Assessment of a drug candidate for the treatment of two rare orphan diseases: Morquio type B disease and GM1 gangliosidosis

• Research unit: INNOSUISSE
• Project number: 16639.1 PFLS-LS

Short description (German)

Assessment of a drug candidate for the treatment of two rare orphan diseases: Morquio type B disease and GM1 gangliosidosis

Short description (English)

Assessment of a drug candidate for the treatment of two rare orphan diseases: Morquio type B disease and GM1 gangliosidosis

Abstract (German)

Morquio type B disease and GM1 gangliosidosis are two rare orphan genetic disorders caused by mutated forms of the enzyme beta-galactosidase. A selective beta-galactosidase inhibitor was identified as a potential rescuer of the mutated enzyme as it helps the folding of the enzyme in vitro. The current project proposes to synthesize this potent drug with improved routing, to measure its efficacy on patient cells and to assess its in vivo bioavailability.

Abstract (English)