The California Institute for Regenerative Medicine (CIRM) has achieved a historic milestone with the FDA's accelerated approval of KRESLADI, a one-time gene therapy developed by Rocket Pharmaceuticals for severe leukocyte adhesion deficiency-I (LAD-I) in children without a bone marrow donor. This marks the first approved therapy funded by California taxpayer dollars through CIRM.
LAD-I is a rare genetic disorder that impairs the immune system's ability to fight infections, leading to recurrent, life-threatening bacterial and fungal infections. Previously, the only treatment option was bone marrow transplant, which carries significant risks. KRESLADI corrects the defective gene in the patient's own blood-forming stem cells, enabling the body to produce healthy white blood cells without the need for a donor.
CIRM invested $5,867,085 to support a clinical trial site at UCLA Mattel Children's Hospital led by Dr. Donald Kohn. The global Phase 1/2 study showed a 100% survival rate one-year post-treatment among all nine patients, aged 5 months to 9 years. Six patients were treated at the CIRM-funded UCLA site, while three were treated in London and Madrid.
This approval underscores CIRM's commitment to advancing regenerative medicine, as outlined by President and CEO Jonathan Thomas, who thanked California voters for their support and praised clinical trial participants. Chief Science Officer Rosa Canet-Avilés highlighted the significance for Californians and anticipated more FDA-approved stem cell and gene therapies in the future.
The milestone coincides with CIRM's Rare Disease Acceleration Through Platform Innovation and Delivery (RAPID) program, which aims to accelerate therapies for rare diseases. Although individually rare, these conditions affect over 30 million people in the U.S., with 95% lacking approved treatments.
The approval also demonstrates the effectiveness of CIRM's network of supported clinics, which deliver cell and gene therapy clinical trials and approved therapies statewide. As part of its requirements, Rocket Pharmaceuticals will ensure the therapy is accessible to Californians, reflecting CIRM's commitment to benefiting the state's residents.
This achievement represents years of collaboration among scientists, patients, families, clinicians, advocacy groups, and regulators. Since its creation by California voters in 2004, CIRM has been entrusted with $8.5 billion to accelerate stem cell and gene therapies. For more information, visit CIRM's website.
