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Newsletter March 2021

News: EMA Launches Safety Review After Gene Therapy Trial Patient Develops Blood Cancer
The European Medicines Agency (EMA) has launched a safety review of bluebird bio’s thalassaemia drug Zynteglo, a conditionally licensed gene therapy in Europe. This review comes after researchers identified an acute myeloid leukaemia (AML) case in a patient who received bb1111 (LentiGlobin), an investigational gene therapy for sickle cell disease (SCD), in a Phase I/II study. This therapy relies on the same viral vector found in Zynteglo which delivers a gene into cells.

News: Gene therapy trials for sickle cell disease halted after two patients develop cancer
A company has stopped its clinical studies of a promising gene therapy for the blood disorder sickle cell disease after two people who participated developed leukemia-like cancer. Bluebird bio is now investigating whether a virus it uses to deliver a therapeutic gene caused the cancers, reviving old concerns about the risks of this approach. It’s also possible the cancers stemmed from chemotherapy the patients received to prepare their bodies for the gene’s delivery. “This is really a sad development whatever the cause,” says Donald Kohn of the University of California, Los Angeles, who has led gene therapy trials for sickle cell and other diseases.

Conference: Cell & Gene Therapy Manufacturing & Commercialization Asia 2021
Delivering science, technologies and partners to accelerate stem cell, CAR-T and gene therapies to market.
The full agenda is now available for Cell & Gene Therapy Manufacturing & Commercialization Asia 2021, April 5-8, 2021. Due to the continuing pandemic, this year's event will be combined with our BioProcess International Asia event, free-to-attend and delivered in a 100% virtual format. Attendee registration will open up later this month (March 2021), so sign up for our email updates below to receive agenda and registration notifications.

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Conference: Virtual World Orphan Drug Congress USA 2021
Over 1,000 attendees from 50+ countries will gather virtually April 28th for the online edition of the of World Orphan Drug Congress USA!
This conference has become the industry defining meeting on Advanced Therapies for rare diseases, featuring discussions on clinical development, manufacturing and commercialization of cell and gene therapies, as well the annual gathering for artificial intelligence and digital health solutions transforming the rare disease industry!
Becoming the largest rare disease and orphan drug conference globally has allowed us to bring together leading industry, government and regulatory authorities, patient advocacy groups, payers, investors and solution providers who rely on World Orphan Drug Congress USA to meet, brainstorm and dialogue on ways to advance orphan drug development and improve access to life-saving therapies — all without having to leave their homes.

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Conference: Genetic Vaccine Development for Infectious Diseases Summit
With genetic vaccines launching at record speeds, the first Genetic Vaccine Development for Infectious Diseases Summit, May 18-20, 2021, will focus on overcoming the translational and clinical challenges of developing genetic vaccines targeting infectious diseases, including Influenza, Lassa fever, Ebola, MERS, HIV, Zika, and COVID-19.
With a focus on mRNA, DNA, and engineered viral vector-based vaccines, this niche 3-day agenda features a world-class speaker faculty on the front line of genetic vaccine development, including BioNTech, Pfizer, Moderna, AstraZeneca, Inovio and CureVac.
Discover updates on long-term clinical efficacy and safety, immune responses, novel delivery methods and genetic platforms, and network with thought-leaders paving the way for next-generation vaccine technologies for infectious diseases.

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Conference: Gene Therapy Analytical Development Europe
Optimising Analytical Methods to Guarantee Safe, High Quality, Consistent & Efficacious Gene Therapy Products, Gene Therapy Analytical Development, 25-27 May, 2021, is the Comprehensive Gene Therapy Analytical event.
Join 150+ analytical pioneers to discover the realities of working with tools like dPCR, AUC and HPLC, as well as how and where these tools can be most effective in process development, and what the European regulators expect in order to grant approval. Whether you’re working with AAV or lentiviral vectors, this is your opportunity to enhance your existing analytical methods and explore innovative new tools to support safe and effective gene therapy development.

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Conference: Gene Therapy for Muscular Disorders
The first Gene Therapy for Muscular Disorders Summit arrives on your screens on May 25-27, 2021!
Join your fellow industry leaders including PTC Therapeutics, Solid Biosciences, Pfizer, AskBio & Novartis to overcome the translational and clinical challenges of developing genetic therapies targeting muscular disorders, including Duchenne Muscular Dystrophy (DMD), Limb-Girdle Muscular Dystrophy (LGMD), Pompe Disease and X-Linked Myotubular Myopathy.
With clinical holds lifting there has been more investment and clinical progress in the gene therapy for muscular disorders space than ever before, yet the consequences of toxicity are not a distant memory. Despite the successes, there are still withstanding challenges for gene therapy developers targeting the muscle when it comes to optimizing delivery and navigating toxicity to develop truly safe and transformative therapeutics for muscular disorders patients.
With a focus on AAV and gene editing technologies, this niche 3-day agenda has been specifically designed to cover immunogenicity challenges and mitigate toxicity with high doses targeting the muscle to successfully demonstrate durability with your gene therapy product.

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- 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 March 2021, Macrophage Therapy Symposium, Online event
- 23 - 25 March 2021, Innate Killer Summit 2021, Online event
- 23 - 25 March 2021, Gene Therapy Patient Engagement, Online event
- 24 - 25 March 2021, Gene and Cell Therapies CMC & Vector Manufacturing Online Summit, Online event
- 30 March - 1 April 2021, Gene Therapy Comparability, Online event
- 5 - 8 April 2021, Cell Therapy Manufacturing Asia & BioProcess International Asia, Online even
- 20 - 21 April 2021, Formulation & Delivery Europe: Gene and Cell Therapies, Online event
- 27 - 29 April 2021, Neuroimmunology Drug Development Summit, Online event
- 28 April 2021, World Orphan Drug Congress USA Virtual, Online event
- 11 - 14 May 2021, American Society of Gene and Cell Therapy (ASGCT) 24th Annual Meeting, Online event
- 18 - 20 May 2021, Genetic Vaccine Development for Infectious Diseases Summit, Online event
- 25 - 27 May 2021, Gene Therapy Analytical Development Europe, Online event
- 25 - 27 May 2021, Gene Therapy for Muscular Disorders, Online event
- 26 - 28 May 2021, International Society for Cell and Gene Therapy (ISCT) New Orleans virtual meeting, Online event
- 8 - 10 June 2021, Next Generation Gene Therapy Vectors, Online event
- 29 - 30 June 2021, Formulation & Delivery US: Gene and Cell Therapies, San Diego, CA
- 13 - 15 July 2021, Epigenetic Therapeutic Targets Summit, Online event
- 19 - 21 July 2021, 2nd Gene Therapy for Neurological Disorders Europe, Online event
- 16 - 19 August 2021, 13th Annual The Bioprocessing Summit, Boston, MA
- 24 - 26 August 2021, 13th Annual Next Generation Dx Summit, Washington DC
- 25 - 27 August 2021, World Orphan Drug Congress USA, Maryland, USA
- 31 August - 2 September 2021, CAR-TCR Summit USA, Boston, MA
- 1 - 3 September 2021, World Smart Bioprocessing: Pharma 4.0 Summit, Berlin, Germany
- 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
- 19 - 21 October 2021, XXVIII Congress of the European Society of Gene and Cell Therapy (ESGCT), Brussels, Belgium
- 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
- 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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