PO-001

PO-001 is an investigational oral sustained-release formulation of cysteamine bitartrate developed by Patient One for the treatment of cystinosis, a rare autosomal-recessive lysosomal storage disease. Cystinosis is caused by mutations in the CTNS gene, which encodes the lysosomal cystine transporter cystinosin, leading to toxic intracellular cystine accumulation. Cysteamine bitartrate acts as a cystine-depleting agent; it enters the lysosome and reacts with cystine to form cysteine and a cysteine-cysteamine mixed disulfide. This disulfide can then exit the lysosome via the PQLC2 transporter, effectively bypassing the defective cystinosin pathway. Unlike existing immediate-release (Cystagon) or delayed-release (Procysbi) formulations, PO-001 utilizes a non-pH-dependent coating to achieve a slow, time-dependent release throughout the gastrointestinal tract, aiming to reduce peak-trough variability in plasma concentrations and improve patient tolerability.

Other names

cysteamine bitartrate sustained-releasesustained-release cysteamine bitartrate