disease modeling
and drug discovery

The 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 resources that integrate data, standards, and AI-enabled tools to support regulatory decisions and accelerate adoption across research, industry, and clinical development.

Join + Support

This work is made possible by Founding Members:

Supporting Members

Xellar Biosystems

news

ISSCR News

ISSCR Submits Comments Opposing Proposed U.S. Regulations that would Harm Merit-Based Science and International Collaborations

The ISSCR submitted comments on proposed regulations from the U.S. Office of Management and Budget (OMB) that would reshape how the U.S. federal government funds scientific research. Among other changes, the proposed regulations would de-emphasize merit-based review of scientific grant applications and instead establish a political review process, enable mid-stream grant cancellations without cause, and place restrictions or significant bureaucratic burdens on international research collaborations. As outlined in the comment letter, these proposed changes would have harmful impacts to the progress of stem cell research and its translation to the clinic. Given this, the ISSCR respectfully urged OMB to withdraw the proposed regulations.

Take Action to Preserve Merit-Based Science and International Collaborations

The White House Office of Management and Budget (OMB) has proposed a new rule titled “Regulation for Federal Financial Assistance” that will significantly reshape how the U.S. government funds and manages scientific research, including international collaborations, through the National Institutes of Health (NIH) and other agencies. All ISSCR members, regardless of whether you are U.S.-based or not, are eligible to provide comment on the proposed policy.

Though the rule is extensive and applies to all federal grantmaking, there are several areas where input from our community is critical.

The ISSCR Joins Coalition Letter in Support of the National Institutes of Health (NIH)

The ISSCR joined more than 110 organizations in the cell and gene therapy sector to urge congressional appropriators to provide at least $51.303 billion for the National Institutes of Health (NIH) for fiscal year 2027. This funding level would represent a robust investment in important biomedical research and a meaningful increase over enacted fiscal year 2026 levels. NIH funding remains foundational to the continued advancement of stem cell research. Strong support for NIH will ensure the continued translation of stem cell research into the clinic and to the lives of patients. In addition to the funding request, the letter urges the timely distribution of NIH funding to ensure biomedical research can continue without disruption.

The ISSCR Releases Summary of Meeting With UK Regulators on the Use of AI to Develop Stem Cell Therapies

The International Society for Stem Cell Research (ISSCR) held its second Broader Scope Scientific Advice meeting with the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) on October 29, 2025, focusing on the use of artificial intelligence (AI) in developing stem cell therapies.

The session explored key topics including the current state and challenges of AI, machine learning, and digitalization systems for automation in cell and gene therapy; applications of AI in autologous iPSC-derived RPE cell therapy; and AI-based image analysis for in-process characterization and decision-making during iPSC generation.

The ISSCR Urges Continued NIH Investment in Human Embryonic Stem Cell Research to Sustain Scientific Progress and Therapeutic Innovation

The International Society for Stem Cell Research (ISSCR) has submitted a formal response to a Request for Information (RFI) issued by the National Institutes of Health (NIH) regarding its pause on new submissions to the NIH Human Embryonic Stem Cell (hESC) Registry and considerations to reduce reliance on hESCs in federally supported research.

‍Representing nearly 5,000 scientists, clinicians, ethicists, and industry leaders worldwide, the ISSCR underscores that continued investment in hESC research is essential to sustaining scientific progress and advancing life-saving therapies.

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