StemCell Therapy

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

Originally posted here:
Orgenesis and Theracell to Launch Point of Care Cell and Gene Therapy Centers within HYGEIA Group's Hospital Network in Greece - GlobeNewswire

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

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

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

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

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

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

The optimistic outlook is seen in an adorable 10-year-old girl named Jojo, who became the first patient to receive a gene therapy treatment, called AXO-AAV-GM1, during a human clinical trial this summer at the National Institutes of Health in Maryland. Auburn's College of Veterinary Medicine and the University of Massachusetts Medical School developed the treatment that has moved from helping cats with GM1 to hopefully helping children.

"Jojo is doing well and has experienced no major complications," said Dr. Doug Martin, professor in the Department of Anatomy, Physiology and Pharmacology in Auburn's veterinary college and the Scott-Ritchey Research Center. "Seeing all of the effort come together to help patientswho haveno treatment options today gives us great hope."

Auburn scientists for several decades have researched treatments to improve and extend the lives of cats affected by GM1. Martin is leading Auburn's effort, which was started by his mentor, Professor Emeritus Henry Baker.

To move the treatment toward human medicine, Martin developed a partnership with UMass Medical School researchers Drs. Miguel Sena-Esteves and Heather Gray-Edwards, an Auburn alumnaand they have worked collaboratively for 15 years, combining animal and human medicine studies to cure rare diseases that affect both animals and humans. In December 2018, the gene therapy was licensed to Axovant Gene Therapies Ltd., a clinical-stage company developing innovative gene therapies.

"This treatment is extremely promising because it has worked well in GM1mice and cats, and it is delivered by a single IV injection that takes less than an hour," Martin said. "We're hopeful that the treatment makes a real difference for patients and their families.

"The NIH is hoping to begin treating three or four more children in the next few months. As the trial progresses and more patients are treated, we'll have a good idea of whether the gene therapy helps children as much as it has helped the animals."

The NIH clinical trial is led by Dr. Cynthia Tifft, deputy clinical director at the National Human Genome Research Institute. "GM1 gangliosidosis is a devastating disease in young children, for which there are no currently approved treatment options. The development of a safe and effective gene therapy for these patients would be a welcome advancement in the field of pediatric lysosomal storage disorders affecting the brain," Tifft said.

For Auburn graduates Sara and Michael Heatherly of Opelika, whose son Porter was the first known case of GM1 in Alabama and died in 2016, the knowledge of a treatment is one of mixed emotions.

"We are excited to know there is hope for the future of children diagnosed with GM1," Michael Heatherly said. "We are thankful for everyone who has dedicated their time, resources and careers to move this treatment forward and to Axovant for bringing all of their work to life and making it a reality for GM1 patients.

"We understood early on the research would not help Porter, but we wanted to help spread the word of the research and the progress that was being made."

The Heatherlys gave Auburn researchers a reason to hope, and work harder for a cure. To honor the family, which held fundraisers for several years to support the research, the College of Veterinary Medicine's Scott-Ritchey Research Center incorporated Porter's likeness in a creative identity for the center.

SOURCE Auburn University

http://www.auburn.edu

See more here:
Auburn University research leads to gene therapy that provides hope for children with deadly disease - PRNewswire

LONDON, Dec. 3, 2019 /PRNewswire/ -- Alacrita, a leading life science consulting firm, with offices in the United States and the United Kingdom, announces the departure of Rob Johnson who leaves the company to take on a new role at a gene therapy startup. Anthony Walker, co-founder of Alacrita and managing partner, will take over leadership activities of the U.S. office.

Rob is a co-founder of Alacrita and was instrumental in establishing and growing the firm's U.S. presence. He guided it over the past seven years, leading the expansion of its consulting team there as well as of its portfolio of U.S. clients in the biotech and pharmaceutical sectors.

This continues a long history of Alacrita involvement in start-up companies. Alacrita consultants have played leading roles in a number of startup enterprises including Onyvax, Biotica Technology, Leucid Bio and other companies that have been spun out of university clients. Alacrita has worked with over 35 academic institutions on commercialization of early stage technology, including writing multiple business plans for successful spin-outs.

"Rob has been a driving force at Alacrita, leading our U.S. team and playing a major role in the progression of our firm in the United States. As we have grown there, his leadership has been effective and steady. Beyond his deep life-science expertise and leadership skill, he is a wonderful colleague who I have worked with for over 20 years. We could not be more excited for him in his new role," said Anthony Walker, Alacrita managing partner and co-founder.

"We are grateful for Rob's leadership of our U.S. operations. Rob worked tirelessly to help us build Alacrita into the transatlantic firm that it is today. We truly wish him well in his new endeavor," said Simon Turner, Alacrita managing partner.

"My time at Alacrita will always be meaningful. I am grateful for the incredible team that I have worked with all these years, for all that we have achieved together, as well as for the chance to help so many of our clients succeed with their projects. As I move on to a new challenge and chapter, I know I leave my post in capable hands that will continue the firm's strong trajectory upward," said Rob Johnson.

Anthony Walker, who takes over direction of the firm's U.S. office, leverages more than 30 years of experience, including over a decade spent buildingand managing a biotechnology company and nearly 20 years as a management consultant to the pharmaceutical and biotech industries.

Alacrita conducts more than 250 successful client assignments every year.The firm's team combines extensive industry experience (strategic, technical and commercial), advanced functional capabilities and a track record of success across the domain. Learn more by visiting http://www.alacrita.com.

For further information, please contact:

Anthony Walker, Managing Partner, U.S. Email: usa@alacrita.com Telephone: +1-617-714-9696 Address: 303 Wyman Street, Waltham, MA 02451

Simon Turner, Managing Partner, Europe Email: europe@alacrita.com Telephone: +44-(0)207-691-4915 Address: London BioScience Innovation Centre, 2 Royal College St, London, United Kingdom

Original post:
Alacrita's Rob Johnson Departs for Gene Therapy Space - P&T Community

Shares of Regenxbio (RGNX) have risen by 190% since I initially uncovered and recommended a position in this gene therapy pioneer in 2016. The stock has appreciated by just 52% since my February 2018 update piece.

With the recently announced acquisition of collaboration partner Audentes Therapeutics (BOLD) by Astellas Pharma for $2.7 billion (also a prior recommendation), I thought it's time to revisit this gene therapy "picks and shovels" play.

Chart

Figure 1: RGNX daily advanced chart (Source: Finviz)

When looking at charts, clarity often comes from taking a look at distinct time frames in order to determine important technical levels to get a feel for what's going on. In the above chart (daily advanced), we can see a downtrend taking place from April into Q3, with share price bottoming in the low 30s in September. Recently, the stock received a boost after news of Audentes Therapeutics being acquired (had licensed NAV technology for various indications including XLMTM, Crigler-Najjar and CPVT). Readers might recall that when collaboration partner AveXis was acquired for $8.7 billion in 2018, its suitor Novartis (NVS) was required to pay them $100 million upfront as an accelerated licensee payment (while remaining on the hook for $80 million in commercial milestones plus royalties on Zolgensma). A move to highs last witnessed in 2018 would equate to a bit less than a double from today's levels.

In my last update piece, I touched on the following keys to our bullish thesis:

Figure 3: Internally developed pipeline (Source: corporate presentation)

Figure 4: Collaboration partner pipeline (Source: corporate presentation)

Let's take a look at recent events and how they've affected our thesis here.

In late July Regenxbio and Swiss biotech firm Neurimmune announced an exclusive license to develop novel AAV gene therapies using NAV vectors to deliver human antibodies against targets implicated in chronic neurodegenerative diseases, including tauopathies. Both companies will be jointly responsible and share development costs equally, with each having co-development and co-commercialization options (or can elect to receive a phase-based worldwide royalty instead of continued development investment).

On July 31st Regenxbio announced a license agreement with Pfizer (PFE), specifically non-exclusive global license to NAV AAV9 vector for commercialization of gene therapies for the treatment of Friedreich's ataxia. In return for these rights, Regenxbio received an upfront payment, and has the potential to receive ongoing fees, development and commercial milestone payments, and royalties on net sales of products incorporating their IP.

On October 30th, it was disclosed that the company exercised its option under the previously announced option and license agreement to Clearside Biomedical's (CLSD) in-office SCS Microinjector for the delivery of adeno-associated virus-based therapeutics, including RGX-314 delivery to the suprachoroidal space to potentially treat wet age-related macular degeneration, diabetic retinopathy, and related conditions where chronic anti-vascular endothelial growth factor (VEGF) treatment is currently the standard of care. Deal terms were modest, involving a small upfront payment, up to $34 million in development milestones, up to $102 million in sales milestones and mid-single digit royalties on net sales of products incorporating SCS Microinjector.

