The California Institute for Regenerative Medicine achieved a landmark milestone as the first therapy developed with California taxpayer dollars received approval from the U.S. Food and Drug Administration. KRESLADI, developed by Rocket Pharmaceuticals, treats severe leukocyte adhesion deficiency-I in children who lack suitable bone marrow donors. This approval fulfills CIRM's commitment to developing life-saving stem cell and gene therapies, according to agency President and CEO Jonathan Thomas, who acknowledged California residents for providing funding and recognized clinical trial participants as heroes whose participation benefits the broader disease community.
Leukocyte adhesion deficiency-I is a rare genetic disorder that compromises a child's immune system, leading to recurrent, life-threatening infections that often resist antimicrobial treatment and require frequent hospitalizations. Previously, the only available treatment was bone marrow transplantation, which carries risks of serious long-term complications. KRESLADI represents a different approach by correcting the defective gene in the patient's own blood-forming stem cells, enabling the body to produce healthy white blood cells. This method avoids the complications associated with bone marrow transplants from donors.
CIRM invested $5,867,085 to support a clinical trial site at UCLA Mattel Children's Hospital under Dr. Donald Kohn. The global Phase 1/2 study demonstrated a 100% survival rate one year post-treatment for all nine enrolled patients aged 5 months to 9 years with severe LAD-I. Six patients received treatment at the CIRM-funded UCLA site, while three others were treated at facilities in London and Madrid. Rosa Canet-Avilés, CIRM Chief Science Officer, emphasized this development validates California's investment in regenerative medicine and provides new options for children with LAD-I. She anticipates more FDA-approved stem cell and gene therapies benefiting people worldwide who have limited treatment alternatives.
This milestone coincides with CIRM's launch of the Rare Disease Acceleration Through Platform Innovation and Delivery program to accelerate therapies for rare conditions. 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 1 in 10 Americans has a rare disease, with 95% lacking approved therapies. The successful trial occurred within CIRM's network of clinics that deliver cell and gene therapy clinical trials and approved treatments across California. Rocket Pharmaceuticals will ensure accessibility to this therapy for Californians as part of CIRM's requirement that funded therapies benefit state residents.
