Rejuvenate Bio Secures $4M from California Regenerative Medicine Institute

Rejuvenate Bio, a biotechnology firm headquartered in San Diego, has been awarded a $4 million grant from the California Institute for Regenerative Medicine (CIRM). The funding aims to support the development of RJB-0402, a one-time gene therapy designed to treat desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM).

RJB-0402 is an innovative gene therapy that uses an AAV8 vector to deliver the FGF21 protein specifically to the liver. This protein targets several key pathological drivers of DSP ACM, a condition that affects the heart’s electrical and mechanical functions, leading to severe arrhythmias and heart failure. The conventional gene replacement approach is not feasible for DSP ACM due to the large size of the DSP gene, making alternative strategies like RJB-0402 essential. FGF21 is known for its beneficial effects on cardiac health, combating issues such as ventricular arrhythmias, inflammation, and fibrosis, which are common in DSP ACM. The success of RJB-0402 could potentially pave the way for treating other forms of arrhythmogenic cardiomyopathy (ACM).

Dan Oliver, CEO and Co-Founder of Rejuvenate Bio, emphasized the gravity of DSP ACM, describing it as a rare and debilitating condition that often manifests in young adults, posing a high risk of sudden cardiac death and heart failure. Currently, there are no therapies available that can modify the course of the disease. "Our gene therapy RJB-0402 addresses a significant unmet medical need," Oliver stated.

Noah Davidsohn, Ph.D., Chief Scientific Officer and Co-Founder of Rejuvenate Bio, expressed gratitude for CIRM’s funding and support, which he believes will expedite the progression of RJB-0402 into clinical trials. Davidsohn is optimistic about the transformative potential of RJB-0402, which could become the first disease-modifying treatment for DSP ACM patients, addressing an urgent medical need.

Nonclinical studies conducted on mouse models of ACM have shown promising results for RJB-0402. The gene therapy significantly improved cardiac structure and function, and markedly reduced the incidence of arrhythmias, normalizing premature ventricular contractions to levels seen in non-affected mice. These results provide a strong scientific basis for further development of RJB-0402 as a therapeutic option that could positively impact patients' survival, symptoms, and quality of life.

Dr. Abla Creasey, Vice President of Therapeutics Development at CIRM, highlighted the significance of this research. According to Dr. Creasey, a one-time gene therapy for this rare cardiac condition could have a profound impact on patients suffering from DSP ACM. She expressed optimism about supporting Rejuvenate Bio in their mission to bring this revolutionary therapy to those in need.

Desmoplakin Arrhythmogenic Cardiomyopathy (DSP ACM) is a rare and severe autosomal recessive disease characterized by life-threatening ventricular arrhythmias and progressive cardiac dysfunction. This dysfunction is due to fibrofatty replacement of the ventricular myocardium, which can eventually lead to heart failure. The disease involves multiple variants in desmosomal genes, critical components of the cardiac intercalated disc, essential for the electrical and mechanical cohesion of heart cells. While DSP ACM's exact prevalence is uncertain, it is estimated to affect between 30,000 and 50,000 people in the United States.

Rejuvenate Bio is committed to advancing gene therapies for chronic age-related diseases. The company has developed a pipeline of gene therapies that target clinically validated genes and employ a delivery method ensuring durable and well-tolerated expression. Originating from research at Harvard Medical School's Wyss Institute, Rejuvenate Bio aims to treat chronic diseases in humans and animals effectively.

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