Figure 5: Widespread retinal transduction observed in both subretinal and suprachoroidal delivery of AAV8 in non-human primates (Source: Corporate Presentation)

Keep in mind that Regenxbio is currently suing the FDA, as they disclosed a clinical hold for RGX-314 suggesting the agency opted for this action "without notice or explanation". The clinical hold is related to issues associated with delivery systems currently being used (seems agency is not a fan of subretinal injections, which allows for a more direct approach but can have a complicated adverse event profile (involves potential events such as intraocular inammation, retinal detachment, ocular hemorrhage, etc). A more convenient intravitreal approach is being utilized by Adverum Biotechnologies (ADVM). While such differences may not be that big of a deal in clinical trials, you can bet that when and if such gene therapies make it to the market, those that offer the ideal mix of high convenience and best safety profile will be the most likely to win this high stakes race.

Figure 6: Subretinal injection versus intravitreal injection (Source: Adverum corporate presentation)

For the third quarter of 2019, the company reported cash and equivalents of $417.1 million as compared to net loss of $34.6 million. Research and development costs nearly doubled to $35.7 million, while SG&A increased to $12.4 million

Regarding RGX-314 in treating wet AMD and diabetic retinopathy, we are informed of the clinical hold related to use of third-party commercially available devices used to deliver the gene therapy candidate. Specifically, the company states that all 42 patients have been dosed in the phase 1/2a study and that the partial clinical hold on IND is not related to the gene therapy candidate itself. Management guides for phase 2b study in wet AMD to get underway in Q1 2020, around which time IND will be filed for the study in diabetic retinopathy. We are reminded that data here continues to be encouraging, with no drug-related serious adverse events and subjects in Cohort 5 showing reduction of over 80% from the mean annualized injection rate during the 12 months prior to receiving RGX-314. Additionally, durable effects on vision and retinal thickness had been demonstrated over 1.5 years in the third cohort, with 50% of subjects remaining free of anti-VEGF injections more than 1.5 years after RGX-314 administration.

Figure 7: Cohort 5 injections pretreatment and after being treated with RGX-314 (Source: Corporate Presentation)

Management continues to state that they are evaluating in-office delivery of RGX-314 to the suprachoroidal space and will unveil plans for this route of administration next year.

As for RGX-501 for the treatment of Homozygous Familial Hypercholesterolemia (HoFH), interim data from Cohort 2 is expected at the end of the year (keep in mind this time they are using corticosteroid prophylaxis). Also at the end of the year, we'll get an interim data update on the phase 1/2 study of RGX-121 for the treatment of MPS II.

As these gene therapy companies are increasingly valued on the basis of their manufacturing capabilities as reflected in recent buyouts (including Audentes Therapeutics), it's worth noting that Regenxbio's new cGMP production facility (allows for production of NAV Technology-based vectors at scales up to 2,000 Liters) is expected to be operational in 2021.

As for partnered efforts, Novartis revealed Q3 Zolgensma sales of $160 million resulting in royalty revenue to Regenxbio of $9.2 million. Audentes Therapeutics' AT132 in XLMTM is well on its way to potential regulatory approval with BLA to be filed mid-2020.

To conclude, even after the recent bounce this gene therapy "picks and shovels" has continued upside ahead. With 20+ partnered product candidates, one can almost think of it as a mutual fund of sorts as investors gain exposure to various rare disease programs as well as programs targeting more prevalent conditions. On the other hand, the company has much more to prove with wholly-owned programs, as the anti-VEGF market is known for being intensely competitive with multiple "next-generation" approaches being tried out by larger companies aiming to garner a slice of this lucrative pie. Consider Baker Brothers' recent royalty deal, purchasing a 4.5% rate for $225 million (assumes KSI-301 worth over $5 billion using back of the envelope math). Another overlooked catalyst is phase 1 data for Danon Disease program with collaboration partner Rocket Pharmaceuticals (RCKT) in 2020, a lucrative indication of high unmet medical need considering estimated US+ EU prevalence of 15,000 to 30,000 patients.

For readers who are interested in the story and have done their due diligence, Regenxbio remains a Buy and I suggest patiently accumulating dips over the next couple of quarters.

Additional dilution in the near term does not look likely considering the current cash position and operational runway. Disappointing data for wet AMD and other programs not to mention setbacks in the clinic (especially delays or safety/tolerability concerns) would weigh on the stock. Failure to get the recent clinical hold lifted would weigh on the stock and the company's competitive position in the wet AMD market (and related indications). Competition for certain indications should not be ignored, especially by peers with significantly greater resources.

Author's Note: I greatly appreciate you taking the time to read my work and hope you found it useful. Consider clicking "Follow" next to my name to receive future updates and look forward to your thoughts in the Comments section below.

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Disclosure: I am/we are long CLSD, RCKT. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: Disclaimer: Commentary presented is NOT individualized investment advice. Opinions offered here are NOT personalized recommendations. Readers are expected to do their own due diligence or consult an investment professional if needed prior to making trades. Strategies discussed should not be mistaken for recommendations, and past performance may not be indicative of future results. Although I do my best to present factual research, I do not in any way guarantee the accuracy of the information I post. I reserve the right to make investment decisions on behalf of myself and affiliates regarding any security without notification except where it is required by law. Keep in mind that any opinion or position disclosed on this platform is subject to change at any moment as the thesis evolves. Investing in common stock can result in partial or total loss of capital. In other words, readers are expected to form their own trading plan, do their own research and take responsibility for their own actions. If they are not able or willing to do so, better to buy index funds or find a thoroughly vetted fee-only financial advisor to handle your account.

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Regenxbio: Revisiting This Gene Therapy 'Picks And Shovels' Play - Seeking Alpha

DAYTON, Ohio, Dec. 3, 2019 /PRNewswire/ -- CSafe Global understands temperature control, gained from 40 years of experience in developing innovative temperature-controlled packaging - from solutions designed to transport vital pediatric vaccines to crucial life-saving cancer drugs. CSafe is known for protecting what matters most to pharmaceutical companies, so that patients receive what matters most to them.

CSafe recognizes the importance of thermally protecting shipments of life-enhancing medications as they are shipped to patients in need around the world. The criticality of temperature protection within the emerging Cell and Gene Therapy (CGT) space is further amplified due to the value and uniqueness of CGT products and the complicated supply chain. The new AcuTemp Plus Series has been developed with this in mind, and will confidently meet the evolving and increasingly demanding customer expectations for temperature performance, solution quality, and system robustness required of packaging solutions to protect shipments of novel cell and gene therapies.

"We are proud to expand our cold chain packaging offerings for the Cell and Gene Therapy market, and have focused and invested significantly in R&D to develop best-in-class solutions to meet the demanding product requirements for this emerging class of medication," stated Patrick Schafer, CEO of CSafe Global. "Test results of the new AcuTemp Plus Series have been very impressive with temperature performances outlasting other market available solutions by a large margin."

The AcuTemp Plus Series of packaging solutions has been designed with proprietary, high-performance ThermoCorVacuum Insulated Panels (VIP) to guarantee precise end-to-end control of internal payload temperatures. In addition to industry-leading performance, the innovative range has been developed with simplicity in mind, benefiting from optimized system components and solution design. The new series is available in multiple size configurations and temperature profiles, along with different integrated track and trace options to meet customer needs. Furthermore, the AcuTemp Plus Series is supported with a fully managed, end-to-end service infrastructure leveraging CSafe's industry-unique retest and reuse program - REPAQ.

With a market-leading portfolio of both active and passive temperature-management solutions, it is CSafe's mission to bring peace of mind to every life-science customer.

CONTACT: Josh Angliss, jangliss@csafeglobal.com

View original content:http://www.prnewswire.com/news-releases/csafe-expands-cold-chain-offerings-for-the-cell-and-gene-therapy-market-with-launch-of-high-performing-acutemp-plus-series-of-temperature-controlled-packaging-300968270.html

SOURCE CSafe Global

Original post:
CSafe expands cold chain offerings for the Cell and Gene Therapy market with launch of high-performing AcuTemp Plus Series of temperature-controlled...

New GMP conditioning of PEIpro-GMP designed to support clinical phase and commercialization stages of production

Strasbourg, France December 3, 2019 Polyplus-transfection(R) SA, the leading biotechnology company that supports gene and cell therapy by supplying innovative transfection solutions, today announces the availability of the first GMP-grade transfection reagent PEIpro(R)-GMP in additional conditioning that will ensure additional flexibility and cost-efficiency.

Polyplus-transfection commercialized PEIpro-GMP at the end of 2018. This was the first transfection solution for the gene and cell therapy industry that is compliant with global cGMP viral vector manufacturing requirements. Polyplus-transfection is now expanding the PEIpro-GMP packaging options from a 1 liter single-use bag to several conditioning options from 10 milliliters up to 1 liter in both single-use bags and bottles.

The use of closed-systems and single-use technologies for clinical trials and commercial manufacturing of viral vectors continues to increase. Polyplus-transfection is now providing two sizes of PEIpro-GMP in bags to ensure aseptic connections to successful perform large-scale transfection in closed sterile systems. Polyplus-transfection now additionally supplies PEIpro-GMP in bottle conditioning that will allow the adaption to different manufacturing scale-up and scale-out strategies. These increasing scale-out strategies require the performance of multiple smaller-scale transfections in multiple cell culture units at the same time. Demand for transfection reagents has thus increased for the smaller as well as for the larger conditioning options. The increases in options will therefore result in additional flexibility for therapeutic developers in the setup of cost-efficient viral vector manufacturing processes.

