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Newsletter May 2020

News: FDA Gives Fabry Gene Therapy, FLT190, Orphan Drug Status
The U.S. Food and Drug Administration (FDA) has designated FLT190, an investigational gene therapy for Fabry disease, an orphan drug to support and speed its development and possible review, Freeline Therapeutics, its developer, announced. The therapy is being evaluated in a Phase 1/2 trial, called MARVEL1 (NCT04040049), that may be enrolling up to 15 adult male patients at three sites in Europe.

News: Gene therapy for color blindness passes first phase of human testing
The results of a first human trial testing a gene therapy for complete color blindness have been published in the journal JAMA Ophthalmology. The research suggests the experimental gene therapy is safe, and potentially efficacious, opening the door to larger human trials in the future.

Conference: Gene Therapy Immunogenicity
Gene therapies are revolutionizing the therapeutic landscape for rare and inherited diseases. For the field to progress, however, there are several unique and complex challenges. Perhaps the most prominent of these challenges is the immune response to gene therapy delivery. The inaugural Gene Therapy Immunogenicity Summit, 4-6 August 2020 in Boston, is the industry’s definitive forum enabling you to better measure, modulate and predict immune response to your gene therapy candidate. Uniting industry leaders across the gene therapy space, this multi-disciplinary forum will enable you to develop more effective and safe gene therapies.
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Conference: Gene Therapy for Ophthalmic Disorders
More companies than ever before are realizing the potential of gene therapies to treat a wide range of ophthalmic disorders but in practice there are still hurdles to overcome. A variety of unique challenges must be addressed to effectively bring gene therapies to patients, from developing preclinical models that accurately translate to the clinic to validating clinical endpoints that will satisfy regulators. Gene Therapy for Ophthalmic Disorders, 4-6 August 2020 in Boston, will unite industry leaders across the ophthalmology space to overcome the specific delivery and drug development challenges faced by companies developing gene therapies for ophthalmic diseases.
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Conference: Neuroimmunology Drug Development Summit
The Neuroimmunology Drug Development Summit will now take place on 5-7 August 2020 in Boston. Organizations on the cutting-edge of this rapidly developing field, including Biogen, Merck, Denali, Third Rock Ventures and Alector, will meet over 3 days and 2 in-depth workshops to overcome their translational challenges and progress their therapeutic to the clinic.
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Conference: Cell Engager Summit
The annual Cell Engager Summit will now take place on 18-20 August 2020 in Boston. Join over 25 world-class speakers from the organizations on the cutting edge of bispecific and multi-specific cell engager research. Over 3 days, 2 workshops and over 14 hours of content, the cell engager community will come together to advance the next generation of immune cell engaging antibody therapies for solid tumor indications.
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Conference: World Orphan Drug Congress USA
The World Orphan Drug Congress USA, 24-26 August 2020 in Fort Washington, MD focuses on the most pressing challenges and opportunities to bring rare disease therapies to patients faster. Bringing together a global gathering of 1,500 leaders in orphan drugs from 50 countries, World Orphan Drug Congress USA will feature over 260 presentations covering all aspects of orphan drug development and rare disease research.
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- 15 - 19 June 2020, 9th International Symposium on Monolith - DNA/RNA, vaccines, gene therapy vectors, Portoroz, Slovenia
- 8 - 9 July 2020, Formulation and Drug Delivery conference - Cell & Gene Therapy Biomanufacturing: Insights, Key Strategies & Novel Approaches, London, UK
- 8 - 9 July 2020, Onco Cell Therapy Summit USA, Boston, MA
- 21 - 23 July 2020, RNAi-Based Therapeutics Summit, Boston, MA
- 21 - 23 July 2020, Bioprocessing Summit Europe, Barcelona, Spain
- 4 - 6 August 2020, Gene Therapy Immunogenicity Summit, Boston, MA
- 4 - 6 August 2020, Gene Therapy for Ophthalmic Disorders, Boston, MA
- 5 - 7 August 2020, Neuroimmunology Drug Development Summit - Translating Neuroimmunological Mechanisms into Clinically Effective Neurodegenerative Targeting Therapeutics, Boston, MA
- 18 - 20 August 2020, 2nd Annual Cell Engager Summit, Boston, MA
- 24 - 26 August 2020, Innate Killer Summit 2020, San Diego, CA
- 24 - 26 August 2020, World Orphan Drug Congress USA, Gaylord, National Harbor, Maryland
- 24 - 28 August 2020, 12th annual Bioprocessing Summit, Boston, MA
- 31 August - 4 September 2020, PEGS: The Essential Protein Engineering and Cell Therapy Summit - Cell and Gene Therapies, Boston, MA
- 1 - 3 September 2020, Gene Therapy Analytical Development Europe, London, UK
- 7 - 9 September 2020, 15th Annual World Advanced Therapies Congress, London, UK
- 14 - 17 September 2020, CAR-TCR Summit USA, Boston, MA
- 15 - 17 September 2020, Bioanalytical strategies for Cell & Gene Therapies workshop, Málaga, Spain
- 16 - 17 September 2020, Oligonucleotide Therapeutics and Delivery conference, London, UK
- 21 - 22 September 2020, Bioproduction Summit on Advances in AAV for Gene Therapy & Protein Production, San Francisco, CA
- 22 - 24 September 2020, Advanced Therapies Manufacturing Strategy Summit, Boston, MA
- 22 - 25 September 2020, Patient Centric Cell Therapy Supply Chain Summit, Philadelphia, PA
- 1 – 3 October 2020, 2020 Cell Therapies and Immunotherapy Conference, Naples, Italy
- 6 – 7 October 2020, Cell Series UK 2020 - 6th Annual Cell & Gene Therapy Manufacturing Congress, London, UK
- 15 - 16 October 2020, Global Cell and Gene Therapy Innovation Leaders Shanghai Forum - Asia Pharma R&D Leaders Summit 2020, Shanghai, China
- 20 – 21 October 2020, Cell & Gene Therapy Research & Development Congress Asia 2020, Taipei, Taiwan
- 20 – 23 October 2020, XXVIII Congress of the European Society of Gene and Cell Therapy (ESGCT), Edinburgh UK
- 26 - 27 October 2020, 2nd Annual Gene and Cell Therapy: Quality Developments to Commercialization Summit, Munich, Germany
- 26 - 28 October 2020, Allogeneic Cell Therapies Summit 2020, Boston, MA
- 3 - 4 November 2020, 6th Annual Biopharma Development and Production (BDP) Week - 2nd Annual Cell and Gene Therapy Manufacturing, Shanghai Hongqiao, China


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, Zika Virus Net and Corona Virus Net.
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