Category Archives: Gene therapy

Merit has completed construction on the 16 million expansion of Cell and Gene Therapy Catapults manufacturing centre in Stevenage, doubling the capacity of the facility which plays a crucial role in bringing advanced cell and gene therapies to patients.

Merit used its advanced offsite technology and specialist expertise in delivering controlled cleanroom environments to engineer pre-assembled modules at its manufacturing centre in Cramlington, UK. This reduced the impact of works as well as overall build time, allowing existing GMP manufacturing at the Stevenage site to continue without interruption.

Tony Wells, managing director, Merit, said: Our close collaboration with the team at CGT Catapult was key to the successful delivery of this complex project. We worked alongside them throughout the programme using our proven expertise in next generation cleanroom manufacturing technology and advanced offsite fabrication capabilities to deliver high-quality cleanrooms that meet the stringent standards of the technical specification.

Backed by over 75 million of funding, including investment from the UK Governments Industrial Strategy Challenge Fund; the department of Business Energy and Industrial Strategy, from Innovate UK, the UKs innovation agency, and from the European Regional Development Fund, the centre is providing the infrastructure and expertise to enable companies to develop their manufacturing capabilities and systems for large scale, commercial cell and gene therapy supply. Companies currently collaborating at the centre are Adaptimmune, Autolus, Cell Medica, Freeline Therapeutics and TCR2 Therapeutics.

Keith Thompson, CEO, Cell and Gene Therapy Catapult, said We are very pleased to be able to double the existing capacity of the CGT Catapult manufacturing centre with help from our partners Innovate UK and the European Regional Development Fund. We would like to thank Merit for supporting the build out of this phase, it has been rapid using pre-prepared fallow space and off-site prefabricated building techniques. Thanks to their expertise the new capacity will come online quickly, leveraging the existing MHRA licences, installed quality and operational systems alongside the expertise developed by the Catapult and our collaborators at the centre.

Merit is one of the UKs leading engineering and construction specialists and prides itself on the ability to design and construct controlled environment solutions for the bioscience and pharmaceutical sectors. The company operates from offices across the UK with its in-house team of designers and manufacturing experts who are all specially trained in highly regulated industries where the quality of workmanship is vital.

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Expansion of 16m Cell and Gene Therapy Manufacturing Centre completed - BQ Live

To develop Ocugens orphan drug designated gene therapy candidate in its modifier gene therapy platform

Ocugen, Inc., a UK based clinical stage biopharmaceutical company focused on innovative therapies that address rare and underserved eye diseases, has entered into a strategic partnership with China based CanSino Biologics on Ocugens gene therapy pipeline product candidates for inherited retinal diseases, which are currently in development with Schepens Eye Research Institute of Massachusetts Eye and Ear, an affiliate of Harvard Medical School.

Under this strategic collaboration, CanSinoBIO will provide all CMC development and clinical supplies for the development of OCU400, Ocugens first gene therapy product candidate in its modifier gene therapy platform. CanSinoBIO maintains the option to support commercial manufacturing for Ocugen. The agreement also provides commercialization rights to CanSinoBIO in Greater China.

OCU400 has received two different orphan drug designations (ODD) from the U.S. FDA. The first, for the treatment ofNR3E3mutation-associated retinal degeneration and, most recently, for the treatment ofCEP290mutation-associated retinal disease.

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CanSino partners with Ocugen to work on gene therapy - BSA bureau

An encounter with gene therapy in action inspires Andrew Lewis to believe that healing a sick planet is within reach but depends on all of us.

At a recent North Bay Bob Dylan tribute, I happened to meet a woman who worked as a biochemist at a Bay Area pharma outfit. She manufactures blood proteins used to treat hemophilia. But as we talked, she excitedly announced that one day her work would go away.

She explained that blood coagulant requires 12 distinct proteins. The genome of hemophiliacs, it turns out, are unable to manufacture protein eight. But its now possible to engineer a virus that contains the missing DNA sequence. And if we introduce the virus to hemophiliac marrow, the DNA of the infected marrow will repair and gain the ability to manufacture the missing protein. It all sounded miraculous and strange - voodoo even.

