Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials. For resources pertaining to regenerative cell therapy and clinical treatment, check out R3 Stem Cell.

Posted on: 12 October 2024, source: pharmaceutical-technology.com
The side effects of bluebird bio’s gene therapy Skysona (elivaldogene autotemcel) have been thrust back into the spotlight after new data shows seven children who took part in its clinical studies went on to develop a type of blood cancer. The findings, from a study published in The New England Journal of Medicine (NEJM) yesterday (9 October), show that seven out of 67 patients under 18 years of age who took part in a Phase II and Phase III trial for Skysona developed haematologic cancers. The patients received the one-time autologous hematopoietic stem cell-based gene therapy as a treatment for early cerebral adrenoleukodystrophy (CALD) – a rare and fatal neurodegenerative disease.

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Posted on: 1 October 2024, source: prnewswire
The ISCT Institute of Training & Development, a division of the International Society for Cell and Gene Therapy (ISCT), has become the first cell and gene therapy (CGT) organization to receive globally recognized accreditation from the ANSI National Accreditation Board (ANAB) for excellence in workforce development and training. This prestigious accreditation underscores ISCT's leadership in addressing critical skill gaps within the rapidly evolving CGT sector, while ensuring that ISCT's courses are standardized, globally recognized, and accredited.

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Posted on: 26 August 2024, source: fabrydiseasenews.com
The first patient has been dosed in uniQure’s U.S. Phase 1/2a clinical trial of AMT-191, its gene therapy candidate for Fabry disease. The clinical trial, an open-label study (NCT06270316) focused on safety and early efficacy, is still recruiting about 12 adult male patients at a site in Fairfax, Virginia. Eligible men will have had a suboptimal response to enzyme replacement therapy (ERT). An open-label trial means that both researchers and participants know what treatment is being administered.

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Posted on: 30 July 2024, source: Insight News
Macquarie University researchers have identified a gene therapy that could help treat glaucoma, with potential applications for other neurodegenerative conditions such as Alzheimer’s disease. The protein Tau is essential to the function of cells in the brain and central nervous system, but when over-produced under certain conditions, it forms tangles that clog the cells’ internal structures. These tangles have also been found in Alzheimer’s disease patients. The protein has also been found in retinal cells, with research by Macquarie University’s Vision Science Group finding that it also likely plays a role in glaucoma.

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Posted on: 2 July 2024, source: GEN
Gene therapy has a reputation as a one-time intervention that has a lifelong effect. It’s an all-or-nothing proposition. But what if it wasn’t? What if gene therapy could be dialed up or down, on a daily basis, with a simple pill? The possibility has inspired MeiraGTx to develop a riboswitch technology that is designed to allow for the precise, dose-responsive control of gene expression by oral small molecules. The riboswitch technology is just part of MeiraGTx’s work in gene therapy. The company has technologies for the optimization of adeno-associated virus (AAV) vectors and for the design of promoter sequences. Also, the company has an internally developed manufacturing platform process and several production facilities. Finally, the company has several gene therapy programs in late-stage clinical trials.

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Posted on: 20 May 2024, source: CBS News
One of the youngest children in the world to receive a new type of gene therapy to treat genetic deafness can now hear for the first time in her life. The family of the toddler taking part in a medical trial has called the change in their daughter "mind-blowing." Opal Sandy, now 18 months old, was born with total deafness due to a fault in the OTOF gene, which makes a protein called Otoferlin. Otoferlin enables communication between cells of the inner ear, or cochlea, and the brain. As part of a trial run by Cambridge University, Opal received an infusion of a working copy of the OTOF gene in her right ear. The surgical procedure took only 16 minutes and was carried out just before she reached her first birthday.

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Posted on: 6 May 2024, source: The New York Times
A 12-year-old boy in the Washington, D.C., area faces months of procedures to remedy his disease. “I want to be cured,” he said. On Wednesday, Kendric Cromer, a 12-year-old boy from a suburb of Washington, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition. For the estimated 20,000 people with sickle cell in the United States who qualify for the treatment, the start of Kendric’s monthslong medical journey may offer hope. But it also signals the difficulties patients face as they seek a pair of new sickle cell treatments.

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