StemCell Therapy

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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Premium Insights of Gene Therapy Market Size-Share Analysis and System Production (2020-2026) | Addressing the Potential Impact of COVID-19 by ZMR -...

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

Oct. 5, 2020 08:09 UTC

HARLOW, England--(BUSINESS WIRE)-- Diamond Pharma Services (Diamond), a leading technical services and regulatory affairs consulting group, has announced that it provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight Biologics (GenSight), including the preparation, authoring support, agency communication and submission of GenSights first Marketing Authorisation Application to the European Medicines Agency (EMA), for its novel ocular gene therapy LUMEVOQ. The EMA decision is expected in H2 2021.

LUMEVOQ (Lenadogene nolparvovec) is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary Optic Neuropathy (LHON) caused by mutation in the ND4 mitochondrial gene. LHON mainly affects young males, and the ND4 mutation results in the worst visual outcomes, with most patients becoming legally blind. There is a high unmet medical need for LHON patients, of which there are 800-1200 in the EU and the US annually.

Headquartered in Paris, France, GenSight is a biopharma company focused on developing and commercialising innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Diamond has provided GenSight with regulatory, pharmacovigilance, quality and compliance support for LUMEVOQ leading up to the MAA assessment.

Maureen Graham, Managing Director, Regulatory, Diamond Pharma Services, said: We are pleased GenSight Biologics chose to work with our team of experts at Diamond to provide European Regulatory, Pharmacovigilance, Quality and Compliance support for LUMEVOQ, its first MAA submission, and the first for a gene therapy treating a mitochondrial disease. It has been a personal ambition of mine to have the opportunity to work on a gene therapy within the ophthalmic arena, and GenSight has allowed Diamond that opportunity and that privilege.

This submission adds to Diamonds broad experience in providing support to companies developing advanced therapy medicinal products (ATMPs), which includes over 50 programmes at various stages of development, and two MAA approvals - Glybera and Yescarta.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201005005339/en/

Original post:
Diamond Pharma Services Supports GenSight Biologics in Submitting Its First Marketing Authorisation Application, for Ocular Gene Therapy LUMEVOQ -...

The Food and Drug Administration (FDA) has granted Fast Track designation to the investigational gene therapy candidate, PF-06939926 (Pfizer), for the treatment of Duchenne muscular dystrophy (DMD).

PF-06939926 is a recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of human dystrophin gene under the control of a human muscle-specific promoter. The Company has chosen the rAAV9 capsid due to its potential to target muscle tissue.

The designation was based on data from an ongoing phase 1b study evaluating the safety and tolerability of a single intravenous infusion of PF-06939926 in 9 ambulatory boys with DMD aged 6 to 12 years. Preliminary results showed that PF-06939926 was well tolerated during the infusion period and dystrophin expression levels were sustained over a 12-month period.

The Company plans to launch a double-blind, placebo-controlled phase 3 study to evaluate the efficacy and safety of PF-06939926 in boys with DMD. The study will include patients who are at least 4 years old and less than 8 years old; all participants will need to be on a daily dose of glucocorticoids for at least 3 months prior to enrolling and to stay on daily glucocorticoids for the first 2 years of the study. The primary outcome of the study (change from baseline in North Star Ambulatory Assessment) will be assessed at 52 weeks; patients will be followed for 5 years after treatment.

The FDAs decision to grant our investigational gene therapy PF-06939926 Fast Track designation underscores the urgency to address a significant unmet treatment need for Duchenne muscular dystrophy, said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development. We are working to advance our planned phase 3 program as quickly as possible.

The FDAs Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.

For more information visit pfizer.com.

Pfizer receives FDA Fast Track designation for Duchenne muscular dystrophy investigational gene therapy. https://www.businesswire.com/news/home/20201001005382/en/Pfizer-Receives-FDA-Fast-Track-Designation-for-Duchenne-Muscular-Dystrophy-Investigational-Gene-Therapy. Accessed October 2, 2020.

