Category Archives: Gene therapy

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them or one of the couple of Americans who arrived later got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

We dont have too much time, Andre Kalil, the trials lead investigator, told Endpoints News. Everythings moving really fast.

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'The headlines are the headlines, but': BioMarin talks up potential savings as hemophilia gene therapy launch looms - Endpoints News

Alia (2nd from left) and her father (left) with Dr Arif Khan (3rd from left), paediatric ophthalmologist and ocular geneticist at Cleveland Clinic Abu Dhabi, and other hospital doctors at a press conference in Abu Dhabi. Alia and her sister, Hessa, were the first to receive gene therapy in the UAE. Picture by Samihah Zaman, Image Credit:

Abu Dhabi: Two Emirati sisters from Abu Dhabi have become the first to receive gene therapy in the UAE, it was announced in the capital today.

The therapy was conducted with the aim of saving the vision of Alia, 13, and Hessa, 11, who suffered from retinal damage due to a defective gene.

The vision-saving surgery was performed at Cleveland Clinic Abu Dhabi in January (2020), and doctors said at a press conference that they are hopeful it will slowly improve the vision of both patients.

"The prospect of slowly losing one's vision from an untreatable condition is traumatic for both children and their parents. This genetic therapy means we can now replace the faulty gene in the eye, saving and even improving the vision of an individual who would otherwise have eventual irreversible blindness," said Dr Arif Khan, paediatric ophthalmologist and ocular geneticist at the hospital.

Only centre in region with procedure

Cleveland Clinic Abu Dhabi is only one of 10 centres worldwide, and the only one in the region, that is qualified to offer this procedure - the first gene therapy that was approved by the Food and Drug Administration in the United States, doctors said. But its availability opens the doors for more gene therapies to be offered to treat conditions as varied as diabetes and spinal muscular atrophy, they added.

Weve travelled abroad multiple times to find some treatment for our daughters, and this procedure has been a godsend. It has only been a month since the therapy however, and we are hoping for much more improvement to their vision over time, Fatima, the girls mother, told Gulf News.

The procedure

Alia and Hessa were born with RPE65-related retinal dystrophy, a genetic dystrophy in which the RPE65 protein is lacking because a child inherits two copies of the defective RPE65 from both parents. It is a recessive disease and therefore rare, and known to affect one in 200,000 people worldwide. But the condition is also more common in the region because of the limited genetic pool and cultural preferences for consanguineous marriages.

Patients with the condition face gradual damage to their retinas, the photosensitive layer at the back of the eye, and could eventually end up with irreversible blindness.

The gene therapy to treat the condition uses a vector - a bioengineered non-pathogenic virus to deliver normal copies of the RPE65 gene to the eye.

The procedure takes only about an hour, but it is very delicate. The retina can be considered an offshoot of the brain because its cells are very thin, like brain tissue. We use advanced microscopes to create openings in the white of the eye, then use a specialized canula to reach under the retina and inject the vector into the specific area, which can be as small as one-tenth of a millimeter, explained Dr Emad Abboud, chief of the department of the posterior segment at the hospitals Eye Institute.

Post-surgical recovery takes only about a week, but outcomes take a while to become obvious.

As Dr Khan explained, the main benefits for patients receiving the therapy is an improvement of vision in low-light conditions, and an improvement of the visual field.

Vision is a complex sense, involving photosensitivity, visual field, visual acuity, colour sensitivity, motion sensitivity, and the ability to navigate. But [at the least], this therapy prevents the progressive deterioration of the retina, he explained.

What the family said

My handwriting has definitely gotten better, Alia told Gulf News when asked about how the therapy has helped.

I cant tell what else will get better but I already feel that this has been life-changing, she added.

According to Fatima, she noticed her daughters had visual impairment when they were as young as two months old.

The doctor pointed out to me that they were not focusing or making eye contact. So we were aware of the condition. It definitely made life very challenging, especially as their development was delayed, the mother explained.

