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.

Next evolution of CAR T-cell therapy is solid tumors
While chimeric antigen receptor (CAR) T-cell therapy has become a game changer for some patients with blood cancer, the next area of evolution for the...

11 new developments in cell and gene therapy
There have been a number of recent developments in the cell and gene therapy field. Bruce Carlson, publisher of Cell and Gene Therapy Business Outlook,...

New gene therapy platform shows potential against certain muscle diseases
Scientists have engineered a new family of adeno-associated viruses (AAVs) to deliver therapeutic genes to muscle tissue at 100 to 250 times lower doses...

Jaguar Gene Therapy nabs $139M to develop AAV9-based gene therapies
Jaguar Gene Therapy has closed $139 million in funding to advance its preclinical pipeline of adeno-associated virus 9 (AAV9)-based gene therapies.

Jaguar Gene Therapy launches, targeting rare genetic diseases
Jaguar Gene Therapy has launched after being in stealth mode with the goal of accelerating the development, manufacturing, and commercialization of novel...