Gene Therapy Net Newsletter |
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News: Gene therapy approved by FDA for certain boys with rare genetic condition
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A new gene therapy treatment gained FDA approval last month to treat boys ages four and five with Duchenne muscular dystrophy, a rare and deadly genetic disorder that leads to muscle degeneration. Connor Stoll from Chicago was the first patient in the nation to receive the infusion as part of a clinical trial at Nationwide Children’s Hospital in January 2018.
Read more…
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Conference: Gene Therapy Potency Summit
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The Inaugural Gene Therapy Potency Summit (August 29-31) is dedicated to providing you with tools to identify, characterize and measure potency, enabling you to take tactical insights back to your team. The content will focus on the “sweet spot” of drug development where potency is most critical - pre-clinical development through to commercialization.
This is the only industry-led event dedicated to covering all challenges associated with potency for gene therapy drug developers specifically. We will unite with 60+ analytical, bioassay, Quality Control, regulatory and CMC experts to bring together content that gives actionable insights by combining both strategic and technical content. Hear experts speak from companies such as the FDA, Biogen, Ultragenyx Pharmaceutical, bluebird bio, Johnson & Johnson, and Spark Therapeutics. Register now…
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Conference: 2nd Next-Generation Lipid-Based Nanoparticle Delivery Summit
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2023 is already shaping to be a landmark year for lipid-based delivery technology development and the 2nd Next-Generation Lipid-Based Nanoparticle Delivery Summit returns next month (August 29-31) to unite 140+ Delivery, Formulation, Engineering & Analytical leaders all racing to uncover the potential of lipid-carrier system properties. This is your definitive industry-led meeting to discover new lipid-based nanoparticle formulations and engineer delivery systems for emerging payloads beyond RNA for targeted & extrahepatic delivery, overcoming the limitations of conventional LNPs. Join AstraZeneca, Beam Therapeutics, Genentech, Novartis, Pfizer and more to expedite the next-generation of lipid-based nanoparticle delivery systems closer to patients this August! Register now… |
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Conference: 4th Annual Gene Therapy Immunogenicity Summit
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Optimize Immunogenicity Risk Assessments, Improve Monitoring of Immune Responses & Actively Mitigate Against Adverse Immune Reactions The challenges posed by the immune response is one of the biggest obstacles to developing safe, effective, and durable gene therapies. Over the last two decades, drug developers have observed and begun developing solutions to a wide range of immune responses to AAV vectors. The 4th Annual Gene Therapy Immunogenicity Summit (September 5-7) is your opportunity to connect with likeminded peers from gene therapy drug developers, along with leading academics, regulators, and key service providers, all making strides with improving risk assessments, monitoring, and mitigating against AAV gene therapy immunogenicity. Registering for 2023 will grant you access to keynote plenaries from leading gene therapy figures, deep-dive discussions into pressing preclinical and clinical developments, and highly appraised networking opportunities with 150+ of your peers, all to leave you with the actionable tools, insights, and connections you need to enhance your immunogenicity-focused work. Register now… |
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Conference: 5th Exosome Based Therapeutic Development Summit
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Are you looking to accelerate candidates toward commercialization within exosome-based therapeutics?
Join the 5th Exosomes Based Therapeutic Development Summit (September 5-7) to deep dive into:
- Emerging standardized analytical strategies for optimized characterization, purification, and isolation
- Key pre-clinical and clinical updates across a range of therapeutic areas
- The latest advancements in targeted delivery and therapeutic payloads
- The challenges of large-scale manufacturing
- Navigating the complex regulatory landscape
Register now…
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Conference: RNA Leaders USA Congress
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RNA Leaders is a high-quality therapeutic conference, focusing on the development of mRNA, RNAi, ASOs, oligonucleotides, vaccines, microRNAs, genome editing, wider nucleic acids & RNA targets. For investors and pharma, it offers the chance to peruse the science and meet biotechs working on a variety of different product types within an emerging space. For biotechs, it offers the chance to learn how others are meeting field-wide challenges like regulation and delivery and hear about the work being done beyond their immediate space. Join us in Boston for RNA Leaders USA Congress – 6-7 September 2023. You’ll hear the latest advances in RNA chemistry, get clinical updates from the companies developing new therapies and source the partners who are helping to deliver drugs to patients. Use code GTN15 for discount of 15%.
Register now…
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Conference: 3rd Annual Mitochondria-Related Drug Development Summit
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The 3rd Annual Mitochondria-Related Drug Development Summit (September 12-14) returns for the first in-person meeting of 80+ industry experts to explore the unique opportunities of targeting mitochondrial dysfunction in rare and prevalent diseases.
This September, unlock the breakthroughs in our understanding of mitochondrial biology, structure, and function, to accelerate drug development. Recent IND approvals and clinical successes prove the near-endless potential of mitochondria to treat cardiovascular, renal, and neurodegenerative diseases.
Join us as we bring together rare mitochondrial disease experts and prevalent disease drug developers, from companies such as AbbVie, Eli Lilly, AstraZeneca, Capacity Bio, and Astellas, for a new era of targeting mitochondrial dysfunction. Register now…
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Conference: Cell Therapy Analytics Online Symposium 2023
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Cell Therapy Analytics Online Symposium 2023 (19 September) encompasses novel characterisation techniques & robust analytical tools to ensure safe, quality therapeutic cell products are advanced through to the clinic. Engage in scientific discussions on the latest tools and technologies for advancing cell therapy analytical development from a panel of prominent leaders and scientists. View the agenda.