Musicians picked up their guitars, took seats at the keyboards and drum kit and the lilting chime of Mr. Tambourine man filled the room.

Fifteen months ago, a young Greta Thunberg left school and held a sign outside the Swedish Parliament. She stood alone. Skolstrejk fr Klimatet her sign read. Hardly a year later, millions of young people in hundreds of cities and communities in every corner of the globe gathered to strike for climate change.

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Gene Therapy for the Planet | Perspectives | Perspectives - KQED

Three new animal-model tests of a potential gene therapyfor oculopharyngeal muscular dystrophy (OPMD), known as BB-301, are planned, Benitec Biopharmaannounced. Results are expected tosupport a request for a Phase 1 clinical trial in patients.

OPMD results from a faulty PABPN1 gene, leading to a protein that can form insoluble clumps linked with muscle weakness.

BB-301 uses a DNA-directedRNA interference(ddRNAi) strategy. Delivered via a modified, harmless adeno-associated virus (AAV), the therapy is intended to silence and replace the mutant protein. It suppresses its production and provides a source of normal, or wild-type, protein to target cells.

A 2017 study showedthat this approach significantly reduced the amount of PABPN1 aggregates, restored muscle strength and eased muscle fibrosis (scarring) in mice. Subsequent experiments in cells from OPMD patients confirmed these results.

The preclinical research, to be conducted in a canine disease model, will help to optimize methods of administration, confirm the efficiency of the AAV vector in introducing a healthy gene into key muscle cells, assess optimal dosing, and further characterize toxicological data necessary for regulatory filings and clinical trial design.

According to the company, these three planned dog studies will support the submission of an Investigational New Drug (IND) application, an essential step in getting regulatory approval to start the trial. Pre-IND application meetings with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and Health Canada took place in 2017.

Through our continued focus on BB-301 for the treatment of [OPMD], our team has an unprecedented opportunity to develop a novel genetic medicine that could facilitate clinically meaningful patient benefit, Jerel A. Banks, MD, PhD, Benitecs executive chairman and CEO, said in a press release.

The preclinical studies will be done in partnership with a medicine and surgery team with several decades of experience in OPMD treatment, Benitec added.

The FDA granted orphan drug status to BB-301 as a potential OPMD treatment in January 2018. The EMA awarded it similar status in early 2017.

Jos is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimers disease.

Total Posts: 307

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

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Final Tests Planned into Potential OCMD Gene Therapy, Could Support Trial in Patients - Muscular Dystrophy News

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Newswise When Zion Grant was 3 months old his mother Daisy Dodd noticed he wasnt tracking her with his eyes. Zion would fixate on a light in the room, but not on her face.

Genetic testing confirmed Zion hadLeber congenital amaurosis, a rare eye condition that usually starts in childhood and worsens over time. Cells in the eye crucial for vision diminish, and by age 20, mostwith thisform of retinal dystrophy are legallyblind.

But Zions diagnosis would come during a period of hope and progress in the field of gene therapy aimed at curing blindness.

Progress in treating Leber congenital amaurosis is something physicians and families have long worked for through research andclinical trials.

Leber congenital amaurosisamaffects the retina, the tissue in the back of the eye that detects light and color. The condition occurs in 2 to 3 out of 100,000 newborns in the United States, according to theNational Institutes of Health.

Vision problems associated with the eye condition include night blindness and diminished visual fields.

With the boys diagnosis in mind, in 2014, Zions family moved from Tennessee to Michigan to establish a relationship with theretinal dystrophy clinicat theUniversity of Michigan Kellogg Eye Center, where clinical trials were underway and new treatments were on the horizon.

Kellogg is one of just 10 centers in the country to offerLuxturna, which in 2017 became the first genetherapy approved by the U.S. Food and Drug and Administration for an inherited disease.

In clinical trials, the treatment produced dramatic results, restoring the ability of patients to see things they couldnt see before, like stars in the night sky and their parents faces.

