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: 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.

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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.

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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.

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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.

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Posted on: 11 March 2020, source: Gene Therapy Net
Please note that due to the evolving COVID-19 situation, several conferences and meetings will be cancelled or postponed. Gene Therapy Net is continually monitoring COVID-19 updated guidelines from WHO and CDC. Check the individual conference websites regularly to be informed well. At this time, there is no plan to cancel or postpone ASGCT’s 23rd Annual Meeting in May, the ESGCT Spring School in April, or the ESGCT meeting in October. The following meetings have been postponed: NVGCT, DG-GT Symposium, SFTCG annual meeting, Advanced Therapies 2020.

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Posted on: 20 February 2020, source: pharmatimes.com
The NHS has reported treating its first patient with Novartis’ Luxturna (voretigene neparvovec) a “revolutionary” new gene therapy that can restore eyesight, as part of its NHS Long Term Plan. The therapy is for those born with an inherited retinal disorder - Leber’s Congenital Amaurosis (LCA) - who have poor sight which swiftly deteriorates, with many ultimately losing their vision completely in childhood.

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Posted on: 1 February 2020, source: FDA
This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA has approved four gene therapy products, which insert new genetic material into a patient’s cells. The agency anticipates many more approvals in the coming years, as evidenced by the more than 900 investigational new drug (IND) applications for ongoing clinical studies in this area. The FDA believes this will provide patients and providers with increased therapeutic choices. In that spirit, today, the FDA is announcing the release of a number of important policies: six final guidances on gene therapy manufacturing and clinical development of products and a draft guidance, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.

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