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Investigational Gene Therapy Fast Tracked for Duchenne Muscular Dystrophy - Monthly Prescribing Reference

NEW YORK, Oct. 05, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that the Company will present at upcoming conferences and will announce six-month safety and efficacy data from the second cohort of its AXO-Lenti-PD program the morning of Tuesday, October 6, 2020. Additionally, as previously announced the Company will hold a Parkinsons Disease R&D Day on Friday, October 30.

Information on the upcoming presentations can be found below:

Chardan 4th Annual Genetic Medicines Conference

2020 ARM Virtual Cell and Gene Meeting on the Mesa

AXO-Lenti-PD Parkinsons Disease R&D Day

A live webcast of the presentations will be available in the Events section of Axovant's website at http://www.axovant.com. Replays will be available for approximately 30 days following the conferences.

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:

Media & Investors

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

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Axovant to Participate in Upcoming October Conferences and Provide AXO-Lenti-PD Program Update the Morning of October 6th - BioSpace

Global Cancer Gene Therapy Market: Overview

Cancer could be defined as uncontrolled cell growth in the body leading to organ malfunction. If untreated, it can lead to death. Uncontrolled growth of cell is managed by the body in several ways, one of them is by deploying white blood cells to detect and eradicate these cancerous cells. It has been discovered that the immune system could be manipulated to influence cancerous cells to destroy itself.

Read Report Overview https://www.transparencymarketresearch.com/cancer-gene-therapy-market.html

Radiation and chemotherapy therapy have consistent and reliable effects to decrease cancerous cells in the body. Recently, immunotherapy for hematological cancers has experienced a recognition and is of interest for many researchers Scientists have developed methods to isolate, replicate, and develop cancer-destroying cells from the patients blood cancer and injecting those cells back for the destruction of their cancers, with durable remissions.

New options for the treatment is needed to be developed if order to achieve elimination of cancer suffering and death by 2020. According to NCI, 5-year survival rate for cancers such as lung (15%), glioblastoma (5%), pancreatic (4%), and liver (7%) remains very low. Current available treatments have several side effects, the systemic toxicity due to chemotherapy results in nausea, mild cognitive impairments, and mouth ulcerations, in addition to long-term side effects such as increasing risk of developing other types of cancers. Therefore, new and innovative treatment methods are required to reduce the suffering of cancer patients.

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Global Cancer Gene Therapy Market: Drivers and Restraints

The emerging field of cancer Gene Therapy offers varied potential treatments. Gene therapy involves a range of treatment types, which use genetic material to alter cells (either in vivo or in vitro) to help cure the disease. Cancer Gene Therapy shown efficacy in various in vitro and preclinical testing. Preclinical testing for cancer gene therapy has been performed on glioma, pancreatic cancer, liver cancer, and many other cancers.

Increase in prevalence of cancer, rise in government funding and initiatives, growth in pipeline of cancer gene therapy products, and collaborations to develop and launch gene-therapy products are some factors driving the market. According to NCBI researchers, development of genetically-modified T-cell therapies for treatment of cancer has had maximum clinical impact among other gene therapies. However, high treatment cost is a major limitation in the cancer gene therapy market. The reason behind the huge cost for cancer gene therapy is the necessity of rigorous, exhaustive clinical trials; also treatment by cancer gene therapy differs from person to person depending upon the genetic acceptance of every patient, unlike other drugs thereby limiting the market growth.

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Global Cancer Gene Therapy Market: Key Segments

Based on type, the cancer gene therapy market is segmented into gene transfer immunotherapy and oncolytic virotherapy. Immunotherapy uses genetically modified cells and viral particles to stimulate the immune system to destroy cancer cells. Immunotherapy include treatment with either cytokine gene delivery or tumor antigen gene delivery. Oncolytic virotherapy uses viral particles, which replicate within the cancer cell causing the death of the cell. It is an emerging treatment modality that is expected to shows great promise, particularly in metastatic cancer treatment. It includes treatment with adenovirus, retrovirus, lentivirus, herpes simplex virus, adeno-associated virus, simian virus, alphavirus, and vaccinia virus. Gene transfer is the newest treatment modality that is expected to introduce new modified genes into cancerous cell or associated tissue for destruction of cell or to slow down cancer growth. This technique is flexible as a wide variety of vectors and genes are used for clinical trials with positive outcomes. As gene therapy advance, they could be used alone or in combination with other treatments to control the disease. Gene transfer or gene replacement is performed using naked/plasmid vectors, electroporation, sonoporation, magnetofection, and gene gun.

