StemCell Therapy

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Sarepta is best known for its RNA technology platform, which has led to two approved though also controversial drugs for Duchenne muscular dystrophy.

Yet, Sareptais also deeply invested in gene therapy, having developed an extensive list of more than two dozen experimental treatments, six of which have reached human testing.

Rare diseases have been an early target in this rapidly growing field. The two gene therapies approved in the U.S., Roche's Luxturna and Novartis' Zolgensma, are respectively used to treat an uncommon form of blindness and a muscle disease that occurs in about 1 in every 10,000 births.

Sarepta'stherapies target a wide variety of rare diseases, including Duchennemuscular dystrophy, Pompe disease, and types of Limb-girdle muscular dystrophy. Taking a stake in AavantiBio, with its work in Friedreich'sataxia, could expand Sarepta'sreach even further. The disease affects approximately 1 in every 40,000 people, according to the National Organization for Rare Disorders, which would equate to around 8,200 patients in the U.S.

"Our equity participation in AavantiBio serves our strategy to build our gene therapy engine through targeted investment in potentially life-enhancing therapies,"Doug Ingram, Sarepta'sCEO, said in a statement Thursday.

AavantiBio joins a couple large, powerful companies in the hunt for a gene therapy to treat Friedreich's ataxia. Pfizer and Novartis are each working on their own programs.

Outside of gene therapy, Reata Pharmaceuticals disclosed last year positive datafrom a study that tested an oral drug, known as omaveloxolone,in patients with Friedreich's ataxia. Reata said it intends to file the drug for approval based on those results.

AavantiBiowill be headquartered in the greater Boston area, putting it close by Cambridge, Massachusetts-based Sarepta. Cumbo, along with his role as CEO, will take one of AavantiBio'seight board of directors seats. Cumbohas, during the span of his career, helped launch 11 specialty products across multiple drug companies, including Sarepta, Gilead and Vertex, where he built a sales team for the hepatitis C drug Incivek.

Co-founders Byrne and Corti will be on the board too, alongside two independent directors and representatives from the investor group of Perceptive, Bain and RA.Louise Rodino-Klapac, senior vice president of gene therapy for Sarepta, will serve as a board observer.

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Sarepta, continuing its gene therapy push, helps launch a startup - BioPharma Dive

Some technologies that have emerged and altered the landscape in recent years include immunotherapy, CRISPR-Cas9 gene editing, and chimeric antigen receptor (CAR) T-cell therapies. Now, another platform technology is maturing from the research laboratory to commercial viability. In 2017, the U.S. Food and Drug Administration (FDA) approved the first directly administered gene therapy for mutations of a specific, disease-related gene. That product, Luxturna, marketed by Spark Therapeutics, delivers a functioning copy of the RPE65 gene to retinal cells using an adeno-associated virus (AAV) as a vector to treat a genetic form of blindness.

This advance has injected new energy into biotech startups seeking to capitalize on gene therapy.

As the field matures, gene therapy companies will eventually need to find a way to sell once-in-a-lifetime cures in a market built around chronic therapies. In some ways, its been easier for our European counterparts and other single-payer economies, [research analyst Mani] Foroohar comments. [They] can rest more assured that the financial benefits of reducing future hospitalizations due to expensive future illness will be captured by the same payor thats providing the upfront outlay.

Just as biotechnology has transformed every aspect of our healthcare system over the last 40 years, gene therapy will challenge, disrupt, and overturn our healthcare pricing and reimbursement paradigms as it becomes an increasingly common and routine treatment approach.

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Resurgence of gene therapy has dramatically altered the the biomedicine revolution - Genetic Literacy Project

Paris-based SparingVision, a genomic medicine company focused on ocular diseases, raised 44.5 million (approximately $52.2 million) in a financing round. Funds will be used to advance the development of the companys treatment for a genetic eye disorder that can lead to vision loss.

SparingVision is developing SPVN06 for the mutation-agnostic treatment of retinitis pigmentosa, the most common inherited retinal degeneration that affects about two million people globally. There is currently no approved treatment to treat all genetic forms of this rare retinal disease that leads to blindness, the company said. According to SparingVision, SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy consisting of one neurotrophic factor and one oxidative stress reducing enzyme which, acting synergistically, aim to slow or stop the degeneration of photoreceptors. Loss of photoreceptors leads to blindness in retinitis pigmentosa. In June, the European Commission granted Orphan Drug designation to SPVN06

In addition to advancing its gene therapy treatment, funds from the financing round will be used to support SparingVisions GMP activities, including the manufacturing of a first clinical batch of the product, as well as regulatory activities. Funds will also be used to begin human trials of the gene therapy, which are set to begin in 2021.

Gene therapy has already been approved by regulatory to approve a type of genetic blindness. Spark Therapeutics, now a division of Roche, won regulatory approval for Luxturna (voretigene neparvovec), a gene therapy for a rare, genetic form of blindness. Luxturna is approved for the treatment of pediatric and adult patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. The disease can lead to vision loss and may cause complete blindness in certain patients. The approval marked the first time the U.S. Food and Drug Administration approved a directly administered gene therapy that targets a disease caused by mutations in a specific gene.

In addition to developing its gene therapy for retinitis pigmentosa, SparingVision said it intends to establish a toehold in the United States and will expand its management team.

The financing round was led by 4BIO Capital and UPMC Enterprises. It was supported by Jeito Capital and Ysios Capital. Current investors Bpifrance and Foundation Fighting Blindness also participated in the round. Torreya Capital, LLC served as exclusive placement agent for the offering.

In addition to the financing, Stphane Boissel, who currently serves as chairman of the board of directors, was named chief executive officer of the company. He takes over from Florence Allouche, a cofounder of the company. Boissel, who previously served as head of corporate strategy at Sangamo Therapeutics, said the support SparingVision received in the financing round demonstrates the excitement about the potential of SPVN06.

With its singular mutation-agnostic approach, SPVN06 could have a much broader commercial potential than most gene therapy products for RP currently in development and will be used as an anchor to build an economically-viable portfolio of therapies in the field of ophthalmology. Our shareholders, both new and existing, are all long-term, strategic and patient-centric investors that share our vision and we are excited to be working with them to achieve our goals, Boissel said in a statement.

