Drug Repurposing for Rare Metabolic Diseases

Cori Disease and SRD5A3-linked Congenital Disorder of Glycosylation (CDG) are two rare and fatal metabolic disorders for which there are no cures or therapies. Both conditions result in early-onset developmental delays, greatly hinder quality of life, and result in a rapid degeneration of motor functions. Due to their rarity, research into these conditions is lagging and therapeutic strategies focus solely on symptom management. Therefore, there is an urgent need to develop and assess new therapeutics for these incurable diseases. Modelis’ mission is to accelerate drug discovery for human genetic diseases and they are investing a large portion of their activities to drug repurposing. The current project will allow us to rapidly identify already-approved drugs for Cori Disease and SRD5A3-CDG using simple worms models of the disease in order to translate them quickly to the clinic

Faculty Supervisor:

Pierre Drapeau;Jodey Alexander Parker

Student:

Partner:

Modelis inc.

Discipline:

Life Sciences

Sector:

Professional, scientific and technical services

University:

Université de Montréal

Program: