CIRM's Historic Breakthrough: First FDA-Approved Therapy from CA Taxpayer Funding

The California Institute for Regenerative Medicine (CIRM) has achieved a historic breakthrough with the first FDA-approved therapy resulting from California taxpayer funding. The U.S. Food and Drug Administration granted accelerated approval to KRESLADI™, a one-time gene therapy developed by Rocket Pharmaceuticals to treat severe leukocyte adhesion deficiency-I (LAD-I) in children without bone marrow donor matches. This milestone represents the culmination of 21 years of CIRM's work and delivers on the promise made to Californians who entrusted the agency with billions in funding. CIRM President and CEO Jonathan Thomas emphasized that this achievement fulfills their commitment to develop life-saving stem cell and gene therapies, crediting both California taxpayers and the heroic clinical trial participants who made this breakthrough possible.

LAD-I is a devastating rare genetic disease where children's immune systems cannot properly fight infections, leading to recurrent life-threatening bacterial and fungal infections that require frequent hospitalizations and respond poorly to conventional treatments. Previously, the only treatment option was bone marrow transplantation, which carries significant risks of serious long-term complications. KRESLADI represents a revolutionary approach that fixes the defective gene in the patient's own blood-forming stem cells, enabling their body to produce healthy white blood cells capable of fighting infections. This innovative method avoids the complications associated with bone marrow transplants by using the patient's own cells, offering new hope for affected children and their families.

CIRM invested $5,867,085 to support a clinical trial site for KRESLADI at UCLA Mattel Children's Hospital, which was run by Dr. Donald Kohn. The global Phase 1/2 study demonstrated remarkable results with a 100% survival rate one-year post-treatment for all nine patients enrolled, who ranged from 5 months to 9 years old with severe LAD-I. Six patients were treated at the CIRM-funded UCLA site, while three others received treatment at sites in London and Madrid. This success comes as CIRM launches its new Rare Disease Acceleration Through Platform Innovation and Delivery (RAPID) program, designed to accelerate therapies for rare diseases like LAD-I. The RAPID program addresses the critical need for treatments, given that rare diseases collectively affect over 30 million Americans, with approximately 95% having no approved therapy available.

The approval of KRESLADI reflects years of scientific research, clinical investigation, and collaboration among patients, families, clinicians, advocacy groups, and regulators. As part of CIRM's ongoing efforts to ensure Californians benefit from therapies funded by the agency, Rocket Pharmaceuticals will provide a pathway for making this treatment accessible to people in California. This milestone also highlights the effectiveness of CIRM's network of supported clinics that deliver cell and gene therapy clinical trials across the state. The successful trial at UCLA took place within this network, demonstrating how CIRM's infrastructure accelerates medical breakthroughs that change lives both in California and around the world.