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SparingVision Nabs 44.5 Million to Support Gene Therapy, Adds New CEO - BioSpace

Luke Timmerman interviews Sana Biotechnology CEO Steve Harr at the GeekWire Summit this week.

Sana Biotechnology CEO Steve Harr shed more light on one of most secretive, heavily funded startups in Seattle and the global biotech industry detailing its plans to create tools that replace and repair human body cells, with the potential to treat various diseases and create new medicines.

Harr spoke with biotechnology journalist Luke Timmerman, founder of The Timmerman Report, this week at the GeekWire Summit. Sana raised more than $700 million this summer in one of the largest venture financing deals in the life sciences industry and one of the biggest rounds on record in Seattle.

Founded in 2019, the 250-person company has an ambitious goal of both repairing cells in the body (gene therapy) and also replacing damaged cells (cell therapy). Its led by several former executives from Juno Therapeutics, another Seattle biotech company that went public in 2014 and sold to Celgene for $9 billion in 2018.

Sana has kept a relatively low profile since launching. It is competing with much larger entities that have deeper pockets and more robust logistics capabilities. But Harr said a startup such as Sana has a key differentiator.

We have one competitive advantage: we can make faster and better decisions, he said. We get there because we have better people, we have greater focus, and we have better communication.

Read on for key takeaways from the conversation.

How Sana started:Harr and his former colleagues at Juno learned a lot about engineering cells and manipulating genes during their startup journey. Juno was among a handful of U.S. companies making cutting-edge cancer immunotherapy treatments.

But they also knew there was more opportunity in a nascent industry of gene and cell therapy.

We wanted to build the transformative or winning company of this next era, of the next 20 years, Harr said. To do that, we had to break the model of what biotech is, which is typically taking an idea and figuring out where to apply it best.

Sana instead is trying to build the platform that can engineer cells and fix them, much like building a computer.

There are a whole host of component parts that go into it, Harr said. We have to aggregate the right technologies.

Harr said too many biotech companies sell solutions in search of problems. He likened it to someone showing up with a tiny screwdriver and looking for a loose screw to fix. Harr sees Sana more as a toolbox that can help build the right medicine for the right patient.

Sana is targeting various disease areas, including cancer, diabetes, genetic disorders, and more. They are relatively diverse, but there are some really fundamental underlying platform and strategy principles that drive each of those, Harr said.

Sanas secret sauce:One key focus for the company is reimagining the delivery system for these therapies how to get DNA, RNA, proteins, etc. into a cell. Ultimately at the core, what were trying to do is really improve delivery, and really figure out how to hide cells from the immune system, Harr said.

Hiding re-engineered or replaced cells from ones immune system is important to prevent the possibility of the body rejecting the new cells.

Harr also talked about delivering therapy via injection, with the body becoming the bioreactor. Its similar to technology built by Moderna and others. You deliver the tools to enable your body to make its own medicine, Harr said.

Manufacturing:Sana is also aiming to innovate how gene and cell therapies are produced and distributed at scale. They are typically expensive Timmerman said CAR T-cell immunotherapies for cancer ran in the $300,000-to-$400,000 range per patient. Figuring out manufacturing costs at scale and making it less than current alternative methods of care for patients will be key to the strength of Sanas business. Harr added that you have to do it in a way thats constructive for the system.

Headcount: Sana employs 250 people spread across offices in Seattle, the Bay Area, and Cambridge, Mass. Having three outposts helps the company attract the best talent, Harr said. One advantage to raising so much capital is being able to hire the best folks. Last month Sana added top scientists Ed Rebar and Terry Fry to the executive team.

If you really hire one of the true world leaders in something, it is pretty amazing how quickly teams form around them, Harr noted.

Money matters: Having more than $700 million in the bank helps Sana in various other ways. Harr said a lot of biotech companies often run what amount to experiments to justify raising more capital. We have the privilege of running experiments to find truth as fast as you want to, he said. And then we want to have the balance sheet, technologies, and people to be able to grapple with whatever the truth is.

Timeline: Harr said the company is on track with its original strategy but does not plan on selling medicine in the next two years. It will progress with multiple medicines in parallel, not one at a time, Harr said.

Leadership advice:During the pandemic and remote work, Harr said hes started to reach out to four-to-six people at Sana each week that he wouldnt normally talk with. He holds half-hour meetings to chat about what they are working on, and what leadership could do to make their life or job better. I found that to be just such an invigorating way to learn whats going on, he said.

[The full interview with Harr, and other GeekWire Summit sessions, are available on-demand exclusively to attendees of the virtual event.Learn more and register here.]

Link:
Sana CEO reveals details about stealthy gene therapy startup that has raised more than $700M - GeekWire

NEW YORK, Oct. 22, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that it will host a virtual R&D Day on Friday, October 30, 2020 at 11:30 AM Eastern time, to discuss the Companys AXO-Lenti-PD gene therapy for Parkinsons disease.

Axovants Parkinsons disease R&D Day will be moderated by Chief R&D Officer, Gavin Corcoran, M.D., and will feature presentations on the current treatment landscape and unmet medical need for people living with Parkinsons disease from the following key opinion leaders:

In addition, the Company will present data from the second cohort of the Phase 2 SUNRISE-PD trial for AXO-Lenti-PD including:

Drs. Adler, Palfi, and Eberling will be joined by Dr. Corcoran to answer questions following the formal presentations.

AXO-Lenti-PD is the only investigational gene therapy for Parkinsons disease that delivers three genes via a lentiviral vector to encode a set of critical enzymes required for endogenous dopamine synthesis, with the goal of improving motor function and restoring steady, tonic levels of dopamine in the brain. The gene therapy aims to provide patient benefit for years following a single administration.

To register for the R&D webcast, please click here.

A live audio webcast of the R&D Day can be accessed through the Events & Presentations section of the company's website at investors.axovant.com. An archived replay of the webcast will be available on the company's website following the event.

