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: 8 March 2010, source: NPR
Twenty-five years ago, it seemed as if gene therapy was on the verge of revolutionizing medicine. But that revolution never occurred, and scientists realized that they had been overly optimistic about how quickly they could develop such therapies. Now, however, there are signs that the field of gene therapy is making definite progress, even if the revolution is still on hold. The concept of gene therapy is simple. Some diseases are caused by damage to a single gene — for example, cystic fibrosis and hemophilia. Give patients the healthy gene, and in theory, the disease is cured.

Posted on: 7 March 2010, source: Voice of America
Researchers have moved a step closer toward fully restoring the eyesight of people with a rare genetic disorder. A new study shows the treatment is safe and effective, and could pave the way for helping cure more common causes of blindness. Leber's congenital amaurosis is an extremely rare condition that causes blindness in approximately 4,000 people in the United States.

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.