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Newsletter June 2012

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News: Gene Therapy Society Looking to NIH to Revisit Role of the Recombinant DNA Advisory Committee
The principal U.S. professional society representing gene therapists is awaiting word from NIH about whether or not there will be re-examination of the role of its Recombinant DNA Advisory Committee (RAC). They aim to limit its longstanding role in clinical trials while continuing its service as a forum for discussing and educating professionals on policy issues. The American Society of Gene & Cell Therapy (ASGCT) touched off the debate earlier this year, when its president and her predecessor conveyed the society’s view that RAC should focus on broader issues rather than review the clinical protocols of individual researchers. They sent a letter to Amy Patterson, M.D., director of NIH’s Office of Biotechnology Activities (OBA), and issued a statement seven days later at a RAC meeting.

News: Targeting DNA: After 20 years of high-profile failure, gene therapy is finally well on its way to clinical approval
The concept is simple: if a mutated gene is causing a problem, replace or supplement it with a new, accurate copy. In theory, such a strategy could not just treat, but cure countless human genetic diseases. In practice, however, developing safe and effective gene therapies has not been easy. Even when identifying a disorder’s genetic basis is fairly straightforward, finding the appropriate delivery vector to target the diseased tissues in the body, while avoiding unintended consequences, has challenged would-be gene therapists for more than 20 years. But more and more researchers are convinced that the technique is on the brink of becoming a common medical practice.

News: RetroSense Therapeutics Completes pre-IND Meeting for RST-001
RetroSense Therapeutics, a gene therapy company dedicated to vision restoration, announced completion of a pre-IND meeting with the Center for Biological Evaluation and Research (CBER) division of the FDA that took place on May 22nd 2012 regarding RetroSense’s lead biologic, RST-001 for vision restoration in retinal degenerative conditions. The purpose of the meeting was to obtain CBER’s guidance for the clinical path to a Biologics License Application (BLA) for RST-001 in the US, and clarity on the steps required for Investigational New Drug (IND) submission. The discussions included manufacturing criteria, the scope and design of the preclinical studies, and the scope and design of Phase I and IIa clinical trials.

Conference: World Cord Blood Conference
The World Cord Blood Congress is North America’s leading commercial cord blood banking and therapy conference. The World Cord Blood Congress is where science and business come together to advance the cord blood banking and therapeutics sectors. Speakers include senior-level executives from private and public cord blood banks, hospitals, biotech companies, academia, and government.


Conference: Stem Cells USA Regenerative Medicine Congress
Stem Cells USA & Regenerative Medicine Congress is the region's largest commercial stem cells conference. Most stem cell companies face two options - go bankrupt or find a partner with deep pockets. Despite there being hundreds of companies with promising technologies, most of them never pass Phase II trials due to lack of funding. This year’s Stem Cells USA & Regenerative Medicine Congress will focus on strategies and business models to help you navigate this increasingly challenging marketplace, and to ensure your stem cell and regenerative medicine gets to market.
New to 2012 - this year's conference will take place alongside the World Cord Blood Congress. What does this mean for you? Double the networking and double the content.


- 28 – 30 June 2012, The 18th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Kumamoto University, Kumamoto, Japan
- 5 – 6 October 2012, International Society for Cell & Gene Therapy of Cancer - 2012 Singapore Conference, Singapore
- 22 – 24 October 2012, International conference on Genetic Syndromes & Gene Therapy, Chicago
- 26 – 29 October 2012, European Society Of Gene & Cell Therapy (ESGCT) XXth annual meeting and the French Society of Cell and Gene Therapy (SFTCG) collaborative Congress 2012, Versailles, France
- 19 – 21 November 2012, International Conference on Genetic Syndromes & Gene Therapy, San Antonio, USA
- 5 - 7 December 2012, DNA Vaccines 2012, San Diego, LA


Gene Therapy Net is the information resource for basic and clinical research in gene therapy, and the site serves as a network in the exchange of gene therapy information and breaking news items. Visitors can keep track of the latest scientific papers, conference announcements, gene therapy jobs, regulations and guidelines. Other information websites created and maintained by the owner of Gene Therapy Net are Dengue Virus Net, Influenza Virus Net and Chikungunya Virus Net.
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