Compounds under development for the treatment of mucopolysaccharidoses

Project Portfolio

Dorphan is developing DO-1, a preclinical chaperone drug candidate for the treatment of GM1-gangliosidosis and mucopolysaccharidosis type IVB, both of which are orphan indications where the beta-galactosidase enzyme is mutated.

DO-1 is a promising pharmacological chaperone molecule, which selectively and potently restores the beta-galactosidase activity in patient cells for at least two thirds of the mutations tested. DO-1 possesses key drug-like properties for the targeted indications, including:

  • Brain and bone penetration
  • Oral bioavailability

The compound is currently under preclinical development to assess its activity and safety prior to initiation of clinical development.