Adeno-associated virus, illustration
Credit: Kateryna Kon/Science Photo Library/Getty Images

Gene therapy company Kriya has acquired Tramontane Therapeutics, which is developing such therapies for metabolic and neurodegenerative diseases. Kriya has prioritized Tramontane’s lead program that uses an adeno-associated virus (AAV) vector targeting Fibroblast Growth Factor 21 (FGF2), which has been established as a clinically-validated biological target in Non-alcoholic steatohepatitis (NASH).

“People with NASH are in desperate need of better treatment options and FGF21 is a clinically-validated target for preventing fibrosis in this disease,” said Fátima Bosch, PhD, professor in biochemistry and molecular biology at UAB, co-founder, president and chief scientific advisor of Tramontane Therapeutics, and Kriya scientific advisory board member.

Tramontane uses AAV vectors to express therapeutic proteins against multiple diseases, including NASH, Type 2 diabetes, obesity, Alzheimer’s disease, Parkinson disease, and other disorders associated with impaired cognition and behavior.

With the transaction, Kriya acquires a portfolio of FGF21 assets including Tramontane’s lead program, an AAV vector designed to express a steady level of the native FGF21 protein. FGF21 has beneficial metabolic effects across several target organs including the liver.

The two companies believe that a one-time intramuscular AAV gene therapy that expresses native FGF21 protein is a novel approach to treating NASH, and has significant potential for better efficacy, safety, tolerability, and pharmacokinetic profile than other products in development.

“We are very impressed with the data associated with the Tramontane FGF21 program, which has consistently established strong efficacy and durability across multiple validated animal models of obesity and NASH,” said Shankar Ramaswamy, MD, co-founder and CEO of Kriya.

“The addition of Tramontane’s FGF21 program strategically aligns with our Metabolic Disease portfolio which also includes a one-time gene therapy candidate for insulin-dependent diabetes,” he added.

Kriya’s gene therapy candidate NASH may have the following benefits: Ease of administration, better tissue distribution and receptor binding, continuous FGF21 expression of FGF21, and multiyear efficacy in the setting of a chronic lifelong disease.

“With the ongoing research that continues in this disease, including innovative approaches like gene therapy, the future outlook for NASH treatments has markedly improved. I believe that a one-time gene therapy would be a quantum leap forward in the management of this chronic disease,” said Bosch.

NASH is the most severe form of nonalcoholic fatty liver disease and affects approximately 17 million people in the United States. It is a condition in which the liver builds up excessive fat deposits causing inflammation and liver cell damage. NASH can progress to more serious disease stages, such as fibrosis, cirrhosis, liver failure, or liver cancer.

Approximately 20 percent of people with NASH will progress to advanced liver disease including cirrhosis and cancer over several years or decades; in advanced stages of the disease, liver transplant may be the only option. There are currently no FDA-approved therapies for halting or reversing the progression of NASH.

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