Renova Therapeutics Announces Potential Expansion of Gene Therapy Platform for Heart Failure, Diabetes and Related Conditions

Renova is exploring new therapeutic opportunities that have the potential to expand the use of Renova’s gene therapy platform to include several pathologies associated with diabetes and heart failure.

Renova Therapeutics, a biotechnology company developing gene therapy-based treatments for cardiovascular and metabolic diseases, announced today current and potential future clinical directions that can expand the use of its gene therapies to treat additional debilitating diseases.

Renova is pushing ahead with its two most advanced therapies and is poised to launch a phase 2b/3 clinical trial for heart failure (HF) and a first-in-human trial for type 2 diabetes (T2DM) upon approval by regulatory agencies.

In addition to its potential to treat type 2 diabetes as demonstrated in preclinical studies, Renova’s Ucn2 gene therapy has potential therapeutic applications for type 1 diabetes, non-alcoholic fatty liver disease (NAFLD) (also known as metabolic dysfunction–associated steatotic liver disease (MASLD)), hypertension and heart failure.


Non-alcoholic fatty liver disease (NAFLD), for example, which is a common cause of liver failure in the U.S.,[1] was present in models of diabetes in the preclinical studies. Ucn2 gene transfer significantly reduced fatty infiltration six to eight weeks after a single intravenous delivery of the therapeutic product.

Type 1 Diabetes

Preclinical data demonstrated that a one-time intravenous injection of AAV8.Ucn2 has beneficial effects in a murine model of insulin deficiency analogous to type 1 diabetes. Beneficial effects included marked improvement in abnormal glucose flux, reduced retinopathy, and reduction in mortality from 94% to 18% (p<.0001). In addition, published preclinical studies have shown the usefulness of Ucn2 gene transfer in heart failure with reduced ejection fraction (EF) and heart failure with preserved EF.

Renova received an NIH STTR Award to investigate the use of Ucn2 gene transfer for type 1 diabetes and associated left ventricular dysfunction.


Hypertension is a major risk factor for both heart failure, stroke and kidney failure leading to dialysis. In 2020, about half of U.S. adults — 122 million patients — had hypertension (high blood pressure).[2] Despite many anti-hypertensive drugs on the market, hypertension continues to contribute to the high incidence of these conditions. These data underscore the challenge of managing hypertension effectively, providing a rationale for gene therapy for this pervasive and difficult to treat disease. Preclinical studies are investigating the use of Ucn2 gene therapy for hypertension.

Renova holds worldwide, exclusive licenses to key patents covering composition matters and delivery methods as well as clinical data. The Company intends to further develop its internal intellectual property (IP), acquire strategic IP, and utilize market exclusivity for its gene therapies granted by the regulatory agencies.

“Despite the many therapies we now have for diabetes, we still see too many patients with inadequate blood sugar control and frequent complications of diabetes. Gene therapy is clearly one of the most promising new approaches to reducing the impact of diabetes,” said Dr. David Horwitz (MD, PhD, MBA), member of Renova’s Scientific Advisory Board. Dr. Horwitz previously served as Global Chief Medical Officer at the Johnson & Johnson Diabetes Institute and is a former member of the FDA advisory panel in clinical chemistry and toxicology.

“Renova is privileged to have Dr. Horwitz as part of its Scientific Advisory Board. His expertise in health care and wealth of knowledge in metabolic diseases is a great asset to the Company in advancing its products and helping patients worldwide,” said Renova CEO Dr. Vijay Mahant.

About Renova Therapeutics

Renova Therapeutics is developing definitive, one-time gene therapies to restore the health of people suffering from chronic diseases. The company is focusing on gene therapy treatments for heart failure and type 2 diabetes, two of the most common and devastating chronic diseases in the world. The company’s lead product candidate, RT-100, is a treatment that delivers a therapeutic gene directly to the heart during a routine outpatient procedure and has the potential to increase heart function in millions of patients with heart failure. The Company’s product pipeline also includes a groundbreaking gene therapy in preclinical stage for sufferers of type 2 diabetes. Renova Therapeutics was founded in 2009 and is led by an experienced management team in biopharmaceuticals and gene therapy. For additional information about the company, please visit

DISCLOSURE NOTICE: The information contained in this release is as of June 10, 2024. Renova Therapeutics assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

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[1] Cotter TG, Rinella M. Nonalcoholic Fatty Liver Disease 2020: The State of the Disease. Gastroenterology. 2020 May;158(7):1851-1864. doi: 10.1053/j.gastro.2020.01.052. Epub 2020 Feb 13. PMID: 32061595.

[2] Martin et. al. 2024 Heart disease and stroke statistics: a report of U.S. and global data from the American Heart Association. Circulation. Published online January 24, 2024. doi: 10.1161/CIR.0000000000001209.

Source: Renova Therapeutics, Inc.