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Based on region, the global cancer gene therapy market is segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa. North America is anticipated to hold the largest market share. The U.S. dominates the cancer gene therapy market owing to its increase in funding for research & development and other government initiatives. Key players in the biotech industry are engaging in research & development of gene therapy products. Moreover, rising demand for DNA vaccines and growing interest of venture capitalists to investment in commercialization of gene-based cancer therapies are likely to propel the market. The cancer gene therapy market in Asia Pacific is anticipated to expand at a rapid pace as in China cancer gene therapy is anticipated to attribute for largest revenue, due to the recent launch of Gendicine and rising healthcare expenditure with strong R&D facilities.

Global Cancer Gene Therapy Market: Key Players

Key players operating in the global cancer gene therapy market are Adaptimmune, ZioPharm Oncology Altor Bioscience, MolMed, bluebird bio, Shanghai Sunway Biotech company limited , MultiVir, Shenzhen SiBiono GeneTech, Corporation.

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Cancer Gene Therapy Market is Expected to Expand at an Impressive Rate by 2025 - The Daily Chronicle

CAMBRIDGE, Mass., Oct. 05, 2020 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, announced today that Phillip Samayoa, vice president of strategy and portfolio development, will present a company overview at the annual Cell & Gene Meeting on the Mesa. The presentation will be available for registered participants to view on demand throughout the conference, to be held virtually Oct. 12-16, at meetingonthemesa.com.

Geoff McDonough, M.D., Generation Bios president and chief executive officer, will participate in a panel on the future of gene delivery. That discussion will be available to view on demand on the conference website starting Oct. 13.

About Generation Bio

Generation Biois an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is designed to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The companys efficient, scalable manufacturing process supports Generation Bios mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.For more information, please visitgenerationbio.com.

Contact:

InvestorsChelcie ListerTHRUST Strategic Communicationschelcie@thrustsc.com910-777-3049

MediaStephanie SimonTenBridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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Generation Bio to Present at 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire

Nearly five months after snagging a conditional approval for its spinal muscular atrophy (SMA) gene therapy in Europe, Novartis is rolling out fresh data to support a launch that has seen its share of challenges.

The company unveiled interim data from a phase 3 trial of the gene therapy in babies born with the genetic disease. Of the 33 patients treated so far in the European trial, 21 had achieved milestones in motor skills during a mean follow-up period of 10.6 months that the disease would normally prevent, the company said. For example, six could sit without assistance for more than 10 seconds and 20 could control their head movements.

Most of the children in the study who entered it without requiring ventilation remained free of ventilatorsupport, and 67% were able to eat without help, Novartis said during the World Muscle Society 2020 Virtual Congress. Most of the children received a higher score on a widely used test of neuromuscular functioning than untreated SMA patients typically do, the company added.

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Shephard Mpofu, M.D., chief medical officer of Novartis Gene Therapies, said in a statement that the data boosts previous clinical trial results supporting the use of Zolgensma in patients with type 1 SMA, the most common form of the disease. With more than 600 patients now treated, including some more than five years post-treatment and more than five years old, these data further reinforce the transformative benefit a one-time dose of Zolgensma has on SMA patients, Mpofu said.

RELATED: New Zolgensma 'inflection point' is here as Novartis snags EU nod for SMA gene therapy

Novartis effort to win approval for Zolgensma in Europe ran into one hurdle back in April 2019, when an infant in the trial died of brain damage and respiratory issues. An autopsy later revealed that the death was unrelated to Zolgensma.

