Colin Ross

BSc, PhD, MSc

Investigator, BC Children's Hospital
Associate Dean, Research, Faculty of Pharmaceutical Sciences

The goal of my research is to develop new treatments for patients with rare diseases. One strategy that we are working on is to develop new gene therapies. Another approach that we are working on is to understand why certain patients respond positively to a medication, while others receive no benefit, or worse, develop debilitating or life-threatening adverse drug reactions (ADRs) to the same dose of medication. We work collaboratively with clinicians and researchers from across Canada and around the world.

1. We successfully developed the first gene therapy to achieve regulatory approval. That work has led to many new gene therapies coming onto the market and many more in development. We are now working on several approaches to reduce the high costs of gene therapies by developing more efficient products and improved manufacturing.

2. We are developing new approaches for gene therapy that utilize genome editing for improved effectiveness and reduced costs.

3. We have recruited patients that have suffered specific serious ADRs, as well as control patients that have recived the same drugs without ADRs. We use high-throughput genomics and next-generation DNA sequencing technologies to identify the genetic factors of severe ADRs of key clinical importance.

The research involves many approaches from statistical, computational and molecular genetics, to molecular biology, and cell biology. The program’s mission is to translate these findings into new diagnostics and therapeutics that will improve the quality of lives of patients and families.