CIRM grant $4.8m to fund gene therapy for rare disease

6 December 2022. CIRM grant will fund novel gene therapy that aims for single lifelong treatment of Friedreich’s ataxia, a progressive neuromuscular disorder; a second CIRM grant will advance efforts to leverage UC San Diego research on another rare disease. 

I n 2020, Cherqui and her team, in a new study, found that specifically described how CRISPR-Cas9 gene editing of hematopoietic stem cells from patients with FA could work. Hematopoietic stem cells are capable of developing into all types of blood cells. CRISPR-Cas9 is a technology that allows scientists to edit parts of the genome by removing, adding or altering sections of the DNA sequence. 

 In November 2022, the California Institute for Regenerative Medicine (CIRM) awarded Cherqui and the team a grant of $4.8 million, to move this approach closer to clinical trials. The funding will be used to develop a therapy based on gene-edited hematopoietic stem and progenitor cells derived from FA patients, which would be re-infused as a one-time, lifelong treatment.

CIRM grant $4.8m to fund gene therapy for rare disease