Drug intelligence / Profile preview

saposin c

Development stage
Preclinical
Lead developer
Bexion Pharmaceuticals
Modality
Recombinant Proteins and Enzymes
Administration
Intravenous
01

Overview

Saposin C is a lysosomal protein derived from the precursor prosaposin and functions as an essential activator for the enzyme glucocerebrosidase (GCase), which is deficient in Gaucher disease. It facilitates GCase activity by remodeling lysosomal membranes to make lipid substrates accessible for hydrolysis. Saposin C also plays roles in lipid antigen presentation via CD1b molecules and may act as a neuroprotective factor by inhibiting α-synuclein aggregation. Recombinant non-glycosylated Saposin C is being investigated as part of BXQ-350, an experimental anti-neoplastic therapy that combines Saposin C with dioleoylphosphatidylserine (DOPS). Its primary indications under investigation include cancer and neurodegenerative diseases such as Parkinson’s disease[1][2][3][5].

Other names
co-β-glucosidasea1 activatora-1 activatora 1 activatorglucosylceramidase activatorsphingolipid activator protein 2sap-2sap2sap 2
02

Targets

GBA1 (Glucosylceramidase)

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