Key Highlights
- Rejuvenate Bio awarded $4M by CIRM for RJB-0402 development.
- RJB-0402 targets DSP ACM, a severe and rare cardiac disease.
- Novel AAV8 gene therapy drives liver-specific FGF21 protein expression.
- RJB-0402 shows promise in nonclinical studies for improving cardiac function.
Source: Business Wire
Notable Quotes
- “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. There is currently no disease modifying therapy for patients with this disease, and our gene therapy RJB-0402 addresses a significant unmet medical need.” — Dan Oliver, CEO & Co-Founder at Rejuvenate Bio
- “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. 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.” — Noah Davidsohn, Ph.D., Chief Scientific Officer & Co-Founder at Rejuvenate Bio
- “Our goal is to move the most promising research forward. 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.” — Dr. Abla Creasey, Vice President of Therapeutics Development at CIRM
SoHC's Take
Rejuvenate Bio’s recent $4M grant from the California Institute for Regenerative Medicine (CIRM) marks a significant milestone in the development of RJB-0402, a novel gene therapy for the treatment of desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM). This disease, characterized by severe and life-threatening cardiac dysfunctions, currently lacks disease-modifying therapies. The innovative approach of RJB-0402, which leverages AAV8 gene therapy to express the FGF21 protein specifically in the liver, shows promising potential. The nonclinical studies demonstrating improved cardiac function and reduced arrhythmia burden underline the therapy’s potential impact. This funding will undoubtedly accelerate the transition of RJB-0402 into clinical trials, potentially offering the first effective treatment for patients suffering from DSP ACM.