Moving back to Michigan and being introduced to the new therapy was a sign that we were in the right place, says Dodd. Our care team at Kellogg gave Zion the chance to see like a normal kid again, and we couldnt be more grateful.

Cagri Besirli, M.D., Ph.D.is Zions doctor and a Kellogg ophthalmologist who specializes in pediatric retinal illnesses.

These therapies are provided to our youngest patients so they do not have to endure more vision loss later in life, says Besirli. For many families, this genetic therapy gives them hope for the future.

The gene therapy process

Gene therapy provides a working copy of a gene responsible for producing a protein that makes light receptors work in the eye.

The gene therapy approved by the FDA was developed for those with a mutation in the RPE65 gene. Treatment involves surgery to inject a functioning copy of the gene directly under the retina using a fine needle. Zion received the first injection in early January and the second later that month.

I knew I had to do what was best for my kid, and giving him the best sight possible was what Zion needed the most, says Dodd. Thats why we went forward with the Luxturna treatment.

Kari Branham, M.S., a genetic counselor at Kellogg, worked with Zions family to help themunderstand the genetic basisfor Zions condition.

We have seen such amazing progress with these conditions over the last 15-20 years,says Branham. We used to tell patients and their families that we would have to wait and see what happens, but now we can actually do something to help.

By going through the gene therapy process, Branham says the team is hopeful that this has changed Zions prognosis.

The treatment is designed to stop or slow the death of specialized cells in the retina, called photoreceptors, that send visual information to the brain.

Patients who have Leber congenital amaurosis have night blindness, says Besirli. One of the first treatment effects after receiving Luxturna is that (patients) are telling us that they function much better in dark.

They can play outside much longer, they can navigate around the house and dont need nightlights anymore and can participate in indoor sports. Thats been a huge change in their lives.

Changing the trajectory

Seven months after treatment, Zion, now age 7, and his family are back to their normal routine in Montrose, Mich., and monitor his progress during follow-up appointments at Kellogg.

Zion says hes looking forward to playing football and, with improved vision -- playing outside at night with his brothers.

We hope that with Zion we have changed the trajectory for him to the point that in his 20s he wont have significant vision loss we see with him now, says Branham.

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7-year-old receives FDA-approved gene therapy - Newswise

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NORFOLK, Va. -Kaeli Prices piercing cry is a joyful noise to her mom and dad.

She'll be screaming right in your face and all we can think is that we're just so thankful that we can hear her now, said Kelli Price, Kaelis mother.

When Kaeli was born in March, you could barely hear her cry and more signs signaled something wasnt right.

We started noticing she wasn't holding her head up like some babies would normally do, said her mother. So we started to worry a little bit.

Their pediatrician in Belvidere, North Carolina began to worry, too. He sent them to see Dr. Crystal Proud, a pediatric neurologist at Childrens Hospital of The Kings Daughters. The doctor specializes in Spinal Muscular Atrophy. Its the top genetic killer of infants.

The babies that were diagnosed with this disorder passed away from losing the ability to breathe because those muscles became so weak, explained Dr. Proud.

The doctor said the source of the disorder is a missing or mutated gene which keeps the babies muscles from developing. Most diagnosed with SMA die by the age of two. Ten babies a year are diagnosed with the deadly condition in Virginia. At just eight weeks old, testing confirmed Kaeli Price was one of them.

We were overwhelmed, said Kaelis mother.

We felt powerless, said Brandon Price, Kaelis father.

Dr. Proud started Kaeli on a drug called Spinraza, which has shown promise in babies with SMA since it was approved by the FDA in 2016. It requires an injection into their tiny bodies every four months for the rest of their lives.

Not long after Kaelis initial treatment on Spinraza, a newly approved gene therapy drug called Zolgensma hit the market. It would essentially give Kaelis body the missing gene, and it would only require one dose. However, the drug came with a price tag of $2.1 million. The Price familys insurance covered it, allowing Kaeli to be the first baby in Virginia to be treated with Zolgensma under FDA approval.

I can't even describe how thankful we are, said Mrs. Price.