Alia and Hessa both crawled and walked late, and have needed assistance getting around. While they kept pace with their peers at school, it took Hercualean efforts from both girls, their parents, shadow teachers and at-home tutors, and required the use of Braille and visual aid resources.

Fatima and her husband, Mubarak, kept looking for treatments for both girls, travelling to Germany, India and Spain.

In 2016, we visited the Cleveland Clinic Abu Dhabi, and it was then that we heard of this revolutionary treatment. It was still not approved by the FDA, but we kept dreaming, she said.

It has been a month since the surgery and the doctors said they are more photosensitive now and that this is a good sign. Of course, as a mother, I would love for them to eventually have complete vision, Fatima added.

What is the therapy?

The UAE Ministry of Health and Prevention (MoHAP) approved the gene therapy for RPE65-related retinal dystrophy, Luxturna, in June 2019.

In a statement released at the time, the MoHAP said its registration of Luxturna was only the third global registration of the drug at the health authority level, and was aimed at paving the way for future gene-based therapies for complex conditions.

The United States Food and Drug Administration only approved Luxturna, developed by gene therapy developer, Spark Therapeutics, and the Childrens Hospital of Philadelphia, in late 2017.

The gene therapy to treat the condition uses a vector - a bioengineered non-pathogenic virus to deliver normal copies of the RPE65 gene to the eye with the defective gene.

While the Cleveland Clinic Abu Dhabi did not provide the cost of treatment, international media reports put it at $425,000 (Dh1.56 million) per eye.

Benefits of gene therapy

Dr Khan said at the press conference that us familiar with a few more families with the RPE65-related retinal disorder.

Over time, gene therapy could be used to treat many conditions. The eye is uniquely suited for these therapies because it is a self-contained organ. But at least five other genetic therapies are in development, some at the human trial level, he explained.

Consider gene delivery to be like providing a protein factory to the body. So for instance, some diabetic patients require regular insulin injections. If a gene could be engineered to make insulin, and it could be delivered to the patient, he wouldnt need these regular injections, Dr Khan added.

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UAE's first gene therapy improves eyesight of two Emirati sisters - Gulf News

The IPO market is feeling the effects of the coronavirus outbreak with a surge in the VIX volatility index weighing on IPO activity. Since 2015, a week in which VIX volatility has surpassed 35 has been followed by a week averaging two US IPO pricings. Our observational trend continues through 2020, as one biotech entered the public market this past week.  Six IPOs and three SPACs submitted initial filings with the SEC.

Passage Bio (PASG), a preclinical biotech developing gene therapies, priced at the high end of the range to raise $216 million at an $840 million market cap. The deal raised 72% more in proceeds than Passage Bio originally filed for. The company is furthering the research from UPenn’s Gene Therapy Program, which is headed by co-founder James Wilson. Bolstered by the recent performances of other large early stage biotech IPOs, Passage Bio finished up 23%, another sign that biotechs are pushing back against the effects of the coronavirus. Chinese medical information platform Zhongchao (ZCMD) began trading on Monday after raising $12 million in an IPO on Friday 2/21. The company was flat after its first day on the Nasdaq and is currently down 2%.

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IPO Weekly Recap: Yes, the IPO Market has caught the coronavirus - Nasdaq

Early proponents of genome sequencing made misleading predictions about its potential in medicine.

An emergency room physician, initially unable to diagnose a disoriented patient, finds on the patient a wallet-sized card providing access to his genome, or all his DNA. The physician quickly searches the genome, diagnoses the problem and sends the patient off for a gene-therapy cure. Thats what a Pulitzer prize-winning journalist imagined 2020 would look like when she reported on the Human Genome Project back in 1996.

The Human Genome Project was an international scientific collaboration that successfully mapped, sequenced and made publicly available the genetic content of human chromosomes or all human DNA. Taking place between 1990 and 2003, the project caused many to speculate about the future of medicine. In 1996, Walter Gilbert, a Nobel laureate, said, The results of the Human Genome Project will produce a tremendous shift in the way we can do medicine and attack problems of human disease. In 2000, Francis Collins, then head of the HGP at the National Institutes of Health, predicted, Perhaps in another 15 or 20 years, you will see a complete transformation in therapeutic medicine. The same year, President Bill Clinton stated the Human Genome Project would revolutionize the diagnosis, prevention, and treatment of most, if not all, human diseases.