Polyplus-transfection has been developing strong supplier-manufacturer relationships over eighteen years, and certainly since the conception of the modern gene and cell therapy industries. As a result, we have unrivalled first hand visibility on the ever-changing needs of the gene and cell therapy markets, said Claire Wartel, PhD, director of quality and compliance, Polyplus-transfection. The focus of the market has evolved to commercialization and late-stage clinical stages. This means the therapeutic production scale and strategy needs to be adaptable to the production of each viral vector, whilst complying with cGMP manufacturing requirements. To provide this critical function in making available therapies to patients, Polyplus-technology is now able to offer a sustainable solution for viral vector platforms. This will benefit gene and cell therapy developers, the wider industry, and most importantly, the patient community reliant on the ongoing development of these therapies.

Claire Wartel recently gave more information on Identifying and mitigating risks in the viral vector supply chain in a podcast interview that can be accessed here.

About Polyplus-transfection SA

Polyplus-transfection SA is a biotechnology company specializing in nucleic acid delivery solutions located close to the University of Strasbourg in Eastern France and a market leader for transfection reagents for cell & gene therapy. Polyplus-transfections vast portfolio of delivery solutions can be used for all types of applications, from research and process development, all the way to clinical trials. For more information, please visit the Polyplus-transfection web site at: http://www.polyplus-transfection.com.

Read the original post:
Polyplus-transfection expands GMP portfolio to deliver flexibility for gene and cell therapy manufacturing - BioSpace

By Murielle Gonzalez 2-Dec-2019

Pharmaceuticals

New building in Stein also hosts the production of difficult-to-manufacture solid dosage forms

A cell processing specialist at work in new cell and gene facility in Stein. Photo as seen on Novartis website

Novartis has announced the opening of a new manufacturing facility for cell and gene therapies in Stein, Switzerland.

Our site in Stein is vital for new launches of solid and liquid drugs, explained Steffen Lang, Global Head of Novartis Technical Operations and member of the Novartis Executive Committee.

Lang continued: The construction of the new manufacturing facility is another investment in the production of breakthrough cell-based therapies that can potentially change the lives of patients.

In addition to manufacturing areas for novel CAR-T cell therapies, the new building also hosts the production of innovative, difficult-to-manufacture solid dosage forms, such as tablets and capsules.

Last September, the first clinical production of a cell and gene therapy badge was successfully completed, Novartis said.

Unlike conventional drug production, cell and gene therapy asks for the manufacture of a personal dose for each patient.

For this purpose, patients who have already undergone various therapies have a small amount of their own blood cells taken, which are then sent to Stein.

Here we enrich part of the white blood cells, the T cells, and genetically modify them so that they can recognize and fight the cancer cells in the patients blood, said Dorothea Ledergerber, project manager of the Stein plant for cell and gene therapies.

The altered cells are then sent back to a hospital and administered to the patient by infusion.

For Ledergerber, Novartis is doing pioneering work here. She explained: We have the unique opportunity to offer patients for whom there have been no other therapeutic options a totally new perspective by using these novel CAR-T cell therapies.

Novartis celebrated the opening in the presence of Federal Councillor Alain Berset and other guests on 28th November 2019.

Read more:
Novartis opens facility for innovative cell and gene therapies in Switzerland - Cleanroom Technology

Eleven drugmakers led by Pfizer and Novartis have set aside a combined $2 billion to invest in gene therapy manufacturing since 2018, according to a Reuters analysis, in a drive to better control production of the world's priciest medicines.

The full scope of Novartis' $500 million plan, revealed to Reuters in an interview with the company's gene therapy chief, has not been previously disclosed. It is second only to Pfizer, which has allocated $600 million to build its own gene therapy manufacturing plants, according to filings and interviews with industry executives.

Gene therapies aim to correct certain diseases by replacing the missing or mutated version of a gene found in a patient's cells with healthy copies. With the potential to cure devastating illnesses in a single dose, drugmakers say they justify prices well above $1 million per patient.

But the treatments are also extremely complex to make, involving the cultivation of living material, and still pose a risk of serious side effects.

Drugmakers say building their own manufacturing plants is a response to rising costs and delays associated with relying on third-party contract manufacturers, which are also expanding to capitalize on demand.

They say owning their own facilities helps safeguard proprietary production methods and more effectively address any concerns raised by the U.S. Food and Drug Administration (FDA), which is keeping a close eye on manufacturing standards.

"There's so little capacity and capability at contract manufacturers for the novel gene therapy processes being developed by companies," said David Lennon, president of AveXis, Novartis's gene therapy division. "We need internal manufacturing capabilities in the long term."

The approach is not without risks.

Bob Smith, senior vice president of Pfizer's global gene therapy business, acknowledged drugmakers take a "leap of faith" when they make big capital investment outlays for treatments before they have been approved or, in some cases, even produced data demonstrating a benefit.

PUSHING THE LIMITS

The rewards are potentially great, however.

Gene therapy is one of the hottest areas of drug research and, given the life-changing possibilities, the FDA is helping to speed treatments to market.

It has approved two so far, including Novartis's Zolgensma treatment for a rare muscular disorder priced at $2 million, and expects 40 new gene therapies to reach the U.S. market by 2022.

There are currently several hundred under development by around 30 drugmakers for conditions from hemophilia to Duchenne muscular dystrophy and sickle cell anemia.

The proliferation of these treatments is pushing the limits of the industry's existing manufacturing capacity.

Developers of gene therapies that need to outsource manufacturing face wait times of about 18 months to get a production slot, company executives told Reuters.

They are also charged fees to reserve space that run into millions of dollars, more than double the cost of a few years ago, according to gene therapy developer RegenxBio.

As a result, companies including bluebird bio, PTC Therapeutics and Krystal Biotech are also investing in gene therapy manufacturing, according to a Reuters analysis of public filings and executive interviews.

They follow Biomarin Pharmaceutical Inc, developer of a gene therapy for hemophilia, which constructed one of the industry's largest manufacturing facilities in 2017.

REGULATORY SCRUTINY

The FDA is keeping a close eye on standards.

This comes amid the agency's disclosure in August that it is investigating alleged data manipulation by former executives at Novartis' AveXis unit.

AveXis had switched its method for measuring Zolgensma's potency in animal studies. When results using the new method didn't meet expectations, the executives allegedly altered the data to cover it up, the FDA and Novartis have said.

One of the former executives, Brian Kaspar, denied wrongdoing in a statement to Reuters. Another, his brother Allan Kaspar, could not be reached for comment.

Novartis and the FDA say human clinical trials, which found Zolgensma effective in treating the most severe form of spinal muscular atrophy in infants, were not affected. Novartis also says its investments in gene therapy production started long before it became aware of the data manipulation allegations.

But the scandal has highlighted the importance of having a consistent manufacturing process for gene therapies, industry executives say.

According to four of them, the FDA has stressed in recent meetings the need for continuity in production processes all the way from the development of a drug to its commercialization.

By bringing production in-house, drugmakers may avoid pitfalls such as the need to switch to a larger facility if contract manufacturers' capacity proves limited, executives say.

The FDA is finalizing new guidelines for gene therapy manufacturing, expected at the end of the year.

"Manufacturing consistency is always a major concern for the agency," FDA spokeswoman Stephanie Caccomo told Reuters.

Highlighting the pressures on the industry, Sarepta Therapeutics, which largely outsources manufacturing, delayed a clinical trial of its Duchenne treatment in August, telling investors it wanted to avoid any questions from regulators about consistency in producing its therapy at commercial scale.

ENOUGH GROWTH FOR ALL?

"Between the trade secrets, the cost schedules and the time lag, it makes a whole lot of sense, if you can do it, to build out your own facilities and more and more gene therapy companies have started to do that," said Krish Krishnan, chief executive of Krystal Biotech Inc.

Krystal, which is developing therapies for rare skin diseases, has built one manufacturing facility and plans to invest more than $50 million in a new one it will start constructing in December.

MeiraGTx, which focuses on gene therapies for eye conditions, estimates it is currently spending roughly $25 million a year on manufacturing, including process development.

Despite such moves, however, contract manufacturers like Lonza and Thermo Fisher are confident their businesses will continue to grow due to the strength of demand.

Thermo Fisher has told investors its Brammer gene therapy manufacturing division, acquired in May, could soon earn $500 million in revenue a year, double its projected 2019 earnings.

Lonza CEO Marc Funk is also optimistic.

"Demand in gene therapy has increased," he said in an interview. "We believe this is going to continue in the coming years."

Continued here:
Pfizer, Novartis lead pharma spending spree on gene therapy production - Japan Today

MUNICH, Germany, Nov. 28, 2019 (GLOBE NEWSWIRE) -- ViGeneron GmbH, a gene therapy company, announced the closing of its series A financing round led by WuXi AppTec and Sequoia Capital China. The proceeds will enable ViGeneron to accelerate its proprietary viral vector-based gene therapy platforms and drive product development in its two lead ophthalmic gene therapy programs.

