Dear Science Advisory Board Member,

The American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting was held this week in Washington, DC, demonstrating how far the field has come in the last quarter of a century. ASGCT 2022's scientific, educational, and plenary sessions all drove home the potential of new cell and gene therapy (CGT) treatments for a wide array of diseases.

President Joe Biden's acting scientific advisor, Francis Collins, MD, PhD, who was awarded ASGCT's inaugural Founder's Award, highlighted recent CGT successes at the meeting but also took the opportunity to call for greater progress in making in vivo genome editing scalable to treat a lot more patients.

Many disorders "won't be managed by an ex vivo therapy approach," said Collins, the former NIH director and head of the groundbreaking Human Genome Project. "Why in vivo? Why gene editing?" he asked. "There are thousands of rare diseases. I want to find something that's scalable." Collins also emphasized that "delivery is the real challenge" and that a "zip code system for human tissues" is needed.

The delivery challenge was also on the mind of Kevin Foust, PhD, senior vice president for preclinical and translational medicine at Jaguar Gene Therapy, who spoke with ScienceBoard this week during ASGCT 2022. "Delivery is really everything," Foust said. "It is the first question we think about, at least on the preclinical side: How are we going to get this to the patient?"

Thomas McCauley, PhD, chief scientific officer at Omega Therapeutics, discussed with ScienceBoard how the precision genomic medicine company is looking to harness "nature's operating system" for controlling gene expression and cell differentiation. "We believe this is applicable across the board really, certainly in monogenic diseases, but also importantly in complex multigenic diseases," McCauley said.

Sangamo CEO Sandy Macrae, PhD, told us that 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 technology is solid tumors. If the industry is successful with creating allogeneic forms of the therapy "that could be given off-the-shelf" to patients urgently, then cancer could eventually become a "chronic disease of living," Macrae said.

Watch next week for more video interviews from ASGCT 2022 with the researchers, clinicians, and companies who are advancing the CGT field.

Best wishes,

Greg Slabodkin

Editor in Chief

editor@scienceboard.net