It is now 2020 and no one carries a genome card. Physicians typically do not examine your DNA to diagnose or treat you. Why not? As I explain in a recent article in the Journal of Neurogenetics, the causes of common debilitating diseases are complex, so they typically are not amenable to simple genetic treatments, despite the hope and hype to the contrary.

The idea that a single gene can cause common diseases has been around for several decades. In the late 1980s and early 1990s, high-profile scientific journals, including Nature and JAMA, announced single-gene causation of bipolar disorder, schizophrenia, and alcoholism, among other conditions and behaviors. These articles drew massive attention in the popular media, but were soon retracted or failed attempts at replication. These reevaluations completely undermined the initial conclusions, which often had relied on misguided statistical tests. Biologists were generally aware of these developments, though the follow-up studies received little attention in popular media.

There are indeed individual gene mutations that cause devastating disorders, such as Huntingtons disease. But most common debilitating diseases are not caused by a mutation of a single gene. This is because people who have a debilitating genetic disease, on average, do not survive long enough to have numerous healthy children. In other words, there is strong evolutionary pressure against such mutations. Huntingtons disease is an exception that endures because it typically does not produce symptoms until a patient is beyond their reproductive years. Although new mutations for many other disabling conditions occur by chance, they dont become frequent in the population.

Instead, most common debilitating diseases are caused by combinations of mutations in many genes, each having a very small effect. They interact with one another and with environmental factors, modifying the production of proteins from genes. The many kinds of microbes that live within the human body can play a role, too.

A silver bullet genetic fix is still elusive for most diseases.

Since common serious diseases are rarely caused by single-gene mutations, they cannot be cured by replacing the mutated gene with a normal copy, the premise for gene therapy. Gene therapy has gradually progressed in research along a very bumpy path, which has included accidentally causing leukemia and at least one death, but doctors recently have been successful treating some rare diseases in which a single-gene mutation has had a large effect. Gene therapy for rare single-gene disorders is likely to succeed, but must be tailored to each individual condition. The enormous cost and the relatively small number of patients who can be helped by such a treatment may create insurmountable financial barriers in these cases. For many diseases, gene therapy may never be useful.

The Human Genome Project has had an enormous impact on almost every field of biological research, by spurring technical advances that facilitate fast, precise and relatively inexpensive sequencing and manipulation of DNA. But these advances in research methods have not led to dramatic improvements in treatment of common debilitating diseases.

Although you cannot bring your genome card to your next doctors appointment, perhaps you can bring a more nuanced understanding of the relationship between genes and disease. A more accurate understanding of disease causation may insulate patients against unrealistic stories and false promises.

Written by Ari Berkowitz, Presidential Professor of Biology; Director, Cellular & Behavioral Neurobiology Graduate Program, at the University of Oklahoma.

Originally published on The Conversation.

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Sequencing the Human Genome Was Supposed to Revolutionize Treatment of Disease Heres Why It Failed - SciTechDaily

Alongside resolutions, the new year tends to come with biopharma acquisitions. The first two months of 2018 brought four deals worth $1 billion or more. The same was true in 2019, which barely got off the ground before a mammoth bid for Celgene was announced.

The early days of 2020, by contrast, have notched just one billion-dollar deal.

Analysts and consultants are mostly waving off this period of lighter activity. "We don't think a lack of deals over a couple of months is really anything notable," said Phil Nadeau of Cowen & Co. "It's probably just a bit of a dry period for no reason at all."

And yet, the slow start could seem oddly timed. It doesn't fit with the early and splashy dealmaking seen in recent years, nor does it align with some investment bank expectations that drug companies might try to ink deals sooner in the year to avoid possible disruptions from the U.S. presidential election.

