FOR IMMEDIATE RELEASE
Contact:
Amy Adams
Senior Director of Communications
press@cirm.ca.gov
South San Francisco, CA, December 11, 2025 – The California Institute for Regenerative Medicine (CIRM) governing board today approved funding of over $160 million for 16 new research awards to develop therapies for a range of rare and common diseases; supported two new funding concepts to educate high school, undergraduate, and graduate students for careers in regenerative medicine; and supported a new strategy for how CIRM will put regenerative medicine therapies within reach of Californians.
“CIRM recognizes that making a medical breakthrough is only the beginning of the complex process of delivering new regenerative medicine therapies to the people of California,” said Rosa Canet-Avilés, PhD, Chief Science Officer. “That’s why CIRM funds regenerative medicine research and clinical trials, while simultaneously strengthening and expanding the educational opportunities for future members of the regenerative medicine workforce, and addressing barriers that might prevent patients in California from benefiting from cell and gene therapies.”
The governing board, formally the Independent Citizens Oversight Committee, sets the agency’s priorities and allocates funding for CIRM’s research, education, and infrastructure programs, including approving new awards and new funding concepts. It is made up of 35 California-based research and biotechnology industry leaders, patient advocates, nurses, and university and institution executives.
Developing cures for Californians
The awards approved today support four clinical trials as part of the CIRM’s clinical program and 12 awards that move discoveries to clinical trials as part of the preclinical development program. These preclinical and clinical research projects target common diseases such as urinary incontinence, heart disease, and chronic pain; and rare diseases such as leukoencephalopathy and others.
When California Proposition 14 passed in 2020 and allocated $5.5 billion for cell and gene therapy development, $1.5 billion of that was intended for diseases of the central nervous system. With that mandate, CIRM has prioritized applications for those diseases. Each of the approved clinical trials in this cycle is for a neurological condition.
Approved clinical projects include those testing approaches for treating Parkinson’s disease and Huntington’s disease, which both involve injecting stem cell-derived neural cells into the brain of patients to replace damaged cells. Programs targeting the rare genetic diseases Dravet Syndrome and CMTJ4 (Charcot-Marie-Tooth disease type 4J) are both testing genetic therapies intended to fix the mutated genes that underlie those conditions.
The 12 preclinical projects that were approved span a wide range of diseases and therapeutic approaches. The preclinical awards include several neurological diseases as well as diseases of the heart, cartilage, lung and muscle. The therapeutic approaches range from off-the-shelf cell therapies to injected CRISPR genetic therapies. CRISPR technology, which earned its discoverers a Nobel Prize in 2020, directly edits mutated genes.
Most genetic therapy approaches deliver the therapy to cells using a modified virus. Two CRISPR approaches funded today deliver the therapy to cells in the body without using a virus, which is a first for CIRM’s active portfolio and could overcome common issues with using viruses for that purpose.
Stem cell-based approaches are similarly diverse in this round of funding and include stem cells taken from the patient’s own tissues and modified in the lab, or involving off-the-shelf approaches where cells are modified in a lab, distributed to clinical trial sites, and injected into the patient.
The successful applicants in CIRM’s Preclinical Development program are:
| Application # | Program Title | Principal Investigator/Institution | Amount |
| PDEV-19137 | Development of an AAV Epigenetic Gene Therapy for Intractable Chronic Pain Disorders | Ana Moreno – Navega Therapeutics | $9,486,864 |
| PDEV-19164 | A First-in-Class CRISPR-CasX Gene Editing Therapy for Lowering Lp(a) to Prevent Cardiovascular Events | Maria Mirotsou – Scribe Therapeutics | $12,714,480 |
| PDEV-19141 | TY1 and Semaglutide to Treat Cardiometabolic HFpEF | David Lefer – Cedars-Sinai | $10,571,220 |
| PDEV-19150 | A first-in-class CRISPR-CasX gene editor silencing APOC3 transcription for the treatment of Severe Hypertriglyceridemia | Brett Staahl – Scribe Therapeutics | $13,000,000 |
| PDEV-19140 | CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia | Stephanie Cherqui – UCSD | $7,423,504 |
| PDEV-19154 | Late-stage development of AS-241, an UNC13A Targeting Antisense Oligonucleotide treatment for Amyotrophic Lateral Sclerosis, for IND-enabling studies | Samuel Alworth – AcuraStem Incorporated | $7,500,000 |
| PDEV-19133 | Stem Cell-Based Cartilage Tissue Regeneration | Darryl D’Lima – Scripps Health | $12,715,000 |
| PDEV-19138 | Noncoding RNA drug for arrhythmogenic cardiomyopathy | Alessandra Ciullo – Cedars-Sinai | $10,419,929 |
| PDEV-19156 | Gene Therapy for Alpha-1 Anti-Trypsin Deficiency | Melissa Calton – 4D Molecular Therapeutics, Inc. | $5,916,702 |
| PDEV-19152 | Advancement of a myotropic, liver-detargeting therapy for LGMD2i/R9 | Tahseen Mozaffar – Cure Rare Disease | $7,350,596 |
| PDEV-19149 | Microglia replacement therapy for CSF1R-related Leukoencephalopathy | Sunil Gandhi – Savanna Bio | $12,993,456 |
| PDEV-19131 | Autologous iPSC-derived progenitor smooth muscle cells for treatment of urinary incontinence | Bertha Chen – Stanford | $7,499,999 |
The successful applicants in CIRM’s Clinical program are:
| Application # | Program Title | Principal Investigator/Institution | Amount |
| CLIN2-19068
|
A Phase 1 trial of RNDP-001, a dopaminergic progenitor drug product, in idiopathic Parkinson’s disease | Howard Federoff – Kenai Therapeutics | $8,000,000 |
| CLIN2-18731
|
Clinical Development of ETX101, a Gene Regulation Therapy for SCN1A+ Dravet Syndrome | Salvador Rico – Encoded Therapeutics | $15,000,000 |
| CLIN2-18595
|
Phase 1B/2A study of the safety and tolerability of human neural stem cells for Huntington’s Disease (REGEN4HD) | Leslie Thompson – UC Irvine | $11,999,933 |
| CLIN2-19061 | Phase I/II Clinical Trial for CMT4J | Keith Gottlieb – Elpida Therapeutics | $8,000,000 |
Building the regenerative medicine workforce
Creating a skilled workforce that can tackle the scientific challenges of today and deliver the innovative solutions of tomorrow is an essential part of CIRM’s mission to accelerate world class science and deliver transformative regenerative medicine treatments.
