Intermediate scale manufacturing and in vivo therapeutic assessment of a promising drug candidate for the treatment of GM1-gangliosidosis

• Research unit: INNOSUISSE
• Project number: 25253.1 PFLS-LS

Short description (German)

Intermediate scale manufacturing and in vivo therapeutic assessment of a promising drug candidate for the treatment of GM1-gangliosidosis

Short description (English)

Abstract (German)

GM1-gangliosidosis is a rare orphan genetic disease caused by mutated forms of the lysosomal enzyme beta-galactosidase. DO-1 is a chaperone drug candidate that has been shown to rescue the defective enzyme activity in ex vivo patient cells. The current project proposes to perform critical steps in the preclinical development of DO-1, consisting in the set-up of its intermediate scale production route and the assessment of its therapeutic efficacy in a mouse model.

Abstract (English)