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…
53K Followers 736 FollowingEarly stage biotech VC. Recovering scientist. Opinions expressed are solely my own and do not express the views or opinions of Atlas Venture.
6K Followers 278 FollowingEIR @GVteam, dreamer, inventor of ABE. Interested in genomic medicine and next-generation application of gene editing tools. Leonardo’s & Isabella’s Mom.
14K Followers 498 FollowingYour source for the latest #Science and #Biotech news. If you're interested in #CRISPR #GeneEditing hit follow! Spaces podcast host🎙- all 28 episodes ⬇️
16K Followers 506 FollowingGene editor + Professor Molecular Therapeutics @berkeleyMCB + Director for Technology and Translation @igisci + @danahercorp Beacon for CRISPR Cures.
30K Followers 989 FollowingCEO @VerveTx $VERV; Imagine if a one-time treatment led to permanent lowering of LDL cholesterol: that’s what we are developing to treat heart attack
25K Followers 769 FollowingScientist, entrepreneur, ex-biopharm exec, former FM radio jock, lifelong hockey fan, aspiring guitar player, #immigrant. CEO @ArrakisTx. Just @gilman on...
318K Followers 2K FollowingPublishing the best of biotech science and business. Find us on Bluesky, Facebook & Instagram. Part of @SpringerNature and @NaturePortfolio.
5K Followers 420 FollowingFormer CEO @NimbusTx. Board member at multiple companies helping teams discover & develop new medicines for patients. Any tweets are solely my own views.
16K Followers 420 FollowingFounding CEO of @Alnylam. Pioneered RNAi therapeutics as a whole new class of medicines for patients. Now bioentrepreneur-at-large. Opinions are my own.
118 Followers 968 Following||🙏Lover of God, Jesus Christ, and the Holy Spirit.||
||💊 Pharmacist by profession, Culinary Artist by passion.||
||Research Pharmacist on a mission 🧪||
704 Followers 5K FollowingSauerkraut enthusiast. I like banging my drum about biochar, UBIs, Score Voting, building better buildings and neighborhoods and base 12 math. And Novavax! LVT!
150 Followers 101 Followinghicon: smart wristband with OLED display and interchangeable social network icons, bluetooth 4.0 LE and gesture recognition. IndieGoGO: http://t.co/nC0YDsWpvA
120K Followers 781 FollowingBiotech reporter @statnews. Dog ❤️er. Polk Award winner. #COYS. Said one analyst: The likes of Adam Feuerstein attack viciously. On Signal: stataf.54
53K Followers 736 FollowingEarly stage biotech VC. Recovering scientist. Opinions expressed are solely my own and do not express the views or opinions of Atlas Venture.
6K Followers 278 FollowingEIR @GVteam, dreamer, inventor of ABE. Interested in genomic medicine and next-generation application of gene editing tools. Leonardo’s & Isabella’s Mom.
14K Followers 498 FollowingYour source for the latest #Science and #Biotech news. If you're interested in #CRISPR #GeneEditing hit follow! Spaces podcast host🎙- all 28 episodes ⬇️
16K Followers 506 FollowingGene editor + Professor Molecular Therapeutics @berkeleyMCB + Director for Technology and Translation @igisci + @danahercorp Beacon for CRISPR Cures.
30K Followers 989 FollowingCEO @VerveTx $VERV; Imagine if a one-time treatment led to permanent lowering of LDL cholesterol: that’s what we are developing to treat heart attack
25K Followers 769 FollowingScientist, entrepreneur, ex-biopharm exec, former FM radio jock, lifelong hockey fan, aspiring guitar player, #immigrant. CEO @ArrakisTx. Just @gilman on...
30K Followers 298 FollowingJournalist @ ApexOnco. The Wolverine Of Biotech / Mr ASH. Once had a blue tick. Interested in: oncology. Not interested in: what stock you bought today.
5K Followers 420 FollowingFormer CEO @NimbusTx. Board member at multiple companies helping teams discover & develop new medicines for patients. Any tweets are solely my own views.
16K Followers 420 FollowingFounding CEO of @Alnylam. Pioneered RNAi therapeutics as a whole new class of medicines for patients. Now bioentrepreneur-at-large. Opinions are my own.
243K Followers 2K FollowingSign up for my new newsletter! (Link below) Also: Co-author of Abundance, host of Plain English, and contributing writer at The Atlantic.
3K Followers 55 FollowingDeputy Secretary of Health and Human Services @HHSGov Acting Director of the Centers for Disease Control and Prevention @cdcgov
79K Followers 298 FollowingMAHA PAC is striving to Make America Healthy Again. We proudly supported RFK Jr's 2024 campaign and continue to back the MAHA movement in 2025 & beyond.
488K Followers 146 FollowingNobel Laureate. Co-Founder & CEO @GoogleDeepMind - working on AGI. Solving disease @IsomorphicLabs. Trying to understand the fundamental nature of reality.
5K Followers 868 FollowingFaculty @DanaFarber, @harvardmed, @broadinstitute, @hhminews investigator. Founder of @foghorntx. Chromatin and gene regulation in human disease.
25K Followers 133 FollowingMedical reporter for the New York Times and author of six books. The latest, Mercies in Disguise, will be published in March of 2017.
24K Followers 120 FollowingGrowSF is the #1 voter guide in San Francisco, trusted by hundreds of thousands of San Franciscans. You deserve a city that works.
17K Followers 17K FollowingAuthor, attorney, translator and adjunct teacher of law ~ and society ~ at @NYUGallatin and @EugeneLang. @MakeTheRoadNY Legal alum.
437K Followers 762 FollowingComplex systems, wicked problems. Society, technology, science and more. @Princeton professor. @NYTimes columnist. My newsletter @insight https://t.co/6Ky01N9JwA
5K Followers 824 FollowingExecutive Coach to Biotech Leaders • Author of the Biotech Leader’s Handbook • Tweets on: biotech, CEO excellence, high-performing teams, culture
1K Followers 508 FollowingCo-founder and Executive Director of Revive & Restore, with a mission to enhance biodiversity through the genetic rescue of endangered and extinct species.
24K Followers 57 FollowingWelcome to the Official X of Wrexham AFC Women | @Wrexham_AFC | Members of the #AdranPremier | Media/player requests - [email protected] #WxmAFC
9K Followers 209 FollowingI wrote that song you sang in that language you don't speak
@deccarecords | @umg | @YamahaMusicUSA
@boosey | @concordmusicpub
https://t.co/snfIXJPXoX
131K Followers 752 FollowingVirologist. Vaccinologist. Science Communicator. — My tweets are my own. My science is the world’s. — @KizzyPhD_Lab @HarvardChanSPH @HHMINEWS @hwaspeakers
3K Followers 2K FollowingI specialize in sickle cell disease care for all ages with the heart of a pediatric hematologist. My tweets are my opinions to be shared and to support others
5K Followers 540 FollowingLead Translator @arenabioworks, formerly Professor of Pathology (on leave, 2023-2025) @HarvardMed, #geneediting, #CRISPR; views expressed are my own