But perhaps most glaring: the downswing comes as many large biotech and pharmaceutical companies are seemingly in need of M&A.

Two of the biggest biotechs, Gilead and Biogen, are under pressure to acquire because they haven't convinced investors that their experimental drugs can make up for problems on the commercial side of the business.

Among the pharmas, Sanofi, Eli Lilly and GlaxoSmithKline are trying to play catch up in the industry's hottest research area, oncology, and have shown a willingness to speed up that process through buyouts. Swiss drug giants Roche and Novartis, meanwhile, paid hefty premiums for footholds in the rapidly evolving gene therapy field.

Even Vertex and Merck & Co., which are considered to be in strong financial and competitive positions, turned to M&A several times last year to seed themselves for future growth.

"It's clear that, given the growth rates among the large companies in the industry, they have to keep filling their pipeline through acquisition, partnership or in-licensing," Nadeau said.

*Transaction value classified by acquiring company type. 2019 data through Nov. 30 of last year

Image Source: Jacob Bell / BioPharma Dive, data from EY

With buyers still on the lookout, industry followers predict 2020 will deliver a healthy level of M&A in spite of the early drought.

What they don't foresee, however, is this year being defined by a mega-deal like the last two were. Instead, bolt-on deals in the $5 billion to $10 billion range will be a "sweet spot" for large pharma acquirers, according to PwC.

Companies hold ample resources to support such deals too, as EY estimates the life sciences industry has more than $1 trillion at its disposal for deals. Notably, that's after a record-setting 2019 in which the industry spent north of $350 billion on M&A.

"There was so much moving and shaking that happened last year. And for me, I just feel like the underlying activity is still there. It just hasn't reached the surface yet," said Glenn Hunzinger, U.S. Pharmaceutical and Life Sciences Deals Leader at PwC.

If and when the activity surfaces, it's likely to target rare disease treatments, cell and gene therapy, and cancer drugs, with the latter proving particularly attractive. A recent Jefferies analysis found two-thirds of small- to mid-sized biotechs that got a cancer drug approved since 2010 were later acquired.

Many potential buyers, though, are in more comfortable financial positions now than they were even a couple years ago, and may not be as open to a less-than-perfect fit when considering a deal.

"Growth has returned to the large cap space, partially through acquisitions, and free cash flow across a number of large-cap companies has never been higher," said Kennen MacKay, an analyst at RBC Capital Markets. These companies now "have the luxury of time on their side, and they can be a little bit more selective."

That's especially true with regard to pricing, according to MacKay, who said the current lull in M&A may have something to do with a rebound in biotech stocks. The XBI, an exchange traded fund of about 130 biotechs, is up about 20% since hitting a relative low point in October. The rise also comes after multiple years in which cancer and gene therapy-focused biotechs have sold for high double-digit and sometimes triple-digit premiums.

"I think there's real reluctance to buy when small-, mid-cap stock prices are really running up," MacKay said.

Nadeau from Cowen, conversely, sees valuation as less of a barrier.

"Occasionally you do hear business development executives or former management teams talk about how valuation has gotten rich and they don't see attractive targets at a right price," he said. "But on the other hand, when you get these business development folks in private, offline, they'll admit that valuations are very flexible too. And if there's a strategic deal that a company wants to do, they can make any valuation work."

On his end, Nadeau isn't reading too much into 2020's slow M&A start. He guesses it's either due to happenstance, or because some of the deals that might have been inked this quarter were tied up early. Between October and December there were at least nine biopharma acquisitions, including five valued at more than $2 billion.

Peter Behner, global transactions leader for EY's Health Science & Wellness business, also doesn't envision the quiet period will last much longer. Whether in pharmaceuticals or other industries, companies don't just go dormant after a productive year of dealmaking, he said.

"I don't have a great explanation," Behner added. "At the same time, I don't have much doubt that the year should be a solid year."

