UCLA receives grant to support development of HSC gene therapy for Angelman syndrome
Jan. 31, 2025
California Institute for Regenerative Medicine (CIRM) has awarded a $5.8 million translational research grant to a scientist at the University of California, Los Angeles (UCLA) to further support the development of a hematopoietic stem cell (HSC) gene therapy to treat Angelman syndrome, including preparation of a pre-IND package submission to the FDA.