborns of therapies, at rates that are sometimes three times as high as states that don't have newborn screening," said Lennon.

While Zolgensma's early market success suggests a one-time gene therapy can be commercialized, Novartis has also benefited from both SMA's rarity and the cost of existing treatment in winning over insurers.

In the U.S., only about 500 babies are born each year with SMA, 60% of whom are diagnosed with the most severe form, called Type 1. That's a much smaller population than other rare genetic conditions like hemophilia and sickle-cell disease, the target of other gene therapy programs in Phase 3 trials.

And Spinraza costs $750,000 for the first year of treatment, and $375,000 thereafter potentially making $2.1 million more palatable if Zolgensma truly is a one-and-done treatment.

Novartis' achievement, however, is marred by a damaging data scandal that's put both the drug and company under scrutiny.

When submitting the application for FDA approval, Novartis included testing data from preclinical mice studies that was manipulated, it turned out, by (or at the behest of) top AveXis officials. Novartis management knew of the falsified data in March, but filed Zolgensma anyway.

In a show of its displeasure and a signal to other developers, the FDA warned Novartis of potential civil or criminal penalties, pending an investigation that has yet to complete. Zolgensma, however, could stay on the market, and the FDA affirmed the therapy's positive benefit-risk profile.

Novartis ousted the AveXis executives it says were to blame, and has sped up the integration of the biotech into its own quality control organization.

"Quality, in the grandest scheme, is always a journey," said AveXis' Lennon. "While we've accelerated this, we don't see an end to our commitment to quality improvement."

Months after addressing that controversy, the FDA placed partially on hold a study of Zolgensma due to concerns over toxicity seen in animals, possibly tied to how the therapeutic gene was delivered. The regulatory action revived previously raised warnings about the risk of inflammation in certain neurons exposed to a virally delivered gene.

In both respects, Zolgensma could be a case study in the risks as well as rewards involved for large pharmaceutical companies looking to get into gene therapy.

"This is one of the first gene therapies," said Gal. "We don't have a lot of experience infusing viral particles into the brain."

The rest is here:
Drug Launch of the Year: Zolgensma - BioPharma Dive

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Biocair Exhibits at Cell Therapy Manufacturing and Gene Therapy Congress 2019 – BioSpace

Wednesday, December 4th, 2019

December 2019

Cambridge, UK: Global courier for cell and gene therapy, Biocair, will exhibit at Cell Therapy Manufacturing and Gene Therapy Congress between 3-6 December 2019. Taking place in Amsterdam, Cell Therapy Manufacturing and Gene Therapy Congress brings together key players from the industry to discuss the critical issues facing the development, scale-up and manufacture of cell-based and gene therapies.

Speaking about the logistics surrounding cell and gene therapy, Gert de Gier, Cell and Gene Business Development Director at Biocair, comments that with cell and gene therapies moving into the clinical trial phase for a wide variety of diseases, the results of these studies and their potential impact to patients are invaluable. Therefore the transportation of the materials and need for a secure supply chain is absolutely critical.

Patient samples can be bio-hazardous, time critical and have an extremely short shelf-life. Biocair has developed a highly customised project-based approach to the challenging requirements of the clinical and commercial cell and gene markets, providing chain of custody provision across every touchpoint, validated and pre-conditioned temperature-controlled packaging for all temperature ranges, total transparency in supply chain design and unrivalled short transit times (12 36hrs).

Biocairs specialist services are precisely documented in standard operating procedures and are regularly audited by its 12, 500 life science customers across the world. For more information, contact your local Biocair office or contact enquiries@biocair.com.

Notes to editors

Biocair is a specialist courier with over 30 years of dedicated experience in the pharmaceutical, biotechnology and life science sectors. The company has built up a unique, client-centric approach by employing scientists in front-line logistics positions and assembling a team of best-in-class industry experts in quality, cold chain and regulatory compliance amongst others. Biocair focuses on providing the most comprehensive service options available whilst delivering flexible, tailored, cost effective logistics solutions to all clients.

Biocair operates across Europe, Africa, Asia and the Americas.

