Thursday, September 07, 2023
Kriya Therapeutics, a biopharmaceutical company specializing in gene therapies for prevalent medical conditions, has announced its acquisition of Tramontane Therapeutics, Inc. Tramontane, previously a private gene therapy firm with a focus on metabolic and neurodegenerative diseases, originated from Universitat Autònoma de Barcelona (UAB) and now operates as a wholly-owned subsidiary under Kriya.
This acquisition grants Kriya access to a range of assets centered around Fibroblast Growth Factor 21 (FGF21), including Tramontane's flagship program. This program utilizes an adeno-associated virus (AAV) vector engineered to consistently express the native FGF21 protein, which has demonstrated positive metabolic effects in various target organs, especially the liver. FGF21 has established itself as a clinically-validated biological target for Nonalcoholic Steatohepatitis (NASH), a condition Kriya has prioritized as its primary FGF21 program.
Both organizations share the belief that a one-time intramuscular AAV gene therapy, designed to express the native FGF21 protein, represents an innovative approach to NASH treatment, potentially offering enhanced efficacy, safety, tolerability, and pharmacokinetic profiles compared to other products in development.
Shankar Ramaswamy, M.D., Co-Founder and CEO of Kriya, expressed enthusiasm about the Tramontane FGF21 program's data, highlighting its consistent efficacy and durability across multiple validated animal models of obesity and NASH. Additionally, he underscored how this addition strategically aligns with Kriya's Metabolic Disease portfolio, which includes a one-time gene therapy candidate for insulin-dependent diabetes.Administering the therapy intramuscularly as a one-time procedure eliminates the need for complex medication regimens, which can enhance patient adherence and convenience.The utilization of native FGF21, devoid of artificial modifications often present in analogs, could improve the distribution of the protein within tissues and its binding to receptors, potentially enhancing its therapeutic effectiveness.The continuous production of FGF21 by the gene therapy ensures a steady and predictable supply of the protein. This contrasts with the variability in pharmacokinetics associated with recurrent injections of recombinant analog products, potentially leading to more reliable treatment outcomes.The gene therapy's potential for providing long-term effectiveness could offer sustained benefits over an extended period. This feature is particularly valuable for managing chronic, lifelong conditions like NASH.
Fátima Bosch, Ph.D., Professor in Biochemistry and Molecular Biology at UAB, Co-Founder, President, and Chief Scientific Advisor of Tramontane Therapeutics, as well as a Kriya Scientific Advisory Board Member, highlighted the pressing need for improved treatment options for those suffering from NASH. She emphasized that FGF21 has been clinically validated as a target for preventing fibrosis in this disease. Dr. Bosch expressed optimism about the ongoing research efforts, including innovative approaches such as gene therapy, which have significantly enhanced the outlook for NASH treatments. She firmly believes that a one-time gene therapy could represent a substantial leap forward in managing this chronic condition.
NASH represents the most severe form of nonalcoholic fatty liver disease (NAFLD) and affects an estimated 17 million individuals in the United States. It occurs when the liver accumulates excessive fat deposits, leading to inflammation and damage to liver cells. NASH can progress to more severe disease stages, including fibrosis, cirrhosis, liver failure, or even liver cancer. Approximately 20 percent of individuals with NASH will advance to advanced liver disease stages, such as cirrhosis and cancer, over several years or decades. In advanced stages of the disease, a liver transplant may become the only viable option. Currently, there are no FDA-approved therapies available for halting or reversing the progression of NASH.