DSP AAV gene therapy (Renovacor)

- Funding will advance development of Rejuvenate’s one-time gene therapy RJB-0402 for the treatment of desmoplakin gene variant arrhythmogenic cardiomyopathy SAN DIEGO--(BUSINESS WIRE)-- Rejuvenate Bio, announced today that the California Institute for Regenerative Medicine (CIRM) has awarded the company a $4M grant to complete IND-enabling activities for the development of RJB-0402 for the treatment of desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM). RJB-0402 is a novel systemically administered investigational AAV8 gene therapy that drives liver specific expression of FGF21 protein, targeting multiple key pathological drivers of DSP ACM. Since the DSP gene is too large for gene replacement therapy, novel strategies are needed to treat DSP ACM. FGF21 has known salutary effects on cardiac dysfunction as well as ventricular arrhythmias, adipogenesis, inflammation, and fibrosis, all of which are DSP ACM hallmarks. Furthermore, clinical efficacy of RJB-0402 in DSP ACM may form the basis for the treatment in other forms of ACM. “DSP ACM is a rare, severe, life-threatening, and debilitating disease that typically manifests in young adults as a high risk of life-threatening ventricular arrhythmias, sudden cardiac death, and progression to heart failure,” said Dan Oliver, CEO & Co-Founder, Rejuvenate Bio. “There is currently no disease modifying therapy for patients with this disease, and our gene therapy RJB-0402 addresses a significant unmet medical need.” “The funding and strategic support from CIRM will accelerate development of our gene therapy candidate RJB-0402 into clinical trials for DSP ACM patients in desperate need of new therapies,” said Noah Davidsohn, Ph.D., Chief Scientific Officer & Co-Founder, Rejuvenate Bio. “We are confident that RJB-0402, a one-time gene therapy, could be a transformative regenerative medicine and the first disease modifying therapy to address the unmet medical need of DSP ACM patients, and the recent funding from CIRM will enable us to initiate our first in human clinical trial.” Nonclinical proof-of-concept studies in the ACM mouse model showed that RJB-0402 significantly improved cardiac structure, function, and markedly reduced the arrhythmia burden, normalizing premature ventricular contractions (PVCs) to wild type levels. The clinical relevance of these efficacy signals provides a scientific rationale for further development as a therapeutic with the potential to meaningfully impact patients’ survival, symptoms, and quality of life. “Our goal is to move the most promising research forward,” said Dr. Abla Creasey, Vice President of Therapeutics Development at the California Institute for Regenerative Medicine (CIRM). “A one-time gene therapy treatment for patients with this rare cardiac disease DSP ACM, would have significant impact for patients with this degenerative disorder. We look forward to supporting Rejuvenate Bio in bringing this regenerative therapy to patients with this degenerative disease.” About Desmoplakin Arrhythmogenic Cardiomyopathy (DSP ACM) DSP ACM is a severe, life-threatening, and debilitating, rare autosomal recessive disease that results in ventricular arrhythmias that are often life threatening and progressive cardiac dysfunction caused by fibrofatty replacement of ventricular myocardium which can lead to heart failure for which no disease modifying therapies exist. While more than 10 different genes have been implicated in this heterogeneous disease, the majority involve one or more variants in desmosomal genes which are key components of the cardiac intercalated disc that are essential for the electrical and mechanical coupling of cardiomyocytes. These multiprotein complexes work together to provide cardiac mechanical integrity and strength as well as electrical communication and synchronization. While the exact prevalence of DSP ACM is unknown, it is estimated to affect an estimated 30,000-50,000 people in the United States. About Rejuvenate Bio Rejuvenate Bio is a biotechnology company dedicated to developing novel gene therapies for chronic age-related diseases. Rejuvenate Bio has built a gene therapy pipeline with huge potential in chronic disease by utilizing clinically validated gene targets and a delivery approach that ensures well tolerated, durable expression. Founded on scientific research developed at the Wyss Institute at Harvard Medical School, Rejuvenate Bio has developed groundbreaking therapies to treat chronic age-related disease in both humans and animals. The company is headquartered in San Diego, CA. For more information, visit . About California Institute for Regenerative Medicine (CIRM) At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies. With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is the world’s largest institution dedicated to helping people by bringing the future of cellular medicine closer to reality. For more information go to .