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Newsletter August 2021

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News: FDA allows Novartis gene therapy trials to resume after nearly 2 year pause
Novartis on Tuesday said it will begin a new study of its gene therapy Zolgensma in older patients with spinal muscular atrophy following the Food and Drug Administration's clearance of administration via a spinal infusion. The regulator had suspended testing of that dosing in October 2019 due to safety concerns.
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News: Gene therapy delivered to the brain shows promise in children with rare neurodegenerative disease
Scientists from Ohio State University have developed a novel method for delivering gene therapy to specific regions of the brain. Now, they have evidence from a small clinical trial in children that the treatment could address a rare, inherited neurodegenerative disease. And they believe their technique could eventually be used to treat more common brain diseases, like Alzheimer’s and Parkinson’s. The Ohio State team developed the gene therapy to treat aromatic L-amino acid decarboxylase (AADC) deficiency, which hampers the body’s ability to make dopamine and serotonin and results in developmental delays and a range of motor and behavioral symptoms. The gene therapy uses a viral vector to carry DNA-expressing AADC to the brain.
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Conference: Cell & Gene Therapy USA - Reuters Event
Getting a cell and gene therapy approved is a huge feat. Years of hard work, evidence generation, and record-breaking science prepare these therapies for success – but it is post-approval, which so few have yet to reach, where the complexities and uniqueness of commercialization could spell tragedy, if done incorrectly.
That's why Reuters Events are proud to bring you Cell & Gene Therapy 2021! Back for its 2nd year (on 28-29 September 2021, virtual), the 2-day online event will bring together over 2000 decision makers to marry science with strategy and get therapies to market most efficiently, post-FDA approval.

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Conference: Advanced Therapies LIVE
We bring together the Advanced Therapies community, 5-6 October 2021 in London, to look at what’s happened in the industry in the last year, what the effect of the COVID-19 outbreak has been, good and bad, and how we evolve from here. This summit will explore, define and attempt to tackle the challenges to development of patient access to ATMPs, and how we move forward into a brighter 2022.
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Conference: Pharma Europe 2021
Join thousands of your peers at the world’s gathering for pharma pioneers and health innovators - https://reutersevents.com/events/barcelona/Reuters Events Pharma 2021, 11 - 22 October, 2021, Virtual (formerly eyeforpharma Barcelona) – the biggest, most senior and cross-functional commercial pharma event. We may be in the year of the vaccine, but we’re just beginning the decade of pharma’s awakening.
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Conference: Advancing Gene Therapy 2021
Meet the industry forerunners at BSC's inaugural Advancing Gene Therapy 2021, 18 - 20 October 2021, Boston, to gain insights into their strategies and program development. The Summit will highlight the challenging field of gene therapeutic approaches. Advances in development and manufacturing will be presented, as well as quality control methods and regulatory aspects: The focus is on the path to safe and beneficial advanced therapies for the patient.
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Conference: 2nd Annual Dry AMD Therapeutic Development Summit
Explore Gene Therapy as a novel area of dry-AMD R&D at the Dry-AMD Therapeutic Development Summit, 19-21 October 19-21, 2021, Virtual Event. This October, over 40 world-class speakers and 100 ophthalmology experts from #AbbVie, #IVERIC Bio, #Apellis, #Novartis, #Gemini Therapeutics and more will come together to accelerate the approval of next-generation therapies for this unmet medical need.
Dry-AMD Therapeutic Development is the only end-to-end forum solely dedicated to targeting inflammatory and regenerative pathways whilst overcoming challenges with drug delivery, safety and validated endpoints to bring about clinically effective dry-AMD therapies.

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Conferences
- 16 - 19 August 2021, 13th Annual The Bioprocessing Summit, Boston, MA
- 25 - 27 August 2021, World Orphan Drug Congress USA, Maryland, USA
- 30 August - 2 September 2021, 6th CAR-TCR Summit, Online event
- 7 - 9 September 2021, Next Generation Cancer Vaccine Development Summit, Online event
- 14 - 16 September 2021, Gene Therapy for Metabolic Disorders Summit, Online event
- 21 - 22 September 2021, Formulation & Delivery UK: Gene and Cell Therapies, London, UK
- 21 - 23 September 2021, 2nd Gene Therapy for Ophthalmic Disorders, Online event
- 22 - 23 September 2021, Oligonucleotide Therapeutics and Delivery conference, London, UK
- 28 - 29 September 2021, Cell & Gene Therapy USA - Reuters Event, Online event
- 5 - 6 October 2021, Advanced Therapies LIVE 2021, London, UK
- 12 - 13 October 2021, Pharma Europe 2021 - Cell & Gene Therapies, Barcelona, Spain
- 18 - 20 October 2021, Advancing Gene Therapy 2021, Boston, MA
- 19 - 21 October 2021, 2nd Annual Dry AMD Therapeutic Development Summit, Online event
- 19 - 21 October 2021, XXVIII Congress of the European Society of Gene and Cell Therapy (ESGCT), Online event
- 26 - 27 October 2021, Global Bioprocessing Summit - ATMP Development, Online event
- 26 - 28 October 2021, 6th annual Oncolytic Virotherapy Summit, Boston, MA
- 27 - 29 October 2021, World Smart Bioprocessing: Pharma 4.0 Summit, Berlin, Germany
- 2 - 4 November 2021, 5th Annual Gene Therapy for Rare Disorders Europe, Online event
- 8 - 10 November 2021, 2nd Annual Inborn Error of Metabolism Drug Development Summit - Gene Therapies, Boston, MA
- 10 - 11 November 2021, Pharma Manufacturing: Gene and Cell Therapies, Online event
- 16 - 17 November 2021, Onco Cell Therapy Summit (OCTS) USA 2021, Boston, MA
- 16 - 18 November 2021, CRISPR 2.0 Summit - Gene editing, Online event
- 22 - 23 November 2021, Ophthalmic Drugs Conference - Gene Therapies, London, UK
- 29 November - 2 December 2021, 3rd Annual Gene Therapy Analytical Development, Online event

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