Alliance for Regenerative Medicine

Another gene therapy approval for the year! This week, the European Commission approved Pfizer's gene therapy for hemophilia B, which was endorsed by the EMA CHMP earlier this summer. This marks the EU's second gene therapy option for patients with severe hemophilia B. The therapy was approved in Canada in December 2023 and in the United States in April 2024. https://lnkd.in/ew-NiGSR

Will we soon see in-vivo CAR-T therapies? The first patient is set to receive this promising approach in an Australian clinical trial for B-cell malignancies. Researchers believe this new therapeutic approach can help provide more treatment options for patients, with some potentially being faster, safer, and cheaper. https://lnkd.in/gjEZ4DWM #CellTherapy #InVivo

An amazing breakthrough in cell therapy took place this month! By the time she reached 21, Janina’s liver and kidneys were failing due to complications from lupus. With few other options, her family turned to CAR-T cell therapy. Within days, most of her severe symptoms receded. Oliver Barnes covers her story and CAR-T cell therapy's promise for treating autoimmune diseases in the Financial Times. https://lnkd.in/gxs7ssg4 #CellTherapy #Lupus

We've just announced the 120+ leading companies and organizations presenting at ARM's 2024 Cell & Gene Meeting on the Mesa! 🧬🌵 View the initial slate 👉 https://lnkd.in/g2VBgWht Collectively, this slate covers several unique products with an extensive range of possible therapeutic applications. In addition to the presenters, hundreds of different organizations on the cutting edge of cell and gene therapy will also be in attendance. Don't miss your chance to meet with senior executives from the sector's leading companies - register for the conference today! https://lnkd.in/eiUpMy4 #CGMesa24

Last call! ⏱ Join us tomorrow for a conversation on Strategic Manufacturing: Build vs Buy for Cell and Gene Therapies from 11:00am - 12:00pm ET. RSVP here: https://lnkd.in/emesZfHe The webinar discusses insights into the strategic considerations behind the “Build vs. Buy” dilemma in manufacturing. Thought leaders from Autolus Therapeutics and JLL will share their experiences determining the optimal manufacturing strategy for cell and gene therapies. Moderator: Joe Judge, Executive Managing Director, JLL Featured Speakers: Matt Jackson, Managing Director, JLL Christopher Vann, Chief Operating Officer, Autolus Therapeutics

What's new this week in the #cellandgenetherapy sector? 📑Newly published clinical data on gene therapy shows promise for muscular dystrophy patients 🧫Allogeneic T-cell therapy to treat EBV-PTLD has FDA BLA accepted; A decision date is set for Jan 15, 2025 🔬Injectable disc progenitor cell therapy for lumbar degenerative disc disease enters Phase III trials See the full stories below and subscribe to our Sector Vector newsletter for more weekly news on cell and gene therapy clinical, business, and manufacturing. 📩 https://lnkd.in/eD7ZNaU Researchers from UW Medicine published new data on a gene therapy for Duchenne Muscular Dystrophy. The therapy shows promise of not only arresting the decline of the muscles but also potentially repairing them. https://lnkd.in/ePWuVcnq This week, Atara Biotherapeutics announced that the FDA accepted a Biologics License Application for its allogeneic T-cell therapy to treat EBV+ PTLD. The therapy has a decision date of Jan 15, 2025. https://lnkd.in/eyE8B-vh DiscGenics announced it has gained FDA acceptance for the clinical protocols and CMC clinical development plan for the Phase III clinical program of its allogeneic, injectable disc progenitor cell therapy for lumbar degenerative disc disease. https://lnkd.in/ekm5kWvb

The 2025 U.S. pipeline for cell and gene therapies has begun. This week, Atara Biotherapeutics announced that the FDA accepted a Biologics License Application for its allogeneic T-cell therapy to treat Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD). The therapy has an assigned decision date of January 15, 2025. If approved, it would be the first therapy in the U.S. for EBV+ PTLD. https://lnkd.in/eyE8B-vh

Join us next week on Tuesday for our next webinar in ARM's Building the Future of Regenerative Medicine series! 🖥 Strategic Manufacturing: Build vs Buy for Cell and Gene Therapies 📆 July 23, 2024 ⏲ 11:00am - 12:00pm ET RSVP: https://lnkd.in/emesZfHe The webinar discusses insights into the strategic considerations behind the “Build vs. Buy” dilemma in manufacturing. Thought leaders from Autolus Therapeutics and JLL will share their experiences determining the optimal manufacturing strategy for cell and gene therapies. Moderator: Joe Judge, Executive Managing Director, JLL Featured Speaker: Christopher Vann, Chief Operating Officer, Autolus Therapeutics

Our 2024 Cell & Gene Congressional Fly-In came to a close yesterday, marking our most successful Fly-In yet! Throughout the course of Tuesday, we covered: ➡ 100+ offices ➡ 23 states Thank you to all of our advocates who came out this week to show support for patients who rely on the promise of cell and gene therapies. We especially thank our speakers and the 100+ offices from Congress and the administration for taking the time to hear our priorities. #CGFlyIn24

It’s a hot day on Capitol Hill, but that’s not stopping our members and advocates! Below are some highlights from today’s meetings with Congressional offices at our 2024 Cell & Gene Congressional Fly-In. #CGFlyIn24