Development of selective nanomolar cyclic peptide ligands as GBA1 enzyme stabilisers.

ABSTRACT: The stabilisation of recombinant glycosidases by exogenous ligands, known as pharmacological chaperones or enzyme stabilisers, has recently garnered great clinical interest. This strategy can prevent enzyme degradation in the blood, reducing required dosages of recombinant enzyme and extending IV injection intervals, thereby reducing side effects, improving patient lifestyles and treatment costs. While this therapeutic approach has been successfully implemented for treating Pompe and Fabry diseases, clinical studies for Gaucher disease using chaperones alone or in combination with enzyme replacement therapy (ERT) have been limited, and no small molecule chaperones have yet been approved for this condition. Developing such therapies requires selective and effective reversible GBA1 ligands.

SUBMITTER: Katzy RE 

PROVIDER: S-EPMC11808397 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature