California grants $7.4 million to advance gene-edited stem cell therapy for Friedreich’ s ataxia

Gen 13, 2026. The California Institute for Regenerative Medicine (CIRM) has awarded $7.4 million to support a University of California San Diego team developing a first-of-its-kind stem cell-based gene therapy for Friedreich’s ataxia, a rare inherited neurodegenerative disease that causes progressive loss of coordination, muscle strength, heart function and overall mobility. The new funding will help the research team complete the final steps required by federal regulators before they can apply to begin a first-in-human clinical trial.

California grants $7.4 million to advance gene-edited stem cell therapy for Friedreich’ s ataxia