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Pharmacquired: Where are all the deals? - BioPharma Dive

~ Up to Two Years of Follow-up in Large Transgenic Huntingtons Disease Model Demonstrates Stable mHTT Protein Lowering ~

~ Novel Preclinical Data Demonstrates Successful Lowering of Pathogenic Exon 1 Fragment ~

~ Additional Data Demonstrates the Potential of MRS as Imaging Biomarker for Huntingtons Disease Gene-Therapy Studies ~

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Feb. 27, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the presentation of multiple new preclinical data on AMT-130, its investigational AAV gene therapy for the treatment of Huntingtons disease (HD), at the 15th Annual CHDI Huntingtons disease Therapeutics Conference in Palm Springs, California.

Our data presentations at CHDI illustrate the increasing potential of AMT-130 to target the highly toxic exon 1 protein fragment, achieve broad vector biodistribution across several animal species and show meaningful activity using the presence of extracellular vesicles as a potential biomarker, stated Sander van Deventer, M.D., Ph.D., executive vice president, research & product development of uniQure. In addition, we highlight the use of magnetic resonance spectroscopy as a potentially important imaging biomarker to measure the restoration of target tissue. Collectively, these findings represent a robust package of new preclinical data to better inform how researchers and clinicians pursue a much-needed treatment for this devastating disease.

Four scientific abstracts submitted by uniQure researchers were accepted for presentation at the conference, of which one is an oral presentation to be featured today. Important findings across several preclinical studies presented at the conference include the following:

Translatable Biomarkers in Gene Therapy for Huntington Disease: Learnings from Pre-clinical Studies

Secreted Therapeutics: Monitoring Durability of microRNA-based Gene Therapies in Huntingtons disease

Lowering the Pathogenic Exon 1 HTT Fragment by AAV5-miHTT Gene Therapy

Exploring the Effects of Intrastriatal AAV5-miHTT Lowering Therapy on Neuronal Function, MRS Signal and Mutant Huntingtin Levels in the Q175FDN Mouse Model of Huntingtons disease

The uniQure data presentations featured at CHDI are available on the investor page of the Companys website, http://www.uniQure.com

About Huntingtons DiseaseHuntingtons disease is a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioral abnormalities and cognitive decline, resulting in complete physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene, that leads to the production and aggregation of abnormal protein in the brain. Despite the clear etiology of Huntingtons disease, there are no therapies to delay the onset or to slow the diseases progression.

About uniQure uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. http://www.uniQure.com

uniQure Forward-Looking StatementsThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, whether AMT-130 will target the highly toxic exon 1 protein fragment, achieve broad vector biodistribution across several animal species or show meaningful activity using the presence of extracellular vesicles as a potential biomarker, and whether magnetic resonance spectroscopy will be an important imaging biomarker to measure the restoration of target tissue. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaborators clinical development activities, clinical results, collaboration arrangements, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQures Quarterly Report on Form 10-Q filed on October 28, 2019. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

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uniQure Presents Multiple New Preclinical Data on AMT-130 at the CHDI's 15th Annual Huntington's Disease Therapeutics Conference - GlobeNewswire

Supplies of Zolgensma, the gene therapy approved last year for spinal muscular atrophy, are tight.

Novartis, which sells the one-time treatment, can currently make about 700 to 800 doses a year at its manufacturing plant in Libertyville, Illinois. That's enough to cover the infants in the U.S. currently eligible to receive Zolgensma but leaves little room for treating a wider group of patients, which the Swiss drugmaker aims to do.

On Thursday, executives from AveXis, the Novartis unit that developed Zolgensma, opened a new facility in Durham, North Carolina, that the company views as a critical cog in its plans to expand supply of the gene therapy.

Initial production will begin this spring. But until the Food and Drug Administration licenses the plant, Novartis won't be able to use product made there for commercial sale. The company expects to gain approval next year.

Between now and then, Novartis also hopes to secure regulatory OKs for manufacturing Zolgensma at a site in Longmont, Colorado, bought last year, and through the contract manufacturer Catalent.