Biographies of R&D Day Panelists:

Dr. Adler has received numerous grants to investigate experimental treatments for Parkinson's disease, essential tremor, dystonia, restless legs syndrome, and chronic traumatic encephalopathy (CTE). He serves as an advisory member to many different international medical societies such as the International Parkinson and Movement Disorder Society, MDS Industry Education and Services Committee, and the American Academy of Neurology Section of Movement Disorders. Dr. Adler has a commitment to education having trained residents, 14 fellows and graduate students, and has given many invited lectures. Dr. Adlers main research interests are investigating tissue diagnostic tests for Parkinsons disease, biomarkers for an early diagnosis of Parkinsons disease and PD with dementia, and identification of new treatments for PD and PD with dementia. He also has been investigating essential tremor, restless legs syndrome, and dystonia. He has published over 400 research papers and reviews, and edited a book entitled Parkinson's Disease and Movement Disorders: Diagnosis and Treatment Guidelines for the Practicing Physician. In 2006, Dr. Adler was awarded the Mayo Clinic Distinguished Investigator of the Year Award.

Dr. Palfi has published extensively on trophic factor- and enzyme-based gene therapy in Parkinsons disease and Huntingtons disease. He is a principal investigator on numerous preclinical and clinical studies and has been involved in studies of many novel agents including implanted brain devices, optogenetic, homeoprotein, trophic factors GDNF, CNTF and dopamine lentiviral vectors.

Dr. Eberling earned undergraduate and graduate degrees in biological psychology from the University of California at Berkeley, later moving to the Lawrence Berkeley National Laboratory where she developed expertise in neuroimaging techniques and gene therapy approaches for Parkinsons disease.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

Contacts:

Investors

Parag MeswaniAxovant Gene Therapies Ltd.(212) 547-2523investors@axovant.com

Media

Josephine Belluardo, Ph.D.LifeSci Communications(646) 751-4361jo@lifescicomms.commedia@axovant.com

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Axovant Gene Therapies to Host Virtual Parkinson's Disease R&D Day on October 30, 2020 - BioSpace

Biogen is cutting its experimental multiple sclerosis (MS) drug opicinumab as well as an spinal muscular atrophy (SMA) candidate amid a third-quarter clear-out.

First, to its MS drug. In its third-quarter financials posted Wednesday morning, the biopharma said: In October 2020 Biogen announced that the phase 2 AFFINITY study of opicinumab in MS did not meet its primary or secondary endpoints and that Biogen has discontinued development of opicinumab.

The anti-LINGO monoclonal antibody has experience with failure: All the way back in 2016, it posted a similar phase 2 flop after not hitting its primary and secondary endpoints for MS in the so-called SYNERGY trial.

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Back then, opicinumab was seeing whether it could have an effect on a multicomponent primary endpoint designed to assess ambulation, upper extremity function and physical disability in patients with relapsing forms of MS. Opicinumab, however, failed to outperform placebo in this regard.

A secondary efficacy endpoint, intended to evaluate the slowing of disability progression, also came up negative. But Biogen dug through the data, and, instead of culling it then and there, decided to carry on, saying: While we missed the primary endpoint, the SYNERGY study results suggest evidence of a clinical effect of opicinumab.

RELATED: Biogen tumbles on PhII MS flop, but refuses to write off drug

Biogen believed the data showed an increased clinical effect of opicinumab versus placebo (when used at the same time as interferon beta-1a intramuscular injection).

That led to the AFFINITY study, started in 2017, which looked at opicinumab as an add-on therapy in patients who are adequately controlled on their anti-inflammatory disease-modifying therapy (DMT), versus the DMT alone.

The primary endpoint of the study, overall response score, looked to assess the improvement and worsening of disability over time. Clearly, it failed to achieve this, though in its third-quarter update it did not reveal further details.

It also quietly announced it was throwing out BIIB089, an SMA gene therapy hopeful that had been on an IND hold due to dorsal root ganglion toxicity. Again, it did not any extra color to this cull in its financials.

The biopharma is now pinning its hopes on another failed drug, aducanumab, which it hopes can get past an FDA advisory committee early next month and a potential approval next year, though this controversial Alzheimers disease asset has been given low odds by analysts of managing that feat.

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Biogen finally culls MS dud opicinumab, adds SMA gene therapy to the garbage heap - FierceBiotech

WATERTOWN, Mass. and RESEARCH TRIANGLE PARK, N.C., Oct. 20, 2020 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc. (NASDAQ: SELB) and Asklepios BioPharmaceutical, Inc. (AskBio), today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to MMA-101 for the treatment of isolated methylmalonic acidemia (MMA) due to methylmalonyl-CoA mutase (MMUT) gene mutations. The FDA grants Rare Pediatric Disease Designation to incentivize development of new treatments for serious and life-threatening diseases that primarily affect children ages 18 years or younger with fewer than 200,000 people affected in the U.S. The Rare Pediatric Disease designation program allows for a Sponsor who receives an approval for a product to potentially qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

This Rare Pediatric Disease designation from the FDA highlights the significant unmet medical need that Selecta and AskBio are seeking to address with MMA-101 for this rare metabolic disorder, said Carsten Brunn, Ph.D., chief executive officer of Selecta Biosciences. When used with AAV gene therapy vectors, Selectas ImmTOR aims to inhibit the immune response to the AAV vector, potentially allowing re-dosing of gene therapies. Ongoing clinical programs will focus on evaluating product candidate performance in patients who may have been underdosed or those who may lose transgene expression over time. Were honored to receive this recognition and look forward to advancing this program in hopes of helping young patients affected by MMA and their families.

MMA is a serious and potentially life-threatening inherited metabolic disorder that presents in patients from newborns to adulthood, said Sheila Mikhail, J.D., CEO and co-founder of AskBio. AskBio is committed to delivering transformative genetic medicines for rare diseases like this one, and the Rare Pediatric Disease designation helps us continue development of MMA-101.

AskBio and Selecta expect to initiate a Phase 1 clinical trial of MMA-101 and ImmTOR for patients with MMA in 1H 2021.

About Methylmalonic AcidemiaMethylmalonic Acidemia (MMA) is a rare monogenic disorder in which the body cannot break down certain proteins and fats. This metabolic disease may lead to hyperammonemia and is associated with long-term complications including feeding problems, intellectual disability, chronic kidney disease and inflammation of the pancreas. Symptoms of MMA usually appear in early infancy and vary from mild to life-threatening. Without treatment, this disorder can lead to coma and in some cases death.

