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Newsletter October 2017

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News: FDA experts offer a unanimous endorsement for pioneering gene therapy for blindness
A pioneering AAV gene therapy from Spark Therapeutics took a giant stride toward an FDA approval yesterday as an outside panel of experts offered their support for getting this game-changing treatment into the market after looking over the data and hearing from some of the severely sight-impaired patients whose lives had been transformed by this therapy. The vote was 16 to 0 favoring the benefit-risk profile of the drug, backing an OK for voretigene neparvovec by the agency’s Cellular, Tissue and Gene Therapies Advisory Committee and providing a compelling reason for the U.S. Food and Drug Administration to follow through with an historic first U.S. approval of a vector-delivered gene therapy.
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News: DNA surgery on embryos removes disease
Precise "chemical surgery" has been performed on human embryos to remove disease in a world first, Chinese researchers have told the BBC. The team at Sun Yat-sen University used a technique called base editing to correct a single error out of the three billion "letters" of our genetic code. They altered lab-made embryos to remove the disease beta-thalassemia. The embryos were not implanted. The team says the approach may one day treat a range of inherited diseases.
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News: Gene therapy reverses multiple sclerosis in mice
A novel gene therapy can reverse the symptoms and progression of disease in a mouse model of multiple sclerosis (MS). The neurodegenerative illness is caused by the body's own immune system degrading and destroying nerve cells. US researchers have used a gene therapy approach in the livers of affected mice to produce more regulatory T-cells and reduce this autoimmune response. 'Using a clinically tested gene therapy platform, we are able to induce very specific regulatory cells that target the self-reactive cells that are responsible for causing multiple sclerosis,' said Dr Brad Hoffman at the University of Florida, College of Medicine in Gainesville, who led the study. 'Most current therapies for autoimmune diseases such as multiple sclerosis are based on general immune suppression, which has various side effects or complications.'
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Conferences
- 17 – 20 October 2017, XXV Congress of the European Society of Gene and Cell Therapy (ESGCT), Berlin, Germany
- 18 – 19 October 2017, Market Access for Cell and Gene Therapies Congress, London, UK
- 31 October – 2 November 2017, Gene Therapy for Rare Disorders Europe, London, UK
- 9 - 10 November 2017, 3rd Annual Genome Editing Congress, London, UK
- 9 - 10 November 2017, 3rd Annual Cell & Gene Therapy Congress, London, UK
- 13 - 15 November 2017, World Orphan Drug Congress, Barcelona, Spain
- 15 - 17 November 2017, 16th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases, Sonoma, California
- 23 - 25 January 2018, Cell & Gene Therapy World event 2018, Miami, Fl
- 30 - 31 January 2018, Combined CAR-T Congress Europe, Berlin, Germany
- 7 - 8 February 2018, Annual Cell and Gene Therapy Innovation Summit, Berlin, Germany
- 8 - 9 February 2018, 3rd Annual Genome Editing & Engineering Conference, San Diego, CA
- 5 - 7 March 2018, International Society for BioProcess Technology 8th Spring Meeting - Viral vectors and vaccines, Norfolk, Virginia
- 14 – 16 March 2018, 9th Biennial Congress of the Spanish Society of Gene (SETGyC) and Cell Therapy, Palma de Mallorca, Spain
- 15 March 2018, British Society for Gene and Cell Therapy (BSGCT) Public Engagement 2018, Oxford, UK
- 15 - 17 March 2018, 7th International Conference and Exhibition on Cell and Gene Therapy, London, UK
- 27 April 2018, Annual conference of the British Society for Gene and Cell Therapy (BSGCT), Cardiff, Wales, UK
- 2 - 5 May 2018, International Society for Cellular Therapy (ISCT) Annual Meeting, Montreal, Canada
- 16 - 19 May 2018, American Society of Gene and Cell Therapy (ASGCT) 21th Annual Meeting, Chicago, IL
- 26 - 28 July 2018, The 24th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Tokyo, Japan
- 13 - 16 August 2018, Gene Therapy Bioproduction Conference, Boston, MA
- 16 – 19 October 2018, XXVI Congress of the European Society of Gene and Cell Therapy (ESGCT), Lausanne, Swizterland
- 23 November 2018, British Society for Gene and Cell Therapy (BSGCT) Annual Conference Autumn 2018, London, UK
- 29 April - 2 May 2019, American Society of Gene and Cell Therapy (ASGCT) 22th Annual Meeting, Washington, DC
- 28 – 31 October 2019, XXVII Congress of the European Society of Gene and Cell Therapy (ESGCT), Barcelona, Spain

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