PPT1 replacement therapy

PPT1 replacement therapy is a preclinical small molecule program developed by Palm Therapeutics for the treatment of CLN1 disease (Neuronal Ceroid Lipofuscinosis Type 1). CLN1 is a devastating lysosomal storage disorder caused by a deficiency in the enzyme palmitoyl-protein thioesterase-1 (PPT1), which is responsible for removing long-chain fatty acids from proteins. The absence of PPT1 leads to the toxic accumulation of palmitoylated proteins, resulting in progressive neurodegeneration. This therapeutic approach utilizes small molecules to restore or mimic PPT1 activity, offering a potential alternative to traditional protein-based enzyme replacement therapies.

Other names

PPT1 small molecule therapyPPT-1 small molecule therapyPPT 1 small molecule therapyCLN1 small molecule therapyCLN-1 small molecule therapyCLN 1 small molecule therapy