ViGenerons pipeline in gene therapy addresses ophthalmic diseases with high unmet medical need, including two programs in development for undisclosed indications where no approved treatment options are currently available.

The companys two novel next-generation gene therapy platforms are geared towards addressing the limitations of existing adeno-associated virus (AAV)-based gene therapies. The vgAAV vector platform, based on novel engineered AAV capsids, enables a superior transduction of target cells and is designed to efficiently cross biological barriers. These attributes allow vgAAV vectors to target a broad spectrum of cell types in the retina and potentially other tissues, such as central nervous system tissue; enabling intravitreal, less invasive treatment administration. For larger genes (>5Kb) the company has developed the innovative REVeRT vector platform. This platform uses an innovative vector approach to pack split genes into individual vgAAV vectors and generate a full-length protein via mRNA trans-splicing.

ViGeneron is a spin-off of the Ludwig-Maximilians-University (LMU) in Munich. The companys founding team includes highly experienced executives and internationally renowned experts with track records in developing retinal gene therapy programs from discovery to clinical stage: Dr. Caroline Man Xu (Co-founder and CEO), Prof. Dr. Martin Biel (Scientific Co-founder), and Prof. Dr. Stylianos Michalakis (Scientific Co-founder).

Dr. Caroline Man Xu, Co-founder and CEO of ViGeneron said: The evolution of medicines from small molecules to proteins has driven increased therapeutic benefits in the past; the next generation of gene therapies holds tremendous promise for patients. We are passionate about bringing innovations to patients. This financing is an important validation of our next-generation gene therapy technology platforms and ophthalmic development programs. With these top-tier investors and a strong ophthalmologic network supporting us, we are now in an excellent position to accelerate our development programs.

Edward Hu, Co-CEO of WuXi AppTec, commented: We are impressed by ViGenerons vgAAV vector platform and the innovative REVeRT vector platform. These gene therapy platform technologies will potentially generate superior gene therapy products to treat a wide range of diseases that are traditionally difficult to treat. We look forward to supporting the company to deliver on its great promises for the patients in need.

About ViGeneronViGeneron is dedicated to developing innovative gene therapies to treat ophthalmic diseases with high unmet medical need, as well as partnering with leading biopharmaceutical players in other disease areas. The companys pipeline is built on two proprietary adeno-associated virus (AAV) technology platforms. The first, vgAAV gene therapy vector platform, allows superior transduction efficiency and intravitreal, less invasive treatment administration. The second, REVeRT vector platform, targets diseases caused by mutations in large genes. Privately-owned ViGeneron was founded in 2017 by a seasoned team with in-depth experience in AAV vector technology and clinical ophthalmic gene therapy programs and is located in Munich, Germany. For further information, please visit http://www.vigeneron.com.

About WuXi AppTecWuXi AppTec provides a broad portfolio of R&D and manufacturing services that enable companies in the pharmaceutical, biotech and medical device industries worldwide to advance discoveries and deliver groundbreaking treatments to patients. As an innovation-driven and customer-focused company, WuXi AppTec helps our partners improve the productivity of advancing healthcare products through cost-effective and efficient solutions. With industry-leading capabilities such as R&D and manufacturing for small molecule drugs, cell and gene therapies, and testing for medical devices, WuXi AppTecs open-access platform is enabling more than 3,700 collaborators from over 30 countries to improve the health of those in need and to realize our vision that "every drug can be made and every disease can be treated".

About Sequoia Capital ChinaThe Sequoia team helps daring founders build legendary companies. In partnering with Sequoia, companies benefit from our unmatched network and the lessons we've learned over 47 years. As The Entrepreneurs Behind The Entrepreneurs, leading venture capital firm Sequoia Capital China is renowed for investing early in many successful companies and focuses on four sectors: TMT, healthcare, consumer/serive, and industrial technology.

Contact:ViGeneron GmbH info@vigeneron.com

Media and Investor Relations:MC Services AGShaun Brown/ Julia von Hummelphone: +49 (0)89 21022880 vigeneron@mc-services.eu

Read the original here:
ViGeneron announces closing of series A financing to drive development of next generation gene therapy pipeline - GlobeNewswire

(Reuters) - Eleven drugmakers led by Pfizer and Novartis have set aside a combined $2 billion to invest in gene therapy manufacturing since 2018, according to a Reuters analysis, in a drive to better control production of the worlds priciest medicines.

FILE PHOTO: A logo for Pfizer is displayed on a monitor on the floor at the New York Stock Exchange (NYSE) in New York, U.S., July 29, 2019. REUTERS/Brendan McDermid

The full scope of Novartis (NOVN.S) $500 million plan, revealed to Reuters in an interview with the companys gene therapy chief, has not been previously disclosed. It is second only to Pfizer (PFE.N), which has allocated $600 million to build its own gene therapy manufacturing plants, according to filings and interviews with industry executives.

Gene therapies aim to correct certain diseases by replacing the missing or mutated version of a gene found in a patients cells with healthy copies. With the potential to cure devastating illnesses in a single dose, drugmakers say they justify prices well above $1 million per patient.

But the treatments are also extremely complex to make, involving the cultivation of living material, and still pose a risk of serious side effects.

Drugmakers say building their own manufacturing plants is a response to rising costs and delays associated with relying on third-party contract manufacturers, which are also expanding to capitalize on demand.

They say owning their own facilities helps safeguard proprietary production methods and more effectively address any concerns raised by the U.S. Food and Drug Administration (FDA), which is keeping a close eye on manufacturing standards.

Theres so little capacity and capability at contract manufacturers for the novel gene therapy processes being developed by companies, said David Lennon, president of AveXis, Novartiss gene therapy division. We need internal manufacturing capabilities in the long term.

The approach is not without risks.

Bob Smith, senior vice president of Pfizers global gene therapy business, acknowledged drugmakers take a leap of faith when they make big capital investment outlays for treatments before they have been approved or, in some cases, even produced data demonstrating a benefit.

The rewards are potentially great, however.

Gene therapy is one of the hottest areas of drug research and, given the life-changing possibilities, the FDA is helping to speed treatments to market.

It has approved two so far, including Novartiss Zolgensma treatment for a rare muscular disorder priced at $2 million, and expects 40 new gene therapies to reach the U.S. market by 2022.

There are currently several hundred under development by around 30 drugmakers for conditions from hemophilia to Duchenne muscular dystrophy and sickle cell anemia. The proliferation of these treatments is pushing the limits of the industrys existing manufacturing capacity. Developers of gene therapies that need to outsource manufacturing face wait times of about 18 months to get a production slot, company executives told Reuters.

They are also charged fees to reserve space that run into millions of dollars, more than double the cost of a few years ago, according to gene therapy developer RegenxBio.

As a result, companies including bluebird bio (BLUE.O), PTC Therapeutics (PTCT.O) and Krystal Biotech (KRYS.O) are also investing in gene therapy manufacturing, according to a Reuters analysis of public filings and executive interviews.

They follow Biomarin Pharmaceutical Inc (BMRN.O), developer of a gene therapy for hemophilia, which constructed one of the industrys largest manufacturing facilities in 2017.

The FDA is keeping a close eye on standards.

This comes amid the agencys disclosure in August that it is investigating alleged data manipulation by former executives at Novartis AveXis unit.

AveXis had switched its method for measuring Zolgensmas potency in animal studies. When results using the new method didnt meet expectations, the executives allegedly altered the data to cover it up, the FDA and Novartis have said.

One of the former executives, Brian Kaspar, denied wrongdoing in a statement to Reuters. Another, his brother Allan Kaspar, could not be reached for comment.

Novartis and the FDA say human clinical trials, which found Zolgensma effective in treating the most severe form of spinal muscular atrophy in infants, were not affected. Novartis also says its investments in gene therapy production started long before it became aware of the data manipulation allegations.

But the scandal has highlighted the importance of having a consistent manufacturing process for gene therapies, industry executives say.

According to four of them, the FDA has stressed in recent meetings the need for continuity in production processes all the way from the development of a drug to its commercialization.

By bringing production in-house, drugmakers may avoid pitfalls such as the need to switch to a larger facility if contract manufacturers capacity proves limited, executives say.

The FDA is finalizing new guidelines for gene therapy manufacturing, expected at the end of the year.

Manufacturing consistency is always a major concern for the agency, FDA spokeswoman Stephanie Caccomo told Reuters.

Highlighting the pressures on the industry, Sarepta Therapeutics (SRPT.O), which largely outsources manufacturing, delayed a clinical trial of its Duchenne treatment in August, telling investors it wanted to avoid any questions from regulators about consistency in producing its therapy at commercial scale.

Between the trade secrets, the cost schedules and the time lag, it makes a whole lot of sense, if you can do it, to build out your own facilities and more and more gene therapy companies have started to do that, said Krish Krishnan, chief executive of Krystal Biotech Inc.

Krystal, which is developing therapies for rare skin diseases, has built one manufacturing facility and plans to invest more than $50 million in a new one it will start constructing in December.

