The California Institute for Regenerative Medicine (CIRM) has reached a significant milestone with the U.S. Food and Drug Administration granting accelerated approval to KRESLADI, a one-time therapy developed by Rocket Pharmaceuticals to treat severe leukocyte adhesion deficiency-I in children. This represents the first FDA-approved therapy to emerge from California taxpayer funding in the agency's 21-year history.
Leukocyte adhesion deficiency-I is a rare genetic disease that compromises a child's immune system, leaving them vulnerable to recurrent, life-threatening bacterial and fungal infections that respond poorly to antimicrobials and often require frequent hospitalizations. Previously, the only treatment option was a bone marrow transplant, which carries risks of serious long-term complications. KRESLADI works by correcting 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 while avoiding the complications associated with bone marrow transplants.
CIRM invested $5,867,085 to support a clinical trial site for KRESLADI at UCLA Mattel Children's Hospital under the direction of Dr. Donald Kohn. The global Phase 1/2 study demonstrated a 100% survival rate one-year post-treatment for all nine patients enrolled, ranging in age from 5 months to 9 years with severe LAD-I. Six patients were treated at the CIRM-funded UCLA site, with three others treated at sites in London and Madrid.
This approval arrives as CIRM actively works to accelerate new therapies for rare diseases through its Rare Disease Acceleration Through Platform Innovation and Delivery (RAPID) program. While individually rare diseases affect relatively few people, collectively they impact over 30 million Americans, approximately half of whom are young children with limited life expectancy. Roughly 1 in 10 Americans has a rare disease, with 95% having no approved therapy available.
The successful trial at UCLA occurred within CIRM's network of supported clinics that deliver cell and gene therapy clinical trials and approved therapies across California. As with all CIRM clinical programs, Rocket Pharmaceuticals will provide a pathway to make this therapy accessible to Californians, ensuring that state residents benefit from treatments funded by taxpayer dollars. The approval reflects years of scientific research, clinical investigation, and collaboration among patients, families, clinicians, advocacy groups, and regulators.
Jonathan Thomas, PhD, JD, CIRM President and CEO, emphasized that this milestone delivers on the agency's commitment to Californians to support the development of new stem cell and gene therapies that save lives. Rosa Canet-Avilés, PhD, CIRM Chief Science Officer, noted that this represents a major step forward for Californians who supported investing in regenerative medicine and expressed optimism that many more FDA-approved stem cell and gene therapies will benefit people in California and worldwide who have no other treatment options.