I hope and I expect that she will sit, stand, and walk. She will go through school and just excel, said Dr. Proud. And at some point well down the road she will have her own family that she can tell the story to.

Zolgensma has come under scrutiny because of its multi-million dollar price tag.

Dr. Proud, who was part of the clinical trials for the gene therapy drug, said it is actually cost effective when you weigh the billions of dollars it takes for clinical trials and the medical bills a child would incur without the drug.

Dr. Proud said beginning this year, all newborns in Virginia will be tested for SMA. The earlier doctors can intervene, preferably within the first few weeks of life, the better their chances for treatment.

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Gene therapy drug priced at $2 million saves North Carolina babys life - WTKR News 3

RESEARCH TRIANGLE PARK Fennec Pharmaceuticals Inc. has appointed Jodi Cook to its board of directors the company announced in a statement Tuesday.

Cook is currently the head of gene therapy for PTC Therapeutics Inc., a global biopharmaceutical company. Before beginning her work at PTC, she helped found Agilis Biotherapeutics, a clinical-stage company focused on gene therapies, where she also served as chief operating officer.

Jodi Cook

On behalf of the board of directors, we welcome theaddition of Jodi Cook, saidKhalid Islam, Fennecs chairman, in a press release. She brings extensive scientific, clinical and executive business experience to the Company. Her background and track record of success will enhance our team as we further advance the commercialization and development strategy of PEDMARK.

PEDMARK is a therapy intended to help children with hearing loss as a result of platinum-based chemotherapy. There is currently no preventative measure for this type of hearing loss. Fennec aims to complete its NDA submission for the treatment in early 2020 with a potential commercial launch of PEDMARK in the second half of 2020.

I am delighted to be joining Fennecs board of directors at this key point of development for PEDMARK, said Cook in the press release. Cisplatin induced hearing loss is a significant unmet medical need, a solution to which has evaded medicine for decades.

This story is from the North Carolina Business News Wire, a service of UNC-Chapel Hills School of Media and Journalism.

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Fennec Pharma names gene therapy exec to its board - WRAL Tech Wire

BASEL, Switzerland, Sept. 25, 2019 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that Parag Meswani, Pharm.D., senior vice president, commercial strategy & operations, will present at the annual Cell & Gene Meeting on the Mesa at 4:30 PM PT on October 3, 2019 in Carlsbad, California.

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a three-day conference featuring more than 80 dedicated company presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies, as well as over 100 panelists and featured speakers.

A live video webcast of all company presentations will be available at: http://www.meetingonthemesa.com/webcast and will also be published on the conference website shortly after the event. Please visit http://www.meetingonthemesa.com for full information including registration.

About AxovantAxovant, part of the Roivant family of companies, is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurological and neuromuscular diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including 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, visitwww.axovant.com.

About RoivantRoivant Sciences aims to improve health by rapidly delivering innovative medicines and technologies to patients. It does this by building Vants nimble, entrepreneurial biotech and healthcare technology companies with a unique approach to sourcing talent, aligning incentives, and deploying technology to drive greater efficiency in R&D and commercialization. For more information, please visitwww.roivant.com.

Contacts:Media and InvestorsParag V. Meswani, Pharm.D.(212) 547-2523media@axovant.cominvestors@axovant.com

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Axovant Gene Therapies to Present at 2019 Cell & Gene Meeting on the Mesa - GlobeNewswire

DUBLIN, Sept. 20, 2019 /PRNewswire/ -- The "Global & US Gene Therapy Market Forecast to 2020" report has been added to ResearchAndMarkets.com's offering.

Gene therapy is one of the most widely researched fields in the healthcare industry. The high potential of gene therapy to cure various chronic diseases makes it a popular research area. Various researches are being performed across the globe to explore the potential of gene therapy for the treatment of incurable diseases, such as diabetes, cancer, and HIV amongst others.