"There is a short-term challenge over the next six to nine months to make sure that we can manage the supplies that are out there," said David Lennon, president of Novartis' AveXis unit, in an interview.

"We feel comfortable where we are, but we'd love to have these other sites onboard to make sure we're really robust and don't face any risks of shutdowns or anything that could impact supply."

Limited supply has also kept Novartis from widening a program set up to make the gene therapy available free of charge to patients in countries where it's not yet approved. The "expanded access" scheme, which was launched in January, randomly allocates doses of Zolgensma for participating patients under the age of two with genetically confirmed spinal muscular atrophy.

This year, Novartis plans to distribute 100 doses through the lottery, which has been criticized as putting a child's life to chance.

"We obviously know that not everyone is happy with the program," said Lennon. "We're still considering what we might do, but we're open to making changes if it makes sense for the community and to meet the goals of the program."

Lennon said he hoped to expand the program as more manufacturing capacity for Zolgensma becomes available.

Novartis has committed upwards of $200 million to building out the site in Durham, which will employ about 400 staff by the end of the year.

Spanning 170,000 square feet, the facility will be used for both commercial Zolgensma manufacturing as well as to support clinical production of other gene therapies that Novartis is developing.

"This is as much an investment in the short term in building out our supply for Zolgensma, as it is for the long term to have the flexibility to deliver on a robust pipeline," said Lennon.

Novartis currently expects to treat about 100 infants every three months in the U.S. under Zolgensma's current label. But it's also working to expand the therapy's approval to treat older children over two using a spinal injection rather than an infusion.

That patient population is significantly larger and will test Novartis' ability to produce a steady supply of the drug, although the FDA has placed a partial "hold" on the study testing the new dosing.

Novartis' launch of Zolgensma is under significant scrutiny, both because of the $2.1 million price tag the drugmaker put on the therapy and due to a data manipulation scandal that engulfed the company last year.

Despite the high cost, insurers have largely covered treatment, leading to strong sales of Zolgensma in its first three quarters on the market.

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With supply tight, Novartis readies gene therapy plant for production - BioPharma Dive

February 20, 2020 -The Institute for Gene Therapies (IGT), a new advocacy foundation focused on policy issues surrounding gene therapies, recently launched with the goal of modernizing US regulatory and reimbursement framework for gene therapies.

According to IGT, the US health system currently spends about 85 cents of every healthcare dollar managing the symptoms of chronic diseases over a patients lifetime. With gene therapies, providers could alter patients non-functioning genes or replace absent ones, leading to long-lasting effects and potentially reshaping the way thousands of diseases are treated.

While traditional biologic and pharmaceutical medicines help manage the symptoms of disease over time, gene therapies target the cause of disease at the DNA level, creating lasting changes in the body. Some gene therapies are also designed to be one-time treatments that offer lifelong benefits.

IGT will work to educate stakeholders across the healthcare system about the potential for gene therapies to treat and cure common and rare chronic diseases, and advocate for policies that help ensure patients who need gene therapies can benefit from them.

Existing regulatory and reimbursement structures were established and adjusted over time to support pharmaceutical and biologic medicines, IGT noted, and need refining to accommodate the potential of gene therapies.

READ MORE: Data Mining Techniques Could Improve Cancer Gene Therapies

Many crippling conditions like Charcot-Marie-Tooth (CMT), which I was diagnosed with before the age of two take hold at a very young age, cut lives far too short or cause ongoing daily suffering, said Susan Ruediger, CEO of the CMT Research Foundation (CMTRF) and member of the IGT Patient Advocacy Advisory Council.

Like so many diseases, CMT currently has no cure. I am proud to stand with other leading patient advocates, members of the research community and companies that are developing gene therapies to help ensure patients can fully realize the benefits of these giant leaps toward treatments and cures.

The FDA has already approved four gene therapy products, and researchers are studying hundreds more in clinical trials for rare and common diseases, including many types of cancer, neuromuscular diseases, blood disorders and infectious diseases, and other conditions.

