Raphael Thys
CIRM funding will support the company through IND-enabling studies of FGF21-boosting gene therapy for genetic heart condition.
Age-targeting gene therapy company Rejuvenate Bio has secured a $4 million grant from the California Institute for Regenerative Medicine (CIRM) to advance development of RJB-0402, a one-time gene therapy for desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM). The company says the new funding will allow it to complete the necessary Investigational New Drug (IND)-enabling activities before it can move into clinical studies in humans.
Founded in 2018, Rejuvenate Bio was spun out from research conducted in George Church’s lab at the Wyss Institute at Harvard Medical School. The company’s approach is rooted in the concept that aging is the predominant risk factor for many chronic diseases.
DSP ACM is a serious and potentially fatal genetic disorder characterized by ventricular arrhythmias and progressive cardiac dysfunction due to fibrofatty replacement of the ventricular myocardium. The disease is primarily caused by mutations in genes critical for the electrical and mechanical coupling of heart muscle cells.
“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 of 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.”
RJB-0402 is an investigational gene therapy using an AAV8 vector to promote liver-specific expression of the FGF21 protein, which targets several pathological aspects of DSP ACM, including cardiac dysfunction, arrhythmias, adipogenesis, inflammation, and fibrosis. Rejuvenate Bio’s preclinical studies in mouse models of ACM demonstrated that RJB-0402 significantly improved cardiac structure and function and reduced arrhythmias to normal levels.
“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,” said Dr Noah Davidsohn, CSO of Rejuvenate Bio.
The broader implications of Rejuvenate Bio’s approach may also extend beyond DSP ACM. The company’s founders previously told us that increasing levels of FGF21 – a protein that declines with age – could also have benefits for overall healthspan and longevity. By elevating FGF21 levels, RJB-0402 aims to restore a more youthful and healthy physiological state, potentially offering therapeutic applications for a wide range of conditions.
With $5.5 billion in funding and over 150 active programs, CIRM is the largest institution dedicated to advancing cellular medicine. It collaborates with both academic and industrial partners to accelerate innovative technologies.
“Our goal is to move the most promising research forward,” said 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, 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.”