First California Taxpayer-Funded Therapy Receives FDA Approval for Rare Childhood Disorder

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 without bone marrow donor matches. This marks the first FDA-approved therapy to emerge from California taxpayer funding in the agency's 21-year history, representing a tangible return on public investment in regenerative medicine. CIRM President and CEO Jonathan Thomas emphasized this achievement fulfills the commitment to Californians to support life-saving stem cell and gene therapies, acknowledging both taxpayers and clinical trial participants whose contributions made this milestone possible.

Leukocyte adhesion deficiency-I is a rare genetic disorder that severely compromises a child's immune system, leaving them vulnerable to recurrent, life-threatening infections that respond poorly to treatment and necessitate frequent hospitalizations. Until now, the only available treatment option has been bone marrow transplantation, which carries significant risks of serious long-term complications. KRESLADI represents a paradigm shift by genetically correcting the defective gene in the patient's own blood-forming stem cells, enabling the body to produce healthy white blood cells capable of fighting infections. This autologous approach eliminates the complications associated with donor bone marrow transplants, offering a potentially safer alternative for affected children.

The clinical development of KRESLADI received substantial support from California taxpayers through CIRM, which invested $5,867,085 to fund a clinical trial site at UCLA Mattel Children's Hospital under the direction of Dr. Donald Kohn. The global Phase 1/2 study demonstrated remarkable results, with all nine enrolled patients aged 5 months to 9 years achieving a 100% survival rate one year post-treatment. Six patients received treatment at the CIRM-funded UCLA site, while three others were treated at facilities in London and Madrid. This success underscores the effectiveness of CIRM's network of supported clinics that deliver cell and gene therapy clinical trials across California, ensuring residents have access to cutting-edge medical interventions.

This approval arrives as CIRM intensifies efforts to accelerate therapies for rare diseases through initiatives like the 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. Notably, about 1 in 10 Americans has a rare disease, and 95% of these conditions lack approved therapies, highlighting the critical need for continued investment and innovation in this area. The approval of KRESLADI demonstrates how targeted funding can address these unmet medical needs while advancing scientific knowledge and therapeutic capabilities.

The milestone reflects years of collaborative effort involving patients, families, clinicians, advocacy groups, and regulatory bodies. As with all CIRM clinical programs, Rocket Pharmaceuticals will establish pathways to ensure this therapy remains accessible to Californians, honoring the agency's commitment to delivering benefits back to the state's residents who funded the research. This achievement validates California's pioneering investment in regenerative medicine and establishes a precedent for future therapies that may emerge from similar public-private partnerships, potentially transforming treatment landscapes for numerous conditions with limited options.