Alliance for Regenerative Medicine

ARM’s Q3 2024 cell and gene therapy sector data is now available! Dive in to view our figures highlighting clinical trials, the number of therapeutic developers, and total sector investment. One new trend to highlight: ?? Sector investment through Q3 2024 has now surpassed total sector investment in all of 2023 View the complete data on our website. ?? https://lnkd.in/ebVuvmKK

Calling all future #cellandgenetherapy leaders! ARM is accepting host company?and?intern?applications for the GROW Internship Program. This program provides crucial, early-career paid opportunities in the regenerative medicine sector to talented undergraduate and graduate students whose backgrounds and life experiences have shaped their unique perspectives. The application deadline is February 15, 2025, with the early selection deadline being December 15, 2024. All majors are encouraged to apply. Learn more: https://lnkd.in/eM5EzPsT #RegenerativeMedicine #GROWRegenMed

Last night, ARM joined senior leaders and policy leaders in the European Parliament for our reception "How to cure the incurable? The promise of ATMPs and overcoming patient access challenge," co-hosted by MEP Stine Bosse and MEP Romana Jerkovi?. Our thanks to the hosts, speakers and attendees for convening to share their perspectives on the unique promise of advanced therapies and how we can ensure access for patients in Europe.

Happening today - Commercial Strategies for Cell and Gene Therapies ??? ?? 11:00am - 12:00pm ET Join ARM and Catalent Pharma Solutions for our last Building the Future of Regenerative Medicine webinar of the year. This live webinar will discuss the critical steps required to successfully bring cell and gene therapies to market. Register here to tune in: https://lnkd.in/gEG8xKXe Moderator: Delara Motlagh Ph.D. Vice President, Global Cell Therapy and Plasmid DNA, Catalent Pharma Solutions Featured Speakers: Rob Allen, Ph.D. Managing Partner and General Manager Europe, Dark Horse Consulting Group Inc. Sarah Creviston Global Vice President, Patient Advocacy and Public Affairs, Novartis Gene Therapies John King Chief Commercial Officer and Head of Business Development, Vor Bio Kristin Yarema, Ph.D. President and CEO, Poseida Therapeutics, Inc.

Our next Scientific Exchange is about to start! Looking to explore ways to advance gene editing platforms? You'll be able to view today's event virtually at no cost. Tune in today at any time by registering here: https://lnkd.in/eMp5gZwZ The event is co-organized by ARM, ISCT, International Society for Cell & Gene Therapy, and Danaher Corporation. It will convene key stakeholders, including senior leaders from the FDA. The 2024 Scientific Exchange seeks to make progress on therapeutic development for rare conditions that are scientifically feasible but commercially non-viable.

We’re thrilled to welcome Tay Salimullah as a fellow in the ARM Fellowship Program, where he will bring invaluable insight to our work on critical topics in Cell and Gene Therapy Ethics and Society. Tay will provide strategic counsel on pivotal areas including pricing, value, and access, advancing industrialization for global reach, addressing ultra-rare disease challenges, and fostering scientific engagement in advanced MedTech. Currently a Venture Partner at 4BIO Capital, Tay previously served on the executive team at Novartis Gene Therapies, where he was instrumental in establishing global, industry-leading capabilities for pioneering a multi-billion-dollar gene therapy and rare disease business. He was also recently appointed to the Board of Directors at Trogenix, a company developing a breakthrough Synthetic Super-Enhancer platform, Odysseus, to enable unprecedented precision in targeting aggressive cancers. We’re eager to collaborate with Tay to drive forward meaningful conversations and leadership on the field’s most complex issues.

ARM, the Institute for Gene Therapies, the National Organization for Rare Disorders, EveryLife Foundation for Rare Diseases, The Leukemia & Lymphoma Society, and 130+ other patient, provider, and research organizations call on Congress to pass three bills by the end of the year to ensure patient access to breakthrough therapies. In a letter addressed to Congressional leadership, we urge the inclusion of three bills in any end-of-year legislative package: ?? The reauthorization of the Rare Pediatric Disease PRV Program ?? The MVP Act (S. 4204 / H.R. 2666) ?? The Accelerating Kids Access to Care Act (S. 2372 / H.R. 4758) To improve patient access, we must incentivize innovation, find sustainable financing mechanisms for new advanced therapy treatments, and alleviate administrative barriers. These three bipartisan bills help address these issues. Congress needs to pass them now! Read the full letter here: https://lnkd.in/ekHdjwND

This week has some notable regulatory news for several gene therapies. ?FDA approves gene therapy for AADC deficiency ??BLA accepted by the FDA for a gene therapy to treat recessive dystrophic epidermolysis bullosa (RDEB) ??First epigenetic therapy to treat an infectious disease enters clinical trials Read the full stories below and subscribe to ARM's Sector Vector newsletter for more weekly news on cell and gene therapy clinical, business, and manufacturing. ?? https://lnkd.in/eD7ZNaU The FDA announced that it approved PTC Therapeutics, Inc.'s gene therapy to treat Aromatic L-amino acid decarboxylase (AADC) deficiency, a fatal rare genetic disorder. This is the first-ever gene therapy approved in the US that is administered directly to the brain. https://lnkd.in/eEsgjEXD The FDA accepted for review Abeona Therapeutics' resubmission of BLA for its cell-based gene therapy, as a potential new treatment for recessive dystrophic epidermolysis bullosa (RDEB). The PDUFA date is April 29, 2025. https://lnkd.in/esq5a4nU Tune Therapeutics announced that it has received clinical trial application approval in New Zealand to initiate a Phase 1b clinical trial for TUNE-401 – an investigational epigenetic silencing therapy designed to treat chronic Hepatitis B. https://lnkd.in/eEvnSYS9 #genetherapy #celltherapy #epigentics

Calling all cell and gene therapy innovators! ?? Apply now to present at ARM's 2025 Meeting on the Med, Europe’s premier cell and gene therapy conference. Showcase your work to over 600 attendees, including industry leaders and tech pioneers. The deadline to apply is December 13—don’t miss your chance! ?? https://lnkd.in/eUTEuU7 #CGMed25 #cellandgenetherapy

The recent wave of cell and gene therapies has brought many promising treatments to the market. Many stakeholders, however, have important questions about the ethical impact of this transformative science on society. ARM CEO Tim Hunt will speak at the 2nd International Conference on the Ethics of Engineering Life on November 18th?to highlight how ARM has been grappling with some of the field’s most complex ethics issues, including: -?How we think about the pricing and value of cell and gene therapies - Enabling global access to cell and gene therapies through industrialization -?The challenges of the ultra-rare disease model with cell and gene therapies -?Scientific engagement and ensuring appropriate guardrails for frontier technologies Find us on the agenda here: https://lnkd.in/eAJTv-bv