BREAKING: Last August, scientists secretly began creating a custom #CRISPR therapy to fix a mutation responsible for one baby’s debilitating disease. He was treated in February and doing better. My deep dive in Endpoints News has the details. endpts.com/philadelphia-d…

CRISPR is used in landmark treatment to correct genetic misspelling of a single patient statnews.com/2025/05/15/cri… via Jason Mast

In a medical milestone, a customized base editor was developed, characterized in human and mouse cells, tested in mice, studied for safety in non-human primates, cleared by U.S. FDA for clinical trial use, manufactured as a complex with an LNP, and dosed into a baby with a severe,

In case you needed a smile this Tuesday: 10-month-old baby KJ, who received a bespoke gene-editing therapy after a six month scientific sprint - "graduated" from the hospital today and went HOME! YAY SCIENCE! Here's his story: wapo.st/451ZQjT

🧬 FDA Cell & Gene Therapy Roundtable: A Story of Frustration, Urgency, and Hope 🧵 Today, something rare happened. The FDA, HHS, NIH, CMS, patients, founders, and investors sat down together, not to check a box, but to fix what’s broken. And if you’ve ever felt like the FDA

Another inspiring update on the KJ story We say that base editing is “programmable” — that is just another way of saying it is both “customizable” and “predictable” Progress in gene editing will make more outcomes like this possible

Thursday morning, I had the privilege of speaking with the Muldoon family, whose 10-month-old son, baby KJ, was discharged Wednesday from Children's Hospital. Baby KJ was diagnosed with a rare urea cycle disorder and received a first-of-its-kind personalized CRISPR gene-editing

Beam announced new safety & efficacy data from its BEACON Phase 1/2 clinical trial of BEAM-101 in patients with sickle cell disease with severe vaso-occlusive crises, which will be shared this afternoon in a poster presentation at the EHA2025 Congress.

Congrats Sek, Verve Therapeutics, and Eli Lilly and Company ! A powerhouse combination in cardiovascular health:

What if science could completely change the treatment of cardiovascular disease—from chronic care to a one-and-done treatment? Today we officially acquired Verve Therapeutics, expanding our pipeline of cardiometabolic health medicines. e.lilly/4kTgoP9