Blockchain Registration Transaction Record

CIRM's Historic Breakthrough: First FDA-Approved Therapy from CA Taxpayer Funding

CIRM achieves historic milestone with first FDA-approved therapy from California taxpayer funding. KRESLADI gene therapy treats rare LAD-I disease in children with 100% survival rate in trials.

This news represents a transformative moment in medical science and public health investment. For families with children suffering from LAD-I and other rare genetic diseases, KRESLADI offers a life-saving alternative to risky bone marrow transplants with a proven 100% survival rate in trials. Beyond the immediate impact on LAD-I patients, this breakthrough validates California's $8.5 billion investment in regenerative medicine and demonstrates that taxpayer funding can successfully translate into FDA-approved therapies. The success of this therapy establishes a precedent for treating other rare genetic disorders using similar gene-editing approaches, potentially benefiting millions of Americans affected by rare diseases. For the biotechnology sector, it showcases how sustained public investment in research infrastructure and clinical networks can accelerate medical innovation. This achievement also strengthens the case for continued funding of regenerative medicine research, as it proves that long-term investment in basic science can yield practical, life-saving treatments. The requirement for accessibility to Californians ensures that the state's taxpayers directly benefit from the therapies they helped fund, creating a model for equitable medical innovation.