Disease Modeling
and Drug Discovery
ISSCR improves how stem cell science is conducted and applied in drug discovery and development. By aligning academia, industry, and regulators, we support validation, benchmarking, and adoption of more predictive, human-relevant models. This work is driven largely by the Consortium on Advanced Stem Cell-Based Models in Drug Discovery and Development.
KEY AREAS
Validation & Benchmarking
Establishing shared standards for evaluating stem cell–based models.Cross-Sector Collaboration
Bringing together academia, industry, and regulators to align approaches.Translational Case Studies
Demonstrating the predictive value of advanced models in real-world contexts.Data & Knowledge Integration
Building shared resources and tools to support adoption and decision-making.
Consortium on Advanced Stem Cell-Based Models in Drug Discovery and Development
A global organization of industry and academic scientists working with regulatory experts to establish standards, develop tools, and build the collaborative infrastructure needed to accelerate responsible integration of stem cell-derived models into preclinical stages of drug development. This effort aligns with recent policy and regulatory shifts toward new approach methodologies (NAMs) and greater emphasis on human-based research. Immediate objectives:
Define regulatory-ready standards and validation frameworks for stem cell–based models, including performance benchmarks, reporting criteria, and fit-for-purpose guidance aligned with real-world regulatory pathways.
Generate qualification-grade evidence at scale by aggregating and benchmarking multi-site data against clinical outcomes, establishing clear contexts of use and acceptance criteria for decision-making.
Build an open, interoperable resource that integrates data, standards, and AI-enabled tools to support regulatory decisions and accelerate adoption across research, industry, and clinical development.
Supporting Members
This work is made possible by Founding Members:
Impact and Recent Work
Global Collaboration
-
Shuibing Chen, PhD, Weill Cornell, USA (Co-chair)
Daniela Cornacchia, PhD, AstraZeneca, Sweden (Co-chair)
Luigi Aloia, PhD, AstraZeneca, UK
Erica Bello, PhD, Milner Institute, University of Cambridge, UK
Kapil Bharti, PhD, NEI/NIH, USA
Hans Clevers, MD, PhD, Hubrecht Institute, Netherlands
David Gnutt, PhD, Bayer AG, Germany
Magdalena Kasendra, PhD, Cincinnati Children’s, USA
Henning Kempf, PhD, GEM CELL hub at Novo Nordisk
Christine Mummery, PhD, LUMC, Netherlands
Steve Murry, PhD, Jackson Labs, USA
Hideyuki Okano, MD, PhD, Keio University, Japan
Li Pang, MD, FDA, USA
Lee Rubin, PhD, Harvard University, USA
Charis Segeritz-Walko, PhD, Novo Nordisk, Denmark
Clive Svendsen, PhD, Cedars-Sinai, USA
Carlos Tristan, PhD, NCATS/NIH, USA
Joseph Wu, MD, PhD, Stanford University, USA
Matthias Zilbauer, MD, PhD, University of Cambridge, UK
-
TBD
-
What began as a passion project has evolved into something more. We’re proud of where we’ve been and even more excited for what’s ahead. What sets us apart isn’t just our process—it’s the intention behind it. We take time to understand, explore, and create with purpose at every turn.
-
Item description
-
Item description