First therapy funded by California taxpayer dollars is approved to treat rare childhood disorder

South San Francisco, Calif., March 27, 2026 - The California Institute for Regenerative Medicine (CIRM) celebrates a major milestone in the agency's 21-year history: the first approved therapy to come out of California taxpayer funding. The U.S. Food and Drug Administration (FDA) has approved KRESLADI™, a one-time therapy developed by Rocket Pharmaceuticals, Inc. to treat severe leukocyte adhesion deficiency-I (LAD-I) in children who have no bone marrow donor match for stem cell transplantation.

"With the first FDA-approved therapy from CIRM funding we are delivering on our commitment to Californians to support the development of new stem cell and gene therapies that save lives," said Jonathan Thomas, PhD, JD, CIRM President and CEO. "Thank you to the people of California who entrusted us with funds to support therapies like this one, and to the patients and their families who participated in clinical trials leading up to this milestone. Clinical trial participants are heroes whose bravery helps the entire disease community have access to improved therapies."

LAD-I is a rare genetic disease where a child's immune system can't properly fight infections, putting them at risk for serious illness. Children with the disease have recurrent, life-threatening bacterial and fungal infections that respond poorly to antimicrobials and require frequent hospitalizations.

The only treatment option available for children with LAD-I has been a bone marrow transplant, which runs the risk of serious long-term complications. With KRESLADI, doctors fix the defective gene in the patient's own blood-forming stem cells, helping their body make healthy white blood cells to fight infections. By using the patient's own cells, the approach avoids complications of a bone marrow transplant.

"The FDA-approved treatment for LAD-I is a major step forward for Californians who supported investing in the promise of regenerative medicine," said Rosa Canet-Avilés, PhD, CIRM Chief Science Officer. "We are thrilled that children with LAD-I have this new option available to them. In the future, we expect to see many more FDA-approved stem cell and gene therapies that will benefit people in California and around the world who have no other options."

CIRM invested $5,867,085 to support a clinical trial site for KRESLADI™ at UCLA Mattel Children's Hospital run by Dr. Donald Kohn. The global Phase 1/2 study of KRESLADI™ demonstrated a 100% survival rate one-year post-treatment for all nine patients enrolled in the trail. Patients were aged 5 months to 9 years with severe LAD-I, of which six were treated at the CIRM-funded UCLA site; the three other patients were treated at sites in London and Madrid.

This milestone comes at a time when CIRM is actively working to accelerate new therapies for rare diseases like LAD-I through its new Rare Disease Acceleration Through Platform Innovation and Delivery (RAPID) programrogram. Although individually rare diseases don't affect many people, cumulatively they affect over 30 million people in the U.S., about half of whom are young children with limited expected lifespan. Roughly 1 in 10 Americans have a rare disease of which 95% have no approved therapy.

The milestone is also a result of CIRM's network of supported clinics that deliver cell and gene therapy clinical trials and approved therapies to people across the state. The successful trial at UCLA took place in one of these supported clinics.

The approval of KRESLADI reflects years of scientific research, clinical investigation, and community collaboration, including patients, families, clinicians, advocacy groups, and regulators. As with all CIRM clinical programs, Rocket Pharmaceuticals will provide a path for making this therapy accessible to people in California. That requirement is part of CIRM's ongoing efforts to make sure people of California benefit from the therapies funded by CIRM.

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.