About Selecta Biosciences, Inc.Selecta Biosciences, Inc. (NASDAQ: SELB) is leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses. With a proven ability to induce tolerance to highly immunogenic proteins, ImmTOR has the potential to amplify the efficacy of biologic therapies, including redosing of life-saving gene therapies, as well as restore the bodys natural self-tolerance in autoimmune diseases. The companys first program aimed at addressing immunogenicity to AAV gene therapies is expected to enter clinical trials in early 2021 in partnership with AskBio for the treatment of methylmalonic acidemia (MMA), a rare metabolic disorder. A wholly-owned program focused on addressing IgA nephropathy driven by ImmTOR and a therapeutic enzyme is also in development among additional product candidates. Selecta recently licensed its Phase 3 clinical product candidate, SEL-212, in chronic refractory gout to Sobi. For more information, please visit http://www.selectabio.com. About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, fully integrated AAV gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. Its pipeline includes clinical-stage programs in Pompe disease and congestive heart failure and a diverse preclinical portfolio of therapeutics targeting neuromuscular, CNS and other diseases, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics, acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics, acquired by Pfizer). AskBios gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary third generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids.

Selecta Forward-Looking StatementsAny statements in this press release about the future expectations, plans and prospects of Selecta Biosciences, Inc. (the company), including without limitation, statements regarding the unique proprietary technology platform of the company, and the unique proprietary platform of its partners, the potential of ImmTOR to enable re-dosing of AAV gene therapy, the potential treatment applications of product candidates utilizing the ImmTOR platform in areas such as gene therapy and MMA, the companys plans to initiate a clinical trial for a product candidate to treat MMA, the ability of the company and AskBio to develop gene therapy products using ImmTOR and AskBios technology, any development plans of the company and AskBio have for product candidates to treat serious and life-threatening diseases and the intention to seek regulatory approval thereof, the novelty of treatment paradigms that the Company is able to develop, the potential of any therapies developed by the company and AskBio to fulfill unmet medical needs, the companys plan to apply its ImmTOR technology platform to a range of biologics for rare and orphan genetic diseases, the potential of the companys intellectual property to enable repeat administration in gene therapy product candidates and products, the ability to re-dose patients and the potential of ImmTOR to allow for re-dosing, the potential to safely re-dose AAV, the ability to restore transgene expression, the potential of the ImmTOR technology platform generally and the companys ability to grow its strategic partnerships, and other statements containing the words anticipate, believe, continue, could, estimate, expect, hypothesize, intend, may, plan, potential, predict, project, should, target, would, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including proof of concept trials, including the uncertain outcomes, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, the unproven approach of the companys ImmTOR technology, potential delays in enrollment of patients, undesirable side effects of the companys product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials, the companys inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the companys recurring losses from operations and negative cash flows from operations raise substantial doubt regarding its ability to continue as a going concern, substantial fluctuation in the price of its common stock, and other important factors discussed in the Risk Factors section of the companys most recent Quarterly Report on Form 10-Q, and in other filings that the company makes with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the companys views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any intention to update any forward-looking statements included in this press release.

AskBio Forward-Looking StatementsThis press release contains forward-looking statements regarding AskBio. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as believes, anticipates, plans, expects, will, intends, potential, possible and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding MMA-101, including the potential timing of the Phase 1 clinical trial for patients with MMA, AskBios pipeline of development candidates; AskBios goal of developing life-saving medicines aimed at curing genetic diseases; the potential benefits of AskBios development candidates to patients.

These forward-looking statements involve risks and uncertainties, many of which are beyond AskBios control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office.

Any of the foregoing risks could materially and adversely affect AskBios business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

For more information please contact:

Selecta: For Investors:Lee M. SternSolebury Trout+1-646-378-2922lstern@soleburytrout.com

For Media: Meredith Sosulski, Ph.D.LifeSci Communications, LLC+1-929-469-3851msosulski@lifescicomms.com

AskBio:Robin FastenauVice President, Communications+1-984-275-2705rfastenau@askbio.com

Link:
Selecta Biosciences and AskBio Receive FDA Rare Pediatric Disease Designation for their Gene Therapy for Methylmalonic Acidemia - GlobeNewswire

In anticipation of our Pfizer Gene Therapy Career Event scheduled for Wednesday, October 28, 2020, BioSpace spoke with Alison Ricci, Pfizers Senior Business HR Partner- Global Sciences, Business HR for Bioprocess Research and Development, and HR Site Lead for the companys four locations in Chapel Hill and Morrisville, North Carolina, Chesterfield, Missouri and Lake Forest, Illinois.

In late August, Pfizer announced it was investing an additional $500 million into its state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina. This is in addition to the companys continuing investment into gene therapy research and development and other areas going on throughout the company, but specifically in its North Carolina sites in Chapel Hill and Kit Creek. The companys presence in North Carolina currently exceeds 3,600 people, with 650 in Sanford. The expanded facility is expected to add over 100 new jobs.

Ricci told BioSpace the company has been investing heavily in gene therapy and in the Raleigh-Durham Research Triangle Park area. They have three facilities in and around the area that focus specifically on gene therapy, including a research facility in Morrisville, its existing clinical manufacturing site in Chapel Hill, and a very large manufacturing site in Sanford.

And in recognition of the continued investment and commitment to gene therapy, the company acquired a 60,000-square-feet building and 16 acres in Durham, which they are renovating into a clinical manufacturing site, essentially doubling our clinical manufacturing capacity.

*Rendering of Durham location

As such, the company has numerous positions in the area currently open with many more expected as the new site comes online.

Right now, we have 30 positions that were going to be advertising for that directly support gene therapy in those three different campuses, Ricci said.

The roles they will be looking to fill include what Ricci calls a multitude of individual contributor, team lead and management roles."

"For instance, weve got our R&D and analytical scientist roles; we have manufacturing engineering roles; we have technical specialist roles; and different types of manufacturing support and technology transfer roles, Ricci said.

They also have training specialist positions because there are a variety of supporting functions that go into the magic of making gene therapy work.

Ricci seemed excited about gene therapy and the part it is playing increasingly in Pfizers core mission. Speaking of Duchenne muscular dystrophy (DMD), increasingly a target of gene therapies, she noted how exciting and important it is to be able to provide a therapy that extends quality of life, that doesnt just treat symptoms, but that potentially marks a minimization of those symptoms and the elongation of life. Thats one of the central core components of who Pfizer is. Our patients are our North Star.

She also emphasized not only the science and motivation of Pfizer as an employer, but its culture.