But then, in October of last year, Novartis was hit with questions from regulators in Europe and Japan about the Zolgensma manufacturing process. Resolving the queries from the EU required inspections, and it pushed back thedecision from the EUs Committee for Medicinal Products for Human Use (CHMP).

Now that Zolgensma is finally approved in Europe, though, Novartis has an opportunity to steal market share from Biogens SMA drug Spinraza. Thats because the European approval allows the Zolgensma to be used in children weighing up to 21 kilograms, which basically covers any child under the age of 5.

Thats a significant difference from the FDA approval, which only covers children under 2 years of ageand it could allow Novartis to offer its therapy to older children currently taking Spinraza.

RELATED: Roche touts 2-year Evrysdi data as oral SMA drug drives early interest

Novartis has an ambitious expansion plan for Zolgensma in the U.S., too, though that has hit some obstacles recently. Its working on a new intrathecal formulation of the drug, in the hopes that the FDA will approve that version for children up to 5 years old. But last week, the company said the FDA will require it to run a phase 3 study of the intrathecal formulation, rather than considering it for approval based on a smaller phase 1/2 trial. That could push the filing to 2023.

Analysts deemed the FDAs decision a major setback. Jefferies analysts had estimated the gene therapy would peak at $2.8 billion in worldwide sales, with the intrathecal version claiming $1 billion of that.

Meanwhile, Novartis is facing potential competition from more than just Spinraza. Roche introduced an oral drug for SMA, Evrysdi, last month. And it just revealed new data from a pivotal trial of that drug that intensify Novartis marketing challenge.

In the Evrysdi study, 88% of infants with type 1 SMA were alive without needing full-time ventilation after two years. Their motor functioning also improved in the second year, with more patients able to sit, stand, turn over or maintain head control.

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Novartis cites 'transformative' data on Zolgensma as it rolls out SMA gene therapy in Europe - FiercePharma

SAN RAFAEL, Calif., Oct. 2, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a pioneer in developing treatments for phenylketonuria (PKU) and gene therapies, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation toBMN 307, an investigational gene therapy for the treatment of individuals with PKU.

Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need, enabling drugs to reach patients earlier. Clinical programs with Fast Track designation may benefit from early and frequent communication with the FDA throughout the regulatory review process. These clinical programs may also be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, which means that completed sections of the Biologic License Application can be submitted for review before the entire FDA application is complete. Both the FDA and European Medicines Agency have granted BMN 307 Orphan Drug Designation.

"Fast Track designation combined with our ability to conduct our clinical studies incorporating material manufactured using a commercial-ready process will further facilitate rapid clinical development of BMN 307 gene therapy," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "We are looking forward to working closely with the FDA, as well as other health agencies, to evaluate the safety and efficacy of this promising investigational gene therapy as we continue our unwavering 15-year commitment to advance the standard of care for people with PKU."

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is commonly found in many foods. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological issues, affecting a person's ability to think and problem solve, and can lead to depression, anxiety, and behavior disturbance impacting quality of life. Due to the seriousness of these symptoms, in many countries, infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to treatment guidelines, PKU patients should maintain lifelong control of their Phe levels.

BMN 307 Clinical Program

Last week, BioMarin announced that it had dosed the first participant in the global Phearless Phase 1/2 study with BMN 307, an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, increase plasma Phe levels, and enable a normalization of diet in patients with PKU.BioMarin is conducting this study with material manufactured with a commercial-ready process to facilitate rapid clinical development and potentially support approval. BMN 307 represents a potential third PKU treatment option in BioMarin's PKU franchise and a second gene therapy development program.

BioMarin's clinical program is composed of two key studies. Phearless, a Phase 1/2 study, will evaluate the safety, efficacy, and tolerability of a single intravenous administration of BMN 307 in patients with PKU. The study consists of a dose-escalation phase, followed by a cohort expansion phase once an initially efficacious dose has been demonstrated. In addition, BioMarin is sponsoring an observational study, Phenom, which includes patients with PKU to measure both established and new markers of disease and clinical outcomes over time.