According to this new research report "Global & US Gene Therapy Market Forecast to 2020, most of the gene therapy researches are being focused on finding the treatment for cancer, followed by genetic diseases and neurological disorders, respectively. In this context, the gene therapy application chapter of the report provides a comprehensive overview of various diseases in which the gene therapy is used, along with the current and future market size of gene therapy for particular disease and its geographical break up. Primarily, the gene therapy market is dominated by oncology applications, with several companies and academic institutions focusing on novel and difficult to treat' cancers. Other therapeutic areas seeking developments in gene therapy include monogenic diseases, cardiovascular diseases, infectious diseases, inherited blindness and neurological diseases. Furthermore, the chapter also provides details regarding the various aspects of the clinical trials in the different gene therapy application by phases, vector types, etc.

The major part of the revenue of gene therapy market is generated from research phase. Most of the gene therapy products are in research phases, only few products have been commercialized till date. The report also provides the sales of major marketed gene therapy products, and the list of the products in clinical/pre-clinical research along with their clinical phases.

The companies operating in the gene therapy market are also receiving various funding, grants, and investment from government bodies and venture capitalist firms which are aiding them to develop new products. The study highlights the applications for which the investments have been received.

North America continues to have the maximum number of clinical trials in the gene therapy segment. This is a major reason for the dominant position of North America in the gene therapy market. Based on the geography, the market is divided into four regions, namely, North America, Europe, Asia, and Rest of the World. The report provides the market for each of the geography, along with its forecast till 2020.

Gene silencing, advanced therapies combining gene therapy and stem cell technology, immunodeficiency syndromes, growing interest of venture capital firms, etc. will propel the industry's growth. An analysis has also been done of a few factors limiting the growth of the industry. The report also provides insights regarding the strategies adopted by the players from 2013 to 2015 for enhancing their market share. Finally, with a view to understand the competitive landscape, the profiles of key market players have been included in the report to present a complete picture of the global gene therapy market.

Key Topics Covered:

1. Analyst View

2. Research Methodology

3. Gene Therapy - An Introduction3.1 Classification of Gene Therapy Techniques3.2 Physical Methods of Gene Transfer3.2.1 Electroporation3.2.2 Hydrodynamic3.2.3 Microinjection3.2.4 Particle Bombardment3.2.5 Ultrasound-Mediated Transfection3.3 Vectors for Gene Therapy3.3.1 Viral Vectors3.3.1.1 Adenoviral Vectors3.3.1.2 Adeno-associated Virus Vectors3.3.1.3 Retroviral Vectors3.3.1.4 Lentiviral Vectors3.3.2 Non Viral Vectors3.3.2.1 Naked DNA/Plasmid Vectors3.3.2.2 Oligonucleotides3.3.2.3 Liposomes, Lipoplexes and Polyplexes3.3.2.4 Gene-Activated Matrix

4. Industry Overview4.1 Market Drivers4.1.1 Failure of Conventional Therapies to Treat Cancers4.1.2 Rising Focus to Accelerate Commercialization of Gene Therapy in Developed Nations 4.1.3 Rising Incidence of Chronic and Life-Threatening Diseases4.1.4 Growing Interest of Venture Capital Firms4.2 Market Restraints4.2.1 Stringent Regulatory Laws & Safety Concerns 4.2.2 Challenges in Commercialization of Gene Therapy4.2.3 High Cost of the Gene Therapy Drugs4.2.4 Pitfalls in Current Technique4.2.5 Production & Manufacturing: A Unique Challenge for Gene Therapies 4.3 Market Opportunities4.3.1 Approval of Gene Therapy Drug in Europe4.3.2 Gene Therapy for Hemophilia B Offers Significant Opportunity 4.4 Industry Trends4.4.1 Gene Silencing: Gaining Momentum4.4.2 Advanced Therapies: Combining Gene Therapy and Stem Cell Technology4.4.3 Immunodeficiency Syndromes: The Focus of Gene Therapy4.4.4 Nanotechnology: Empowering Gene Therapy4.4.5 Gene Therapy: A New Hope to Treat Blindness4.4.6 Gene Therapy: Potential Cure for Cancer4.5 Winning Imperatives4.5.1 Investment in R&D Activities

