Welcome to Gene Therapy Net

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.

FDA Approves First Cell-Based Gene Therapy For Adult Patients with Relapsed or Refractory MCL

Posted on: 4 August 2020, source: FDA
The U.S. Food and Drug Administration approved Tecartus (brexucabtagene autoleucel), a cell-based gene therapy for treatment of adult patients diagnosed with mantle cell lymphoma (MCL) who have not responded to or who have relapsed following other kinds of treatment. Tecartus, a chimeric antigen receptor (CAR) T cell therapy, is the first cell-based gene therapy approved by the FDA for the treatment of MCL. “Tremendous progress has been made in the discovery of new therapies for debilitating diseases that are difficult to treat. This approval is yet another example of customized treatments that use a patient’s own immune system to help fight cancer, while using a scientific advance in this promising new area of medicine,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “We’re seeing continued advances in the field of gene therapy and remain committed to supporting innovation in this promising new area of medicine.”

Two deaths in gene therapy trial for rare muscle disease

Posted on: 30 June 2020, source: ScienceMag
Two boys have died after receiving high doses of a gene therapy treatment for their rare muscle disease, Biopharma Dive reports. The patients, born with x-linked myotubular myopathy, developed liver problems that apparently led to sepsis, according to a 23 June letter to patient groups from trial sponsor Audentes Therapeutics. They were older patients and had existing liver disease; several younger patients who got lower doses of the treatment have done well and now breathe on their own without a ventilator.

Preclinical Study of SLS-004, Potential Parkinson’s Gene Therapy, Starting

Posted on: 3 June 2020, source: Parkinson News
Seelos Therapeutics announced it is beginning, earlier than expected, a preclinical study into its investigational gene therapy candidate for Parkinson’s disease called SLS-004. Nerve cell damage in Parkinson’s is caused by the buildup of toxic forms of the alpha-synuclein protein, and resulting clumps of misfolded proteins known as Lewy bodies. These toxic aggregates damage and eventually kill nerve cells — called dopaminergic neurons — in a region of the brain that regulates muscle movement and coordination.

FDA Gives Fabry Gene Therapy, FLT190, Orphan Drug Status

Posted on: 13 May 2020, source: fabrydiseasenews.com
The U.S. Food and Drug Administration (FDA) has designated FLT190, an investigational gene therapy for Fabry disease, an orphan drug to support and speed its development and possible review, Freeline Therapeutics, its developer, announced. The therapy is being evaluated in a Phase 1/2 trial, called MARVEL1 (NCT04040049), that may be enrolling up to 15 adult male patients at three sites in Europe.

Gene therapy for color blindness passes first phase of human testing

Posted on: 4 May 2020, source: NewAtlas
The results of a first human trial testing a gene therapy for complete color blindness have been published in the journal JAMA Ophthalmology. The research suggests the experimental gene therapy is safe, and potentially efficacious, opening the door to larger human trials in the future.

Wyatt Technology Pioneers Multi-CQA Method for Gene Vectors

Posted on: 13 April 2020, source: Wyatt
Wyatt Technology, the world leader in instrumentation for absolute macromolecular and nanoparticle characterization, announces the release of a novel method suitable for determining multiple critical quality attributes of adeno-associated virus and similar small gene vectors, in a single chromatographic run. The method is based on SEC-MALS, which combines size-exclusion chromatography for separation with multi-angle light scattering for characterization. SEC-MALS was introduced by Wyatt in 1985 and has been a mainstay of biophysical characterization of biotherapeutics such as monoclonal antibodies and virus-like particles. It utilizes Wyatt’s DAWN MALS instrument coupled to an industry-standard SEC system and offers full support for FDA 21CFR(11) compliance.

New gene therapy product Zolgensma to treat spinal muscular atrophy

Posted on: 30 March 2020, source: EMA
The EMA has recommended granting a conditional marketing authorisation in the European Union for the gene therapy Zolgensma(onasemnogene abeparvovec)to treat babies and young children with spinal muscular atrophy (SMA), a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement. There are currently limited treatment options for children with SMAin the EU. Patients also receive physical aids to support muscular functions and help them and their families cope with the symptoms of the disease.