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.

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.

Conference cancellations and postponements

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.

First patient undergoes Luxturna gene therapy on NHS

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.

FDA Continues Strong Support of Innovation in Development of Gene Therapy Products

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.

Dog Study Revives Concerns About Virus Used for Gene Therapy

Posted on: 7 January 2020, source: Science
Adeno-associated viruses are popular vectors for delivering gene therapies to patients’ cells because, researchers believed, the DNA these viruses carry rarely inserts into the host genome. But according to results presented last month (December 9) at the American Society of Hematology (ASH) meeting in Orlando, Florida, this assumption may be wrong. In six dogs that were treated with an AAV-based gene therapy for the blood-clotting disorder hemophilia B, all of them carried the therapeutic DNA within their genomes, and in some cases, the genetic material had inserted near genes known to play a role in cell growth, Science reports.

NJIT to Launch New Programs in Cell and Gene Therapy

Posted on: 23 December 2019, source: NJIT
New Jersey Institute of Technology (NJIT) in collaboration with New Jersey Innovation Institute (NJII), an NJIT corporation, has announced it will soon offer both a Professional Science Masters (PSM) degree program and professional graduate certificate in the rapidly expanding field of cell and gene therapy. The 30-credit M.Sc. degree — available in spring 2020 as part of NJIT’s Department of Chemistry and Environmental Science’s pharmaceutical chemistry master's program — aims to "train the next generation of scientists and engineers in modern aspects of cell and gene therapy, emphasizing cell biology and cellular therapeutic development, regenerative medicine, process development and biomanufacturing, analytical technologies, quality and regulatory compliance.”

Hopkins team invents non-viral system for getting gene therapy into cells

Posted on: 12 December 2019, source: FierceBiotech
One of the most popular methods for inserting therapeutic genes into cells to treat disease is to transport them using a virus that has been stripped of its infectious properties. But those noninfectious viruses can still sometimes touch off dangerous immune responses. A team from Johns Hopkins Medicine is proposing an alternative method for transporting large therapies into cells—including genes and even the gene-editing system CRISPR. It’s a nano-container made of a polymer that biodegrades once it’s inside the cell, unleashing the therapy. The researchers described the invention in the journal Science Advances.