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has developed therapies that have been used to treat approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, and the investigational gene therapy BMN 307 is currently in development. BioMarin has conducted 41 clinical studies in PKU and has sponsored 44 external clinical studies. BioMarin researchers have authored 65 publications in medical and scientific journals on PKU and supported another 57 publications by external researchers.

About Gene Therapy

Gene therapy is a form of treatment designed to address a genetic problem by adding a normal copy of the defective gene. The functional gene is inserted into a vector containing a small DNA sequence that acts as a delivery mechanism, providing the ability to deliver the functional gene to targeted cells. The cells can then use the information from the normal gene to build the functional proteins that the body needs, potentially reducing or eliminating the cause of the disease.

Gene Therapy Manufacturing

BioMarin has leveraged its knowledge and experience in manufacturing complex biological products to design, construct and validate a state-of-the-art vector production facility in Novato, California. This facility is the site of production for both valoctocogene roxaparvovec and BMN 307, investigational gene therapies. Manufacturing capabilities are an essential driver for BioMarin's gene therapy programs and allows the Company to control quality, capacity, costs and scheduling enabling rapid development. Production of BMN 307 with a commercial ready process at scale reduces risk associated with making process changes later in development and may speed overall development timelines significantly.

Ongoing process development efforts and experience gained at commercial scale have led to improvements in productivity and operational efficiency. The ability to scale out the facility with additional equipment combined with the improvements in productivity result in a doubling of overall potential capacity to 10,000 doses per year, combined for both products, depending on final dose and product mix. This improvement in productivity is anticipated to meet potential commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future.

About Phenylketonuria

PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases.The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.For additional information, please visitwww.biomarin.com. Information on such website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the Company's BMN 307 program being eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, the development of BioMarin's BMN 307 program generally, including the impact on the timing and process for regulatory interactions and decisions, BioMarin's gene therapy manufacturing capabilities and the anticipation that the current manufacturing capabilities will meet potential commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future and the impact of using material manufactured at commercial scale in a clinical trial on reducing risk and speeding up overall development timelines. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others:the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; uncertainties inherent in research and development, including unfavorable new clinical data and additional analyses of existing clinical data; the results and timing of current and future clinical trials related to BMN 307; our ability to reproducibly and consistently manufacture sufficient quantities of BMN 307, the possibility that changes may be required to the current manufacturing process; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

Contacts:

Investors

Media

Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

SOURCE BioMarin Pharmaceutical Inc.

https://www.biomarin.com/

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BioMarin, Pioneer in Phenylketonuria (PKU) and Gene Therapy, Receives FDA Fast Track Designation for PKU Investigational Gene Therapy, BMN 307 -...

Gene therapy is no longer an approach for the future. It's a technique used now.

As of January 2020, the FDA has approved four gene therapies for use in the United States and has received more than 900 investigational new drug (IND) applications for clinical studies. At present, more than 3,400 active gene therapy trials are taking place worldwide, according to ClinicalTrials.gov.

None of these trials would have launched without valid preclinical research. One of the key requirements during preclinical research is selection of the appropriate animal model.

Selecting animal models that reflect the population studied and reproduce the target disease state increases the likelihood that studies will meet strict regulatory expectations. Further, strong preclinical research, with accurate data and clinically relevant biomarkers, helps ensure that clinical trials get to the finish line on time and on budget.

"If you choose a too simplistic model or a model that does not accurately recapitulate the disease state you're trying to address, your preclinical data doesn't amount to much,"said Anjli Venkateswaran, PhD, spokesperson for Biomere, a preclinical CRO based in Worcester, Massachusetts. "Selection becomes even more important in gene therapy research, and it's something scientists grapple with daily."

Gene therapy researchers rely on animal models to assess variables such as safety, efficacy, dosage and localization of transgene expression. Traditional inbred and outbred mice the most common laboratory mice are suitable for most pharmaceutical research. However, when the goal is to alter a human genetic defect, scientists need to test their approach in models that contain the human target sequences. This led to the development of genetically engineered mouse models (GEMMs) of which there are many.

