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FDA finalizes neurodegenerative disease gene therapy guidance

Posted on: 2 November 2022, source: Regulatory Affairs Professionals Society
The US Food and Drug Administration (FDA) last week finalized guidance to assist sponsors developing human gene therapy (GT) products for neurodegenerative diseases. In the final version, FDA acceded to industry’s requests to modify its language on the use of crossover trials, provided more detail on the use of comparability studies in assessing the effect of post-approval manufacturing changes on products and eliminated limits for residual product-related impurities.

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Gene Therapy Rapidly Improves Night Vision in Adults with Congenital Blindness

Posted on: 11 October 2022, source: Penn MedicineDNA
Adults with a genetic form of childhood-onset blindness experienced striking recoveries of night vision within days of receiving an experimental gene therapy, according to researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania. The patients had Leber Congenital Amaurosis (LCA), a congenital blindness caused by mutations in the gene GUCY2D

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Codex DNA's BioXp 9600 System debuting at CAR-TCR Summit

Posted on: 20 September 2022, source: Codex DNA
Increasing the power of optimized synthetic biology workflows with expanded throughput and scale: Introducing the BioXpTM 9600 System. Codex DNA – a San Diego-based biotechnology company, and pioneer in automated benchtop synthetic biology systems – will be debuting their next-generation fully-automated synthetic biology benchtop workstation, the BioXp 9600 System, at this year’s CAR-TCR summit.

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BioMarin reports cancer case in hemophilia gene therapy trial

Posted on: 14 September 2022, source: Biopharmadive
Drug regulators have not ordered a trial hold after an analysis suggested leukemia was naturally occurring, company says. BioMarin Pharmaceutical, a California-based biotechnology company, said a patient in a clinical trial of its hemophilia gene therapy Roctavian has developed leukemia, although testing suggests that the case may be naturally occurring..

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Novartis Confirms Deaths of Two Patients Treated with Gene Therapy Zolgensma

Posted on: 15 August 2022, source: genengnews.com
Novartis has acknowledged that two patients have died of acute liver failure following treatment with its Zolgensma® (onasemnogene abeparvovec-xioi), a one-time gene therapy indicated for some forms of spinal muscular atrophy (SMA). As a result, the company said, it will revise Zolgensma’s label to specify that fatal acute liver failure has been reported.

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First patient in the Netherlands successfully treated with stem cell gene therapy

Posted on: 21 July 2022, source: LUMC
Researchers from the Leiden University Medical Center (LUMC) have successfully used stem cell gene therapy to treat a baby with the severe congenital immune disorder SCID. An important milestone: it is the first time stem cell gene therapy of Dutch origin has been administered to a patient, and also the first time it has been used to treat this particular form of SCID worldwide. The treatment was effective and the patient is doing well.

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One-time gene therapy injection could provide HIV treatment that may last a lifetime

Posted on: 12 July 2022, source: ohsu.edu
A new pre-clinical study in nonhuman primates will evaluate an experimental drug’s potential use as a gene therapy that could prevent people who have HIV from having to take daily antiviral drugs for the rest of their lives. The research will be led by Oregon Health & Science University researcher Jonah Sacha, Ph.D., who also serves as a scientific adviser to CytoDyn, the biotechnology company developing the drug, called leronlimab. The study is funded by a five-year grant of up to $5 million that was recently awarded to OHSU by the National Institute of Allergy and Infectious Disease, which is part of the National Institutes of Health.

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Gene therapy to cure leukemia at one-tenth global costs in the works

Posted on: 13 June 2022, source: Business Standard
ImmunoACT, an IIT-Bombay spin-off backed by Hyderabad-based Laurus Labs, is testing a new gene-therapy treatment for cancer, which they claim would not only cure the disease, but also costs one-tenth of the global price. “We are working with CD19 CAR T Cell for leukemia and lymphoma. What we do is we take out some blood and plasma from the cancer patient, and then isolate the T-cells (which are immune cells) from this sample. These cells are then genetically modified so that they eliminate cancer cells. Then this is transfused back to the patient,” explains Rahul Purwar, founder and chairman of ImmunoACT. The product is named H-CAR T-19.

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Gene Therapy Successfully Treats Spinal Cord Injuries Without Side Effects

Posted on: 31 May 2022, source: SciTechDaily
In mouse studies, pain-blocking neurotransmitters produced long-lasting benefits without detectable side effects. An international team of researchers led by scientists at the University of California San Diego School of Medicine reported that a gene therapy that inhibits targeted nerve cell signaling effectively reduced neuropathic pain in mice with spinal cord or peripheral nerve injuries with no detectable side effects.

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Updates on gene therapy for ‘bubble boy’ disease and cellular immunotherapy at ASGCT

Posted on: 15 May 2022, source: ASGCT
St. Jude Children’s Research Hospital scientists will discuss research on cellular therapies for pediatric cancer and more at the annual gathering of the professional society for gene and cellular therapy researchers.

