Rejuvenate Bio, a pioneering biotechnology firm specializing in gene therapies, announced today the reception of a $4 million grant from the California Institute for Regenerative Medicine (CIRM) to propel the development of its innovative gene therapy, RJB-0402, aimed at treating desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM).
Desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM) is a rare, severe disease that poses life-threatening risks such as ventricular arrhythmias and heart failure progression, particularly affecting young adults. Rejuvenate Bio’s RJB-0402 represents a novel approach, leveraging systemically administered AAV8 gene therapy to drive liver-specific expression of FGF21 protein. This therapeutic strategy targets multiple underlying pathological mechanisms of DSP ACM, including cardiac dysfunction, ventricular arrhythmias, inflammation, and fibrosis.
Marking a significant advancement in regenerative medicine, RJB-0402 has shown promising results in preclinical studies, demonstrating substantial improvements in cardiac structure, function, and reduction in arrhythmia burden. This breakthrough therapy not only aims to address the urgent medical needs of DSP ACM patients but also holds potential applications across other forms of arrhythmogenic cardiomyopathy.
Dan Oliver, CEO & Co-Founder of Rejuvenate Bio, emphasized the critical role of this funding in advancing their mission: “DSP ACM represents a significant unmet medical need, with no existing disease-modifying therapies available. RJB-0402 has the potential to be transformative, offering a one-time gene therapy solution that could improve patients’ survival and quality of life.”
Noah Davidsohn, Ph.D., Chief Scientific Officer & Co-Founder, highlighted the strategic impact of CIRM’s support: “The funding from CIRM will accelerate our efforts to initiate clinical trials, marking a pivotal step towards realizing the potential of RJB-0402 as a regenerative therapy. We are committed to advancing our innovative approach to benefit patients suffering from DSP ACM.”
Dr. Abla Creasey, Vice President of Therapeutics Development at CIRM, underscored the significance of supporting groundbreaking therapies: “We are excited to partner with Rejuvenate Bio in advancing RJB-0402, a potential one-time gene therapy for DSP ACM patients. This collaboration underscores our commitment to accelerating promising stem cell and gene therapies to address critical medical needs.”
Editorial Opinion – Funding Impact and Industry Overview:
The $4 million funding injection from CIRM represents more than financial support; it signifies a strategic endorsement of Rejuvenate Bio’s pioneering approach to gene therapy. This investment will enable Rejuvenate Bio to expedite IND-enabling activities and move closer to initiating human clinical trials, crucial steps in bringing RJB-0402 to patients urgently in need.
The field of gene therapy for rare genetic diseases, such as DSP ACM, is rapidly evolving, driven by innovative technologies and increasing recognition of the potential impact on patient outcomes. Rejuvenate Bio’s focus on leveraging gene therapy to modify disease progression rather than merely managing symptoms marks a paradigm shift in cardiovascular medicine. As advancements continue, the collaboration between biotechnology firms and institutions like CIRM will play a pivotal role in accelerating the translation of cutting-edge research into viable therapies.
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