Behind the Headlines, Episode 17: Bespoke CRISPR Therapy, Executive Order Fallout, and More

Behind the Headlines is a bi-weekly panel discussion examining the latest trends, readouts, and other factors that drive pharmaceutical news and innovation. Each episode features consultants, venture capitalists, scientists, patient advocates, and journalists discussing the prior weeks’ top news while seeking to highlight the more enduring lessons that hide behind the headlines.

In episode 17, we’re joined by panelists Jamie Baumgartner, President and CEO of Keystone Symposia on Molecular & Cellular Biology; Jonathan D. Grinstein, PhD, North American Editor for Inside Precision Medicine; and John Wilkerson, Washington correspondent for STAT News. The panelists dove into the beating heart of the life science ecosystem, discussing why researchers are currently attending conferences, what knowledge they are sharing, and what gaps in their knowledge they are looking to fill.

Of note, the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, which was recently held in New Orleans, was the chosen vehicle to announce a world first in personalized CRISPR therapeutics. Scores of people have already benefited from receiving CRISPR-based therapies for genetic conditions such as sickle cell disease. In this case, collaboration among clinicians, scientists, and physicians helped create a custom-tailored therapy to address an infant with CPS1 deficiency. “This compromised his ability to process the nitrogen-containing compounds produced when the body breaks down protein. As a result, his blood had high levels of ammonia, a compound that is particularly toxic to the brain,” according to a news briefing from Nature. (1) “In just six months, Muldoon received his first dose. After that initial dose, Muldoon could safely eat the amount of protein recommended for his age.” (1). Although a big success, the panel considered what the probabilities are for cost lowering, alongside widespread access deployment.

Policy changes at the Department of Health and Human Services (HHS), funding cuts, and tariffs were also discussed, with Sanofi following suit by announcing a 5-year, $20 billion investment into US manufacturing (2). However, the “most favored nation” proposed executive order caused Roche to announce it is beginning to rethink its own $50 billion investment announcement a week earlier (3, 4). 

References

1. Ledfod, H. World’s first personalized CRISPR therapy given to baby with genetic disease. Nature. May 15, 2025. https://www.nature.com/articles/d41586-025-01496-z

2. Sanofi to invest at least $20 billion in the US through 2030, growing investments in science and expanding domestic manufacturing. Press release. Sanofi. Published May 14, 2025. https://www.prnewswire.com/news-releases/sanofi-to-invest-at-least-20-billion-in-the-us-through-2030-growing-investments-in-science-and-expanding-domestic-manufacturing-302455566.html

3. Roche warns that it needs to reconsider its U.S. investments due to Trump's drug pricing order. Bloomberg. May 16, 2025. https://www.bloomberg.com/news/articles/2025-05-14/roche-warns-us-investments-at-risk-from-trump-drug-price-plans?embedded-checkout=true

4. Franco, M. Trump Administration Revives Most-Favored-Nation Drug Pricing: Here's What to Know. Holland and Knight. May 14, 2025. https://www.hklaw.com/en/insights/publications/2025/05/trump-administration-revives-most-favored-nation-drug-pricing