Continued here:
Biocair Exhibits at Cell Therapy Manufacturing and Gene Therapy Congress 2019 - BioSpace

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Astellas joins gene therapy race with $3B Audentes buy – BioPharma Dive

Wednesday, December 4th, 2019

Dive Brief:

Astellas is paying up to join the gene therapy race.

At $60 per share, the Japanese pharma's takeover offer is more than double the $28.61 at which shares in Audentes traded at Monday's market close a multiple on par with Roche's bid for Spark but higher than the premium Novartis offered for AveXis, and Biogen for Nightstar Therapeutics.

Impressive clinical results, paired with the prospect of speedy development, have made gene therapies an attractive investment for larger drugmakers. And approvals for AveXis' Zolgensma (onasemonogene abeparvovec) and Spark's Luxturna (voretigene neparvovec) on data from only a few dozen patients indicates a FDA willing to clear new therapies on less information than usual, providing the benefit is clear enough.

*Over closing price the day preceding deal announcement SOURCE: Companies

For Astellas, its interest in Audentes appears linked as much to the options buying the biotech opens up as it is to the company's lead drug candidate.

Importantly, Audentes owns a sizable manufacturing plant capable of supporting clinical and commercial gene therapy production. Manufacturing the inactivated viruses used to deliver the one-time treatments has proved a bottleneck for the field, so acquiring capabilities could give Astellas a leg up in the future.

And Audentes this year secured a collaboration with Nationwide Children's Hospital in Ohio a hotbed for gene therapy science that gave the biotech several programs aimed at Duchenne muscular dystrophy and myotonic dystrophy type 1.

That work remains preclinical, however. Nearer term, Audentes offers Astellas a gene therapy candidate that could be submitted to regulators for approval as early as the middle of next year. Called AT132, the therapy is designed to treat x-linked myotubular myopathy, a rare and usually fatal neuromuscular disorder affecting infants and young boys.

Mutations in the MTM1 gene result in missing or dysfunctional myotubularin, a protein essential to skeletal muscle cell development. Without it, affected infants are extremely weak and often require ventilator assistance to breathe. Half of those diagnosed die in the first 18 months of life, according to estimates cited by Audentes.

AT132 proposes a genetic fix for the disease, delivering a functional copy of the MTM1 gene via a type of viral vector known as AAV8. Results from a Phase 1/2 study showed infants treated with the therapy were able to sit, stand and even walk. Importantly, the ten treated patients were able to significantly reduce their dependence on ventilatory support compared to two study participants who were used as a control.

Neuromuscular conditions like XLMTM and DMD require much higher doses of gene therapy than diseases of the eye, brain or liver, making Audentes manufacturing capacity a vital asset.

Audentes is also developing a therapy for Pompe disease, which, like DMD, is targeted by several other companies. A clinical-stage program in the ultra rare condition called Crigler-Najjar syndrome, however, appears to have been put on hold this year by Audentes.

Astellas said it will house Audentes in an independent subsidiary, and plans for the acquisition to form a foundation for a new research focus centered on genetic regulation. A statement on the deal seemed to hint more gene therapy partnerships could be forthcoming, or at least made possible by buying Audentes.

At $3 billion, Astellas' acquisition is the largest buyout of a gene therapy developer since Roche-Spark, as well as the largest in a run of deals brokered by the pharma, which is perhaps best known for its development of the prostate cancer drug Xtandi (enzalutamide).

Astellas' interest in Audenetes could ease some worries that would-be acquirers might put off dealmaking in gene therapy until the Federal Trade Commission reaches a decision in its review of Roche-Spark. Repeated delays have raised eyebrows among investors and analysts, who largely expected the deal to close rapidly.

"In light of the pending [Spark-Roche] transaction, some investors may find Astellas-[Audentes] as another potentially drawn-out and uncertain tie-up,' wrote Joseph Schwartz, an analyst at SVB Leerink, in a note to clients.

"However, our review of Astellas' product portfolio and R&D pipeline suggests minimal overlap with [Audentes]'s focus on neuromuscular diseases."

Schwartz and colleagues were able to identify only two drug candidates that could share a target with Audentes', potentially making a takeover from the much larger Astellas easier to clear.

Excerpt from:
Astellas joins gene therapy race with $3B Audentes buy - BioPharma Dive

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