People typically equate large organizations with a level of bureaucracy, that results in less risk and less innovation opportunities, Ricci said. In my five years with Pfizer Ive seen the culture shift to focus on innovation, inclusion, quality and smart resourcing figuring out how to free up resources so we can focus on bold moves and exciting new therapies allowing our scientists to stretch and grow, and make a difference for the patients that count on us.

The company has also structured rewards and recognition around those types of approaches, not only for people theyre bringing into the organization, but with its current staff.

Were still maintaining our focus on a combination of productivity and doing it right the first time while encouraging innovation, taking bold moves and trying new things," Ricci said. "Because thats the best way that good science is done, and patients win in the process.

She also notes that the Pfizer Gene Therapy Career Event is a great opportunity to have direct contact not only with Pfizer hiring managers, but with colleagues who are working in those same roles theyre hiring for.

Its a unique experience in terms of really understanding who Pfizer is and what we have to offer, Ricci said.

And, of course, the company is hiring throughout its worldwide operations in support of not only its COVID-19 clinical research trials and manufacturing processes, but in the numerous other areas the company focuses on.

The expansion in Sanford, North Carolina is only a part of the companys focus on building out capacity. It has committed about $5 billion in U.S.-based capital projects over the next several years.

But the North Carolina area is expanding end-to-end capabilities in gene therapy. The Kit Creek facility focuses on small scale production, from 2L flasks up to 250L bioreactors to develop the early processes that will later be used in larger scale manufacturing. The Chapel Hill facility is where the process is optimized, and staffers work at a 250L scale while developing and implementing quality control measures that include Good Manufacturing Practice (GMP) standards. The Sanford facility is designed for high-quality, efficient supply of gene therapies at clinical- and commercial-scale.

Ricci also points to Paul Mensah, Pfizers Vice President of Bioprocess Research and Development.

He has a nice, pragmatic style and approach in terms of how he continues to foster and leverage innovation, but also the science of how everyone works together to collaborate and deliver. He instills that in the leaders on the team. Ricci said.

Were continuing to focus in terms of career development and the aspirations of each colleague. There are opportunities for colleagues at Pfizer to do anything they want within the realms of the organization." Ricci said. "You can be on the frontlines of early stage development, you can work on the late stage, you can have opportunities for clinical manufacturing. You can run the gamut in terms of different roles with the scientific profile within Pfizer.

Originally posted here:
Pfizer: Expanding North Carolina Gene Therapy Facility and Adding Jobs - BioSpace

Irvine, Calif., October 19, 2020 A breakthrough study, led by researchers from the University of California, Irvine, results in the restoration of retinal and visual functions of mice models suffering from inherited retinal disease.

Published today in Nature Biomedical Engineering, the paper, titled, Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing, illustrates the use of a new generation CRISPR technology and lays the foundation for the development of a new therapeutic modality for a wide range of inherited ocular diseases caused by different gene mutations.

In this proof-of-concept study, we provide evidence of the clinical potential of base editors for the correction of mutations causing inherited retinal diseases and for restoring visual function, said Krzysztof Palczewski, PhD, the Irving H. Leopold chair and a distinguished professor in the Gavin Herbert Eye Institute, Department of Ophthalmology at the UCI School of Medicine. Our results demonstrate the most successful rescue of blindness to date using genome editing.

Inherited retinal diseases (IRDs) are a group of blinding conditions caused by mutations in more than 250 different genes. Previously, there was no avenue available for treating these devastating blinding diseases. Recently, the FDA approved the first gene augmentation therapy for Leber congenital amaurosis (LCA), a common form of IRD which originates during childhood.

As an alternative to gene augmentation therapy, we applied a new generation of CRISPR technology, referred to as base editing as a treatment for inherited retinal diseases, said first author Susie Suh, assistant specialist in the UCI School of Medicine Department of Ophthalmology. We overcame some of the barriers to the CRISPR-Cas9 system, such as unpredictable off-target mutations and low editing efficiency, by utilizing cytosine and adenine base editors (CBE and ABE). Use of these editors enabled us to correct point mutations in a precise and predictable manner while minimizing unintended mutations that could potentially cause undesirable side effects, said co-first author Elliot Choi, also an assistant specialist in the UCI Department of Ophthalmology.

Using an LCA mouse model harboring a clinically relevant pathogenic mutation in the Rpe65 gene, the UCI team successfully demonstrated the therapeutic potential of base editing for the treatment of LCA and by extension other inherited blinding diseases. Among other results, the base editing treatment restored retinal and visual function in LCA mice to near-normal levels. Base editing was developed at the Broad Institute of MIT and Harvard in the lab of David Liu, PhD.

After receiving treatment, the mice in our study could discriminate visual changes in terms of direction, size, contrast and spatial and temporal frequency, said Palczewski. These results are extremely encouraging and represent a major advance towards the development of treatments for inherited retinal diseases.

Gene therapy approaches to treating inherited retinal diseases are of special interest given the accessibility of the eye, its immune-privileged status and the successful clinical trials of RPE65 gene augmentation therapy that led to the first US Food and Drug Administration-approved gene therapy. Now, as demonstrated in this study, base-editing technology can provide an alternative treatment model of gene augmentation therapy to permanently rescue the function of a key vision-related protein disabled by mutations.

This research was supported in part by grants from the National Institutes of Health, the Research to Prevent Blindness Stein Innovation Award, Fight for Sight, the Eye and Tissue Bank Foundation (Finland), The Finnish Cultural Foundation, the Orion Research Foundation, the Helen Hay Whitney Foundation, US Department of Veterans Affairs, and a Research to Prevent Blindness unrestricted grant to the Department of Ophthalmology, University of California, Irvine.

About the University of California, Irvine: Founded in 1965, UCI is the youngest member of the prestigious Association of American Universities. The campus has produced three Nobel laureates and is known for its academic achievement, premier research, innovation and anteater mascot. Led by Chancellor Howard Gillman, UCI has more than 36,000 students and offers 222 degree programs. Its located in one of the worlds safest and most economically vibrant communities and is Orange Countys second-largest employer, contributing $5 billion annually to the local economy. For more on UCI, visit http://www.uci.edu.

Media access: Radio programs/stations may, for a fee, use an on-campus ISDN line to interview UCI faculty and experts, subject to availability and university approval. For more UCI news, visit news.uci.edu. Additional resources for journalists may be found at communications.uci.edu/for-journalists.

