The U.S. Food and Drug Administration has granted accelerated approval to KRESLADI, a one-time gene therapy developed by Rocket Pharmaceuticals to treat severe leukocyte adhesion deficiency-I in children without bone marrow donor matches. This marks the first FDA-approved therapy to emerge from funding by the California Institute for Regenerative Medicine, representing a significant milestone in the agency's 21-year history of supporting regenerative medicine research.
Leukocyte adhesion deficiency-I is a rare genetic disorder where children's immune systems cannot properly fight infections, leading to recurrent life-threatening bacterial and fungal infections that respond poorly to antimicrobial treatments and require frequent hospitalizations. The only previous treatment option 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 bodies to produce healthy white blood cells capable of fighting infections while avoiding transplant complications.
CIRM invested $5,867,085 to support a clinical trial site at UCLA Mattel Children's Hospital under Dr. Donald Kohn's direction. The global Phase 1/2 study demonstrated a 100% survival rate one year post-treatment for all nine patients aged 5 months to 9 years with severe LAD-I, including six treated at the CIRM-funded UCLA site and three at international locations. This achievement reflects years of scientific research, clinical investigation, and collaboration among patients, families, clinicians, advocacy groups, and regulators.
The approval arrives as CIRM accelerates development of therapies for rare diseases through its new Rare Disease Acceleration Through Platform Innovation and Delivery 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, making this development particularly significant for the broader rare disease community.
As part of CIRM's ongoing efforts to ensure Californians benefit from therapies funded by the agency, Rocket Pharmaceuticals will provide access pathways for this treatment within California. The successful trial occurred within CIRM's network of supported clinics that deliver cell and gene therapy clinical trials and approved therapies across the state, demonstrating the infrastructure's effectiveness in advancing medical breakthroughs.
This milestone validates California's investment in regenerative medicine and establishes a precedent for future therapies developed through public funding mechanisms. The development signals potential for similar approaches to address other rare genetic disorders, potentially transforming treatment paradigms for conditions that currently lack effective options. For families affected by LAD-I and similar conditions, this approval represents not just a new treatment option but validation of the clinical trial participation that makes such medical advances possible.
