March 27, 2025 the California Institute for Regenerative Medicine (CIRM) voted to fund our late stage preclinical project (CLIN1) wherein we will study a therapeutic option for Angelman Syndrome (AS) in preparation for clinical trials.
Angelman syndrome is a rare genetic disorder that results from loss of function of the UBE3A gene resulting in global developmental delay, intellectual disability, epilepsy, sleep difficulties, and ataxic gait. There is currently no cure for Angelman syndrome and treatments can only help manage symptoms. Therefore, we will be advancing a gene-modified stem cell therapy involving modifying a patient’s own hematopoietic stems cells with a corrected Ube3a gene. Our hope is that transplanted cells will deliver functional Ube3a protein resulting in improvement, halting, or reversal of Angelman syndrome symptoms.
We are very grateful to have received this funding from CIRM. Read more on this and other funded projects here: https://www.cirm.ca.gov/about-cirm/newsroom/press-releases/cirm-approves-new-and-updated-funding-programs-awards-26-5-million-for-clinical-research-and-development/