About the UCI School of Medicine: Each year, the UCI School of Medicine educates more than 400 medical students, and nearly 150 doctoral and masters students. More than 700 residents and fellows are trained at UCI Medical Center and affiliated institutions. The School of Medicine offers an MD; a dual MD/PhD medical scientist training program; and PhDs and masters degrees in anatomy and neurobiology, biomedical sciences, genetic counseling, epidemiology, environmental health sciences, pathology, pharmacology, physiology and biophysics, and translational sciences. Medical students also may pursue an MD/MBA, an MD/masters in public health, or an MD/masters degree through one of three mission-based programs: the Health Education to Advance Leaders in Integrative Medicine (HEAL-IM), the Leadership Education to Advance Diversity-African, Black and Caribbean (LEAD-ABC), and the Program in Medical Education for the Latino Community (PRIME-LC). The UCI School of Medicine is accredited by the Liaison Committee on Medical Accreditation and ranks among the top 50 nationwide for research. For more information, visit som.uci.edu.

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UCI-led study reveals significant restoration of retinal and visual function following gene therapy - UCI News

MALVERN, Pa., Oct. 22, 2020 (GLOBE NEWSWIRE) -- Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases, today announced Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen, will speak on the importance of strategic partnerships in gene therapy including product consistency, identifying the right partner as well as providing examples of successful partnerships at the 4th Annual Gene Therapy for Rare Disorders Europe Conference from October 26-28, 2020 presented digitally.

Presentation Details:Title:The Importance of Strategic Partnerships in Gene TherapyDate: Wednesday, October 28, 2020Time: 11:00 AM (GMT)Location: DigitalRegister: https://genetherapy-europe.com/take-part/register/

About Ocugen, Inc.Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug one to many and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. For more information, please visit http://www.ocugen.com.

Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (the SEC), including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release.

Corporate Contact:Ocugen, Inc.Sanjay SubramanianChief Financial OfficerIR@Ocugen.com

Media Contact: LaVoieHealthScience Katie Gallagherkgallagher@lavoiehealthscience.com+1 617-792-3937

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CEO Dr. Shankar Musunuri to Discuss the Importance of Gene Therapy Strategic Partnerships at 4th Annual Gene Therapy for Rare Disorders Europe...

Wishing on a star is one of the most magical rites of childhood:

Star light, star bright,The first star I see tonightI wish I may, I wish I might,Have the wish I wish tonight.

But for one little Canadian boy named Sam, that simple activity was beyond his reach. Thats because 8-year-old Sam couldnt see the stars.

Sam suffers from the rare disorder retinitis pigmentosa, a form of progressive blindness caused by genetic retinal degeneration that results from mutations in the RPE65 gene.

You lose perception of light, Dr. Elise Heon, of Sick Kids Hospital, explained to CTV News. You end up in darkness and [its] slowly progressive, its relentless, your visual field shrinks and shrinks and shrinks and shrinks.

Sams sight was extremely limited, especially at night. Images most people take for grantedthe stars, an airplane streaking across the sky, or even our own shoeswere beyond the scope of his vision. Until recently, there was no effective treatment for his condition.

RELATED:Scientists Use New Holy Grail Gene Therapy to Heal Damage Caused By Heart Attacks and it Could Save Millions

Now, however, thanks to a new form of gene therapy, many patients, including Sam, are seeing huge improvements in their eyesight. The science behind the protocol is impressive.

After being modified with a healthy copy of the gene, an inactivated virus is injected directly into the retina. (Each eye is injected only once.) The healthy gene then goes to work, enabling cells to produce a protein that converts light into electrical signals, which in turn, facilitates improved vision and prevents further progression of the disease.

The targeted gene therapy protocol, developed in the U.S., was recently green-lit for use in Canada, but with Sams sight failing, he and his mom, Sarah Banon, traveled to America last year to get him treatment.

Within a weeks time, Banon began to notice progress and says Sams condition has continued to improve over the course of the year since he underwent the procedure.

She reports her son has gained incredible confidence. He dresses without help. Hes able to see, even when its dark, and he no longer requires lights on when its cloudy outside.

MORE: In World First, Gene Therapy Trial Restores Vision in Patients With Genetic Form of Blindness

Now he is able to function as a normal child, she told CTV. This is a story of hope A child told it is what it is, and now, when he looks up at night, he can see stars.

And when Sam wishes on those stars, hell know in his heart that sometimes, wishes really can come true.

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8-Year-old Sees Stars for the First Time After His Blindness is Treated With Gene Therapy - Good News Network

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Oct. 21, 2020 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company focused on developing adeno-associated virus (AAV) based gene therapies for the treatment of rare inherited diseases, today announced that Sue Washer, President & Chief Executive Officer, and Dave Knop, PhD, Vice President, Process Development and Manufacturing, will participate in the Cantor Fitzgerald Key Considerations for Gene Therapy Manufacturing and Platforms virtual panel at 10:00 a.m. ET on Wednesday, October 28, 2020.

The panel will focus on the critical components and capabilities to gene therapy manufacturing, including a discussion on AGTCs proprietary manufacturing processes associated with AAV, and key company catalysts expected over the next 12 months. The call will be moderated by Kristen Kluska, Biotechnology Research Analyst of Cantor Fitzgerald.

Presentation Information:Panel: Key Considerations for Gene Therapy Manufacturing and PlatformsDate: Wednesday, October 28, 2020Time: 10:00 a.m. 11:30 a.m. ET

To register for the event, please click here.

About AGTCAGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Companys most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its pre-clinical programs build on the Companys industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

IR/PR CONTACTS:David Carey (IR) or Glenn Silver (PR)Lazar FINN PartnersT: (212) 867-1768 or (646) 871-8485david.carey@finnpartners.com or glenn.silver@finnpartners.com

Corporate Contact:Bill SullivanChief Financial OfficerApplied Genetic Technologies CorporationT: (617) 843-5728bsullivan@agtc.com

Stephen PotterChief Business OfficerApplied Genetic Technologies CorporationT: (617) 413-2754spotter@agtc.com

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AGTC to Participate in Cantor Fitzgerald Virtual Panel: Key Considerations for Gene Therapy Manufacturing and Platforms - GlobeNewswire

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input theyre looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and its clear from the first round of posted responses that some of the top players including J&J and Pfizer would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Bo Cumbo jumps from the top commercial post at Sarepta to the helm of a gene therapy startup with some influential backers, big plans and $107M -...