Today, the governing board approved concepts for two educational programs—EDUC8, which combines the Bridges Award and COMPASS Program, and the EDUC3 SPARK Program, which provides immersive summer research internships for high school students. These concepts extend existing programs with some updates to give trainees additional opportunities.
The Bridges awards help undergraduate and master’s-level students from state universities and community college access research training opportunities in academic and biotechnology industry labs. The COMPASS Program provides academic and mentoring support to early-stage undergraduate students, with an emphasis on identifying untapped talent and fostering new perspectives within the scientific workforce.
The new EDUC8 program includes both Bridges and COMPASS, as well as an option for an applicant to have a combined program. The umbrella program reduces the administrative burden for those programs with both tracks. EDUC8 also includes expanded training opportunities that came out of surveys and conversations with industry members to learn about their staffing needs.
“Training students and early career scientists so they have the skills and resources they need to work in the fields of stem cell research is a critical part of developing and delivering regenerative medicine therapies,” said Kelly Shepard, PhD, Director of Discovery & Education programs at CIRM.
To date, CIRM’s education and workforce programs have trained more than 4,600 students, interns, and fellows in the fields of regenerative medicine. Students from these programs come from many regions around California and bring unique knowledge and perspectives that inform and enhance their contributions to the field.
Putting therapies within reach
CIRM recognizes that bringing new regenerative medicine therapies to the people of California is a multistep process. Discovering and developing transformative cell and gene therapies is one of those steps, but additional work is needed to make those new therapies available to the people who can benefit from them.
Many patients, especially those in underserved and geographically harder to reach communities, face barriers to access to medical treatments. To address this issue, CIRM’s Access & Affordability (A&A) Strategy was developed to ensure that Californians can access CIRM-funded treatments equitably.
The strategy, which the board approved today, provides a framework for how CIRM will deliver cell and gene therapies to Californians by integrating clinical infrastructure programs, payer engagement, affordability planning, and partnerships into the full lifecycle of programs.
The strategy includes objectives for embedding accessibility and affordability in clinical infrastructure such as Alpha Clinics and Clinical Care Centers of Excellence – that bring clinical trials closer to people who need them. It also calls for engagement with public and private payers to develop policy solutions for patient access. Finally, it embeds early planning requirements into R&D programs with the goal of helping awardees develop viable strategies for reducing patient out-of-pocket costs and supporting long-term therapy access.
The board also heard an update on CIRM’s Patient Support Program, that helps reduce financial barriers to participating in clinical trials, starting with a $15.6 million Patient Assistance Fund. This program offers financial support that helps patients and their families cover the cost of meals, travel, lodging, and transportation associated with clinical visits. The program is currently expanding from an initial three trial sites to include all Alpha Clinic locations and CIRM’s clinical programs.
“All of the effort, research, and resources devoted to developing new regenerative medicine therapies will not be successful if only a select few people can access and afford them,” said Maria Gonzalez Bonneville, Chair of the Access & Affordability Working Group and Board Vice Chair. “A patient’s home address and income should not determine their treatment options. CIRM is committed to expanding equitable access to clinical trials, prioritizing underserved communities statewide.”
About the California Institute for Regenerative Medicine (CIRM)
CIRM was created by the people of California to fund stem cell and gene therapy research with the goal of accelerating treatments for patients with unmet medical needs. With $5.5 billion in funding allocated through both Proposition 71 in 2004 and Proposition 14 in 2020, CIRM supports stem cell and gene therapy discoveries from inception through clinical trials, trains a workforce in California to fill jobs in the state’s thriving biotech and biomedical research industry, and creates infrastructure to make clinical trials accessible for people throughout California. All of CIRM’s research, workforce development, and infrastructure programs are designed to benefit the people of California, whose vision created the agency. For more information, visit www.cirm.ca.gov.
