The U.S. Food and Drug Administration has granted accelerated approval to KRESLADI, a one-time therapy developed by Rocket Pharmaceuticals to treat severe leukocyte adhesion deficiency-I in children without bone marrow donor matches. This marks the first approved therapy to emerge from California taxpayer funding through the California Institute for Regenerative Medicine, representing a significant milestone in the agency's 21-year history.
Jonathan Thomas, PhD, JD, CIRM President and CEO, stated that this approval delivers on the commitment to Californians to support the development of new stem cell and gene therapies that save lives. He acknowledged the people of California who entrusted the agency with funds and the clinical trial participants whose bravery helps the entire disease community access improved therapies.
Leukocyte adhesion deficiency-I is a rare genetic disease where a child's immune system cannot properly fight infections, putting them at risk for serious illness. Children with the disease experience recurrent, life-threatening bacterial and fungal infections that respond poorly to antimicrobials and require frequent hospitalizations. The only previous treatment option was bone marrow transplantation, which carries risks of serious long-term complications.
KRESLADI works by fixing the defective gene in the patient's own blood-forming stem cells, helping their body produce healthy white blood cells to fight infections. This approach avoids complications associated with bone marrow transplants by using the patient's own cells. Rosa Canet-Avilés, PhD, CIRM Chief Science Officer, described the FDA-approved treatment as a major step forward for Californians who supported investing in regenerative medicine, expressing excitement that children with LAD-I now have this new option available.
CIRM invested $5,867,085 to support a clinical trial site for KRESLADI at UCLA Mattel Children's Hospital run by Dr. Donald Kohn. The global Phase 1/2 study demonstrated a 100% survival rate one-year post-treatment for all nine patients enrolled, aged 5 months to 9 years 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 milestone occurs as CIRM actively works to accelerate new therapies for rare diseases through its new Rare Disease Acceleration Through Platform Innovation and Delivery program. Although individually rare diseases affect relatively few people, cumulatively they impact over 30 million people in the United States, approximately half of whom are young children with limited expected lifespans. Roughly one in ten Americans has a rare disease, with 95% having no approved therapy.
The approval also results from CIRM's network of supported clinics that deliver cell and gene therapy clinical trials and approved therapies across California. The successful trial at UCLA took place in one of these supported clinics. The approval reflects years of scientific research, clinical investigation, and community collaboration involving patients, families, clinicians, advocacy groups, and regulators. As with all CIRM clinical programs, Rocket Pharmaceuticals will provide a path for making this therapy accessible to people in California, part of CIRM's ongoing efforts to ensure Californians benefit from therapies funded by the agency.