"Any mouse that is genetically changed or altered to be an appropriate model for disease falls under this umbrella,"Dr. Venkateswaran said. "These models express the gene target and recapitulate some, if not all, of the disease pathophysiology."

As animal model providers gain access to next-generation sequencing, genome-engineering tools and other technology, they can develop more customized models. "Older technology was based on homologous recombination,"said Tom Pack, PhD, senior scientist for Axovant, a New York City-based clinical-stage gene therapy company. "Now we've moved into an era of CRISPR-Cas9, where you have more efficient and sophisticated technology for manipulating DNA, and you can more closely mimic human mutations. You can also focus on specific organs or tissue types with DNA recombinase-based and intersectional genetics approaches to design more targeted therapies more rapidly."

For all the technological advances, certain studies may require a different type of model. "There are limitations as to how much you can humanize mice,"Dr. Venkateswaran said. "Also, mice behave very differently physiologically from humans, and they have a different blood-brain barrier, which can result in differences in gene therapy delivery to the brain. Because of their anatomical differences, large-animal models are emerging in gene therapy."

Large-animal models, including nonhuman primates, may suit certain studies more specifically than humanized mice. For example, pig models have been used effectively in cystic fibrosis studies because their respiratory is more similar to humans than mouse models.

However, large-animal models have limitations. They are more expensive than mouse models and require specialized scientists and technicians and adequate lab space. That's why Dr. Venkateswaran recommends that, generally, researchers should start small and move up.

"You need to get some confidence in your gene therapy's performance,"she said. "When data looks good in in vitro models and in mouse models, then, depending on the therapy, consider testing in relevant large animal models."

Axovant has two pediatric rare-disease studies in the pipeline: both fatal diseases with no other treatment options available. These delicate situations require Axovant to be especially thoughtful about model selection.

"You not only want to mimic the genetic condition, you want to be able to practice the same surgical techniques you would use to deliver the therapy as well as look at some of the same clinical biomarkers,"Dr. Pack said. "With a larger-animal model, you can optimize everything clinically before you start clinical trials."

To determine the appropriate animal model(s) for preclinical gene therapy studies, researchers must weigh both scientific and practical considerations.

Scientific considerations include:

Practical considerations include:

Gene therapy researchers have a lot to consider when designing preclinical research. Appropriate animal model selection is one of the first and most important steps to help move that research into clinical trials.

Fortunately, a select group of CROs have preclinical research expertise to help expedite the process. That includes access to complex genetically modified mouse models and other animal models, as well as scientific experts that understand the biology of these models. Many CROs, including Biomere, also have housing and breeding programs and experience with testing multiple types of therapies (CRISPR-mediated gene editing, AAV and other viruses, RNA therapeutics (RNAi andmiRNA), ceDNA, antisense oligos etc.).

"When you get it right in preclinical studies, you improve the success rate in the clinic,"Dr. Venkateswaran said. "A more thoughtful approach will lead to more robust therapies, which ultimately leads to more patient lives saved." To learn more about model selection for gene therapy studies, download Biomere's white paper.

Need a preclinical partner for a gene therapy study? Visit Biomere.com to contact one of its scientists about your preclinical study today.

Meyerholz DK. Lessons learned from the cystic fibrosis pig.Theriogenology. 2016;86(1):427-432. doi:10.1016/j.theriogenology.2016.04.057

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Preclinical done right: The importance of using relevant animal models in gene therapy studies - BioPharma Dive

Gene therapy - which aims to treat diseases by essentially inserting a gene into a patients cells rather than via drugs or surgery - was a hot trend in the biotech space, but investor interest in the sector appears to have died down considerably, on account of high R&D spending and delays companies have seen in launching revenue-generating products. Our indicative theme of Gene-Based Therapy Stocks is down by about -23% year-to-date, underperforming the S&P 500 which is up by about 2%. However, with valuations declining, these companies could be attractive bets for investors as years of investments potentially start to pay off. These companies could also be acquisition candidates for big pharma. Below is a bit more about these companies and how they have fared this year.

