Batten CLN1 Inhibitor

Batten CLN1 Inhibitor is a small molecule drug candidate being developed by Collaborations Pharmaceuticals for the treatment of CLN1 disease, also known as Infantile Neuronal Ceroid Lipofuscinosis (a form of Batten disease). The drug targets Palmitoyl-protein thioesterase 1 (PPT1), an enzyme whose deficiency leads to the accumulation of palmitoylated proteins and subsequent neurodegeneration. Although CLN1 is a deficiency disease, this candidate is characterized as an inhibitor or modulator of PPT1, potentially serving as a pharmacological chaperone to stabilize the enzyme or acting through a distinct regulatory pathway. It is currently in the preclinical in-vitro stage of development.

Other names

CLN1 inhibitorCLN-1 inhibitorCLN 1 inhibitorPPT1 inhibitorPPT-1 inhibitorPPT 1 inhibitor