CIRM Supports Innovative Gene Therapy for Rare Heart Disorder

/ California Institute for Regenerative Medicine (CIRM)
X-ray image of acute arrythmic heart
Image Source: American Heart Association Journals

 
The California Institute for Regenerative Medicine (CIRM) awarded $4 million to support a gene therapy for rare life-threatening genetic disorder.

A rare heart disorder

Known as desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM), the genetic disorder is a rare heart disorder.  
 
This preclinical award went to Deborah D. Ascheim, MD, and her team at Rejuvenate Bio. The team is advancing RJB-0402, an innovative gene therapy designed to address the root causes of DSP ACM. This disorder stems from a mutation in the DSP gene, which disrupts connections between heart muscle cells and leads to irregular heartbeats and, over time, potential heart failure.

RJB‑0402 uses AAV8, a virus commonly used in gene therapy, to deliver the gene that produces the FGF21 protein to the liver. FGF21 supports heart function and reduces irregular heartbeats, fat buildup, inflammation, and scarring—key issues in DSP ACM.

“Our goal is to advance the most promising research,” says Dr. Abla Creasey, Vice President of Therapeutics Development at CIRM. “A one‑time gene therapy for DSP ACM could make a significant impact. We look forward to supporting Rejuvenate Bio in bringing this therapy to patients.”

CIRM’s support

CIRM’s funding and strategic support play a critical role in accelerating the development of RJB‑0402. This investment will help move the therapy from preclinical studies into clinical trials, offering a new treatment pathway for patients in urgent need.

Noah Davidsohn, PhD, Chief Scientific Officer and Co‑Founder of Rejuvenate Bio, highlights the therapy’s potential: “We believe RJB‑0402, a one‑time gene therapy, could be a regenerative medicine breakthrough and the first disease‑modifying therapy for DSP ACM. CIRM’s funding will allow us to launch our first‑in‑human trial.”

CIRM remains committed to advancing world‑class science and delivering regenerative medicine treatments equitably. Developing RJB‑0402 marks a significant step toward that mission and offers new hope for people affected by DSP ACM.

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