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Newsletter December 2020

News: NIH project aims to maximize efficiency of AAV platform for gene therapy
Maximizing the efficiency of the adeno-associated virus (AAV) platform for gene therapy is the aim of a new pilot project of the National Institutes of Health (NIH). The NIH Platform Vector Gene Therapy (PaVe-GT) project is reported in the peer-reviewed journal Human Gene Therapy. Click here to read the full-text article free through December 16, 2020. "PaVe-GT is an experimental translational science initiative that aims to leverage the power of platform vectors and disease relatedness to help deliver on the promise of gene therapy to patients with rare diseases," states the NIH PaVe-GT team.

News: Gene Therapy in One Eye Improves Vision in Both Eyes
It’s not clear why the patients with Leber hereditary optic neuropathy, a mitochondrial disorder that causes blindness, also experienced the modest benefits in their untreated eye. In a Phase 3 gene therapy trial intended to improve vision among patients with Leber hereditary optic neuropathy, recipients gained somewhat better sight in both eyes even though only one was treated. The results and an investigation into possible explanations for the findings were published December 9 in Science Translational Medicine.

Conference: 28th Annual Molecular Med Tri-Con Virtual Conference & Expo
Join international thought leaders to discuss the latest research and technologies in COVID-19 diagnostics, vaccines and biotherapeutics, point-of-care diagnostics, biomarkers and liquid biopsy, data analytics and artificial intelligence. In addition to the leading research and technology presentations you have come to expect at the Tri-Con, February 16-18, 2021, now available for on-demand viewing, we have added interactive panel discussions and structured networking opportunities, for a comprehensive 3-day program designed to maximize collaboration and innovation
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Conference: CAR-TCR Digital Summit Europe
The CAR-TCR Summit Europe, February 16-18, 2021, is the end-to-end meeting curated for CAR & TCR drug developers actively working to engineer an affordable CAR and TCR technologies to patients. Providing the most comprehensive agenda with exclusive insights from the innovators breaking the ceiling in T-cell engineering, manufacturing, safety management & patient accessibility.
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Conference: 4th Annual Gene Therapy for Rare Disorders Summit
In a year of disruption and uncertainty, the gene therapy field continues to surpass expectations, with investment in the space higher than it has ever been. However, it is clear that there are still unique hurdles to overcome to realize the commercial potential of these gene therapies.
The 4th Annual Gene Therapy for Rare Disorders, February 22-25, 2021, is dedicated to virtually addressing these manufacturing, clinical, commercial, and regulatory challenges in order to bring gene therapies to patients more quickly and effectively. With 4 tracks of dedicated content, 8 interactive workshops, 1 focused discussion day, and plenty of opportunities to network across four days, join us on our bespoke digital platform.

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Conference: Cell & Gene Therapy Day
The Cell & Gene Therapy Day takes place virtually, February 23, 2021 and reports on the latest cell and gene therapy progress with a focus on solid tumors.
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Conference: Immuno-Oncology 360°
The IO360˚ takes place virtually, February 23-26, 2021 and convenes stakeholders spanning the science and business communities to report on the latest data impacting immuno-oncology.
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Conference: 9th Neurodegenerative Drug Development Summit
The 9th Annual Neurodegenerative Drug Development Summit, February 23-25, 2021, is returning in 2021 as an exclusive virtual platform serving to advance the discovery and translation of neurodegenerative therapeutics.
Whether you are working in a team dedicated to Parkinson’s, Alzheimer’s, Lewy Body Dementia or a rare neurodegenerative disorder, this intimate, industry-led forum is not only an opportunity to learn from those pioneering this space, but to network and build meaningful partnerships with over 150 neurodegenerative drug development experts. After all, collaboration in this industry is pivotal!
Across 3 action-packed, case-study driven days, and 2 parallel tracks of learning, we present a unique opportunity to join us online in 2021 and overcome the challenges preventing you and your team from translating promising preclinical research into evidenced clinical benefit.

