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.

 

Posted on: 30 January 2010, source: ASGCT
The AAV2 vector reference standard material (an AAV2-GFP viral vector) is now available from ATCC (http://www.atcc.org). The AAV2 RSM is intended for use in calibrating internal (laboratory-specific) reference materials and assays for recombinant AAV viral gene transfer products, with the purpose of making data from different pre-clinical and clinical studies more comparable.

Posted on: 25 January 2010, source: TradingMarkets.com
Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, has reached another important milestone in the official marketing authorisation process for its lead product Glybera, AMT's proprietary product for lipoprotein lipase deficiency (LPLD). The submission of the Glybera Marketing Authorisation Application (MAA), announced earlier, has cleared the validation stage with The European Medicines Agency (EMA, formerly known as EMEA). The EMA will now commence its formal review of Glybera.

Posted on: 24 January 2010, source: The European Medicines Agency
The European Medicines Agency has published a Concept paper on the revision of the note for guidance on the quality, pre-clinical and clinical aspects of gene transfer medicinal products (EMA/CHMP/GTWP/BWP/234523/2009). This Concept Paper proposes a revision of the Note for Guidance on the Quality, Preclinical and Clinical Aspects of Gene Transfer Medicinal Products (CPMP/BWP/3088/99) that came into effect in 2001. This revision will address the issues identified from clinical experience and provision of Scientific Advice on gene therapy medicinal products and will lay down detailed and updated requirements for the quality, non-clinical and clinical aspects of gene therapy medicinal products.

Posted on: 14 January 2010, source: ScienceDaily
Researchers at the University of Minnesota have discovered a molecular security system in human cells that deactivates and degrades foreign DNA. This discovery could open the door to major improvements in genetic engineering and gene therapy technologies. Led by Reuben Harris, associate professor of biochemistry, molecular biology and biophysics in the College of Biological Sciences, the report's findings will be published online by Nature Structural and Molecular Biology on Jan. 10.

Posted on: 15 December 2009, source: Proactive Investors UK
Gene therapy developers, Oxford BioMedica (LSE: OXB) announced its StarGen therapy has received orphan treatment designation from the European Medicines Agency (EMEA). As a result the treatment will have ten years of marketing exclusivity and reduced regulatory fees. StarGen’s clinical development is expected to start in 2010, in collaboration with Sanofi-Aventis (NYSE: SNY). Stargardt Disease is a rare disease that causes cells on the macula area of the retina to stop working, which leads to problems with central vision. At first it makes vision unclear, distorted or blurred. After a longer period, Stargardt's can cause a ‘blank patch’ in the centre vision. There are currently no treatments available for Stargardt disease.