Alliance for Regenerative Medicine

Could some blood cancer patients one day skip chemotherapy before receiving CAR-T cell therapy? Early trial results published in Cell suggest we’re heading towards that reality. A Phase 1 trial led by researchers at the LIT - Leibniz Institute for Immunotherapy in Germany tested a modified CAR-T approach using T memory stem cells, a specific cell type that can renew itself, persist for years, and differentiate into many other T cell subsets. The results showed that patients who received T-cell therapy without chemotherapy achieved higher complete response rates than those who received standard CAR-T. The science of cell and gene therapy continues to advance, with the potential to transform treatment. Read the full story: https://lnkd.in/ecJkyRD2 #CARTTherapy #CellTherapy

As we approach the middle of 2026, what does the cell and gene therapy clinical pipeline look like in the US and EU? The US currently has two therapeutic products approved, while the EU has one. Notably, the US has 10+ possible regulatory submissions this year, showing the strength of the near-term pipeline. In terms of possible milestones, we could see the first-ever approvals of a CAR-T cell therapy to treat an autoimmune disease and an in-vivo gene editing therapy in 2027. You can dive into the full therapeutic pipeline in ARM’s Q1 2026 Sector Snapshot here: https://lnkd.in/eFqV4UDv #cellandgenetherapy

Last week, the UK Medicines and Healthcare products Regulatory Agency (MHRA) launched a public consultation on a proposed new Rare Disease Therapies Framework in partnership with the Rare Disease Consortium. More than 3.5 million people in the UK are living with a rare disease. Yet fewer than 5% of these conditions currently have an approved treatment. This initiative aims to create a more flexible, patient-centered regulatory system that reflects the realities of rare disease development while maintaining the highest safety standards. ARM commends the MHRA for its collaborative approach to enabling support for innovative therapies and will coordinate a joint response to the MHRA on behalf of the cell and gene therapy community. The consultation is open until July 30. Learn more and see the full framework here: https://lnkd.in/eARQe6hh #raredisease #cellandgenetherapy #ATMP

"In some patients, they can even hear a whisper." In April, Regeneron announced that its gene therapy to treat rare forms of inherited deafness had been approved by the FDA. But how does it work? As noted on All Things Considered, NPR’s Rob Stein breaks down how the scientific approach treats patients, sharing the story of Travis, a young baby born deaf, who heard his mother’s voice for the first time. https://lnkd.in/exRiXNz8 #GeneTherapy

Join us on June 19, 2026, in Paris, France, for a one-day workshop that will bring together 100+ senior executives, CMC leaders, regulatory authorities, and market access experts to define industrial readiness for platform genetic medicines. The workshop will be co-hosted with our event partner, GenoTher. Register here ➡️ https://lnkd.in/eJkvBUdu Sessions will address control strategies and comparability; advanced analytics and genome integrity; scalable manufacturing across modalities; EMA perspectives on platform frameworks; the EU Biotech Act and speed to market; and how technical maturity drives capital formation. #cellandgenetherapy #workshop

"The Heart-2 results provide early clinical evidence that a single dose of VERVE-102 may mimic the LDL-C lowering effects of PCSK9 cardioprotective variants, potentially transforming cardiovascular care from chronic management to a one-time treatment." Clinical data released this week by ARM member Eli Lilly and Company show that its gene therapy, which uses a base editor, lowered LDL-C by 62% in patients enrolled in its Phase 1b Heart-2 clinical trial. The data demonstrate promising potential for a one-time treatment to durably lower high cholesterol and for gene editing to treat prevalent conditions like cardiovascular disease. Read the full release: https://lnkd.in/e4B3EAqe

📣 Last Call: Applications for ARM Advisory Groups will close tomorrow, Wednesday, May 27. Advisory Groups bring together select members to help shape ARM’s policy, advocacy, and strategic initiatives across the regenerative medicine sector. We also encourage members to consider our new EU Government Relations Advisory Group, focused on EU policy and regulatory engagement. To learn more and apply, visit our website here: https://lnkd.in/edD59WU3 Please note: Applications are open only to ARM members. ARM member companies may have only one representative per AG, and individuals may participate in only one group at a time. #cellandgenetherapy

CAR-T cell therapy is revolutionizing the treatment of blood cancers. Just ask Jurassic Park actor Sam Neill. In 2023, he was diagnosed with T-cell lymphoma and underwent intensive chemotherapy, which failed to put the cancer into remission. He shared last month that his cancer had miraculously gone into remission after participating in a CAR-T cell therapy clinical trial in Sydney. The Guardian details Sam’s treatment journey, showing how CAR-T works and why access is becoming a more crucial question for patients with few treatment options. https://lnkd.in/gTy6SiSP #celltherapy

Will the first-ever CAR-T cell therapy soon be approved for an autoimmune disease? Earlier this month, ARM member Kyverna Therapeutics announced that it has initiated a rolling Biologics License Application with the FDA for its CAR-T cell therapy to treat Stiff Person Syndrome. Pending requests for natural history data from the FDA, the application could be fully submitted by the end of the year, setting up a potential approval in 2027. Read more about the story on Fierce Biotech: https://lnkd.in/eafabJYS #celltherapy

At #CGMed26, ARM CEO Tim Hunt reviewed how, in Q1, ARM observed a concerning trend of ‘methodological purity’ at the FDA that resulted in a number of Complete Response Letters and reversals for promising CGT products. That current trend, among other regulatory concerns, has been top of mind for developers, and a recent article by Andrew Joseph in STAT highlights the sector’s discourse on the FDA's recent shifts and broader regulatory trends in cell and gene therapy at ARM's 2026 Cell & Gene Meeting on the Med. https://lnkd.in/excxv3jv In response, ARM and our community have shined a spotlight on these concerns, and have been pushing for a course-correction at the FDA that includes: 1. Honoring Previous Alignments 2. Transparency & FDA Advisory Committees 3. Philosophy of Regulatory Flexibility 4. Bolstering Scientific Leadership