MeiraGTx (MGTX.O), which focuses on gene therapies for eye conditions, estimates it is currently spending roughly $25 million a year on manufacturing, including process development.

Despite such moves, however, contract manufacturers like Lonza (LONN.S) and Thermo Fisher (TMO.N) are confident their businesses will continue to grow due to the strength of demand.

Thermo Fisher has told investors its Brammer gene therapy manufacturing division, acquired in May, could soon earn $500 million in revenue a year, double its projected 2019 earnings. Lonza CEO Marc Funk is also optimistic.

Demand in gene therapy has increased, he said in an interview. We believe this is going to continue in the coming years.

Reporting by Carl O'Donnell in New York and Tamara Mathias in Bengaluru; Editing by Tomasz Janowski, Michele Gershberg and Mark Potter

Read the original here:
Pfizer, Novartis lead $2 billion spending spree on gene therapy production - Reuters

27 Nov 2019

Twenty years ago, researchers took fibroblasts from the skin of eight Alzheimers patients, engineered them to produce nerve-growth factor, and slid them into each volunteers basal forebrain. They hoped the neurotrophin would halt or slow the neurodegeneration that robbed them of their memories, indeed their lives. The gamble failed and since then, scientists have shown little zest for gene therapy in neurodegenerative disorders. That is changing. As evident at this years Society for Neuroscience conference, held October 1923 in Chicago, gene therapy is back. Buoyed by success in treating spinal muscular atrophy in infants, scientists are flush with new ideasand funding.

What was once considered risky, expensive, and unlikely to succeed is now seen by many as risky, expensiveand quite likely to succeed. A growing number of scientists think gene-based therapies may have the best chance of slowing, or even preventing, neurodegeneration, especially for disorders caused by mutations in a single gene. SfN hosted a press briefing on gene therapy, plus many projects are active throughout the field beyond those showcased at the conference. There was no breaking clinical trial news at the annual meeting, but the scope and challenges of such therapies were outlined at the briefing moderated by Rush University s Jeff Kordower, Chicago, as well as a translational roundtable moderated by Asa Abeliovich, Columbia University, New York. Abeliovich recently co-founded Prevail Therapeutics, New York.

Going viral. Researchers are tweaking the capsid of adeno-associated viruses to optimize gene therapies for a multitude of disease. Shown here, AAV2.

From Zolgensma to Alzheimers? If the failure of the nerve growth factor therapy tempered enthusiasm for gene therapy (Mar 2018 news), then the success of AVXS-101, aka Zolgensma, reignited it. Developed by scientists at Nationwide Childrens Hospital, Columbus, Ohio, and AveXis, Bannockburn, Illinois, AVXS-101 uses an adeno-associated virus to deliver billions of copies of the survival motor neuron 1 gene to the brain. A small pilot trial tested the therapy in babies with spinal muscular atrophy (SMA) Type 1, the severest form of this neurodevelopmental disease. Lacking functional SMN1, these infants face progressive muscle weakness. Most die before their second birthday; those who live need a ventilator to breathe.

In Phase 1, AVXS-101 dramatically improved motor function of 15 treated infants; all were living 20 months later when historical data predicted only one would survive. Twelve babies who received the highest dose grew stronger within months, most sitting independently and rolling over. They hit the highest score on a scale of motor function, whereas untreated babies deteriorated. By 20 months, two of the treated babies had begun to walk (Mendell et al., 2017). The Food and Drug Administration approved zolgensma in May 2019. At SfN in Chicago, Petra Kaufmann, AveXis, played videos of the first patients treated with AVXS-101. Some four years later, they are walking, running, and appear to be playing almost normally. A video of a little girl walking downstairs with nary a hint of having SMA Type I visibly moved the audience.

Scientists say its a game-changer. It is really the tremendous success with SMA that has renewed interest in gene therapy, said Clive Svendsen, Cedars-Sinai Regenerative Medicine Institute, Los Angeles. Speaking with Alzforum before SfN, Bart De Strooper, Dementia Research Institute, London, said the same. The success in SMA patients of both gene therapy and antisense therapy has revived interest in the whole area, De Strooper said. Nowadays, researchers tend to lump gene therapy and antisense therapy under one moniker, i.e., gene-based therapy. The SMA antisense therapy nusinersen also works in babies with SMA Type 1 and is FDA-approved (Nov 2016 news; May 2018 conference news). Unlike gene therapy, antisense therapy needs to be delivered indefinitely.

How About Neurodegenerative Disease?At SfN, scientists outlined strategies for treating adults who face years of decline due to Alzheimers, amyotrophic lateral sclerosis, frontotemporal dementia, Huntingtons (HD) and Parkinsons diseases (PD), or other synucleinopathies. Some are being tested in clinical trials, others are in preclinical development. Some target specific losses or gains of function, others aim to rescue dying neurons more broadly. Scientists also believe that working on rare childhood diseases of lysosomal storage may give them an opening to treat this common phenotype in age-related neurodegeneration, as well.

Just this October, an ApoE gene therapy trial started enrolling. Led by Ronald Crystal at Weill Cornell Medical College, New York, it will inject adeno-associated virus carrying the gene for ApoE2 into patients with early to late-stage AD who inherited two copies of ApoE4. The idea is to flood their brains with the protective allele of this apolipoprotein to try to counteract the effects of the risk allele. AAV-rh10-APOE2 will be injected directly into the subarachnoid cisternae of participants brains. The Phase 1 trialwill recruit 15 patients with biomarker-confirmed AD. Beverly Davidson, Childrens Hospital of Philadelphia, has a similar ApoE2 gene therapy in preclinical development.

At SfN, Abeliovich detailed Prevails programs for forms of PD and for frontotemporal dementias that are caused by risk alleles. A trial has begun for a glucocerebrosidase-based gene therapy. The enzyme GCase is essential for lysosomes to function properly. People who have loss-of-function mutations in both copies of the GBA1 gene develop Gauchers, a lysosomal storage disease. The severest form starts in babies, most of whom die before age 2. Milder forms cause later-onset Gauchers, while heterozygous mutations in GBA1 increase risk for Parkinsons, making restoration of GCase an obvious strategy for PD. Some researchers are trying to develop ways to boost activity of the mutated enzyme (e.g., Oct 2019 news), whereas Abeliovich and colleagues have constructed AAV-9 vectors to deliver normal GBA1 into the brain to restore GCase production.

In preclinical studies, the AAV9-GBA1 construct PR001 rescued both lysosomal and brain function in models of GCase deficiency and of Parkinsons, Abeliovich said. In mice fed the GCase inhibitor conduritol epoxide (CBE), PR001 injected into the brain ventricles beefed up GCase activity and reduced glycolipid accumulation, which is a sign that lysosomes are functional. A single dose worked for at least six months. Similar results were seen in a commonly used model of Gauchers that expresses the V394L GBA mutation and only weakly expresses prosaposin and saposins, lysosomal proteins that metabolize lipids. In these 4L/PS-NA mice, PR001 made increased levels of active GCase, fewer lipids accumulated, and the mice were more mobile on a balance beam. 4L/PS-NA mice also accumulate -synuclein, the major component of Lewy bodies in PD and other synucleinopathies. In these mice, and also in A53T -synuclein mice made worse with CBE, PR001 halved the amount of insoluble -synuclein, Abeliovich reported at SfN.

In search of the right dose for humans, the scientists next turned to nonhuman primates. They injected PR001 into the cisterna magna in hopes AAV9 would broadly distribute throughout the brain. At the highest dose, 8 x 1010 capsids per gram of brain weight, exposure in the brain was similar to that seen in the mice. The virus permeated the spinal cord, frontal cortex, hippocampus, midbrain, and putamen.

Also in October, Prevail scientists began recruiting for a Phase 1/2 double-blind, sham-controlled trial to test this gene therapy in 16 people with moderate to severe PD, who have mutations in one or both copies of their GBA1 genes. Six patients each will receive a low or high dose of PR001A. Blood and CSF biomarkers to be analyzed at three and 12 months, and at follow-up, include GCase, lipids, -synuclein, and neurofilament light chain. Participants will also undergo cognitive, executive, and motor-function tests and brain imaging. A Phase 1/2 trial of PR001 in neuronopathic Gauchers, which affects the brain and spinal cord, will start soon, Abeliovich said.

Other groups are boosting dopamine production in Parkinsons by way of gene therapy. VY-AADC,developed by Voyager Therapeutics, Cambridge, Massachusetts, packages the gene for L-amino acid decarboxylase (AADC), which converts L-dopa into dopamine, in an AAV-2 vector that is delivered into the brain. Two Phase 1 open-label trials are testing safety and efficacy. Both the PD-1101 and PD-1102 trials use MRI to guide injections of the vector bilaterally into the putamina of 15 or 16 patients, respectively. According to preliminary results presented at the annual meeting of the American Academy of Neurology this past May, the virus penetrated half of the putamen and AADC activity, as judged by 18F-DOPA PET, increased by 85 percent in the latter study. Seven of eight treated patients reported improvement after a year, along with longer on time on L-DOPA, and shorter off time. Off time is the period when L-DOPA effects wear off and patients experience loss of motor control. RESTORE-1, a Phase 2 study of 42 patients, started in 2018 and will run to the end of 2020.

