California Institute for Regenerative Medicine (CIRM) Clinical Trials Program

California Institute for Regenerative Medicine (CIRM) Clinical Trials Program is sponsored by California Institute for Regenerative Medicine (CIRM). Provides funding for stem cell and gene therapy-based projects through all stages of clinical trial development.

CIRM – California's Stem Cell and Gene Therapy Agency CIRM-funded therapy receives FDA approval The first approved therapy to come out of California taxpayer funding addresses the rare immune disorder LAD-I. New Application Selection Process CIRM’s Board approved a new application selection process that uses program-specific guiding principles across the application lifecycle.

Photo Credit: Barbara Ries of UCSF Advancing California's scientific vision through stories of impact and breakthrough science. CIRM is dedicated to accelerating world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world.

Photo Credit: UCLA Broad Stem Cell Research Center CIRM-funded therapy receives FDA approval The first approved therapy to come out of California taxpayer funding addresses the rare immune disorder LAD-I. UCLA gene therapy offers children with LAD-I new hope Diagnosed with LAD-I at age eight after years of hospitalizations, Marley Gaskins’ immune system is restored after participating in a CIRM-funded clinical trial.

CIRM-funded clinical trial gives siblings a second chance at life The Langenhop children, all diagnosed with LAD-I, now have healthy immune systems after participating in a CIRM-funded clinical trial at UCLA. Accelerating Cures for Californians—and Beyond The California Institute for Regenerative Medicine (CIRM) is a state agency created by California voters to accelerate stem cell and gene therapies for people with unmet medical needs.

Since 2004, Californians have entrusted CIRM with $8. 5 billion to accelerate promising discoveries through clinical trials, train a regenerative medicine workforce, strengthen the state’s biotechnology economy, and expand access to transformative treatments. Accelerating world class science CIRM is a catalyst for discovery, supporting cutting-edge cell and gene therapies to advance the field of medicine.

Through our research and development programs, CIRM continues to accelerate the translation of groundbreaking discoveries into therapies that have the potential to make a real impact on the lives of Californians and patients worldwide.

Learn how we advance research CIRM supports the full spectrum of research, from early discovery research to clinical trials, while building infrastructure to advance innovation and providing education programs to train the next generation of scientists.

Supports innovative early-stage research projects Advances research for pre-clinical studies or broad end use Supports clinical stage projects Provides opportunities to diverse, next-generation scientists Addresses infrastructure challenges in regenerative medicine Making an impact across California and the world CIRM drives new possibilities in science and promotes economic growth by creating jobs, funding infrastructure, and growing a diverse workforce to drive progress in the regenerative medicine field.

Investments in CIRM-funded research by biotech, pharma and industry partners Interns, fellows, and trainees supported through educational and training programs Bringing Therapies to Californians CIRM is committed to making groundbreaking cell and gene therapy treatments accessible to people who need them.

See patient access programs To date, CIRM has funded and supported more than 100 cell and gene therapy clinical trials, each aiming to find treatments for diseases and conditions that affect people across California and worldwide. Browse CIRM-funded clinical trials CIRM-Funded Clinical Trials Calling the next generation of scientists CIRM is passionate about catalyzing growth in regenerative medicine while championing inclusivity.

Our education and training programs provide internships and opportunities to inspire scientists and trainees at various levels. Explore training programs Stories from The Stem Cellar First CA taxpayer-funded therapy approved for rare childhood disorder The FDA has approved KRESLADI™, a groundbreaking genetic therapy funded by CIRM, to treat severe LAD-I in children, offering a life-saving option where none previously existed.

Closer to Cures: Stanford researcher Crystal Mackall discusses progress on clinical trials for rare cancers of the brain and spine with no known cure. In 2023, the California Institute for Regenerative Medicine (CIRM) funded a clinical trial led by Stanford researcher Crystal Mackall, MD, to investigate a possible therapy for rare brain and spinal tumors that arise from a specific genetic mutation, known as H3K27M.

CIRM-funded clinical trial gives siblings a second chance at life The Langenhop children, diagnosed with a rare, life-threatening immune disease, had their immune function successfully restored through a CIRM-funded gene therapy trial by Rocket Pharmaceuticals.