Ensuring appropriate delivery of the correct replacement gene to the necessary cells without excessive toxicity is critical, according to Foust.
"It is the first question we think about, at least on the preclinical side: How are we going to get this to the patient? It factors into your capsid that you choose. It factors into the amount of material that you'll need in terms of dosing," Foust told ScienceBoard.net at the American Society of Gene & Cell Therapy (ASGCT) 2022 annual meeting in Washington, DC. "Delivery is really everything."
Watch the video below to learn more.