RT-200 (Urocortin 2 gene transfer)
RT-200 for type 2 diabetes is a first-of-its-kind IV-injected, single-dose investigational paracrine gene therapy designed to provide substantially improved, sustained outcomes in type 2 diabetes patients by normalizing blood glucose, supported by:
- Increased insulin sensitivity and glucose disposal
- Long-lasting resolution of abnormal glucose homeostasis
- Reduction in fatty infiltration of the liver
RT-200 possesses advantages over current diabetes medications: It is insulin-sensitizing, circumvents the need for daily insulin injections, and reduces weight gain and fatty infiltration of the liver.
How it works
RT-200 delivers a therapeutic gene – urocortin 2 – using a single intravenous injection, a common procedure that can be administered by medical professionals in any doctor’s office. The technology has been shown to safely and efficiently deliver the urocortin 2 gene therapy in mice. The human gene encoding urocortin 2 is delivered via an adeno-associated virus (AAV) that is able to enter cells – primarily in the liver – but cannot reproduce itself.
Preclinical data indicate that gene transfer, via a one-time intravenous injection of a viral vector encoding urocortin 2, increases insulin sensitivity and glucose disposal, provides long-lasting resolution of abnormal glucose homeostasis, and reduces fatty infiltration of the liver in two mouse models of insulin resistance. These findings indicate a promising strategy for treating clinical type 2 diabetes and unlock the potential for treatment of other cardiovascular and metabolic diseases, including nonalcoholic fatty liver disease (nonalcoholic steatohepatitis – NASH). In similar protocols in which mice were dosed initially prior to being fed a high-fat diet, treated mice did not develop type 2 diabetes in their lifetime – signaling a potential for a preventative therapy.
Once all preclinical studies with urocortin 2 gene transfer are completed, Renova Therapeutics plans to submit an Investigational New Drug application with the U.S. Food and Drug Administration in which patients with type 2 diabetes are enrolled to receive RT-200 in well-controlled and safely conducted clinical trials.
Renova Therapeutics is studying RT-200 for the treatment of type 2 diabetes, the first indication for which we will be seeking approval.