Gene Therapy Net Newsletter |
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News: Cell & Gene Therapy Space Gears Up for First CRISPR Approval
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Halloween brings to mind pumpkins and trick or treat, but for the 100,000 Americans suffering from sickle cell disease, this past October 31 had a different meaning when an FDA advisory panel deemed the first potentially curative SCD therapy safe enough for clinical use, bolstering hopes of an FDA approval in December. It would also be the first-ever approval for CRISPR, the genetic modification technique that won its discoverers the Nobel Prize in 2020 for their 2012 breakthrough.
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News: Gene therapy restores hearing in children with rare form of deafness
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Several deaf children can hear after receiving gene therapy—a first for the approach—a team at Fudan University reported today at a meeting in Belgium. The children were born deaf because they inherited two defective copies of the gene for a protein called otoferlin that helps the inner ear’s hair cells transmit sound to the brain. In an attempt to restore this function, researchers injected harmless viruses carrying DNA for a working copy of the otoferlin gene into the children’s ears. Four of five patients treated now have some hearing, the MIT Technology Review reports.
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Conference: mRNA Technologies, Therapeutics & Vaccines Digital Week
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Join us from December 5th – 6th for our complimentary mRNA Technologies, Therapeutics & Vaccines Digital Week. Gain exclusive insight into key concepts such as advanced discovery, development of mRNA therapeutics, novel technologies, and innovative production processes. Don’t miss the opportunity to hear from an expert group, from industry experts to service providers.
Register now…
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Conference: PAT & Real Time Quality
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In today's dynamic pharmaceutical landscape, drug developers and manufacturers face the challenge of achieving sustainable and cost-efficient processes while ensuring the highest product quality. Hence, we’re excited to announce the return of the 2nd PAT & Real Time Quality Summit (December 5-7, 2023), which provides a unique platform to explore cutting-edge strategies and innovations that drive efficient production, enhance quality control and accelerate time-to-market.
Register now…
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Conference: 5th CNS Drug Delivery Summit
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As neuroscience R&D surges with investment to solve the field’s most pressing challenge, access to the brain, the 5th CNS Drug Delivery Summit (December 5-7, 2023) will provide an unmissable showcase of the latest innovations to revolutionize therapeutic administration routes to the brain and optimize dose specificity.
Join neuroscientists, neurobiologists, neuro-oncologists, protein engineers, biochemists, and CNS device experts from the likes of Roche, AbbVie, Takeda, Lundbeck, Sanofi, Biogen, Alnylam Pharmaceuticals, and Bristol Myers Squibb this December as they reunite to translate their novel brain delivery technologies into the clinic.
Register now…
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Conference: 23rd annual Peptalk conference
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Selecting the optimal carrier system for delivery has a profound impact on the quality and characteristics of the final product. Given the diverse array of applications for this technology, the need to elevate vector quality and capacity remains a growing imperative. Yet, the vector landscape poses several challenges. Enduring issues encompass immunogenicity, constrained cargo capacity, precise targeting, purification intricacies, and navigating complex regulatory frameworks. We invite you to participate in the Vector Design and Delivery pipeline (January 16-19, 2024), where we delve into pioneering approaches for refining both viral and non-viral vectors, striving for enhanced efficiency, scalability, and innovative solutions to confront the most formidable obstacles in the domain. Join us in this pursuit of transformative breakthroughs in vector design and delivery.
Register now…
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Conference: Advanced Therapies Week
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Advanced Therapies Week (January 16-19, 2024) is dedicated to helping biotech progress on their commercialization journey, as well as pushing the industry one step closer to delivering life changing treatments to patients. The event is about creating moments of natural connection for the cell and gene industry that spark long-lasting relationships, stretching far beyond transactional. Conga lines on a luxury yacht, sunrise yoga on Miami Beach and catching up with friends, old and new. These are the memorable moments that go beyond what you might expect.
Register now…
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Conference: Gene Therapy Regulatory Affairs Summit
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The Gene Therapy Regulatory Affairs Summit (January 23-25, Boston MA) is the first and only forum to address the regulatory hurdles across non-clinical, clinical & CMC functions from pre-IND through to approval and satisfy regulatory requirements for gene therapies by demonstrating safety and efficacy.
It is a must-attend meeting for those working in the field. This is your opportunity to leverage knowledge from regulatory bodies and the leading regulatory personnel across big pharma and innovative biotech to discuss the hottest topics, identify regulatory bottlenecks and offer actionable insights from AstraZeneca, Health Canada, Ultragenyx, Spark Therapeutics, Pfizer, Bridge Bioscience, Adverum, Regeneron and more.