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AAV Durability: The Road to a One-Shot Cure

Posted on: 15 April 2022, source: Hansonwade
Inconsistent long-term clinical data has highlighted the need to better understand the durability of AAV gene therapy. At Hanson Wade Intelligence, our Market Research division, we have classified 121 AAV gene therapy trials into 4 distinct groups based on the target tissue type – liver, muscle, CNS and ocular tissues. This allowed us to summarise available data on clinical durability as well as the key findings we observed across tissues, trials, doses and serotypes. We hope that this report will provide gene therapy developers with valuable insights on the durability of the current generation of AAV gene therapies.

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Gene Therapy Sector Needs Better Cell Lines for Vector Production

Posted on: 4 April 2022, source: Genengnews
Gene therapy developers targeting diseases requiring high dose therapies face a challenge. Current vector production cell lines and genetic manipulation techniques are too costly for large-scale manufacturing, according to new research. The study, by scientists at Portugal’s Instituto de Biologia Experimental e Tecnológica (iBET), looked at areas of gene therapy production where innovation is needed.

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Gene Therapy Turns to the Use of Synthetic Biology Principles

Posted on: 4 March 2022, source: Genengnews
A new university spinout company is hoping to use synthetic biology to transform gene therapy manufacture. SynGenSys, from the University of Sheffield, plans to tap into engineering design principles to create synthetic promoters to target therapeutic genes to the correct patient tissues. “Typically, people use viral promoters, such as cytomegalovirus as their go-to promoter, but it’s a really complex piece of DNA active in many human tissues,” says David C. James, PhD, professor of bioprocess engineering and co-founder of SynGenSys.

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Lentiviral vector-based gene therapy demonstrates long-term safety and efficacy for Wiskott-Aldrich Syndrome

Posted on: 11 February 2022, source: MedicalXpress
Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.

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Nanoscope Therapeutics announces FDA clearance of IND for MCO-010 gene therapy in Stargardt patients

Posted on: 26 January 2022, source: Ophthalmology Times
Nanoscope Therapeutics Inc. announced that it has received IND clearance from the FDA to begin a Phase 2 trial of its Multi-Characteristic Opsin (MCO-010) ambient-light activatable optogenetic monotherapy to restore vision in Stargardt patients.

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Avrobio stops work on rare disease gene therapy after unexpected study results

Posted on: 6 January 2022, source: Biopharmadive
Avrobio, a Cambridge, Massachusetts-based gene therapy developer, will unexpectedly stop work on one of its most advanced treatments, citing disappointing results from a clinical trial that contrast with earlier, more promising findings. In a statement Tuesday, Avrobio said it would stop recruiting patients into a mid-stage study testing its gene therapy for Fabry disease, a rare inherited disorder, and deprioritize the research program. Monitoring will continue for the 14 Fabry patients who have received Avrobio's therapy so far.

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New Bespoke Gene Therapy Consortium Is Welcome Development

Posted on: 10 December 2021, source: Pharmaceutical Executive
The creation of the Bespoke Gene Therapy Consortium (BGTC) in the US is welcome news, especially if it achieves its ambition to accelerate the delivery of more gene therapies for rare diseases. The new body is headed and funded by FDA and the National Institutes of Health (NIH), with other stakeholders including small biotechs, major pharma brands, and non-profit organizations.

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DMD Gene Therapy Candidate RGX-202 Earns Orphan Drug Status

Posted on: 30 November 2021, source: Bionews
RGX-202, a one-time experimental gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The designation is given to investigational therapies to treat rare medical conditions or diseases that affect fewer than 200,000 people in the U.S. Orphan drug status provides benefits to support the process of developing new therapeutics, including tax credits for costs associated with clinical trials, exemptions from certain fees, and marketing exclusivity for seven years if the therapy achieves FDA approval.

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NIH and FDA Lead Coalition to Accelerate Gene Therapy Development for Rare Diseases

Posted on: 31 October 2021, source: NIH
The National Institutes of Health (NIH) announced the formation of the Bespoke Gene Therapy Consortium (BGTC) on Oct. 27th, 2021. In partnership with FDA, ten pharmaceutical companies, and five non-profits, this coalition is aimed at accelerating development of gene therapies for rare diseases.

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FDA: Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations

Posted on: 13 October 2021, source: FDA
This guidance provides FDA’s current thinking on determining sameness of human gene therapy products under FDA’s orphan drug regulations for the purpose of orphan-drug designation and orphan-drug exclusivity.

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Fourth Boy Dies in Trial of Astellas Gene Therapy Candidate

Posted on: 19 September 2021, source: genengnews.com
Astellas Pharma has acknowledged that a fourth boy has died in the troubled Phase I/II trial through which its Astellas Gene Therapies (formerly Audentes Therapeutics) has been evaluating its adeno-associated virus (AAV) gene therapy candidate AT132 in patients with X-linked Myotubular Myopathy (XLMTM). Unlike the other three boys who all died last year in the Phase I/II ASPIRO trial (NCT03199469), the fourth—whose age has not been disclosed—received the lower dose of 1.3×10e14 vg/kg.

