Renova Therapeutics Eyes Expanding Gene Therapy for Heart Failure and Diabetes

Renova Therapeutics, a biotechnology company based in Carlsbad and San Diego, California, has announced new clinical directions for its gene therapy-based treatments aimed at cardiovascular and metabolic diseases. The company's ongoing efforts signal a significant expansion in the therapeutic applications of its gene therapies.

Renova is advancing two main therapies and is set to initiate a phase 2b/3 clinical trial for heart failure and a first-in-human trial for type 2 diabetes, pending regulatory approvals. Notably, the company’s Ucn2 gene therapy shows promise for treating numerous conditions beyond type 2 diabetes, including type 1 diabetes, non-alcoholic fatty liver disease (NAFLD), hypertension, and heart failure.

Preclinical studies have indicated that Ucn2 gene therapy could be effective for type 1 diabetes, which is an autoimmune condition that leads to insulin deficiency. In these studies, a single intravenous injection of AAV8.Ucn2 significantly improved glucose regulation, reduced retinopathy, and lowered mortality from 94% to 18% in mouse models. Additionally, Ucn2 gene transfer demonstrated therapeutic potential in heart failure with both reduced and preserved ejection fraction.

NAFLD, also known as metabolic dysfunction-associated steatotic liver disease (MASLD), is a prevalent cause of liver failure in the United States. In preclinical models of diabetes, Ucn2 gene transfer notably reduced fatty infiltration in the liver within six to eight weeks of administration.

Hypertension, affecting approximately half of the adult population in the U.S., remains a significant risk factor for heart failure, stroke, and kidney failure despite numerous available treatments. Renova's preclinical investigations are exploring the application of Ucn2 gene therapy to effectively manage hypertension.

The company holds exclusive global licenses for key patents on composition and delivery methods as well as clinical data. Moving forward, Renova aims to enhance its intellectual property portfolio, acquire strategic patents, and leverage market exclusivity for its gene therapies as granted by regulatory authorities.

Dr. David Horwitz, a member of Renova's Scientific Advisory Board, emphasized the promising potential of gene therapy in managing diabetes, noting the limitations of current treatments in achieving adequate blood sugar control and preventing complications. Dr. Horwitz brings extensive experience from his previous roles, including Global Chief Medical Officer at the Johnson & Johnson Diabetes Institute and as a former member of the FDA advisory panel in clinical chemistry and toxicology.

Renova’s CEO, Dr. Vijay Mahant, acknowledged the value that Dr. Horwitz brings to the company’s efforts in advancing its gene therapy products and improving patient outcomes globally.

Founded in 2009, Renova Therapeutics focuses on developing definitive, one-time gene therapies to restore health in individuals with chronic diseases. The company’s lead product candidate, RT-100, is designed to enhance heart function in patients with heart failure through a therapeutic gene delivered directly to the heart. Additionally, Renova's product pipeline includes a pioneering gene therapy for type 2 diabetes, currently in the preclinical stage.