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Newsletter September 2021

News: New gene-delivering workhorse could make gene therapy safer, more effective for muscle diseases
Genetic muscle diseases lead to progressive muscle wasting and often early death, with few treatment options and no cure. Some gene therapies that use a harmless virus to deliver a functioning copy of a disease-causing gene to cells have shown promise in clinical trials for a subset of muscular dystrophies, but have faced challenges. High doses of the gene-carrying virus are needed to reach the muscles throughout the body and the viruses used in these trials often end up in the liver more than in the muscle. This has led to high levels of the virus in the liver, severe adverse side effects, and even death in some trial participants. Researchers at the Broad Institute of MIT and Harvard and Harvard University have developed a new family of adeno-associated viruses (AAVs) - the gene-delivering workhorse of gene therapy - that improve targeting of the muscle tissue, which could be safer and more effective for patients with muscle disease.

News: Lysogene Announces First Patient in the United States Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis
Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced dosing of the first patient in the United States with LYS-GM101 investigational gene therapy at CHOC Hospital (CHOC) in a global adaptative-design clinical trial (NCT04273269) for the treatment of GM1 gangliosidosis. This trial is an interventional, multi-center, single-arm, two-stage adaptive-design study evaluating the intracisternal delivery of a recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human β-galactosidase gene (GBL1). The clinical trial includes a safety phase and a confirmatory efficacy phase. The trial will enroll 16 patients with a diagnosis of early or late infantile GM1 gangliosidosis at sites in the US and Europe.

Conference: Global Bioprocessing Summit - A Virtual Event
Global Bioprocessing Virtual Summit, 26-27 October 2021, is a leading forum for process development, analytical and formulation scientists and industry experts to share day-to-day challenges and practical solutions for the development, manufacture and quality of antibodies, vaccines and gene therapies. Conferenzia World’s Global Bioprocessing Summit will provide a common platform for all stakeholders to build up a relationship and discuss on addressing global health manufacturing challenges, optimizing technologies and strategies such as scaling up, single-use technology, digitalization and automation to accelerate bioprocessing.
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Conference: Oncolytic Virotherapy Summit
In its most exciting year yet the field of oncolytic virotherapy has seen the highest number of clinical read-outs, the emergence of next generation viruses, and never-before-seen combination therapies. Now in its 6th year, the Oncolytic Virotherapy Summit, 26-28 October 2021 in Boston, is the industry’s definitive guide to turning viral vehicles into clinically effective therapeutics across oncology indications. With 3 days of in-depth cutting-edge case studies and the highest calibre of industry discussions, The OV Summit will provide the roadmap to oncolytic virotherapy market success. Uncover fresh studies from pharma and biotech companies of all sizes, as well as clinicians and leading academics for a complete picture of the current challenges and opportunities.
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Conference: 2nd Supply Chain & Logistics Summit
As an increasing number of candidates enter the clinic and promise to be a mainstay in the future of cancer treatment, CGT pose unique supply chain challenges. From time and temperature sensitivity, to patient-facing services and integrated platforms, a seamless vein-to-vein supply chain is central in maintaining cell immunotherapy safety and efficacy at scale and maximizing the patient experience. This October (26-28 October 2021) join industry experts from large pharma, biotech, standards agencies and supply chain and logistics service providers. Hear 16+ real-world case studies led by supply chain, strategic sourcing, apheresis, and technical operations experts. Ensure effective forecasting, risk management, quality assurance and service at scale to meet clinical to commercial demand.
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Conference: Smart Bioprocessing & Pharma 4.0 Summit
CParity’s World Smart Bioprocessing: Pharma 4.0 Summit, 27-29 October 2021 in Berlin, is launching a world platform across the Pharma & Biopharmaceutical Manufacturing community to present their technological advances, to boost innovation, and exchange insights on current challenges. The event will gather top leaders in a 3-day event, aiming to promote a high level of participation to provide the appropriate environment for dialogue with peers from pharma, biotech, schools, and pharma suppliers to maximize our efforts and profit in the industry
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Conference: MarketsandMarkets Cell & Gene Therapy Manufacturing Virtual Conference
Over the last decade, the field of cell therapy has rapidly grown, and it holds enormous promise for treating many diseases. There are still factors like manufacturing maze, investment, logistics and regulatory challenges which prevents the cell and gene therapies to be widely used. We are once again putting together a unique platform to provide the exact solutions to these robust manufacturing and bioprocessing challenges, at our 3rd Annual MarketsandMarkets Cell & Gene Therapy Manufacturing Virtual Conference. We have created a virtual engaging model that will be beneficial for speakers, attendees and sponsors/exhibitors, taking place on 28-29 October 2021. Learn from the leading experts of the industry about strategies, technologies and innovations in the area of bioprocessing of cell-gene therapies
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Conference: 5th Annual Gene Therapy for Rare Disorders Europe
The 5th Annual Gene Therapy for Rare Disorders Europe, 2-4 November 2021, is dedicated to overcoming manufacturing, clinical, and commercialisation challenges drug developers face when delivering gene therapies to market. In the context of unprecedented clinical activity, rapidly approaching approvals and investment at an all-time high, Gene Therapy for Rare Disorders Europe will leverage the experience from the first to market pioneers, as well as unveiling the strategies the next generation of drug developers are employing to improve the efficacy, safety and commercial viability of your gene therapy pipeline. Across three content-packed days, this conference will unite leading experts from innovative biotechs, large pharma, academia and key service provider companies, all with the specific focus of solving complex commercial challenges to deliver on the promise of gene therapies for rare disease patients
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Conference: Biomarkers for Rare Diseases Summit
As reports predict the global biomarker test market will exceed $16bn in coming years, the ability to identify and utilize predictive, diagnostic and prognostic biomarkers is a priority for drug developers. In rare disease, affecting over 400 million worldwide and where clinical insights exist for only around 400 of the 7000+ disease identified, biomarkers are a valuable tool to accelerate drug development where there is current great unmet need. With this, we are excited to announce the launch of the inaugural Biomarkers for Rare Diseases Summit, 9-10 November 2021.
Uniting world leaders developing novel therapeutics for rare disease, we will discuss learnings, challenges and future directions at the only summit dedicated to approaches to identify, test and proof clinical biomarkers in rare disease for regulatory approval. From identifying disease driving biomarkers, robust assay validation, harnessing latest technologies and demonstrating therapy efficacy, we bring you the definitive platform to accelerate bench to bedside rare disease development towards regulatory filing and patient populations.

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- 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
- 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
- 22 - 23 November 2021, Ophthalmic Drugs Conference - Gene Therapies, London, UK
- 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


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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