Long-Lived Gene Therapy. When a Parkinsons disease patient died eight years after neurturin gene therapy, the trophin was still being expressed in their putamen (top left) and substantia nigra (bottom left), where it corresponded with tyrosine hydroxylase activity (right). [Courtesy of Jeff Kordower.]

Also in PD, Kordower and colleagues plan to re-evaluate neurturin-based gene therapy. Previously, the gene for this neurotrophin was delivered in an AAV2 vector into the brains of Parkinson patients in Phase 1 and 2 trials. This did not improve motor function. Even so, in Chicago Kordower showed that in two patients who died eight and 10 years later, the inserted gene was still expressing neurturin and that dopamine levels were higher on the injected than the contralateral side of the substantia nigra/putamen. This shows us that long-term gene expression can be achieved in the human brain, said Kordower (see image above). He believes that by focusing delivery with ultrasound, or tweaking the capsid itself, he may be able to generate enough gene expression to improve function.

Separately, AAV-GAD, a gene therapy for PD that showed promise in Phase 2 (Mar 2011 news) was acquired by MeiraGTx, New York, which will continue to develop it in the U.S. and Europe, according to founder Samuel Waksal (Nov 2018 news).

For its part, Prevail has a gene transfer construct for frontotemporal dementia in the pipeline, as well. Called PR006, it carries GRN, the gene encoding progranulin, on an AAV9 vector. GRN mutations cause familial FTD and, much like GBA mutations, do their dirty work via lysosomal dysfunction. In Chicago, Abeliovich reported that PR006 boosted progranulin release from neurons derived from FTD-GRN patients, nearly doubling their levels of mature Cathepsin D, the lysosomal protease that chops progranulin into granulins and indicates healthy lysosomes. In progranulin knockout mice, PR006 restored brain GRN expression and progranulin secretion into the CSF. Abeliovich said he expects a Phase 1/2 clinical trial in FTD patients to start in early 2020.

The biotech company Passage Bio, Philadelphia, is planning for clinical trials early next year with its AAV-GRN vector. MeiraGTx, New York, is banking on a different approach for FTD. They have developed an AAV carrying UPF1, which encodes regulator of nonsense transcripts 1. This protein helps clear out aberrant RNAs through a process call nonsense-mediated decay. MeiraGTx hopes this will restore homeostasis to RNA processing. AAV-UPF1 will be trialed for FTD and all forms of ALS bar those caused by mutations in SOD1. For SOD ALS, Novartis, Basel, Switzerland, and REGENXBIO, Rockville, Maryland, have a vector in preclinical testing.

For his part, Svendsen is taking a different approach. His lab tackles ALS with ex vivo gene therapy. The idea is to engineer clinical-grade human stem cells to produce glial-derived growth factor, and inject them into the spinal cord, much like the early NGF studies did in AD. Svendsen hopes the cells will churn out enough of the neurotrophin to protect spinal cord motor neurons. In a Phase 1/2a trial, 18 ALS patients have received these cells into one side of their spinal cords, such that each person serves as his or her own control. If this works, they would regain mobility only on the injected side. The trial finished in October; Svendsen expects results to come out in a few months. In a follow-up study, the scientists are trying to do the same with induced pluripotent stem cells. This would allow them to transplant autologous cells into patients, avoiding immune rejection

Other groups are deploying gene therapy as a way to improve immunotherapy, shield neurons from stress, or even generate neurons from astrocytes to make up for those lost to neurodegeneration.Tom Fagan

Read the rest here:
Time to Try Again: Gene-Based Therapy for Neurodegeneration - Alzforum

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Link:
Novartis opens new cell and gene therapies facility in Switzerland - Pharmaceutical Business Review

Pfizer and Novartis lead eleven drug manufacturers setting aside a combined $2bn to invest in gene therapy manufacturing since 2018.

According to a Reuters analysis, this strategy aims to better control production of the worlds priciest medicines.

The full scope of Novartis $500mn plan, revealed to Reuters in an interview with the companys gene therapy chief, has not been previously disclosed. It is second only to Pfizer, which has allocated $600mn to build its own gene therapy manufacturing plants, according to filings and interviews with industry executives.

Gene therapies aim to correct certain diseases by replacing the missing or mutated version of a gene found in a patients cells with healthy copies. With the potential to cure devastating illnesses in a single dose, drugmakers say they justify prices well above $1 million per patient.

But the treatments are also extremely complex to make, involving the cultivation of living material, and still pose a risk of serious side effects.

Drugmakers say building their own manufacturing plants is a response to rising costs and delays associated with relying on third-party contract manufacturers, which are also expanding to capitalize on demand.

They say owning their own facilities helps safeguard proprietary production methods and more effectively address any concerns raised by the U.S. Food and Drug Administration (FDA), which is keeping a close eye on manufacturing standards.

Theres so little capacity and capability at contract manufacturers for the novel gene therapy processes being developed by companies, said David Lennon, president of AveXis, Novartiss gene therapy division. We need internal manufacturing capabilities in the long term.

SEE ALSO:

The Medicines Manufacturing Innovation Centre to be built in Scotland

Ricoh: The advantages of 3D printing across the healthcare sector

AstraZeneca experiences significant sales growth due to introduction of new drugs

Read the latest issue of Manufacturing Global here

Pushing the Limits

The approach is not without risks. The rewards are potentially great, however.

Gene therapy is one of the hottest areas of drug research and, given the life-changing possibilities, the FDA is helping to speed treatments to market.

It has approved two so far, including Novartiss Zolgensma treatment for a rare muscular disorder priced at $2 million, and expects 40 new gene therapies to reach the U.S. market by 2022.

There are currently several hundred under development by around 30 drugmakers for conditions from hemophilia to Duchenne muscular dystrophy and sickle cell anemia. The proliferation of these treatments is pushing the limits of the industrys existing manufacturing capacity.

Developers of gene therapies that need to outsource manufacturing face wait times of about 18 months to get a production slot, company executives told Reuters. They are also charged fees to reserve space that run into millions of dollars, more than double the cost of a few years ago, according to gene therapy developer RegenxBio.

As a result, companies including bluebird bio, PTC Therapeutics and Krystal Biotech are also investing in gene therapy manufacturing, according to a Reuters analysis of public filings and executive interviews.

They follow Biomarin Pharmaceutical Inc, developer of a gene therapy for hemophilia, which constructed one of the industrys largest manufacturing facilities in 2017. The FDA is keeping a close eye on standards.

Regulatory Scrutiny

This comes amid the agencys disclosure in August that it is investigating alleged data manipulation by former executives at Novartis AveXis unit.

AveXis had switched its method for measuring Zolgensmas potency in animal studies. When results using the new method didnt meet expectations, the executives allegedly altered the data to cover it up, the FDA and Novartis have said.One of the former executives, Brian Kaspar, denied wrongdoing in a statement to Reuters. Another, his brother Allan Kaspar, could not be reached for comment.

Read more:
Pfizer and Novartis lead $2bn spending on gene therapy production - Manufacturing Global

Traditional gene therapy has seen numerous challenges during its decades of development, but scientists seem to have finally figured out how to get the treatment to work with regulatory approvals forNovartis' (NYSE:NVS) Zolgensma and bluebird bio's (NASDAQ:BLUE) Zynteglo this year. The process involves inserting genes into diseased cells to express missing or mutated proteins.

Storming onto the scene over the past few years, CRISPR/Cas9, championed by CRISPR Therapeutics (NASDAQ:CRSP), Editas Medicine (NASDAQ:EDIT) and Intellia Therapeutics (NASDAQ:NTLA), offered hope for more precise gene editing. At the very least, the process can insert the gene into a precise location in the genome. More impressive -- and something that traditional gene therapy can't readily do -- CRISPR/Cas9 offers the possibility of deleting problematic genes or making specific changes to mutated genes to restore their functions.

Image source: Getty Images.

CRISPR/Cas9 appeared to be working well in preclinical models, and last week, investors got a first look at how the therapy is working in humans with CRISPR Therapeutics and its development Vertex Pharmaceuticals (NASDAQ:VRTX) announcing results for the first two patients treated with CTX001.

One patient with a blood disorder called transfusion-dependent beta thalassemia (TDT) required 16.5 transfusions per year over the two years before being treated with CTX001, but nine months after treatment, the patient was transfusion independent with high expression of fetal hemoglobin, the gene inserted into the patients' cells.

The other patient had sickle cell disease (SCD) with an average of seven vaso-occlusive crises (VOCs) per year over the two years before the study started. Four months after being treated with CTX001, the patient was free of VOCs, which are caused by sickle-shaped red blood cells that block blood vessels. Like the beta thalassemia patient, the SCD patient had expression of fetal hemoglobin.