Register now…
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Conference: Allogeneic Cell Therapies Summit Europe
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Join Caribou Biosciences, Cellectis, AdaptImmune and 60+ allogeneic experts at the 3rd Allogeneic Cell Therapies Summit Europe (January 29-31, 2024, London, UK) to help you overcome your current challenges and prepare your pipeline for success. Seize this unique opportunity to navigate the intricacies of European and global regulations. Gain insights from investment experts at Swarm Oncology and MPC Therapeutics on what it takes to secure investment for European drug developer. Obtain valuable and actionable strategies to scale up your manufacturing processes, ensuring you harness the full potential of off-the-shelf cell therapies. Register now…
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Conference: 2nd Next Generation Ophthalmic Drug Delivery Summit
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The 2nd Annual Next Generation Ophthalmic Drug Delivery Summit is returning to San Francisco this January (January 30 – February 1, 2024) to unite 90+ device engineers, biochemists, preclinical and clinical experts as they discuss how they are optimizing longer-lasting and less invasive delivery to the back of the eye. Join 23+ expert speakers from the likes of AbbVie, Boehringer Ingelheim, Bayer, Genentech, Janssen, Novartis, and Clearside Biomedical at the only industry-dedicated event focusing on overcoming key translational challenges of developing and implementing novel delivery technology as they progress their ophthalmic pipelines to the clinic!
Register now…
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Upcoming conferences and meetings
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- 15 - 16 November 2023 - USA Clinical Trials Innovation & Outsourcing Programme - New York, NY
- 16 - 17 November 2023 - Global Bioprocessing Summit - Gene & Cell Therapies - Berlin, Germany
- 27 - 30 November 2023 - 5th Annual Gene Therapy Analytical Development Summit 2023 - Boston, MA
- 28 - 29 November 2023 - International Bioprocessing and Biologics Forum - Berlin, Germany
- 28 - 30 November 2023 - Donor Selection & Cell Source Summit - Cell & Gene Therapies - San Francisco, CA
- 28 - 30 November 2023 - Cell Therapy for Autoimmune Disease Summit - Philadelphia, PA
- 28 - 30 November 2023 - Advanced Purification for Complex Biologics Summit - Philadelphia, PA
- 28 - 30 November 2023 - 4th Annual CRISPR 2.0 Congress - Boston, MA
- 4 - 6 December 2023 - Cell Therapy Analytical Development 2023 - Boston, MA
- 5 - 6 December 2023 - mRNA Technologies, Therapeutics & Vaccines Digital Week - Online
- 5 - 7 December 2023 - 5th CNS Drug Delivery Summit - Boston, MA
- 5 - 7 December 2023 - 2nd Annual PAT & Real Time Quality Summit 2023 - Gene Therapies - Boston, MA
- 6 - 7 December 2023 - mRNA Assay Development Summit - Boston, MA
- 4 – 7 December 2023 - Cell & Gene Therapy Manufacturing & Commercialization Europe - Dublin, Ireland
- 11 - 13 December 2023 - 2nd Annual LNP Immunogenicity & Toxicity Summit - Boston, MA
- 13 - 14 December 2023 - Gene Therapy Process Development Summit - Philadelphia, PA
- 16 - 19 January 2024 - Advanced Therapies Week - Gene Therapies - Miami, FL
- 16 - 19 January 2024 - 23rd annual Peptalk conference - San Diego, CA
- 23 - 25 January 2024 - Gene Therapy Regulatory Affairs Summit - Boston, MA
- 29 - 31 January 2024 - 3rd Allogeneic Cell Therapies Summit Europe - London, UK
- 30 January - 1 February 2024 - 2nd Next Generation Ophthalmic Drug Delivery Summit - San Francisco, CA
- 7 - 8 February 2024 - 7th Annual Cell & Gene Therapy Innovation Leaders Summit 2024 - Barcelona, Spain
- 13 - 15 February 2024 - 4th Gene Therapy Comparability Summit - Boston, MA
- 20 - 22 February 2024 - 6th Annual CRISPR AgBio Congress - Raleigh, NC
- 22 - 23 February 2024 - GENAP Summit 2024 - Gene & Cell Therapy - Geneva, Switserland
- 27 - 29 February 2024 - 7th CAR-TCR Summit Europe - London, UK
- 13 - 14 March 2024 - RNA Leaders Europe Congress - Boston, MA
- 11 - 12 April 2024 - FormuTech Summit 2024 - mRNA Process Development & Manufacturing - Madrid, Spain
- 8 - 11 May 2024 - American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting - Baltimore, MD
- 3 - 7 June 2024 - 10th MSS - Chromatography, Gene Therapy, Vaccines - Portorož, Slovenia
- 27 - 28 June 2024 - RESCON Europe 2024 - Inhalation & Respiratory Drug Delivery - Barcelona, Spain
- 27 - 28 June 2024 - Injecta Summit 2024 - Pre-filled Syringes, Injectable Drug Devices - Barcelona, Spain
- 24 - 27 October 2024 - 31th Annual Meeting of the European Society of Gene & Cell Therapy (ESGCT) - Rome, Italy
- 21 - 22 November 2024 - RESCON Summit 2024 - Inhalation & Respiratory Drug Delivery - New York, NY
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