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 (LAD-I) in children. This marks the first FDA-approved therapy to emerge from California taxpayer funding since CIRM's establishment 21 years ago.
LAD-I is a rare genetic disorder that compromises a child's immune system, leaving them vulnerable to recurrent, life-threatening bacterial and fungal infections that respond poorly to antibiotics and often require frequent hospitalizations. Previously, the only available treatment was bone marrow transplantation, 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 without the complications associated with donor transplants.
CIRM invested $5,867,085 to support a clinical trial site for KRESLADI at UCLA Mattel Children's Hospital, led by Dr. Donald Kohn. The global Phase 1/2 study demonstrated a 100% survival rate one year post-treatment for all nine patients enrolled, ranging 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 facilities in London and Madrid.
This approval arrives as CIRM intensifies efforts to accelerate therapies for rare diseases through its new Rare Disease Acceleration Through Platform Innovation and Delivery (RAPID) program. While individual rare diseases affect relatively few people, collectively they impact over 30 million Americans, approximately half of whom are young children with limited life expectancy. Approximately 95% of rare diseases currently lack approved therapies.
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 part of CIRM's requirements, Rocket Pharmaceuticals will establish a pathway to make this therapy accessible to Californians, ensuring that state residents benefit from therapies funded by taxpayer dollars. The approval represents years of scientific research, clinical investigation, and collaboration among patients, families, clinicians, advocacy groups, and regulators.