The growth of innovative research and product development in the field of gene therapy is exciting to us as physicians, scientists and regulators, FDA Commissioner Stephen M. Hahn, MD, said in a statement.

We understand and appreciate the tremendous impact that gene therapies can have on patients by potentially reversing the debilitating trajectory of diseases. These therapies, once only conceptual, are rapidly becoming a therapeutic reality for an increasing number of patients with a wide range of diseases, including rare genetic disorders and autoimmune diseases.

READ MORE: Genetic Sequencing Study Identifies 102 Genes Associated with Autism

The FDA has also released six final guidance documents on gene therapy manufacturing and clinical development of products. These documents incorporate input from stakeholders across the healthcare industry and make important strides in designing a modern structure for gene therapy development.

As the regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs, said Peter Marks, MD, PhD, director of the FDAs Center for Biologics Evaluation and Research.

Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review; including how these products work, how to administer them safely, and whether they will continue to achieve a therapeutic effect in the body without causing adverse side effects over a long period of time.

IGT will being together experts across the healthcare community, including corporate leaders, patient advocacy groups, and academic and scientific stakeholders, to ensure health policies reflect the latest advancements in gene therapies. The institute will also work to remove barriers that limit patient access to these therapies and promote sustainable, long-term solutions.

Experts will learn more about the value gene therapies bring to patients, their families, and the healthcare system as a whole. IGT will help advance chronic disease treatments and get to the root cause of some of the most debilitating, expensive conditions that affect patients throughout their lives.

READ MORE: 77% of Americans Are Optimistic About Genetics Research, Potential

The incredible scientific advancements in this space present unique opportunities to directly improve and save the lives of patients suffering from debilitating diseases, said IGT Chairman, and former Congressman Erik Paulsen.

This is not some far-off future patients are already benefiting from the first FDA-approved gene therapies. But we need policy to move faster toward this new reality where we can treat the causes of many diseases. The Institute for Gene Therapies and our members believe unique regulatory and reimbursement structures need to be established, novel development pathways need to be embraced and new value-based arrangements need to be tested.

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New Gene Therapies Institute Aims to Address Regulatory Barriers - HealthITAnalytics.com

Audentes Therapeutics, an Astellas company, announced it is building a gene therapy manufacturing plant in Sanford, North Carolina.

The company is investing $109 million in a new 135,000-square-feet facility, with the initial phase to take place over about 18 months. It is planned to go into operation in 2021. It is expected to create more than 200 new jobs in Lee County, North Carolina. Hiring is expected to start this year.

Our investment in large-scale manufacturing has always been a cornerstone of our strategy to develop and ultimately deliver our important genetic medicines to patients as rapidly as possible, said Natalie Holles, president and chief executive officer of Audentes. This new facility in Sanford will support the next phase of our growth as we establish a robust, global supply chain and expand our therapeutic and geographic scope as a part of the Astellas group of companies. We are excited to join the vibrant biopharmaceutical research and manufacturing community that the state of North Carolina has established.

Audentes is headquartered in San Francisco and focuses on gene therapy. It was acquired by Tokyo-based Astellas Pharma in January.

No specifics were given about what the site will manufacture. In October 2019, Audentes announced positive data from ASPIRO, the clinical trial of AT132 in patients with X-Linked Myotubular Myopathy (XLMTM). AT132 is an AAV8 vector that contains a functional copy of the MTM1 gene. XLMTM is a serious, life-threatening, rare neuromuscular disease marked by extreme muscle weakness, respiratory failure and early death.

The company indicates it hopes to submit a Biologics License Application (BLA) for AT132 to the U.S. Food and Drug Administration (FDA) later this year.

An announcement ceremony was held in the industrial shell building the company is buying in the Central Carolina Enterprise Park. It is about 45 miles southwest of Raleigh.

Gov. Roy Cooper stated, With our powerhouse research centers and highly skilled workforce, biotech pioneers recognize North Carolinas role as a leader in the life sciences. Lee County is a perfect fit for Audentes as they seek to become a global leader in genetic medicines.

