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Newsletter October 2021

News: Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations
This guidance provides FDA’s current thinking on determining sameness of human gene therapy products under FDA’s orphan drug regulations for the purpose of orphan-drug designation and orphan-drug exclusivity.

News: Fourth Boy Dies in Trial of Astellas Gene Therapy Candidate
Astellas Pharma has acknowledged that a fourth boy has died in the troubled Phase I/II trial through which its Astellas Gene Therapies (formerly Audentes Therapeutics) has been evaluating its adeno-associated virus (AAV) gene therapy candidate AT132 in patients with X-linked Myotubular Myopathy (XLMTM). Unlike the other three boys who all died last year in the Phase I/II ASPIRO trial (NCT03199469), the fourth—whose age has not been disclosed—received the lower dose of 1.3×10e14 vg/kg.

Conference: Onco Cell Therapy Summit (OCTS)
Join 110+ companies with active cell therapy pipelines in-person at the Onco Cell Therapy Summit in Boston on 16-18 November 2021. #OCTS2021
Register now...

Conference: CRISPR 2.0
In the time since its discovery, CRISPR has rapidly become an established tool in the genomics toolbox. Now the question is: What next? The online CRISPR 2.0 Congress, 16-18 November 2021 will showcase only the latest & greatest innovations by uncovering how the next generation of tools are enhancing efficacy, minimizing off-target effects & enabling efficient delivery to specific tissues.
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Conference: 3rd Gene & Cell Therapy: Quality Developments to Comm summit
Gene therapies, CAR-T Cell therapies, and other ATMPs are advancing the development, while continue facing CMC, analytical, process development, and manufacturing hurdles. The link between manufacturing and commercialization of gene and cell therapy is becoming more visible. Join virtually & in-person Gene and Cell Therapies: CMC, CGT & Vector Manufacturing Summit, 18-19 November 2021 in Munich, Germany. Attend the two-days experiences with the leading practitioners and consultants, full of case studies, 1:1 networking, and interactive Q&A. Get answers to your burning questions, explore new technologies, and extend partnerships in the area.
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Conference:Ophthalmic Drugs Conference
The 4th Annual Ophthalmic Drugs Conference taking place on the 22nd-23rd of November 2021, in London will explore new discoveries in the treatment of ocular disease, innovations in combination technologies, and the utterly unique challenges that are faced in the treatment of one of the most complex organs in the body.
The two-day agenda will offer peer-to-peer networking opportunities with leaders in the ophthalmic sphere, from Heads of Drug Development and Senior Directors of Ophthalmology, all the way to academic forerunners in the research into ocular therapy.

Register now...

Conference: 3rd Annual Gene Therapy Analytical Development
Optimizing Cell-Based, Molecular & Biophysical Analytical Methods to Guarantee Safe, High Quality, Consistent & Efficacious Gene Therapy Products that Meet Regulatory Expectations.
The online 3rd Annual Gene Therapy Analytical Development Summit, 29 November - 2 December 2021 will unite large pharma and innovative biotechs to develop robust analytical tools to guarantee the consistency, quality and safety of gene therapy products. Focused specifically on addressing the unique analytical challenges posed by AAV, lentiviral and non-viral gene therapy products, this technical scientific conference will unite the ‘boots on the ground’ scientists from the leading companies grappling with these challenges first-hand.

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Conference: Pharma Manufacturing
Join over 300 senior representatives across two days of cutting-edge presentations on the biggest issues and opportunities in drug product manufacturing and digital transformation at the Pharma Manufacturing conference, 1-2 December 2021. World leaders in operations and engineering will discuss process development, continuous manufacturing, automation, as well as facility development, working towards industry 4.0 and operational excellence through advanced data use.
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Conference: 3rd Gene Therapy for Neurological Disorders
Returning to Boston this 6-9 December 2021, the 3rd Gene Therapy for Neurological Disorders meeting will welcome discovery, preclinical, translational and clinical industry scientists to share, learn and network for the shared goal of successfully and safely delivering gene therapies to the CNS
Register now...

Conference: Synthetic Biology-Based Therapeutics Summit
This year, the synthetic biology market has seen fledgling companies rise to IPO smashing power players, as the demand for high-quality, engineered biomolecules and controllable therapeutics grows exponentially. The therapeutic potential of novel synthetic units is huge, but challenges remain in demonstrating proof-of-concept, developing assays, functional characterization, manufacturing scale-up, and identifying therapeutic applications for commercial development. With an agenda curated by leaders in synthetic biology-based therapies at Synlogic, Merck, Bayer, Senti Bio, MIT and GSK, the online Synthetic Biology-Based Therapeutics Summit, 7-9 December 2021, will provide strategic insights to deploy synthetic biology as a tool for discovery, through to therapeutic applications.
Explore innovations in circuit design and implementation, harnessing the power of computation and AI to better design functioning circuits and uncover novel components which elicit therapeutic benefit. Tackle manufacturing challenges to scale up synthetic biology-based therapeutics with gold-standard quality control and characterization.

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- 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 - 28 October 2021, 6th annual Oncolytic Virotherapy Summit, Boston, MA
- 26 - 28 October 2021, 2nd Supply Chain & Logistics Summit for gene therapies, Online event
- 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
- 9 - 10 November 2021, Biomarkers for Rare Diseases Summit - Gene Therapy, Online event
- 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
- 18 - 19 November 2021, Gene and Cell Therapies: CMC, CGT & Vector Manufacturing Summit, Munich, Germany
- 22 - 23 November 2021, Ophthalmic Drugs Conference - Gene Therapies, London, UK
- 22 - 24 November 2021, Accelerating of Gene & Cell Therapy online training, Online event
- 29 November - 2 December 2021, 3rd Annual Gene Therapy Analytical Development, Online event
- 6 - 9 December 2021, 3rd Gene Therapy for Neurological Disorders, Boston, MA
- 7 - 9 December 2021, Synthetic Biology-Based Therapeutics Summit, Online event
- 14 - 15 December 2021, Neurodevelopmental Drug Development Summit, Online event
- 1 - 2 February 2022, Formulation & Delivery US: Gene and Cell Therapies, San Diego, CA
- 28 - 30 March 2022, Advancing Gene Therapy 2022, Boston, MA
- 13 – 15 April 2022, European Society of Gene and Cell Therapy (ESGCT) Spring School, Athens, Greece
- 21 - 22 April 2022, Global Bioprocessing Summit - ATMP Development, Berlin, Germany
- 16 - 19 May 2022, American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting, Washington, D.C.
- 13 - 17 June 2022, 9th Monolith Summer Symposium – mRNA, Vaccines, Gene Therapy Vectors, Portoroz, Slovenia
- 10 – 14 October 2022, XXIX Congress of the European Society of Gene and Cell Therapy (ESGCT), Edinburgh, 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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