Alliance for Regenerative Medicine

This year, CAR-T therapies became second-line treatments for some blood-cancer indications. Soon, the approach could become a first-line treatment. In October, Kite Pharma announced that its CAR-T therapy to treat high-risk large B-cell lymphoma received an RMAT Designation from the FDA as a first-line treatment. https://lnkd.in/e2JXwAah #CellTherapy

Announcing the next Scientific Exchange! On November 20th, ARM, ISCT, International Society for Cell & Gene Therapy, and Danaher Corporation will convene key stakeholders to explore how to advance gene editing platforms. This Scientific Exchange with the FDA seeks to make progress on therapeutic development for rare conditions that are scientifically feasible but commercially non-viable. Successful platforms hold promise to deliver faster and broader patient access to novel gene therapies while maintaining a favorable risk-benefit balance and appropriate regulatory rigor. We will review case studies from academics and industry developers focused on gene editing, with the objective of defining repeatable platforms that streamline development and regulatory requirements across multiple therapies for rare populations. The meeting will be live streamed for public viewing. Registration and meeting materials can be accessed here: https://lnkd.in/eMp5gZwZ

Exciting News from New York! In October, the state officially launched the next phase of Long Island’s cell and gene therapy initiative, bolstering innovation and expanding research infrastructure across the region. This investment will help accelerate groundbreaking discoveries and revolutionize patient care in cell and gene therapy. https://lnkd.in/g6fgeeQU #cellandgenetherapy

ARM CEO Tim Hunt will deliver a state-of-the-industry briefing on the gene therapy space at the AAV Innovation Summit on November 6th in Cambridge, MA. This exclusive half-day summit brings together top industry and academic experts in cell and gene therapy. Check out the agenda: https://lnkd.in/eRwvZhg7 #Cellandgenetherapy #AAV

Have you heard the news? 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 deadline to apply 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

The fall season brings new milestones and more big news for the #cellandgenetherapy sector. 🧬New partnership to advance next-generation gene therapies for neurological diseases 🤝Over $200 million in new financing deals to support cell and gene therapy clinical programs 💉First patient dosed in trial for in vivo CAR gene therapy to treat B-cell malignancies 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 Dyno Therapeutics is forming a new strategic partnership with Roche to advance AAV gene therapy vectors for neurological diseases. Dyno is to provide further access to its platform and sequence design technologies, enabling in vivo gene delivery. https://lnkd.in/etXMUBhs Interius BioTherapeutics announced that the first study participant has been dosed in its first-in-human Phase 1 clinical trial of a CAR gene therapy for the treatment of B-cell malignancies. https://lnkd.in/duJA-fja AvenCell announced it had raised $112 million in Series B financing this week. The funding will be used to support clinical validation of its switchable universal CAR-T cell therapy platform. https://lnkd.in/eivannxn Be Biopharma announced this week that it secured $82 million in funding for its leading candidate, an engineered B-cell therapy, as it transitions to a clinical-stage company. https://lnkd.in/ez6DRUxH March Biosciences announced this week the close of a $28.4 million oversubscribed Series A financing to support its CAR-T cell therapy trial for T-cell lymphoma. https://lnkd.in/gySMtkWc

How can we create a sustainable and robust pipeline for rare diseases and gene therapies in Europe? This morning at the last day of the 2024 World Orphan Drug Congress Europe, Paolo Morgese, ARM's Vice President for Public Affairs Europe, moderated the closing keynote panel featuring leading experts in market access, regulatory affairs, and clinical research. The lineup included: Paolo Morgese, Vice President, Public Affairs, Europe, Alliance for Regenerative Medicine (Moderator) Victor Maertens, Government Affairs Manager, EUCOPE - European Confederation of Pharmaceutical Entrepreneurs Francis Pang, SVP Global Market Access and International Geographic Expansion, Orchard Therapies Ltd Thomas Butt, Executive Director, Health Economics & Outcomes Research, BioMarin Pharmaceutical Inc. Daria Julkowska, Scientific Coordination, European RD Research Alliance, International Rare Diseases Research Consortium (IRDiRC) Our thanks to the organizing team for a great conference and welcoming ARM's perspectives on topics shaping the future of cell and gene therapies for rare diseases. #cellandgenetherapy #rarediseases #WODC2024

Kendrick Cromer has just made history as the first commercial patient to complete gene therapy for sickle cell disease. While his journey came with challenges, this milestone marks a significant step forward for gene therapy and those living with sickle cell disease. #GeneTherapy https://lnkd.in/d3UmKfaA

Earlier today, Elisabetta Zanon, ARM's Director of European Policy and Advocacy, took part in the opening keynote panel of the 2024 World Orphan Drug Congress in Barcelona to discuss how Europe can remain competitive in the cell and gene therapy and rare disease sector. She was joined by a lineup of leaders in government, civil society, and industry. Stelios Kympouropoulos, MEP, European Parliament Fabienne Bartoli, Inspector General, French National Authority for Health Virginie Bros-Facer, CEO, EURORDIS-Rare Diseases Europe Toon Digneffe, Head, Public Affairs & Public Policy, Europe & Canada, Takeda Our thanks to the organizing team at the World Orphan Drug Congress Europe for a great opening to the conference. Be sure to catch ARM's Paolo Morgese for the morning keynote panel on October 25. #WorldOrphanDrugCongress #WODC2024 #CellandGeneTherapy

We're announcing the release of ARM's latest whitepaper on advanced manufacturing strategies to increase the reach of cell and gene therapies.   As the field progresses and the number of product approvals continues to grow, there is an increasing demand to incorporate higher-volume production to deliver potentially life-saving therapies to expanding patient populations.   ARM's May 2024 workshop brought together therapeutic developers, technology developers, regulatory representatives, and other key stakeholders from across the industry to focus on three primary areas of opportunity: ⚙️ Automation & advanced processes/analytics 💡 Artificial intelligence/machine learning (AI/ML) 🏭 Decentralized manufacturing models A preview of the whitepaper can be found here: https://lnkd.in/ec8rgh6H The paper is free for ARM members on our community portal. Non-members can separately purchase full access to the whitepaper.