Blindness is a global problem where a person cannot see anything, including light. Researchers find novel ways like gene therapy to impart vision, and they initially experimented with mice. They claimed to restore vision in totally blind mice with the help of gene therapy.

Opsins are a group of proteins, that is light sensitive through the presence of chromophore retinal found in the photoreceptor cells of the retina. These opsins only covalently bind to vitamin-A based retinaldehyde in both rods and cones. This starts a cascade of signals when the light activates the photoreceptor cells in the retina. This causes pulsations, which propagates by creating an impulse through the optic nerve to the brain.

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Several disease conditions like macula degeneration, retinitis pigmentosa were causing damage to the photoreceptor cells, which in turn leading to blindness.

Read Also: The water industry can be made more sustainable by a better understanding of biofilms

Scientists said that they can still restore vision even if the photoreceptor cells are impaired. This is by a group of neurons, which we say bipolar cells, can help to evade blindness. When photoreceptor cells failed, other neuronal cells would remain intact.

This is possible when we insert the MCO1 opsin gene to the intact bipolar cells in the retina, which is downstream to the photoreceptors. Hence, the ability to see objects or light enhances.

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This gene therapy could overcome other treatments such as replacement of opsin, invasive surgery, and all that. Also, this treatment is effective in causing 20/60 vision in patients. Clinical trials are necessary to depict a clear picture of showing how it works when it comes to fast-moving objects, and whether it affects vision quality.

Experiments with mice did not yield any harmful effects upon the administration of genes. If everything goes well, they can successfully implement the treatment, and it will definitely be an alternative to the retinal prosthesis such as retinitis pigmentosa.

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Source: Medical Xpress

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Gene therapy to restore vision in mice - News Landed

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Avrobio has licensed a Hunter syndrome lentiviral gene therapy from the University of Manchester in the U.K. The deal positions Avrobio to join Regenxbio and Sangamo Therapeutics in the race to develop gene therapies for use in patients with the rare disorder.

Hunter syndrome, also known as mucopolysaccharidosis Type II, is driven by a mutation that limits a patients ability to break down sugar molecules generated as a byproduct of cell activity. The buildup of the molecules causes progressive damage to organs such as the brain, affecting the development of children with the lysosomal disorder.

Takedas approved drug Elaprase gives Hunter syndrome patients the enzyme they need to break the molecules down, but its inability to cross the blood-brain barrier renders it ineffective against the CNS manifestations of the disease.

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Avrobio has become the latest company to identify gene therapy as a potentially better way to treat Hunter syndrome. The biotech, which is active in other lysosomal disorders, has agreed to pay the University of Manchester $8 million upfront for the global rights to a stem cell gene therapy that is set to enter the clinic in the second half of next year.

Brian Bigger, Ph.D., a professor of cell and gene therapy at the U.K. university, developed the candidate and co-authored a paper about it in 2018. The paper describes work on a braintargeted hematopoietic stem cell gene therapy designed to give patients the lysosomal enzyme iduronate-2-sulfatase (IDS).

Avrobio will make the therapy, now called AVR-RD-05, by modifying a patients own hematopoietic stem cells with a transgene for IDS expression and a protein tag intended to improve stability of the enzyme. If AVR-RD-05 works as hoped, the cells will engraft in the bone marrow and make copies of themselves that also carry the transgene.

The differentiation of the cells into components of the immune system could enable AVR-RD-05 to cause a lasting, body-wide increase in the presence of the enzyme at the root of Hunter syndrome. Avrobio will fund a 9.1 million ($11.8 millon) investigator-sponsored phase 1/2 trial to assess whether the gene therapy performs as hoped. The biotech is also on the hook for up to $80 million in milestones.

Avrobio will face competition if it gets AVR-RD-05 all the way to market. Takeda is the incumbent, Regenxbio and Sangamo have gene therapies in the clinicalthough the latter has underwhelmed so farand Denali Therapeutics is developing an IDS enzyme designed to cross the blood-brain barrier.

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Avrobio bags gene therapy to join Regenxbio and Sangamo in race - FierceBiotech

Professor Robert MacLaren on the exciting potential of CRISPR gene editing and patients seeing stars for the first-time following treatment

Professor Robert MacLaren, of Oxford University, is leading work to develop gene therapies for inherited retinal conditions through Nightstar Therapeutics. The company was acquired in 2019 by US biotechnology company, Biogen, for $US 877 million.

One night I was at my computer and a patient emailed me to say he could see a night star. This was a positive thing. I said, If you are still there can you go and check which eye it was. He came back and said, It is definitely the eye that I had the gene therapy in.

Subsequently, when we developed the gene therapy company, the name NightStar was an obvious one because since then several of our patients have described seeing night stars after gene therapy. It symbolizes looking up at the heavens and hope for the future, but there is also that practical element of achieving better vision at night.

Although I am an ophthalmologist treating patients, when we are looking at the mechanisms of the disease, we get right down to the molecular level, looking at how the DNA is assembled and how it is processed.

It is very exciting as a physician in any specialty to be able to treat disease at the genetic level. Ultimately if the disease occurs at the genetic level and you treat it there, you are treating the origin of the disease itself. If you are just giving drugs to try and correct the genetic disease or compensate for it, it may help but that is not a real treatment. It provides insight into the mechanisms of life itself seeing how DNA is processed and how the genes evolve.

You can treat potentially any disease with that, whereas gene replacement therapy is limited to small genes. This is still good but ideally if you correct the mutation it is a better treatment than replacing the whole gene.

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Gene therapy: It provides insight into the mechanisms of life itself - AOP

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.

If approved, Lenti-D (elivaldogene autotemcel or eli-cel) could transform the prospects of people with CALD, the most severe form of the neurodegenerative disease ALD that usually emerges in boys during early childhood and causes physical and mental disabilities as well as behavioural problems.

Around 40% of patients develop the cerebral form of ALD, which in turn affects around one in 17,000 live births.

A few weeks ago, Bluebird reported new data from the phase 2/3 STARBEAM trial of Lenti-D which showed that 87% of CALD patients were still alive and free of major functional disabilities after at least two years follow-up.

The EU filing comes ahead of a filing for eli-cel in the US, which Bluebird says should take place sometime towards the middle of next year, having been delayed by the coronavirus pandemic.

