Science for Health keynote: Interview with gene therapy pioneer Katherine High

The 2022 edition of Science for Health, co-sponsored by MEDVIA, focuses on gene therapy. Dr Katherine High will open the event on 21 November in Brussels with an overview of the field and how it is evolving.

Katherine High has contributed to a long list of firsts in the field of gene therapy. As President of Spark Therapeutics, she led the team that developed Luxturna ­– the first FDA-approved gene therapy in the world and the first to be approved in both the US and Europe. Prior to that, her trailblazing clinical work with adeno-associated viral (AAV) vectors helped paved the way for many subsequent gene therapies.

Today, Dr High continues her cutting-edge work as president of therapeutics at AskBio, a wholly owned subsidiary of Bayer AG. MEDVIA partner BioVox spoke with Dr High about the evolution of this fascinating field, which has the potential to provide transformational therapies for so many people with otherwise untreatable genetic diseases.

Let’s start with the basics: What is gene therapy?

Dr High: “Fundamentally, gene therapy treats or prevents genetic diseases by correcting problems in a person’s DNA. Often this is done by delivering a healthy version of a missing or defective gene into the cells in a specific type of tissue, such as the liver or the retina. One of the most common delivery methods is to use a viral vector to insert the healthy gene into the target cell.”

See the rest of this interview at BioVox

Science for Health takes place online and in Brussels on 21 November. See the full program and sign up here