RJB-0402

RJB-0402 is an investigational, one-time gene therapy developed by Rejuvenate Bio for the treatment of desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM), a rare and life-threatening genetic heart disorder. The therapy uses an adeno-associated virus serotype 8 (AAV8) vector to deliver the gene encoding fibroblast growth factor 21 (FGF21) specifically to hepatocytes in the liver. This results in sustained overexpression of FGF21, which has demonstrated beneficial effects on cardiac function, including reducing ventricular arrhythmias, inflammation, fibrosis, and fat accumulation—key pathological drivers of DSP ACM. Unlike mutation-specific gene replacement therapies that are limited by the large size of the DSP gene and only address certain mutations, RJB-0402’s approach is not mutation-specific and may benefit a broader patient population with ACM. Preclinical studies have shown that a single dose significantly improves cardiac structure and function while normalizing premature ventricular contractions in mouse models[1][5][6][7][9].