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New gene-delivering workhorse could make gene therapy safer, more effective for muscle diseases

Posted on: 13 September 2021, source: Broad Institute of MIT and Harvard
Genetic muscle diseases lead to progressive muscle wasting and often early death, with few treatment options and no cure. Some gene therapies that use a harmless virus to deliver a functioning copy of a disease-causing gene to cells have shown promise in clinical trials for a subset of muscular dystrophies, but have faced challenges. High doses of the gene-carrying virus are needed to reach the muscles throughout the body and the viruses used in these trials often end up in the liver more than in the muscle. This has led to high levels of the virus in the liver, severe adverse side effects, and even death in some trial participants. Researchers at the Broad Institute of MIT and Harvard and Harvard University have developed a new family of adeno-associated viruses (AAVs) - the gene-delivering workhorse of gene therapy - that improve targeting of the muscle tissue, which could be safer and more effective for patients with muscle disease.

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Lysogene Announces First Patient in the United States Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis

Posted on: 30 August 2021, source: BioPharma Dive
Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced dosing of the first patient in the United States with LYS-GM101 investigational gene therapy at CHOC Hospital (CHOC) in a global adaptative-design clinical trial (NCT04273269) for the treatment of GM1 gangliosidosis. This trial is an interventional, multi-center, single-arm, two-stage adaptive-design study evaluating the intracisternal delivery of a recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human β-galactosidase gene (GBL1). The clinical trial includes a safety phase and a confirmatory efficacy phase. The trial will enroll 16 patients with a diagnosis of early or late infantile GM1 gangliosidosis at sites in the US and Europe.

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FDA allows Novartis gene therapy trials to resume after nearly 2 year pause

Posted on: 10 August 2021, source: BioPharma Dive
Novartis on Tuesday said it will begin a new study of its gene therapy Zolgensma in older patients with spinal muscular atrophy following the Food and Drug Administration's clearance of administration via a spinal infusion. The regulator had suspended testing of that dosing in October 2019 due to safety concerns.

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Gene therapy delivered to the brain shows promise in children with rare neurodegenerative disease

Posted on: 16 July 2021, source: FierceBiotech
Scientists from Ohio State University have developed a novel method for delivering gene therapy to specific regions of the brain. Now, they have evidence from a small clinical trial in children that the treatment could address a rare, inherited neurodegenerative disease. And they believe their technique could eventually be used to treat more common brain diseases, like Alzheimer’s and Parkinson’s. The Ohio State team developed the gene therapy to treat aromatic L-amino acid decarboxylase (AADC) deficiency, which hampers the body’s ability to make dopamine and serotonin and results in developmental delays and a range of motor and behavioral symptoms. The gene therapy uses a viral vector to carry DNA-expressing AADC to the brain.

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A year after getting UniQure's gene therapy, hemophilia patients are still doing better

Posted on: 23 June 2021, source: BioPharma Dive
One year after receiving an experimental gene therapy developed by the Dutch drugmaker UniQure, patients with hemophilia B aren't having nearly as many bleeding issues as they used to have. In hemophilia, genetic mutations prevent the body from making proteins needed to clot blood. People with the less common, "B" form of the disorder are missing a protein known as Factor IX. UniQure's medicine is meant to provide a working version of the gene so patients can generate their own clotting protein, and rely less — or ideally, not at all — on so-called replacement factor treatments.

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Decades after a tragic failure, gene therapy successfully treats a rare liver disease

Posted on: 19 May 2021, source: Science
Twenty-two years ago, one of scientists’ first attempts at gene therapy ended in tragedy when a young man died. The story of Jesse Gelsinger, who had a rare liver disorder, became a textbook example of irresponsible medical research. For years, the case hobbled efforts to treat diseases by adding new DNA to a sick person’s cells. Now, a fresh effort to cure Gelsinger’s disease is bearing fruit, in the latest sign of the field’s resurgence.

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New gene therapy restores dystrophin protein in patients with Duchenne muscular dystrophy

Posted on: 4 May 2021, source: News-Medical.net
UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients. The approach, described online today in the journal Science Advances, could lead to a treatment for DMD and inform the treatment of other inherited diseases.

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New approach to CAR T gene therapy yields efficient and longer-lasting response against HIV

Posted on: 5 April 2021, source: News-Medical.net
A UCLA research team has shown that using a truncated form of the CD4 molecule as part of a gene therapy to combat HIV yielded superior and longer-lasting results in mouse models than previous similar therapies using the CD4 molecule. This new approach to CAR T gene therapy -- a type of immunotherapy that involves genetically engineering the body's own blood-forming stem cells to create HIV-fighting T cells -- has the potential to not only destroy HIV-infected cells but to create "memory cells" that could provide lifelong protection from infection with the virus that causes AIDS.

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EMA Launches Safety Review After Gene Therapy Trial Patient Develops Blood Cancer

Posted on: 13 March 2021, source: BioSpace
The European Medicines Agency (EMA) has launched a safety review of bluebird bio’s thalassaemia drug Zynteglo, a conditionally licensed gene therapy in Europe. This review comes after researchers identified an acute myeloid leukaemia (AML) case in a patient who received bb1111 (LentiGlobin), an investigational gene therapy for sickle cell disease (SCD), in a Phase I/II study. This therapy relies on the same viral vector found in Zynteglo which delivers a gene into cells.

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