Sarepta Therapeutics is a commercial-stage biopharmaceutical company that develops RNA-targeted therapeutics and gene therapy products. The company recently provided some positive data on its investigational gene therapy for Duchenne muscular dystrophy. The stock is up 11% year-to-date.

SRPT

Voyager Therapeutics is a clinical-stage biotech company that is developing gene therapies for Parkinsons disease, Huntingtons disease, and other conditions. The stock is down by about -20% year-to-date.

REGENXBIO Inc.: is a clinical-stage biotechnology company working on gene-based therapies for Retinal diseases, Hunter and Hurler syndromes. The stock is down by about -33% this year.

uniQure is primarily focused on gene-based therapy for Hemophilia and is currently in the late-stage of clinical trials and another program focuses on Huntingtons disease. The stock is down by about -49% year-to-date.

What if youre looking for a more balanced portfolio instead? Heres a high-quality portfolio to beat the market, with over 100% return since 2016, versus 50% for the S&P 500. Comprised of companies with strong revenue growth, healthy profits, lots of cash, and low risk, it has outperformed the broader market year after year, consistently.

See allTrefis Price EstimatesandDownloadTrefis Datahere

Whats behind Trefis? See How Its Powering New Collaboration and What-Ifs ForCFOs and Finance Teams |Product, R&D, and Marketing Teams

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Gene Therapy Stocks Continue To Underperform. Are They Worth A Look? - Forbes

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A question hanging over gene therapies, which attempt a once-and-done fix for genetic diseases, is will their benefits endure? Monday, Sarepta Therapeutics told a scientific conference that the first four children who got its investigational gene therapy, for Duchenne muscular dystrophy, all continued to show better-functioning muscles after two years.

Durability is an important consideration for a onetime treatment, said Sareptas head of gene therapy, Louise Rodino-Klapac. She spoke on a conference call, part of an online version of the annual congress of the World Muscle Society, where scientists reported their progress against the often-deadly muscular dystrophies.

Sarepta (ticker: SRPT) said that a battery of tests that measure daily functions like walking and fatigue showed the children doing even better than they had at the one-year mark. The assessment is an average of 17 tests, and not every child showed improvement on every measure.

Perhaps that is why Sarepta stock slipped 0.5% on the day, to $143.20, in a rising stock market.

Analysts may have also been anxious to hear whether Sarepta had resolved questions raised by the U.S. Food and Drug Administration about the production process to be used in a Phase 3 study of the gene therapy. Agency demands have slowed the timetables of other gene- therapy developers, and its questions for Sarepta are delaying the launch of its pivotal trial.

Chief executive Doug Ingram said Sarepta was ready with a process to produce therapies for a clinical trial and subsequent commercial demand. Were working with the agency right now to resolve any questions that they might have, said Ingram, get their blessing and be starting that trial as soon as is possible.

RBC Capital analyst Brian Abrahams found the data encouraging. He rates Sarepta stock at Outperform, with a price target of $200 a share.

We view the results as continuing to support true functional benefits from the companys gene therapies, Abrahams said in a Monday note. These studies werent controlled, he cautioned, so investors are looking forward to Sareptas results from a randomized, controlled study for which results should be available in the first quarter of 2021.

On Sareptas heels is Pfizer (PFE), with clinical trials of a gene therapy for muscular dystrophy whose benefits it has measured out to one year.

At the conference, Sarepta also showed data from early-stage trials of a gene therapy for another group of inherited muscle diseases, known as Limb-girdle muscular dystrophies. Functional tests of the first three patients showed them maintaining their improvement over 18 months.

CEO Ingram reminded the audienceand perhaps any listening regulatorsthat families are impatient for these muscular dystrophy trials conclusions.

This is truly -- Im not being hyperbolic, but I say in a very real sense, at least from our perspective, a matter of life and death, he said. We have to get this trial started. Kids are waiting for this therapy.

Write to Bill Alpert at william.alpert@barrons.com

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Sareptas First Gene-Therapy Test Patients Look Good, 2 Years Out - Barron's