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
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.
Thursday morning, I had the privilege of speaking with the Muldoon family, whose 10-month-old son, baby KJ, was discharged Wednesday from @ChildrensPhila. Baby KJ was diagnosed with a rare urea cycle disorder and received a first-of-its-kind personalized CRISPR gene-editing…
🧬 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
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
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
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 @US_FDA for clinical trial use, manufactured as a complex with an LNP, and dosed into a baby with a severe,…
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 @endpts has the details. endpts.com/philadelphia-d…
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