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Newsletter November 2015

News: Gene-Edited Cells Reverse Cancer in Baby Girl
Layla was born in the United Kingdom, and was diagnosed with what's known as acute lymphoblastic leukaemia - an aggressive cancer of the bone marrow - at just three months old. Following the diagnosis, the child was admitted to London's Great Ormond Street Hospital intensive care unit. Two months later, once the doctors confirmed that the leukaemia cells had all been removed, she was given another bone marrow transplant. The disease returned seven weeks later. The improvement on Layla using the treatment according to the doctors was "staggering" and "miraculous".

News: FDA Grants Gene Therapy Orphan Drug Status for Rare Genetic Disorder
Agilis Biotherapeutics announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to the investigational gene therapy AGIL-AS for the treatment of Angelman syndrome (AS), a rare neuro-genetic disorder characterized by severe intellectual and developmental disability

Conference: Spring Symposium of the Netherlands Society of Gene & Cell Therapy
The Spring Symposium of Netherlands Society of Gene and Cell Therapy (NVGCT) will take place on March 10 and 11, 2016 at congrescentrum de Werelt in Lunteren, The Netherlands. The theme is gene delivery. Several stablished keynote speakers have already confirmed their presence. In addition several educational lectures will be presented.

Keynote speakers:
Ernst Wagner (München): non-viral/polymeric gene delivery systems
Matthew Wood (Oxford) : Delivery of oligonucleotide therapies for neuromuscular diseases.

Educational lectures:
Renko de Vries (Wageningen): artificial self-assembling viruses
Joost van den Berg (NKI): cell-based therapies
Niels Geijsen (Hubrecht): physical transfection techniques/

Recent clinical successes have boosted the interest for Gene Therapy. The number of trials both for acquired and inherited diseases will increase significantly in the near future. Because of this and the changes in legislation the organization has decided to also organize an educational session in collaboration with UniQure, the COGEM, Bureau GGO, ARM and the CCMO, on how to fill in the applications to help your with the obtaining the permits in an efficient way and to start performing your GT trial in the Netherlands without delays.


Conference: World Orphan Drug Congress USA 2016
The World Orphan Drug Congress USA is the largest global industry gathering for the orphan drug industry and rare disease community where pharma, biotech, patient groups, academia, regulators, payers and investors gather to talk about strategies to expedite orphan drug development. This year’s event will be co-located with the Rare Disease Advocacy World, which grows the breadth of the event even further and empowers patient groups like never before.

Date: April 20-22, 2016
Venue: The Washington Hilton, Washington, DC

Executive speakers from Center for Rare Disease Therapies, Children's National Medical Center, Chilean Public Health institute, European Medicines Agency (EMA), FDA, Food & Drug Administration, India, Foundation Fighting Blindness, Argentina, International Pemphigus & Pemphigoid Foundation (IPPF), Japan Patients Association, Keck Graduate Institute of Applied Life Sciences, Malaysian Rare Disorders Society, MAPI Group, Mexican Federation of Rare Diseases, Ministry of Health, Malaysia, National Association of Rare Diseases, Brazil, National Institute of Public Health, Japan, National Institutes of Health, New York University Langone Medical Center, NORD, Novartis, NYU Langone Medical Center, Parent Project Muscular Dystrophy, Patient Crossroads, Perelman School of Medicine, University of Pennsylvania, Philippine Society for Orphan Disorders, Stanford University School of Medicine, The Children’s Hospital at Montefiore, University of California-Irvine Medical Center, and more are already confirmed for this year’s event.

The industry will be there to attend VIP conference sessions and learn more on:
• Training the next generation of experts
• Patient registries
• Collaboration and partnerships between multiple stakeholders
• Accurate diagnosis and improving patient outcomes
• Expanded access & 'right to try'
• European & other international policy trends
• Marketing trends & medical affairs
• Fostering drug development & preparing for competition
• Pricing & reimbursement
• Putting patients in the center of drug development
• Drug discovery & development
• Regulatory, states, toxicology - why patients need to be experts
• Bioethics and patients' rights
• Rare disease patient advocacy organizations

Register now...

- 18 – 20 November 2015, 14th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases, Sonoma, CA
- 2 – 3 December 2015, Cell Therapy Manufacturing & Gene Therapy Congress, Brussels, Belgium
- 25 - 27 January 2016, Phacilitate Cell & Gene Therapy World 2016, Washington DC, MD
- 3 - 4 March 2016, 18th International Conference on Cell and Gene Therapy - ICCGT 2016, Singapore
- 9 - 11 March 2016, French Society of Cell and Gene Therapy (SFTCG) Annual Congress, Marseille, France
- 10 - 11 March 2016, Spring Symposium of the Netherlands Society of Gene & Cell Therapy (NVGCT), Lunteren, The Netherlands
- 15 April 2016, Annual conference of the British Society for Gene and Cell Therapy (BSGCT), London, UK
- 4 - 7 May 2016, American Society of Gene and Cell Therapy (ASGCT) 19th Annual Meeting, Washington, DC
- 19 – 21 May 2016, 5th International Conference and Exhibition on Cell & Gene Therapy, San Antonio, USA
- 25 - 28 May 2016, International Society for Cellular Therapy (ISCT) 22th Annual Meeting, Singapore
- 20 - 22 April 2016, World Orphan Drug Congress USA 2016, New York, NY
- 28 – 30 July 2016, The 22th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Tokyo, Japan
- 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


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 and Chikungunya Virus Net.
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