The employees at the new factory are expected to earn an average salary of $83,900, which is a little over twice the Lee County average of $41,800. If Audentes hits hiring milestones, it will qualify for a state Job Development Investment Grant worth up to $3.7 million.

The county and the city of Sanford are also offering $5.7 million incentives, which includes almost $400,000 in training support from the North Carolina Community College System.

Audentes chose the location over California, Massachusetts and Colorado.

In every interaction, I was impressed with Audentes patient-centric approach to developing their AAV-based gene therapy to transform the lives of affected patients and families, said Laura Rowley, NCBiotechs director of life science economic development. She led the Centers outreach activity with Audentes. Their decision to grow in North Carolina reflects the Research Triangle regions specialized training capabilities and strengths in gene therapy and biomanufacturing. The passion and focus of the Audentes team makes me confident that they will be an outstanding addition to North Carolinas gene therapy community.

Audentes is acting as the Center of Excellence for Astellas newly founded Genetic Regulation Primary Focus.

Audentes Therapeutics is joining one of the nations top life science clusters, said Anthony M. Copeland, North Carolinas Commerce Secretary. North Carolina has the largest biomanufacturing workforce in the nation and a growing concentration of gene therapy scientists, researchers and workers.

The site of the new plant is quite close to Pfizers new gene therapy campus, which is under construction. That $600 million research and manufacturing facility has a 230-acre campus in Sanford and will employ 340 people.

See the article here:
Audentes to Build $109 Million Gene Therapy Factory in North Carolina - BioSpace

Dive Brief:

Bluebird's commercial operations are just getting off the ground. In its latest earnings report, the Cambridge, Massachusetts-based biotech detailed how it has inked agreements with health insurers in Germany that should provide coverage for LentiGlobin, which is sold under the brand name Zyntegloin Europe, for up to 50% of eligible beta-thalassemia patients. Bluebird expects the first commercial patient to be treated before July.

Across the Atlantic, U.S. patients are looking at a longer timeline before LentiGlobin becomes available. Stifel analysts wrote in a note to clients that they don't foresee any stateside patients receiving the therapy commercially in 2020 "given what we anticipate will be a complicated negotiation process with payors."

Analysts at Raymond James, meanwhile, downgraded Bluebird to a "Market Perform" rating, writing that "execution issues on the regulatory, clinical and manufacturing side outweigh our support for the innovative drug products."

As Bluebird works through the latest delay in beta-thalassemia,it will also be preparing for an expanded research program in sickle cell. The company already intended to kick off a late-stage study in sickle cell patients with a history of vaso-occlusive crises in the first half of 2020. With Tuesday's earnings presentation, though, came plans to initiate a second late-stage study sometime this year, which will evaluate LentiGlobin's effects in about 18 children with sickle cell and elevated stroke risk.

A sickle cell approval, though a ways off, could boost Bluebird's bottom line. Beta-thalassemiais rarer in U.S. than other parts of the world, and certainly less common than sickle cell. According to estimates cited by the National Organization of Rare Disorders, roughly 3,300 U.S. patients have beta-thalassemiaversus the 100,000 who have sickle cell.

An expanded program could provide more evidence of LentiGlobin's benefit in this larger patient pool.Yet the updates don't seem to have alleviated investor concerns. Bluebird shares were down nearly 10% in late Wednesday morning, trading around $80 apiece.

"LentiGlobin in Sickle Cell Disease remains a bright spot, in our view, but with [late-stage studies] expected to get underway this year, we don't expect investor sentiment to change anytime soon," Stifel analysts wrote.

The investment bank models Zyntelgo bringing in $12 million worth of revenue in 2020 from the beta-thalassemia indication, increasing to $53 million in 2021 and $390 million by 2030. Conversely, it models $48 million in 2022 from the sickle cell indication, increasing to almost $2 billion by 2030.

Original post:
Bluebird's gene therapy hits another delay, this time in the US - BioPharma Dive