If approved, eli-cel would provide a one-shot treatment for CALD, holding back the progressive breakdown in the protective myelin that sheathes neurons.

It would be the first alternative to a stem cell transplant to treat the disease, a therapy that can provide significant improvements and even halt progression in some patients if given early enough.

However it requires high-dose chemotherapy to destroy the bone marrow, and that poses significant risks to patients in its own right, and can also lead to graft-versus-host disease, a potentially life-threatening complication in which the bone marrow donors immune cells attack the recipients cells and tissues.

CALD is caused by mutations in the ABCD1 gene located on the X chromosome, which provides instructions for the production of the ALD protein.

ALD protein is needed to clear toxic molecules called very long-chain fatty acids (VLCFAs) in the brain, and if mutated causes the VLCFAs to accumulate and damage the myelin sheath.

Using eli-cel, the patients own stem cells are modified in the lab to produce a working version of the ABCD1 gene, producing functional ALD protein that can help to flush VLCFAs from the body.

CALD is a devastating disease, often marked by rapid neurodegeneration, the development of major functional disabilities, and eventual death, said Gary Fortin, head of severe genetic disease programmes at Bluebird.

If approved, eli-cel would represent the first therapy for CALD that uses a patients own haematopoietic stem cells, potentially mitigating the risk of life-threatening immune complications associated with transplant using cells from a donor, he added.

Aside from STARBEAM, which will follow treated patients for up to 15 years, Bluebird is also conducting the phase 3 ALD-104 trial of eli-cel in CALD, which is due to generate results in 2024.

The EU filing for eli-cel comes shortly after Bluebirds development partner received a 27 March 2021 FDA review date for anti-BCMA CAR-T cell therapy ide-cel, a potential therapy for multiple myeloma.

The biotech already has approval in Europe for Zynteglo, a gene therapy for haematological disease beta thalassaemia, and is due to file its related therapy LentiGlobin for sickle cell disease next year. The two therapies have been tipped to generate $1.5 billion-plus in peak sales by some analysts.

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EMA starts rapid review of Bluebird's gene therapy for rare disease CALD - - pharmaphorum

The FDA has lifted the clinical hold on a phase 1/2 clinical trial of Solid Biosciences gene therapy treatment for Duchenne muscular dystrophy (DMD). Solid Bio secured clearance to resume dosing in the trial after making manufacturing changes to cut the number of viral particles given to patients.

SGT-001, the adeno-associated viral (AAV) vector-mediated gene transfer therapy being tested in the phase 1/2 trial, has suffered a series of setbacks since entering the clinic, most recently when the FDA put the study on hold in response to a case of acute kidney injury. The FDA imposed the hold 11 months ago. In July, Solid Bio said the FDA wanted to see more data before lifting the hold.

The request led Solid Bio to share further information on its gene therapy manufacturing process and its latest safety and efficacy data. The additional information proved sufficient to persuade the FDA to lift the clinical hold.

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Solid Bio will resume dosing using a gene therapy made under a revised manufacturing process. The new process is intended to remove most empty viral capsids, thereby enabling Solid Bio to cut total viral load without reducing the dose. The focus on viral load reflects concerns systemic delivery of AAV vectors can damage organs and cause inflammation.

To get the FDA to lift the hold, Solid Bio shared data from a quantitative, in vitro microdystrophin expression assay designed to show the comparability of SGT-001 manufactured under the old and new processes. Solid Bio shared those results in response to the FDAs request for information in July.

The biotech is taking other precautions to manage the potential risk posed by SGT-001. Solid Bio has capped the maximum weight of the first two patients to receive SGT-001 after the hold lifts at 18 kg. As the dose of SGT-001 is determined by weight, heavier patients receive more vector genomes. The adverse events seen in some gene therapy trials, such as the deaths in Audentes Therapeutics trial, have happened in patients who were heavier and therefore received a higher viral load.

Solid Bio is further mitigating the potential for SGT-001 to cause harm by amending the protocol to include the prophylactic use of eculizumab, the anti-complement inhibitor sold by Alexion as Soliris, and C1 esterase inhibitor, while also increasing the prednisone dose in the month after treatment.

The protocol changes position Solid Bio to resume its pursuit of DMD gene therapy leader Sarepta Therapeutics, which suffered a setback of its own last month when the FDA asked it to use an extra potency assay in a planned clinical trial. Pfizer is also in the race but, like Solid Bio, has run into safety issues that could give Sarepta an edge.

Shares in Solid Bio, which had slumped to $2 apiece, rose 70% in response to the end of the hold.

Link:
FDA lifts clinical hold on Solid Bio gene therapy trial - FierceBiotech

ALDERSON, W.Va. (WVVA) A Greenbrier County teen is about to become one of the first five patients in history to receive an experimental gene therapy for Hurler's Syndrome.

The Alderson native is in many ways your typical 13-year-old. "I like to help my dad feed cows....and donkeys," said Kendra Goins.

But life hasn't always been easy for Kendra. The extremely rare condition makes it impossible for her body to breakdown certain sugars. In addition to causing damage to her organs, the condition makes it difficult for her body to grow.

But whenever anyone has anything to say about it, her sister, Kiristen is always the first time stand up.

"Me with my big head is always the one to jump in," said Goins, who said she has gotten into quite a few quarrels over the issue at school in defense of her sister.

She worries though that she won't be able to jump in when Kendra heads to California soon for a clinical trial. Kendra is heading into the treatment with her first bone marrow transplant nearing the end of its course.

"I've spoken with experts across the country from the chemists who made the drug to the doctor who has used it. This looks like her golden ticket," said Kendra's mother Sheryl Goins.

Administered through the brain, the goal of the gene therapy is to help her body produce the enzymes she needs to survive.

While the cost of the clinical trial and airfare is covered, the family said they need help with expenses they will incur during their three-month stay.

To learn more about how you can help, visit https://www.gofundme.com/f/kendra039s-gene-therapy?utm_source=facebook&utm_medium=social&utm_campaign=p_cp%20share-sheet&fbclid=IwAR3yzHCexch5a_awjaYy06ijB28zMXJ-72WkfQ-SPEwMQgy5s8x_carlz34

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Greenbrier County teen to be among first in nation to receive gene therapy for Hurler's Syndrome - WVVA TV