Register now…
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Conference: 2nd Cell Therapy Analytical Development Europe
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Following the success of last year’s inaugural meeting, the 2nd Cell Therapy Analytical Development Summit Europe heads to Amsterdam this year (19 - 21 September) as the only cell therapy-focused, industry-facing, European-specific summit in the space - view the program here. Join 100+ industry experts this September as they look to advance multi-region access, achieve consistent and scalable assay development and drive analytical standards to bring safe, high-quality cell therapies to patients across the European territories. Hear from expert speakers across 3 jam-packed days of data-driven presentations and gain control of your manufacturing processes, assay development and analytical strategy with key thought leaders from the likes of Takeda, Novo Nordisk, Cellectis and Novartis. Register now… |
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Conference: Viral Vector Characterization & Analytical Development
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Viral Vector Characterization & Analytical Development Summit, September 25-27, 2023 in Boston. Uniting Characterization, Analytical, Formulation, Quality, Engineering, CMC and Process leaders, this industry-led summit is your only for cross-learning opportunities across AAV’s, LV’s & RV’s and more – so you can benchmark your work against rapid-analytical method development transforming your peers’ workflows, turnaround, and consistency.
Register now…
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Conference: Smart Bioprocessing & Pharma 4.0 Summit
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Cparity’s World Bioprocessing Summit is launching a world platform across the Pharma & Biopharmaceutical Manufacturing community to present their technological advances, 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. This Summit program comprises complex case studies, workshops, panels, and breakouts. It will combine forward-thinking leaders from different industries to discuss a range of topics on Bioprocessing and Smart manufacturing. This event (27th - 29th September) is aimed at approaching the main challenges, innovations, and new technologies in Bioprocessing & pharmaceuticals.
There is a special discount coupon of 20% for delegates that register with discount code is “bioprocess”.
Register now…
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Upcoming conferences and meetings
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- 18 - 20 July 2023 - 3rd Tumor Myeloid-Directed Therapies Summit - Boston, MA
- 25 - 27 July 2023 - 4th Gamma Delta T Therapies Summit - Boston, MA
- 26 - 28 July 2023 - 3rd Annual mRNA-Based Therapeutics Summit - Boston, MA
- 9 August 2023 - Drug Discovery and Development - Cell & Gene Therapies - London, UK
- 14 - 17 August 2023 - The Bioprocessing Summit 2023 - Boston, MA
- 29 - 31 August 2023 - 3rd Inner Ear Disorders Therapeutics Summit - Gene & Cell Therapies - Boston, MA
- 29 - 31 August 2023 - mRNA Analytical Development Summit Europe - Berlin, Germany
- 29 - 31 August 2023 - Gene Therapy Potency Summit - Boston, MA
- 29 - 31 August 2023 - 2nd Next-Generation Lipid-Based Nanoparticle Delivery Summit - Boston, MA
- 29 August - 1 September 2023 - 8th CAR-TCR Summit - Boston, MA
- 30 - 31 August 2023 - Viral Vector Formulation & Process Development Summit Europe - Amsterdam, Netherlands
- 5 - 7 September 2023 - 4th Annual Gene Therapy Immunogenicity Summit - Boston, MA
- 5 - 7 September 2023 - 5th Exosome Based Therapeutic Development Summit - Gene Therapies - Boston, MA
- 6 - 7 September 2023 - RNA Leaders USA Congress - Boston, MA
- 12 - 14 September 2023 - 3rd Annual Mitochondria-Related Drug Dev. Summit - Gene Therapies - Boston, MA
- 18 - 21 September 2023 - Cell & Gene Therapy Manufacturing & Commercialization US - Boston, MA
- 18 - 21 September 2023 - BioProcess International - ATMP - Boston, MA
- 19 September 2023 - Cell Therapy Analytics Online Symposium 2023 - Online event
- 19 - 21 September 2023 - 2nd Cell Therapy Analytical Development Europe - Amsterdam, The Netherlands
- 25 - 27 September 2023 - Viral Vector Characterization & Analytical Development - Boston, MA
- 27 - 29 September 2023 - Smart Bioprocessing & Pharma 4.0 Summit - Cell & Gene Therapies - Berlin, Germany
- 3 – 5 October 2023 - 4th Annual Gene Therapy for Ophthalmic Disorders Summit - Boston, MA
- 12 - 13 October 2023 - Biomarkers US 2023 - San Diego, CA
- 18 – 19 October 2023 - World Drug Safety Congress Americas 2023 - Boston, MA
- 24 – 27 October 2023 - Annual Congress of the European Society of Gene and Cell Therapy - Brussels, Belgium
- 31 October - 2 November 2023 - 14th World Orphan Drug Congress Europe - Barcelona, Spain
- 6 - 9 November 2023 - 5th Gene Therapy for Neurological Disorders Summit - Boston, MA
- 7 - 8 November 2023 - Cell Series UK 2023 - London, UK
- 14 - 15 November 2023 - Precision Oncology Europe 2023 - Berlin, Germany
- 14 - 15 November 2023 - Biomarkers Europe 2023 - Berlin, Germany
- 4 – 7 December 2023 - Cell & Gene Therapy Manufacturing & Commercialization Europe - Dublin, Ireland
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