Scientific and Clinical Hub for Orphan Drug Development

With rapid and cost-effective genome sequencing becoming the norm, many causal mutations for inherited genetic diseases are being rapidly determined. The discovery of new genes for inherited diseases is enabling rapid genetic and chemical genetic platforms to be used to discover drug targets and drugs/drug-like molecules as potential treatment options for patients with inherited diseases. This project is focusing on the identification of compounds that could potentially treat human genetic diseases, with a focus on childhood blinding disorders, and muscular dystrophies – the expertise of the industrial partner AGADA Biosciences. The research will identify novel drugs/drug-like molecules for the treatment of patients suffering from these inherited diseases.

Faculty Supervisor:

Christopher McMaster


Taryn Reid


AGADA Biosciences


Pharmacy / Pharmacology


Medical devices




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