CIRM $4 million awarded to support innovative Gene Therapy for Rare Heart Disorder

The California Institute for Regenerative Medicine (CIRM) recently awarded $4 million to support the development of a one-time gene therapy for desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM), a rare and life-threatening genetic disorder.

This preclinical award to Deborah D. Ascheim, MD, and her team at Rejuvenate Bio are advancing RJB-0402, an innovative gene therapy designed to address the root causes of DSP ACM. This disorder arises from a mutation in the DSP gene, disrupting the connections between heart muscle cells and leading to irregular heartbeats and potential heart failure over time.

RJB-0402 utilizes AAV8, a type of virus commonly used in gene therapy, to deliver the gene responsible for producing the FGF21 protein to the liver. FGF21 supports heart function and mitigates issues such as irregular heartbeats, fat buildup, inflammation, and scarring-common problems in DSP ACM.

"Our goal is to support the most promising research forward," says Dr. Abla Creasey, Vice President of Therapeutics Development at CIRM. "A one-time gene therapy treatment for patients with this rare cardiac disease, DSP ACM, could have a significant impact. We look forward to supporting Rejuvenate Bio in bringing this exciting therapy to patients."

CIRM's funding and strategic support are vital in accelerating the development of RJB-0402. This financial backing will help transition the therapy from preclinical stages into clinical trials, providing a new treatment avenue for patients in desperate need.

Noah Davidsohn, PhD, Chief Scientific Officer and Co-Founder of Rejuvenate Bio emphasizes the transformative potential of this therapy: "We are confident that RJB-0402, a one-time gene therapy, could be a regenerative medicine breakthrough and the first disease-modifying therapy to address the unmet medical need of DSP ACM patients. The recent funding from CIRM will enable us to initiate our first-in-human clinical trial."

At CIRM, we are committed to advancing world-class science to deliver transformative regenerative medicine treatments equitably. The development of RJB-0402 represents a significant step forward in achieving this mission, offering hope and potential relief to those affected by DSP ACM.