CIRM approves over $111 million to support discovery and clinical research

FOR IMMEDIATE RELEASE

Contact:
Amy Adams
Senior Director of Communications
[email protected]

South San Francisco, CA, March 27, 2026 - The California Institute for Regenerative Medicine (CIRM) governing board today approved funding of over $80M for six new discovery grants applications and $31M for three clinical trial applications to develop and test potential therapies for a range of rare and common diseases. In addition, the board celebrated the first FDA approved therapy funded by the agency. The therapy for leucocyte adhesion deficiency-I, developed by Rocket Pharmaceuticals, was approved by the FDA during the meeting.

Funding discovery research and clinical trials

The awards approved by the board support three clinical trial (CLIN2) grant applications with a combined approved total of $31M in financial support from CIRM. These grants fund clinical trials for regenerative medicine therapies that address significant unmet medical needs and have the potential to provide transformative benefits to patients, their families, and the healthcare system.

The three approved clinical trial applications include two studies that address rare diseases (mucopolysaccharidosis I and Pitt-Hopkins syndrome) that disproportionately affect children. The third approved clinical trial addresses forms of vision loss.

• Read the summaries
• View the PowerPoint presentation

The successful CLIN2 applicants are:

The awards approved also include six Discovery (DISC4) grant applications with a combined approved total of $80M in financial support from CIRM. These DISC4 grants support studies led by collaborative teams that use a range of technologies and approaches to accelerate foundational insights in diseases of the central nervous system.

The approved discovery projects address both rare and common diseases. These studies include foundational research on the development and regulation of glioblastoma, the most common and aggressive primary brain tumor in adults, and explore the possible genetic and molecular causes of Parkinson's, Alzheimer's, amyotrophic lateral sclerosis, epilepsy, autism, and schizophrenia.

• Read the memo
• Read the summaries
• View the PowerPoint presentation

"Cell and gene therapies have tremendous potential for treating disease, but researchers must answer many scientific unknowns before this potential can be fully realized," said Rosa Canet-Avilés, PhD, Chief Science Officer. "CIRM helps bridge the chasm between a promising scientific discovery and a powerful therapy for treating disease. That's why CIRM's funding and support of the full process from discovery research to clinical trials to delivering the therapy to patients is so vital.

The successful DISC4 applicants are:

Revised review process

The board approved a new application selection process, presented by the CIRM Team for the CLIN2, PDEV, and DISC4 funding programs, that uses guiding principles across program design and review to identify applications that best support CIRM's goals. These principles come from the specific goals set for each funding program and are built into program objectives, the Grants Working Group review criteria, and CIRM Team recommendations.

• Read the memo
• View the PowerPoint presentation
• Read the executive summary

As part of the discussion about funding programs, the board voted to hold one preclinical development (PDEV) funding round per year rather than two. In addition, the board voted that for the next fiscal year the DISC4 funding round will focus on Immune-Tissue Interactions in Disease and Repair.

• Read the PDEV memo
• Read the DISC4 memo

Closer to Cures

In the Closer to Cures speaker series, which features presentations from two CIRM-funded grantees at each ICOC meeting, the board heard two updates. The first was from Diana Farmer, surgeon-in-chief at the children's hospital at the University of California, Davis, and chief of pediatric surgery at Shriners Children's Northern California. The second was from James DeKloe, Distinguished Professor of Biotechnology and Biomanufacturing, who leads CIRM's COMPASS program at Solano College. He was joined by two former students from the program, Jennifer Mayo and Joel Saenz.

Farmer discussed the Cellular Therapy for In Utero Repair of Myelomeningocele (CuRe) trial, which is testing a therapy for spina bifida, a birth defect that affects the spinal cord and is linked to changes in brain structure, leg weakness, and bladder and bowel dysfunction. CIRM has funded several stages of Farmer's research, including preclinical research and phase 1 and phase 2 clinical trials.

Farmer discussed results from a phase 1/2 trial, in which she operated on fetuses diagnosed with spina bifida to repair the damaged spine and place a patch of stem cells on the injury. Based on the strength of early safety results, the Food and Drug Administration approved moving forward with the next phase of the study, which is ongoing.

During the presentation from Solano Community College, former students from the Creating Opportunities through Mentorship and Partnership Across Stem Cell Science (COMPASS) program shared how their experience led to current jobs working at the University of California, Davis GMP facility.

Additional action items

The board also approved reopening the Community Cares Centers of Excellence (CCCEs) applications for a Northern California site. The CCCEs put clinical trials closer to where people in California live, reducing the need to travel to clinical trial sites. They also heard amendments to the funding mechanism for conferences and meetings (EDUC1).

• Read the memo for the Community Cares Centers of Excellence Concept Plan
• Read the memo for the Amendments to the Funding Mechanism for CIRM Conferences and Meetings (EDUC1)

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 $8.5 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 https://www.cirm.ca.gov.