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PCS-01
- Development stage
- Discontinued
- Lead developer
- Proclara Biosciences
- Modality
- Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
PCS-01 (formerly known as NPT189) is a recombinant fusion protein developed by Proclara Biosciences for the treatment of systemic amyloidosis. It utilizes the company's proprietary General Amyloid Interaction Motif (GAIM) technology, which is derived from the gene 3 protein (g3p) of the M13 bacteriophage. GAIM is designed to recognize and bind to the common cross-beta sheet structure shared by various misfolded protein aggregates, regardless of the specific protein involved. PCS-01 is a pan-amyloid disruptor that targets multiple misfolded proteins, including immunoglobulin light chains (AL), transthyretin (ATTR), amyloid-beta, tau, and alpha-synuclein. By binding to these aggregates, the drug promotes their remodeling into non-toxic forms and facilitates their clearance by the immune system. PCS-01 was primarily investigated for systemic amyloidosis indications, reaching Phase 1 clinical trials before development was stalled following the failure of Proclara's lead candidate, NPT088, in Alzheimer's disease.
- Other names
- NPT 189NPT189NPT-189
02
Targets
TTR amyloid fibril (Transthyretin amyloid fibril)Tau aggregates (Microtubule-associated protein tau (pathological form))AL (Amyloid light-chain protein)aggregated α-syn (Alpha-synuclein (α-syn))Amyloid-beta aggregate (N-terminal epitope)
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