The International Society for Stem Cell Research (ISSCR) today opened its sold-out international symposium, Accelerating PSC-Derived Cell Therapies: Starting with the End in Mind, taking place 11-12 December at the Hyatt Regency Boston Cambridge. The meeting convenes a capacity audience of 360 global experts, alongside an at-capacity roster of biotech and pharma sponsors and exhibitors – underscoring the strength, interest and continued momentum in cell therapy development despite recent industry contraction.
As some major pharmaceutical companies scale back internal cell therapy programs, the ISSCR symposium is sending a different message – cell therapy research is showing potential and is accelerating. With new clinical trial results and emerging technologies, the symposium showcases a pipeline advancing toward real therapeutic impact for diseases including Parkinson’s disease, type 1 diabetes, epilepsy, and macular degeneration.
Adding to the excitement, the symposium closes with a keynote from George Church, renowned geneticist, professor at Harvard and MIT, and pioneering force behind modern genomics and synthetic biology.
Across two days, attendees will hear many compelling new clinical and preclinical updates on a variety of topics including:
Restoring Neural Circuitry in Drug-Resistant Epilepsy
Emerging Phase 1/2 clinical data for NRTX-1001, a human PSC-derived inhibitory interneuron therapy for mesial temporal lobe epilepsy will be presented by Yves Maury with Neurona Therapeutics. This breakthrough approach could mark a major turning point in how we treat chronic focal epilepsy and could potentially be applied to other neurological disorders involving focal hyperactive neuronal networks.
Advancing a Next-Generation Retinal Implant for Geographic Atrophy
Jane Lebkowski with Regenerative Patch Technologies, LLC will present long-term results from a Phase 1/2a study and new details from its recently launched Phase 2b PATCH AMD trial evaluating a cryopreserved, hESC-derived RPE implant.
Toward Durable Remission in Type 1 Diabetes
Felicia Pagliuca with Vertex Pharmaceuticals will share progress on its stem cell–derived islet cell therapy platform, designed to reliably replace lost pancreatic beta-cell function through scalable, standardized manufacturing processes.
AI-Designed Cell Fates and Plug-and-Play Manufacturing
Alex Ng with GCTx will introduce an AI-guided forward programming platform powered by the TFome™ library of >1,700 transcription factors. The technology produces high-purity cell types in days – not months – and enables a one-step, reproducible manufacturing strategy for PSC-derived therapeutics.
Three-Year Clinical Results for Parkinson’s Disease Therapy
Joohi Jimenez-Shahed with Icahn School of Medicine at Mount Sinai will present the most recent data from a Phase 1 study of bemdaneprocel, an allogeneic, hESC-derived dopaminergic progenitor therapy for Parkinson’s disease. Dr. Jimenez-Shahed is a paid consultant for BlueRock Therapeutics, LP.
A patient perspectives panel discussion brings together leaders from patient-focused organizations to explore the evolving landscape of stem-cell-based therapies and their path toward clinical impact. Moderated by an expert in the field, the session features Maria L. De Leon, the Michael J. Fox Foundation, USA and Martha Steel, Foundation Fighting Blindness, USA, and Carrie Khouri, Breakthrough T1D, USA, who will share perspectives spanning Parkinson’s disease, blinding retinal disorders, and type 1 diabetes. Together, they will highlight patient needs, translational challenges, and opportunities for cross-disease collaboration to advance responsible, effective cell therapies.
Co-chaired by Claire Henchcliffe, University of California, Irvine, USA and Erin Kimbrel, Astellas Pharma Inc., USA, with organizing committee members from BlueRock Therapeutics, USA, Vertex Pharmaceuticals, USA, reNEW/LUMC, Netherlands, and Cellerator, Denmark, the symposium challenges the traditional “bench-to-bedside” mindset. Instead, it begins with the patient and product requirements, working backward to design development strategies that ensure safety, durability, manufacturability, and global scalability.
The symposium reached full capacity last week, with both attendee registration and exhibition space fully sold out and accompanied by waitlists. The response reflects intense international interest in PSC-derived therapies at a moment when clinical progress is accelerating across multiple disease areas.
About the ISSCR International Symposium
The ISSCR International Symposium, Accelerating PSC-Derived Cell Therapies: Starting with the End in Mind, brings together leading scientists, clinicians, regulators, and innovators advancing ESC- and iPSC-derived therapies. The program emphasizes cross-disciplinary collaboration and real-world clinical and manufacturing insights that are rapidly redefining what is possible in regenerative medicine.
About ISSCR
With nearly 5,000 members from more than 80 countries, the International Society for Stem Cell Research (@ISSCR) is the preeminent global, cross-disciplinary, science-based organization dedicated to stem cell research and its translation to the clinic. The ISSCR mission is to promote excellence in stem cell science and applications to human health.