Fractyl Health today announced positive preclinical proof-of-concept data in an animal model for its gene therapy delivery platform for treating type 2 diabetes.
The company, which develops the Revita diabetes reversal procedure, evaluated its Rejuva program. Rejuva, a novel device and AAV-mediated delivery platform, delivers gene therapy directly targeting the pancreas. This potentially embeds multiple different pharmacologies to address serious metabolic diseases, including type 2 diabetes and obesity.
According to a news release, Fractyl presented data at the World Congress of Insulin Resistance, Diabetes & Cardiovascular Disease (WCIRDC) in Los Angeles last week.
“Today’s Rejuva program preclinical results represent the first demonstration of sustained, dose-dependent efficacy of a gene therapy targeting the GLP-1 receptor pathway in the pancreas in a rodent model of type 2 diabetes and obesity,” said Dr. Harith Rajagopalan, co-founder and CEO of Fractyl Health. “Results show that a single dose islet-targeted GLP-1 receptor agonist gene therapy candidate can induce sustained production of the transgene in pancreatic islets, delay disease progression, improve insulin production, and reduce blood glucose levels for up to ten weeks of follow-up.”
More on the Rejuva system
Rejuva encompasses both AAV-delivered transgenes and device-based delivery systems. Data in the current study include dose-dependent in vivo pharmacology and efficacy data. It covers an AAV-mediated gene therapy of a GLP-1 receptor agonist candidate in a db/db rodent model of type 2 diabetes.
Fracytl plans to present further large animal procedure data for Rejuva in 2023. The company called it “encouraging” feasibility data.
Rajagopalan said the local delivery devices and methods could “potentially enable pancreatic gene therapy to become a reality for the first time.”
“In many gene therapy programs, local delivery is demonstrating the potential to reduce viral load and associated risk of systemic administration, and we believe the same could be true for the pancreas,” said Rajagopalan. “Taken together, our Revita and Rejuva programs have the potential to address root cause pathology in two organs that are diseased in T2D: the gut and the pancreas.”