CA77.1

CA77.1 is a small molecule activator of chaperone-mediated autophagy (CMA), a selective lysosomal degradation pathway. CMA activity naturally declines with age, leading to the accumulation of damaged proteins and cellular dysfunction, which is a hallmark of various age-related pathologies. CA77.1 functions by increasing the levels or stability of the lysosome-associated membrane protein type 2A (LAMP2A), the rate-limiting receptor for CMA on the lysosomal membrane. By restoring CMA activity, CA77.1 facilitates the clearance of proteotoxic aggregates and maintains cellular proteostasis. It has demonstrated efficacy in preclinical models of age-related macular degeneration (AMD), where it restores the function of retinal pigment epithelium (RPE) cells, and is being investigated for its potential in treating neurodegenerative disorders and other conditions associated with protein misfolding.

Other names

CMA activator CA77.1