The results from the first two patients look comparable to Bluebird's Zynteglo, which also treats TDT and SCD by increasing hemoglobin levels. But this was data from just two patients, and investors should still have plenty of questions as we get additional data:

Consistency: One patient in each disease doesn't say much about how well the treatment works in the average patient. What will the efficacy look like after the treatment of a few dozen patients?

Durability: Gene editing and gene therapy are designed to be cures. Do both last forever?

Manufacturing: Bluebird had to adjust its manufacturing procedure to increase expression to treat patients requiring higher expression. Will the initial CRISPR/Cas9 manufacturing procedure work for all patients?

In vivo/ex vivo: That's Latin for in or outside of a living thing -- in this case a human being. CTX001 and Zynteglo are ex vivo treatments because cells are taken from the patient, manipulated to express the gene of interest, and put back into the patient. Novartis has shown that gene therapy can work in vivo with Zolgensma delivered via an injection of a viral vector. Can CRISPR/Cas9 work in vivo in humans? Editas Medicine hopes so, but the company still hasn't advanced a treatment into the clinic.

Last week's data release offers plenty of hope for investors in CRISPR/Cas9 and traditional gene therapy companies should certainly be looking in the rearview mirror at the technology coming up from behind, but it's still way too early to pick a winner between traditional gene therapy and CRISPR/Cas9.

The right answer for investors in biotech companies might end up being to buy both. The upside potential for curing diseases may end up outweighing the downside if one technology doesn't end up working out.

See original here:
CRISPR vs. Gene Therapy Round 1: What Investors Need to Know - The Motley Fool

NEW YORK, Nov. 28, 2019 /PRNewswire/ --

INTRODUCTIONOver the last 12 months, the pharmaceutical industry reported a year-on-year increment of nearly 75% in funding to support the development of various cell and gene therapies. In fact, close to USD 5 billion has been invested into research on gene-based therapies in the previous two decades. Interestingly, over 2,600 clinical studies have been initiated in this field of research, since 1989. The aforementioned numbers are indicative of the rapid pace of development in this upcoming segment of the biopharmaceutical industry. The development of such therapy products require gene delivery vehicles, called vectors, to desired locations within the body (in vivo) / specific cells (ex vivo). The growing demand for such therapies and the rising number of clinical research initiatives in this domain has led to an increase in demand for preclinical and clinical grade gene delivery vectors. Fundamentally, genetic modifications can be carried out using either viral (such as adenovirus, adeno associated virus (AAV), lentivirus, retrovirus, Sendai virus, herpes simplex virus, vaccinia virus, baculovirus and alphavirus) or non-viral (such as plasmid DNA) vectors. Moreover, recent advances in vector research have led to the development of several innovative viral / non-viral gene delivery approaches.

Read the full report: https://www.reportlinker.com/p05828868/?utm_source=PRN

At present, 10+ genetically modified therapies have received approval / conditional approval in various regions of the world; these include (in the reverse chronological order of year of approval) Zynteglo (2019), Zolgensma (2019), Collategene (2019), LUXTURNA (2017), YESCARTA (2017), Kymriah (2017), INVOSSA (2017), Zalmoxis (2016), Strimvelis (2016), Imlygic (2015), Neovasculagen (2011), Rexin-G (2007), Oncorine (2005) and Gendicine (2003). In addition, over 500 therapy candidates are being investigated across different stages of development. The growing number of gene-based therapies, coupled to their rapid progression through the drug development process, has created significant opportunities for companies with expertise in vector manufacturing. Presently, a number of industry (including both well-established companies and smaller R&D-focused initiatives), and non-industry players (academic institutes) claim to be capable of manufacturing different types of viral and non-viral vectors. In addition, there are several players offering novel technology solutions, which are capable of improving existing genetically modified therapy products and upgrading their affiliated manufacturing processes. Considering prevalent and anticipated future trends, we believe that the vector and gene therapy manufacturing market is poised to grow steadily, driven by a robust pipeline of therapy candidates and technical advances aimed at mitigating existing challenges related to gene delivery vector and advanced therapy medicinal products.

SCOPE OF THE REPORTThe "Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market (3rd Edition), 2019-2030 (Focus on AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA and Other Vectors)" report features an extensive study of the rapidly growing market of viral and non-viral vector and gene therapy manufacturing, focusing on contract manufacturers, as well as companies with in-house manufacturing facilities. The study presents an in-depth analysis of the various firms / organizations that are engaged in this domain, across different regions of the globe. Amongst other elements, the report includes: An overview of the current status of the market with respect to the players involved (both industry and non-indutry) in manufacturing viral vectors, non-viral vectors and other novel types of vectors. It features information on the year of establishment, scale of production, type of vectors manufactured, location of manufacturing facilities, applications of vectors (in gene therapy, cell therapy, vaccines and others), and purpose of production (fulfilling in-house requirements / for contract services). An informed estimate of the annual demand for viral and non-viral vectors, taking into account the marketed gene-based therapies and clinical studies evaluating vector-based therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength. An estimate of the overall, installed vector manufacturing capacity of industry players based on information available in the public domain, and insights generated via both secondary and primary research. The analysis also highlights the distribution of the global capacity by vector type (viral vector and plasmid DNA), scale of operation (clinical and commercial), size of the company / organization (small-sized, mid-sized and large) and key geographical regions (North America, Europe, Asia Pacific and the rest of the world). An in-depth analysis of viral vector and plasmid DNA manufacturers, featuring three schematic representations; namely [A] a three dimensional grid analysis, representing the distribution of vector manufacturers (on the basis of type of vector) across various scales of operation and purpose of production (in-house operations and contract manufacturing services), [B] a logo landscape of viral vector and plasmid DNA manufacturers based on the type (industry and non-industry) and the size of the industry player (small-sized, mid-sized and large companies), and [C] a schematic world map representation, highlighting the geographical locations of vector manufacturing hubs. An analysis of recent collaborations and partnership agreements inked in this domain since 2015; it includes details of deals that were / are focused on the manufacturing of vectors, whihc were analyzed on the basis of year of agreement, type of agreement, type of vector involved, and scale of operation (laboratory, clinical and commercial). An analysis of the various factors that are likely to influence the pricing of vectors, featuring different models / approaches that may be adopted by product developers / manufacturers in order to decide the prices of proprietary vectors. An overview of other viral / non-viral gene delivery approaches that are currently being researched for the development of therapies involving genetic modification. Elaborate profiles of key players based in North America, Europe and Asia-Pacific (shortlisted based on scale of operation). Each profile features an overview of the company / organization, its financial performance (if available), information on its manufacturing facilities, vector manufacturing technology and an informed future outlook. A discussion on the factors driving the market and the various challenges associated with the vector production process.

One of the key objectives of this report was to evaluate the current market size and the future opportunity associated with the vector manufacturing market, over the coming decade. Based on various parameters, such as the likely increase in number of clinical studies, anticipated growth in target patient population, existing price variations across different vector types, and the anticipated success of gene therapy products (considering both approved and late-stage clinical candidates), we have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2019-2030. In order to provide a detailed future outlook, our projections have been segmented on the basis of [A] type of vectors (AAV vector, adenoviral vector, lentiviral vector, retroviral vector, plasmid DNA and others), [B] applications (gene therapy, cell therapy and vaccines), [C] therapeutic area (oncological disorders, inflammation & immunological diseases, neurological disorders, ophthalmic disorders, muscle disorders, metabolic disorders, cardiovascular disorders and others), [D] scale of operation (preclinical, clinical and commercial) and [E] geography (North America, Europe, Asia Pacific and rest of the world).

The research, analysis and insights presented in this report are backed by a deep understanding of key insights generated from both secondary and primary research. For the purpose of the study, we invited over 160 stakeholders to participate in a survey to solicit their opinions on upcoming opportunities and challenges that must be considered for a more inclusive growth. Our opinions and insights presented in this study were influenced by discussions held with several key players in this domain. The report features detailed transcripts of interviews held with the stakeholders: Menzo Havenga (Chief Executive Officer and President, Batavia Biosciences) Nicole Faust (Chief Executive Officer & Chief Scientific Officer, CEVEC Pharmaceuticals) Jeffrey Hung (Chief Commercial Officer, Vigene Biosciences) Olivier Boisteau, (Co-Founder / President, Clean Cells), Laurent Ciavatti (Business Development Manager, Clean Cells) and Xavier Leclerc (Head of Gene Therapy, Clean Cells) Joost van den Berg (Director, Amsterdam BioTherapeutics Unit) Bakhos A Tannous (Director, MGH Viral Vector Development Facility, Massachusetts General Hospital) Colin Lee Novick (Managing Director, CJ Partners) Cedric Szpirer (Executive & Scientific Director, Delphi Genetics) Semyon Rubinchik (Scientific Director, ACGT) Alain Lamproye (President of Biopharma Business Unit, Novasep) Astrid Brammer (Senior Manager Business Development, Richter-Helm) Brain M Dattilo (Business Development Manager, Waisman Biomanufacturing) Marco Schmeer (Project Manager, Plasmid Factory) and Tatjana Buchholz (Marketing Manager, Plasmid Factory) Nicolas Grandchamp (R&D Leader, GEG Tech)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGYThe data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market may evolve across different regions and technology segments. Wherever possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include: Annual reports Investor presentations SEC filings Industry databases News releases from company websites Government policy documents Industry analysts' views

While the focus has been on forecasting the market over the period 2019-2030, the report also provides our independent view on various technological and non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various secondary and primary sources of information.

