Key points from article :
Rejuvenate Bio, a gene therapy developer, has received a significant boost of $4 million from the California Institute for Regenerative Medicine (CIRM) to further develop its investigational new drug (IND) candidate RJB-0402 for the treatment of desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM).
Arrhythmogenic cardiomyopathy is a heart condition characterized by the gradual weakening and scarring of the heart muscle, often leading to arrhythmias and heart failure. It is frequently diagnosed in young adults and can significantly impact quality of life. Current treatment options are limited and often focus on managing symptoms rather than addressing the root cause of the disease.
This funding will enable Rejuvenate Bio to conduct additional preclinical studies in mice and non-human primates, scale up manufacturing processes, and engage with potential patients. The company's gene therapy technology leverages a known cardiometabolic-associated protein called FGF21 to address the underlying genetic defect that causes arrhythmogenic cardiomyopathy.
RJB-0402 is administered as one-time treatment to patients. By targeting the underlying genetic defect, it has the potential to provide a more effective and long-lasting treatment. The company's CEO Daniel Oliver highlights the potential of this therapy to address a wider range of cardiovascular and metabolic disorders.
As Rejuvenate Bio continues to advance its research and development efforts, it is poised to make a significant contribution to the field of cardiovascular medicine and improve the lives of patients with arrhythmogenic cardiomyopathy.
