Gene Therapy Net Newsletter |
|

|
|
News: Lentiviral vector-based gene therapy demonstrates long-term safety and efficacy for Wiskott-Aldrich Syndrome
|
|
|
Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.
Read more…
|
|
|
|
|
News: Nanoscope Therapeutics announces FDA clearance of IND for MCO-010 gene therapy in Stargardt patients
|
|
|
Nanoscope Therapeutics Inc. announced that it has received IND clearance from the FDA to begin a Phase 2 trial of its Multi-Characteristic Opsin (MCO-010) ambient-light activatable optogenetic monotherapy to restore vision in Stargardt patients.
Read more…
|
|
|
|
|
Conference: 10th Neurodegenerative Drug Development Summit
|
|
|
Learn from Sanofi, Novartis, Biogen, Roche and more at the 10th Neurodegenerative Drug Development Summit, March 28-30 in Boston! From investigating emerging therapeutics like oligonucleotides, gene therapy and cell therapies against neurodegenerative disease to exploring innovative delivery technology to move across the blood brain barrier, the 10th NDD Summit will unite 40+ CNS leaders to divulge the critical insights you need to realign and optimize your pipeline strategy.
Register now…
|
|
|
|
|
Conference: Advancing Gene Therapy 2022
|
|
|
Meet the industry forerunners at BSC's inaugural Advancing Gene Therapy 2022, March 28-30 in 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.
Register now…
|
|
|
|
|
Conference: 2nd Gene Therapy Comparability Summit
|
|
|
Implement Effective CMC & Analytical Comparability Studies to Maintain Safety, Consistency and Efficacy at Every Stage of Gene Therapy Manufacturing from Phase 1 to Commercialization. The Gene Therapy Comparability Summit, March 29 – April 2 in Boston, returns for the second year providing you with all the practical insights to optimize your gene therapy comparability approach. In the context of scaling up gene therapy development to achieve commercial goals and mounting regulatory scrutiny, the meeting will unite large pharma and innovative biotechs to ensure you can establish comparability at every stage of development, supporting a seamless transition to the larger scales required for commercialization.
Register now…
|
|
|
|
|
Conference: 3rd Gene Therapy for Blood Disorders
|
|
|
In the context of intense scrutiny over the safety and durability of blood disease-targeted gene therapies and a flurry of excitement around novel gene editing technologies, the 3rd Gene Therapy for Blood Disorders Summit, March 29 – 31, will unite the leading large pharma and innovative biotechs to share essential learnings from across this rapidly evolving field, enabling you to progress your pipeline to address the significant unmet need in this field. Focused specifically on hemophilia, beta-thallasemia and sickle cell-directed gene therapies and gene editing approaches, this event is an invaluable opportunity to learn the realities of durability challenges, understand strategies to guarantee safety at every stage of development and get a head-start in understanding the most promising technologies set to revolutionise the field. Incorporating insights from translational, clinical and commercial industry experts, as well as leading clinicians actively involved in late-stage trials, this is your opportunity to investigate the realities of managing immunogenicity for systemically administered gene therapies, establishing less cytotoxic conditioning regimens for ex vivo approaches and prepare for the commercial reality of launching a gene therapy and ensuring there is widespread patient adoption. Register now…
|
|
|
|
|
Conference: 7th Innate Killer Summit
|
|
|
The 7th Innate Killer Summit, March 30 - April 1 in San Diego, is the only industry-dedicated forum overing end-to-end development of NK, gamma delta, NKT, and Macrophage therapies, from discovery to clinical translation, manufacturing, and brand new this year: CMC & Phase Development. As Innate Cell therapies catch up to T cell therapies with monumental investment and show clear therapeutic advantages, whether you work directly on NK or gamma delta T cells or your expertise lies in broader immunotherapy, there has never been a more important time for you and your team to join the only meeting in the conference calendar dedicated to harnessing the innate immune system as the cell therapy of the future.
Register now…
|
|
|
|
|
Conference: 2nd Gene Therapy for Muscular Disorders Summit
|
|
|
Returning to Boston, April 4-7, as its first in-person event, the 2nd Gene Therapy for Muscular Disorders Summit is the field’s only industry-focused event specifically designed to help gene therapy developers successfully target the muscle. With a disease-agnostic agenda, but highlighting novel research in Duchenne’s (DMD), Limb-Girdle (LGMD), Pompe Disease and X-Linked Myotubular Myopathy (XLMTM), the expert industry speakers from the likes of Solid Biosciences, Sarepta Therapeutics, Astellas Gene Therapies and Pfizer will spend 4-days discussing the fields hottest topics from navigating toxicity to state-of-the-art muscular targeting. Uniting over 150 gene therapy experts in Boston, don’t miss your chance to share knowledge, catch up with friendly faces, and make new connections to help you to develop truly transformative gene therapies targeting the muscle.
