CPI-601

CPI-601 is a recombinant human palmitoyl-protein thioesterase-1 (rhPPT1) enzyme replacement therapy (ERT) being developed by Collaborations Pharmaceuticals for the treatment of CLN1 Batten disease. CLN1 is a fatal, ultra-rare pediatric neurodegenerative disorder caused by mutations in the CLN1 gene, which leads to a deficiency in the PPT1 enzyme and subsequent accumulation of palmitoylated proteins. CPI-601 is designed to restore enzymatic activity in the central nervous system. Preclinical studies in mouse and sheep models have demonstrated that intracerebroventricular (ICV) administration of CPI-601 can reduce motor dysfunction and nerve cell death. The therapy has received FDA Orphan Drug and Rare Pediatric Disease designations and is currently in IND-enabling stages.

Other names

Batten CLN1 ERTrecombinant human palmitoyl-protein thioesterase-1rhPPT1rhPPT-1rhPPT 1