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Conference: 2nd Gene Therapy for Blood Disorders
2020 has been an exciting year for gene therapies targeting blood disorders. Despite FDA’s recent rejection of BioMarin’s hemophilia gene therapy program, the clinical landscape is progressing quickly with Pfizer and Sangamo kicking off their phase 3 and bluebird bio receiving European approval for their beta-thalassemia candidate. With pipelines advancing and emerging companies joining the blood disorders field, it is essential for gene therapy developers to communicate and share lessons learned to accelerate their programs to market. The 2nd Gene Therapy for Blood Disorders meeting, March 9-11, 2021, is focusing on the clinical and commercial challenges of developing gene therapies targeting hemophilia, sickle cell disease and beta thalassemia. Evaluate the best strategies for a commercial launch in blood disease and optimize your trial design to ultimately transform your clinical candidates into a commercial reality.
Discount code: 20580GTN (10%)

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- 19 - 21 January 2021, PepTalk 2021 Conference, Online event
- 20 - 21 January 2021, Global Onco Cell Therapy Summit, Online event
- 26 - 28 January 2021, Inaugural TGFβ for Immuno-Oncology Drug Development Summit - Gene Therapies, Online event
- 9 - 11 February 2021, Gene Therapy Medical Affairs Summit, Online event
- 10 - 11 February 2021, RNA Therapeutics 2021, Online event
- 16 - 18 February 2021, 28th Annual Molecular Med Tri-Con Virtual Conference & Expo, Online event
- 16 - 18 February 2021, CAR-TCR Digital Summit Europe, Online event
- 22 - 25 February 2021, 4th Annual Gene Therapy for Rare Disorders, Boston, MA
- 23 February 2021, Cell & Gene Therapy Day, Online event
- 23 - 25 February 2021, 9th Neurodegenerative Drug Development Summit, Online event
- 23 - 26 February 2021, IO360° Summit - Gene Therapies, Online event
- 9 - 11 March 2021, 2nd Gene Therapy for Blood Disorders meeting, Online event
- 15 - 17 March 2021, International Society for BioProcess Technology 10th Spring Meeting - Viral vectors and vaccines, Norfolk, Virginia
- 16 - 17 March 2021, 4th Annual Bioprocessing Summit Europe Virtual, Online event
- 23 - 25 March 2021, Innate Killer Summit 2021, Online event
- 23 - 25 March 2021, Gene Therapy Patient Engagement, Online event
- 30 March - 1 April 2021, Gene Therapy Comparability, Online event
- 14 - 16 April 2021, World Smart Bioprocessing: Pharma 4.0 Summit, Berlin, Germany
- 20 - 21 April 2021, Formulation & Delivery Europe: Gene and Cell Therapies, Online event
- 12 - 15 May 2021, American Society of Gene and Cell Therapy (ASGCT) 24th Annual Meeting, Portland, OR
- 14 - 18 June 2021, 9th International Symposium on Monolith - DNA/RNA, vaccines, gene therapy vectors, Portoroz, Slovenia
- 29 - 30 June 2021, Formulation & Delivery US: Gene and Cell Therapies, San Diego, CA
- 31 August - 2 September 2021, CAR-TCR Summit USA, Boston, MA
- 21 - 22 September 2021, Formulation & Delivery UK: Gene and Cell Therapies, London, UK
- 22 - 23 September 2021, Oligonucleotide Therapeutics and Delivery conference, London, UK
- 12 - 13 October 2021, Pharma Europe 2021 - Cell & Gene Therapies, Barcelona, Spain
- 18 - 20 October 2021, Advancing Gene Therapy 2021, Boston, MA
- 10 - 11 November 2021, Pharma Manufacturing: Gene and Cell Therapies, Online event
- 22 - 23 November 2021, Ophthalmic Drugs Conference - Gene Therapies, London, UK


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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