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Newsletter October 2011

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News: Gene therapy and stem cells unite
Two of the holy grails of medicine - stem cell technology and precision gene therapy - have been united for the first time in humans, say scientists. It means patients with a genetic disease could, one day, be treated with their own cells. A study in Nature corrected a mutation in stem cells made from a patient with a liver disease. Researchers said this was a "critical step" towards devising treatments, but safety tests were still needed.

News: Gene Therapists Celebrate a Decade of Progress
It has taken many years, but researchers may have reached a prized goal in gene therapy: lowering the risk of uncontrolled bleeding in patients with hemophilia. At a meeting last week, researchers reported that six patients who received a virus engineered to carry a gene for a blood-clotting protein called factor IX needed fewer transfusions of the protein for as long as 18 months; some didn't require any transfusions. One patient developed an immune response to the viral vector, but this side effect was successfully treated with drugs.

News: Sangamo gene therapy shows promise in reducing HIV
An early stage trial of Sangamo BioSciences Inc's HIV treatment found that the gene therapy reduced levels of the virus and even eliminated it in one patient with a naturally occurring gene mutation. The very small Phase 1 trial tested the SB-728-T gene therapy, which is designed to disrupt the CCR5 gene used by HIV to infect cells of the immune system. If shown to be safe and effective, the treatment could end the need for the antiretroviral drugs now used to keep the virus that causes AIDS in check by suppressing viral replication in the blood.

Conference: EMA-CAT and ESGCT Joint Workshop; Advanced Therapy Medicinal Products: from Promise to Reality
The CAT-ESGCT satellite workshop ‘Advanced Therapy Medicinal Products: from Promise to Reality’ is where science and regulators come together to advance the commercialization of gene therapy and cell-based medicinal products.
This workshop, organised by the European Medicines Agency's Committee for Advanced Therapies (CAT), will allow attendees involved in researching and developing medicines based on gene therapy or cells to:
• hear how to translate academic research into commercial products;
• understand the regulatory pathways leading to authorisation;
• hear the experiences of organisations involved in getting advanced-therapy medicines to market;
• get practical advice on the authorisation procedure directly from regulators.
The conference features keynote presentations from the EMA, as well as from the United States Food and Drug Administration (FDA), academics, industry’s leading pharmaceutical companies, lively panel debates, interactive roundtables, and case studies. They will be discussing the issues central to defining the regulatory path for translation of research to commercial medicinal products.


Poll: The development of which type of adenoviral vector for (solid) cancer gene therapy treatments is most promising and should be supported?
Gene Therapy Net started a new poll regarding the development of adenoviral vectors for cancer gene therapy. What do you think? Vote now!
1- Suicide gene therapy adenoviral vectors expressing HSV-TK, or
2- Tumor specific replication competent adenoviral vectors (like CrAd), or
3- Immuno stimulating adenoviral vectors (like GM-CSF expressing), or
4- Tumor antigen expressing adenoviral vectors (like Mart-1 for melanoma)

Vote now...

- 27 - 31 October 2011, European Society Of Gene & Cell Therapy (ESGCT) and the British Society of Gene Therapy (BSGT) collaborative Congress 2011, Brighton, UK
- 8 November 2011, 4rd annual Workshop on Genome Engineering, Seattle, WA
- 16 - 18 November 2011, 10th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases. Focus Topic: Cell Trafficking and the Niche, Sonoma, CA
- 18 November 2011, 10th symposium of the Association de Thérapie Génique du Québec (ATGQ), Québec, Canada
- 15 - 16 December 2011, NIH Symposium: RNA Oligonucleotides - Emerging Clinical Applications, Rockville, MD
- 30 January - 1 February 2012, Phacilitate 8th Annual Cell & Gene Therapy Forum 2012, Washington DC, MD
- 2 -5 February 2012, Up Close and Personalized, International Congress on Personalized Medicine, Florence, Italy
- 16 – 20 May 2012, American Society of Gene and Cell Therapy (ASGCT) 15th Annual Meeting, Philadelphia, PA
- 5 – 8 June 2012, International Society for Cellular Therapy (ISCT) 18th Annual Meeting, Seattle, WA
- 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
- 27 - 31 October 2012, The 11th Annual Meeting of the French Society of Cell and Gene Therapy (SFTCG), Versailles, France


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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