CHAPTER OUTLINESChapter 2 is an executive summary of the insights captured in our research. The summary offers a high-level view on the likely evolution of the vector and gene therapy manufacturing market in the short to mid-term, and long term.

Chapter 3 is a general introduction to the various types of viral and non-viral vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages, limitations and applications of currently available gene delivery vehicles. The chapter also provides a brief description of the clinical and approved pipeline of genetically modified therapies. Further, it includes a review of the latest trends and innovations in the contemporary vector manufacturing market.

Chapter 4 provides a detailed overview of around 80 companies, featuring both contract service providers and in-house manufacturers that are actively involved in the production of viral vectors and / or gene therapies utilizing viral vectors. The chapter provides details on the year of establishment, scale of production, type of viral vectors manufactured (AAV, adenoviral, lentiviral, retroviral and others), location of manufacturing facilities, applications of vectors (gene therapies, cell therapies, vaccines and others) and purpose of production (fulfilling in-house requirements / for contract services).

Chapter 5 provides an overview of around 30 industry players that are actively involved in the production of plasmid DNA and other non-viral vectors and / or gene therapies utilizing non-viral vectors. The chapter provides details on the year of establishment, scale of production, location of manufacturing facilities, applications of vectors (gene therapies, cell therapies, vaccines and others) and purpose of vector production (fulfilling in-house requirements / for contract services).

Chapter 6 provides an overview of around 80 non-industry players (academia and research institutes) that are actively involved in the production of vectors (both viral and non-viral) and / or gene therapies. The chapter provides details on the year of establishment, scale of production, location of manufacturing facilities, type of vectors manufactured (AAV, adenoviral, lentiviral, retroviral, plasmid DNA and others), applications of vectors (gene therapies, cell therapies, vaccines and others) and purpose of vector production (fulfilling in-house requirements / for contract services).

Chapter 7 features detailed profiles of the US based contract service providers / in-house manufacturers that possess commercial scale capacities for the production of viral vectors / plasmid DNA. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience and an informed future outlook.

Chapter 8 features detailed profiles of EU based contract service providers / in-house manufacturers that possess commercial scale capacities for the production of viral vectors / plasmid DNA. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience, and an informed future outlook.

Chapter 9 features detailed profiles of Asia-Pacific based contract service provider(s) / in-house manufacturer(s) that possess commercial scale capacities for production of viral vectors / plasmid DNA. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience, and an informed future outlook.

Chapter 10 provides detailed information on other viral / non-viral vectors (including alphavirus vectors, Bifidobacterium longum vectors, Listeria monocytogenes vectors, myxoma virus based vectors, Sendai virus based vectors, self-complementary vectors (improved versions of AAV), and minicircle DNA and Sleeping Beauty transposon vectors (non-viral gene delivery approach)) that are currently being utilized by pharmaceutical players to develop gene therapies, T-cell therapies and certain vaccines, as well. This chapter presents overview on all the aforementioned types of vectors, along with examples of companies that use them in their proprietary products. It also includes examples of companies that are utilizing specific technology platforms for the development / manufacturing of some of these novel vectors.

Chapter 11 features an elaborate analysis and discussion of the various collaborations and partnerships related to the manufacturing of vectors or gene therapies, which have been inked amongst players. It includes a brief description of the purpose of the partnership models (including licensing agreements, mergers / acquisitions, product development, service alliances, manufacturing, and others) that have been adopted by the stakeholders in this domain, since 2015. It consists of a schematic representation showcasing the players that have forged the maximum number of alliances. Furthermore, we have provided a world map representation of the deals inked in this field, highlighting those that have been established within and across different continents.

Chapter 12 presents a collection of key insights derived from the study. It includes a grid analysis, highlighting the distribution of viral vectors and plasmid DNA manufacturers on the basis of their scale of production and purpose of manufacturing (fulfilling in-house requirement / contract service provider). In addition, it consists of a logo landscape, representing the distribution of viral vector and plasmid DNA manufacturers based on the type of organization (industry / non-industry) and size of employee base. The chapter also consists of six world map representations of manufacturers of viral / non-viral vectors (lentiviral, adenoviral, AAV and retroviral vectors, and plasmid DNA), depicting the most active geographies in terms of the presence of the organizations. Furthermore, we have provided a schematic world map representation to highlight the locations of global vector manufacturing hubs across different continents.

Chapter 13 highlights our views on the various factors that may be taken into consideration while pricing viral vectors / plasmid DNA. It features discussions on different pricing models / approaches that manufacturers may choose to adopt to decide the prices of their proprietary products.

Chapter 14 features an informed estimate of the annual demand for viral and non-viral vectors, taking into account the marketed gene-based therapies and clinical studies evaluating vector-based therapies. This section offers an opinion on the required scale of supply (in terms of vector manufacturing services) in this market. For the purpose of estimating the current clinical demand, we considered the active clinical studies of different types of vector-based therapies that have been registered till date. The data was analysed on the basis of various parameters, such as number of annual clinical doses, trial location, and the enrolled patient population across different geographies. Further, in order to estimate the commercial demand, we considered the marketed vector-based therapies, based on various parameters, such as target patient population, dosing frequency and dose strength.

Chapter 15 features an informed analysis of the overall installed capacity of the vectors and gene therapy manufacturers. The analysis is based on meticulously collected data (via both secondary and primary research) on reported capacities of various small-sized, mid-sized and large companies, distributed across their respective facilities. The results of this analysis were used to establish an informed opinion on the vector production capabilities of the organizations across different types of vectors (viral vectors, plasmid DNA, and both), scale of operation (clinical and commercial) and geographies (North America, EU, Asia-Pacific and the rest of the world).

Chapter 16 presents a comprehensive market forecast analysis, highlighting the likely growth of vector and gene therapy manufacturing market till the year 2030. We have segmented the financial opportunity on the basis of [A] type of vectors (AAV vector, adenoviral vector, lentiviral vector, retroviral vector, plasmid DNA and others), [B] applications (gene therapy, cell therapy and vaccines), [C] therapeutic area (oncological disorders, inflammation & immunological diseases, neurological disorders, ophthalmic disorders, muscle disorders, metabolic disorders, cardiovascular disorders and others), [D] scale of operation (preclinical, clinical and commercial) and [E] geography (North America, Europe, Asia Pacific and rest of the world). Due to the uncertain nature of the market, we have presented three different growth tracks outlined as the conservative, base and optimistic scenarios.

Chapter 17 provides details on the various factors associated with popular viral vectors and plasmid DNA that act as market drivers and the various challenges associated with the production process. This information has been validated by soliciting the opinions of several industry stakeholders active in this domain.

Chapter 18 presents insights from the survey conducted on over 160 stakeholders involved in the development of different types of gene therapy vectors. The participants, who were primarily Director / CXO level representatives of their respective companies, helped us develop a deeper understanding on the nature of their services and the associated commercial potential.

Chapter 19 summarizes the entire report. The chapter presents a list of key takeaways and offers our independent opinion on the current market scenario and evolutionary trends that are likely to determine the future of this segment of the industry.

Chapter 20 is a collection of transcripts of the interviews conducted with representatives from renowned organizations that are engaged in the vector and gene therapy manufacturing domain. In this study, we spoke to Menzo Havenga (Chief Executive Officer and President, Batavia Biosciences), Nicole Faust (Chief Executive Officer & Chief Scientific Officer, CEVEC Pharmaceuticals), Jeffrey Hung (Chief Commercial Officer, Vigene Biosciences), Olivier Boisteau, (Co-Founder / President, Clean Cells) and Xavier Leclerc (Head of Gene Therapy, Clean Cells), Laurent Ciavatti (Business Development Manager, Clean Cells), Joost van den Berg (Director, Amsterdam BioTherapeutics Unit), Bakhos A Tannous (Director, MGH Viral Vector Development Facility, Massachusetts General Hospital), Colin Lee Novick (Managing Director, CJ Partners), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Semyon Rubinchik (Scientific Director, ACGT), Alain Lamproye (President of Biopharma Business Unit, Novasep), Astrid Brammer (Senior Manager Business Development, Richter-Helm), Brain M Dattilo (Business Development Manager, Waisman Biomanufacturing), Marco Schmeer (Project Manager, Plasmid Factory) and Tatjana Buchholz (Marketing Manager, Plasmid Factory), and Nicolas Grandchamp (R&D Leader, GEG Tech).

Chapter 21 is an appendix, which provides tabulated data and numbers for all the figures in the report.

Chapter 22 is an appendix that provides the list of companies and organizations that have been mentioned in the report.

Read the full report: https://www.reportlinker.com/p05828868/?utm_source=PRN

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Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market (3rd Edition), 2019-2030 (Focus on AAV, Adenoviral, Lentiviral, Retroviral,...