Register now…
|
|
|
|
|
Conference: Global Bioprocessing Summits San Francisco
|
|
|
Keynotion Global Bioprocessing Summit, April 6-8 in San Francisco, brings together leading bioprocessing scientists and engineers to share day-to-day challenges and practical solutions for the development, manufacturing and quality of antibodies, vaccines and gene therapies. This world class conference is a mixture of Comprehensive Workshop, detailed case studies and interactive discussions in cell culture, scale-up, continuous Biomanufacturing, recovery, purification, process and product characterization, and formulation
Register now…
|
|
|
|
|
Conference: 2nd Gene Therapy Medical Affairs Summit
|
|
|
The 2nd Annual Gene Therapy Medical Affairs Summit, April 19-21 in Boston, is dedicated to exploring the pivotal internal and external role Medical Affairs plays in delivering gene therapies to patients. This is your opportunity to take inspiration from Medical Affairs leaders of the gene therapy world and learn how traditional Medical Affairs approaches are being transformed to meet the unique challenges associated with gene therapy drug development. Gain actionable insights from first-to-market pioneers, who will share how to engage effectively with internal clinical, commercial and regulatory teams as well as external HCPs, regulators and patient advocates to create a cohesive and streamlined launch strategy for complex, high priced Gene Therapy products. The summit will provide a dedicated platform that discusses the role and value of Medical Affairs across each of the phases of the gene therapy development cycle. We will delve into the unique challenges encountered when working with gene therapies, from adapting to the clinical realities of the rare disease space, to handling long-term follow up and post-launch regulatory scrutiny, equipping you with the insights required to launch gene therapy products more efficiently and effectively than ever before. Register now…
|
|
|
|
|
Upcoming conferences and meetings
|
|
|
- 22 - 24 February 2022 - Next Generation CAR-TCR Summit - London, UK
- 7 - 10 March 2022 - 5th Annual Gene Therapy for Rare Disorders - Boston, MA
- 9 - 11 March 2022, From Stem Cells and Genes to Therapies - Joint meeting with the AGCTS and ASSCR, Melbourne
- 14 - 16 March 2022 -11th Spring Meeting ISBioTech - Viral vectors and vaccines - Norfolk, Virginia
- 22 - 24 March 2022 - Bioprocessing Summit Europe - Barcelona, Spain
- 28 - 30 March 2022 - 10th Neurodegenerative Drug Development Summit - Boston, MA
- 28 - 30 March 2022 - Advancing Gene Therapy 2022 - Boston, MA
- 29 - 31 March 2022 - 3rd Gene Therapy for Blood Disorders Summit - Boston, MA
- 29 March - 1 April 2022 - 2nd Gene Therapy Comparability Summit - Boston, MA
- 30 March - 1 April 2022 - Innate Killer Summit - San Diego, CA
- 6 – 8 April 2022 - Global Bioprocessing Summits - Cell & Gene therapy analytics - San Francisco, CA
- 4 – 7 April 2022 - 2nd Gene Therapy for Muscular Disorders Summit - Boston, MA
- 13 – 15 April 2022 - European Society of Gene and Cell Therapy (ESGCT) Spring School - Athens, Greece
- 18 – 20 April 2022 - 4th Neuroimmunology Drug Development Summit - Boston, MA
- 19 – 21 April 2022, 2nd Gene Therapy Medical Affairs Summit, Boston, MA
- 21 - 22 April 2022 - Global Bioprocessing Summit - ATMP Development - Berlin, Germany
- 25 - 27 April 2022 - 3rd Supply Chain and Logistics for Cell Therapies Summit, Boston, MA
- 26 - 28 April 2022 - 5th Neoantigen Summit Europe - T-cell therapies - Amsterdam, the Netherlands
- 9 – 12 May 2022 - 4th Allogeneic Cell Therapies Summit - Boston, MA
- 11 – 13 May 2022 - Global Bioprocessing Summit Europe - Cell & Gene therapy analytics - Frankfurt, Germany
- 16 - 19 May 2022 - American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting - Washington, D.C.
- 17 - 18 May 2022 - Advanced Therapies (ATMP) – Challenges for Routine Applications - Frankfurt am Main, Germany
- 24 - 26 May 2022 - Next Generation RNA Therapeutics Summit - Boston, MA
- 7 - 9 June 2022 - 2nd Annual Gene Therapy Patient Engagement Summit - Boston, MA
- 13 - 17 June 2022 - 9th Monolith Summer Symposium, mRNA, Vaccines, Gene Therapy Vectors - Portoroz, Slovenia
- 14 - 16 June 2022 - Next Generation CAR & T Cell Therapies 2022 event - San Francisco, CA
- 29 - 30 June 2022 - Onco Cell Therapy Summit (OCTS) - Boston, MA
- 11 – 14 October 2022 - XXIX Congress of the European Society of Gene and Cell Therapy (ESGCT) - Edinburgh, UK
|
|
|
|
|