Rejuvenate Bio reposted this
CIRM awarded $4 million to support the development of a one-time gene therapy for desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM). This funding supports Deborah Ascheim, MD, and her team at Rejuvenate Bio in advancing RJB-0402, a therapy designed to address the root causes of DSP ACM. DSP ACM arises from a mutation in the DSP gene, leading to irregular heartbeats and potential heart failure over time. RJB-0402 uses AAV8 to deliver the gene responsible for producing the FGF21 protein, which supports heart function and mitigates issues common in DSP ACM. This award will help transition the therapy into clinical trials, providing a new treatment avenue for patients in need.