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Newsletter June 2016

News: Gene therapy drug approval granted to GSK
Regulators have given one of the world's largest drug companies approval to sell a new gene therapy. The treatment is for an illness called ADA-SCID which prevents babies from fighting off everyday infections. This is the first approval for a genetic therapy granted to a large multinational drug company, GSK. Commentators say the development marks the beginning of many more genetic medicines from so-called "Big Pharma". The condition is extremely rare and affects around two dozen babies each year. Approval of the gene therapy paves the way for the development of treatments for more widespread illnesses such as thalassemia and sickle cell disease. Hundreds of inherited disorders such as cystic fibrosis, muscular dystrophy and many types of blindness are caused by faulty genes.

News: Abeona Therapeutics Doses First Patient in Phase 1/2 Clinical Trial for Sanfilippo Syndrome Type A
Cleveland-based Abeona Therapeutics has dosed its first patient in a Phase 1/ 2 Trial with ABO-102 gene therapy for patients with Sanfilippo syndrome type A (a/k/a Mucopolysaccharidosis Type IIIA or MPS IIIA). ABO-12, a single intravenous injection, is an adeno-associated viral (AAV)-based gene therapy to treat the central nervous system and the peripheral disease manifestations in Sanfilippo patients, representing a new approach to addressing the progressive disease which often prevents children from reaching adulthood. The drug is based on more than 18 years of research and development. There are currently no approved treatments available for Sanfilippo syndrome type A.

- 29 - 30 June 2016, Adoptive T Cell Therapy Congress, Boston, MA
- 28 – 30 July 2016, The 22th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Tokyo, Japan
- 18 – 19 August 2016, Gene Therapy Bioproduction conference, Boston, MA
- 14 -16 September 2016, 22th Annual Meeting of the German Society for Gene Therapy (DG-GT e.V.), Heidelberg, Germany
- 21 -22 September 2016, Phacilitate's Cell & Gene Therapy Europe, Berlin, Germany
- 1 - 5 October 2016, International Meeting on Replicating Oncolytic Virus Therapeutics, Vancouver, Canada
- 5 – 7 October 2016, Cell & Gene Therapy BioProcessing & Commercialization meeting, Boston, MA
- 18 – 21 October 2016, XXIV Congress of the European Society of Gene and Cell Therapy (ESGCT) and the International Society for Stem Cell Research, Florence, Italy
- 21 October 2016, Second European debate on Germline Gene Editing at ESGCT-ISSCR Congress, Florence, Italy
- 3 - 4 November 2016, 2nd Annual Cell & Gene Therapy Congress 2016, London, UK
- 16 – 18 November 2016, 15th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases, Sonoma, California
- 30 November – 1 December 2016, Cell Therapy Manufacturing & Gene Therapy Congress, Brussels, Belgium
- March 2017, International Society for BioProcess Technology 7th Spring Meeting - Viral vectors and vaccines, Washington, DC
- 16 - 17 March 2017, Spring Symposium of the Netherlands Society of Gene & Cell Therapy (NVGCT), Lunteren, The Netherlands
- 29 - 30 March 2017, 19th International Conference on Cell and Gene Therapy - ICCGT 2017, Singapore
- 19 - 21 April 2017, Annual conference of the British Society for Gene and Cell Therapy (BSGCT), Cardiff, Wales, UK
- 3 - 6 May 2017,International Society for Cellular Therapy (ISCT) 23th Annual Meeting, London, UK
- 10 - 13 May 2017, American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting, Washington, DC
- 14 - 19 May 2017, Gordon Research Conference: Virusses & Cells, Lucca, Italy
- 24 - 26 May 2017, 10th Australasian Gene Therapy Society Meeting (AGCTS), Sydney, Australia
- 27 - 28 November 2017, 19th International Conference on Cell and Gene Therapy - ICCGT 2017, Istanbul, Turkey


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, Ebola Virus Net, Chikungunya Virus Net and Zika Virus Net.
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