5. Clinical Trial Assessment & Pipeline Analysis5.1 Clinical Trials5.1.1 By Geography5.1.2 By Indication5.1.3 By Gene Type5.1.4 By Vector5.1.5 By Clinical Trial Phase5.2 Pipeline Analysis

6. Gene Therapy Market - Regulatory Landscape & Reimbursement Scenario6.1 Regulatory Landscape6.1.1 US6.1.2 Canada6.1.3 Europe 6.1.4 Japan6.1.5 China6.1.6 India6.1.7 Australia6.2 Reimbursement Scenario in Global Market

7. Marketed Gene Therapies7.1 Neovasculgen7.2 Glybera7.3 Gendicine, Rexin-G, Oncorine7.3.1 Gendicine7.3.2 Rexin G 7.3.3 Oncorine

8. Gene Therapy Market8.1 Global8.2 US

9. Gene Therapy Market by Application9.1 Oncology9.1.1 Clinical Trials 9.1.2 Prevalence9.1.3 Market Outlook9.2 Cardiovascular Diseases9.3 Infectious Diseases9.4 Neurological Diseases9.5 Genetic Diseases9.6 Others

10. Gene Therapy Market Size by Geography10.1 North America10.2 Europe10.3 Asia10.4 Rest of the World (RoW)

11. Competitive Landscape11.1 Strategies Adopted by Various Players11.1.1 Strategic Collaborations11.1.2 Acquisitions 11.1.3 Funding & Investments11.2 Companies Progress in Gene Therapy In 2015

12. Key Players Analysis (Business Description, Clinical Pipeline, Recent Developments, Strengths & Weaknesses)12.1 Spark Therapeutics, LLC12.2 ViroMed Co. Ltd. dba VM BioPharma12.3 Advantagene Inc.12.4 Bluebird Bio12.5 Sanofi12.6 Vical Inc.12.7 Oxford BioMedica Plc12.8 Genethon12.9 uniQure N.V.12.10 Human Stem Cells Institute12.11 Shanghai Sunway Biotech Co. Ltd.12.12 Sibiono GeneTech Co. Ltd.

For more information about this report visit https://www.researchandmarkets.com/r/g01of6

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Global & US Gene Therapy Market 2019-2020: Immunodeficiency Syndromes - The Focus of Gene Therapy - PRNewswire

Bluebirdbio has announced updated results from a clinical development programmeevaluatingits investigational Lenti-D gene therapy inpatients with cerebraladrenoleukodystrophy (CALD).

The primary efficacy endpoint in the study isthe proportion of patients who are alive and free of major functionaldisabilities at month 24.Of those patients who have or would have reached 24months of follow-up and completed the study, 88% continue to be alive and majorfunctional disabilities-free in a long-term follow-up study.

Further data show that three out of the 32treated patients did not or will not meet the primary efficacy endpoint; twopatients withdrewfrom the study at investigator discretion, and oneexperienced rapid disease progression early on-study resulting in MFDs anddeath.

The company also announced that the 14 patientscurrently on-study have less than 24 months of follow-up and have shown noevidence of MFDs.

With the longest follow-up from the Phase II/IIIStarbeam study now up to five years, the data show that all boys with CALD whoweretreated with Lenti-D and were free of major functional disabilities at 24months continued to be major functional disabilities -free,explained DavidDavidson, chief medical officer of the company.

He continued, Importantly, there were noreports of graft failure or treatment-related mortality, and adverse eventswere generallyconsistent with myeloablative conditioning. These resultssupport the potential of Lenti-D as a treatment for CALD, which we hope maybecome an option for the boys and their families affected by this devastatingdisease.

CALD is the most severe manifestation ofadrenoleukodystrophy (ALD), a rare metabolic disorder. Approximately 35-40% ofboysdiagnosed with ALD will progress to CALD, typically between the ages ofthree and 12 years. It is characterised by a rapidly progressiveneurologicdecline leading to severe loss of neurologic function and death, in mostuntreated patients.

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Bluebird reveals positive gene therapy data - PharmaTimes