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Allosteric Bioscience is developing a first-in-class small molecule inhibitor of GCPII to treat sarcopenia and age-related muscle loss by preserving muscle mass and function. “Statins transformed cardiovascular disease by targeting the cholesterol pathway. Allosteric targets GCPII, a key regulator of muscle metabolism and degeneration.

Research at Johns Hopkins University identified GCPII as a key regulator of metabolic signaling involved in muscle preservation and degeneration. Preclinical studies show that GCPII inhibition preserves muscle mass and function and improves survival by approximately 20% in mouse aging models. Sarcopenia affects hundreds of millions worldwide and has no approved therapies targeting its underlying biology. Allosteric is advancing lead GCPII inhibitors through optimization toward IND-enabling studies and first-in-human clinical trials.

The scientific foundation of the program is based on research conducted in Dr. Barbara Slusher’s laboratory at Johns Hopkins University. In this work, GCPII was identified as a key regulator of metabolic pathways involved in muscle preservation. Experimental studies show that pharmacologic inhibition of GCPII preserves muscle mass and function and improves survival in aging models. These findings support development of small-molecule GCPII inhibitors as a therapeutic strategy to prevent or slow muscle loss. Allosteric holds an exclusive global license from Johns Hopkins University and is advancing the program under a Sponsored Research Agreement with the originating laboratory.

Loss of skeletal muscle mass and strength is a central driver of aging-related decline, leading to frailty, falls, hospitalization, loss of independence, and increased mortality. Sarcopenia is one of the largest untreated conditions in aging. There are no approved therapies that target the biological mechanisms of muscle degeneration. Demand for therapies that preserve muscle biology is increasing as global populations age. Furthermore, the adoption of GLP-1-based therapies for obesity and metabolic disease has highlighted new challenges, such as reductions in lean muscle mass alongside fat loss, driving heightened interest in agents that can preserve muscle biology during weight loss and aging.

Allosteric is developing small-molecule GCPII inhibitors to restore metabolic signaling involved in muscle preservation. Multiple lead compounds are being optimized in collaboration with the Johns Hopkins research team, with backup molecules supporting candidate selection.

Allosteric holds an exclusive global license from Johns Hopkins University covering the GCPII pathway technology underlying its sarcopenia and muscle-loss program. Development is supported through a Sponsored Research Agreement with the originating laboratory. Additional patent filings are in preparation to strengthen protection around GCPII inhibition for muscle degeneration.

Allosteric was founded by repeat biotechnology entrepreneurs and physician-scientists with deep experience in drug discovery, company building, capital formation, and strategic transactions.

Molecular geneticist and serial biotechnology entrepreneur with more than three decades of experience in biomedical research, drug development, and company formation

Entrepreneur and investor with decades of experience in company and capital formation, as well as strategic transactions

Physician-scientist with expertise in oncology, inflammation biology, and metabolic disease

Sarcopenia affects hundreds of millions globally and significantly contributes to healthcare costs associated with hospitalization, fractures, long-term care, and loss of independence. Interest in therapies that preserve muscle biology is rising alongside demographic aging and the growth of longevity medicine. The GCPII pathway may also offer relevance in other muscle-wasting conditions, including ALS and cancer cachexia, creating further opportunities for indication expansion.