Patient-first, America-first: The Case For Global Collaboration
While the rise of Chinese biopharma in the past decade has been meteoric, I’m firmly in the camp that their engagement in the global ecosystem is good for the sector – its more opportunity than threat, great for patients, and is a healthy forcing function to raise the game for everyone
https://t.co/C6mQnRxbt2
Today in @NatureBiotech we report a new suit of PE8 prime editor proteins. PE8 variants were developed from laboratory-evolved PE6 proteins using AI-guided protein redesign. This approach combines recent advances in computational protein design and directed evolution to increase prime editing efficiency, especially in transient therapeutically relevant delivery settings such as mRNA+pegRNA electroporation into primary cells, eVLP delivery of prime editing RNPs, and LNP-mediated mRNA+pegRNA delivery in mice.
https://t.co/bz6PalFvc4
1/11
Today in @NatBiotech, we report the directed evolution of structured RNA motifs that enhance the efficiency of prime editing. Iterated high-throughput pooled screens and mutagenesis of these small RNA elements improved transient pegRNA lifetime.
https://t.co/ZiytYXicaJ
1/11
It's been a busy week at #ASGCT2026. In my "Dispatch," I wrote about how an vivo CAR-T overload, about two startups working on CRISPR therapies for Alzheimer's, and about a missed opportunity to ask hard questions of the FDA. Read more in @endpts: https://t.co/CMI8niS2hU
Genetically altered astrocytes that express chimeric antigen receptors offer a promising immunotherapy system capable of clearing accumulations of amyloid-β in the brains of mice—a hallmark pathological feature of #AlzheimersDisease, according to a new study in Science.
📄: https://t.co/jMTIzVznEV
#SciencePerspective: https://t.co/h1aL5tdltT
Florida State Rep. @AdamAndersonFL lost his beautiful son, Drew. Now he’s trying to help other families dealing with rare disease from suffering the same loss.
https://t.co/WtzJgkFzCV
Below is the story of the first patient treated with a prime-edited therapeutic, developed by @PrimeMedicine in a trial led by Dr. Élie Haddad and his team at CHU Sainte-Justine. This teenager suffered from chronic granulomatous disease (CGD), an immunodeficiency, and now—10 months after treatment—the patient is healthy, stable, and living with a functioning immune system. Tracy Attebury, whose story was previously told by @ginakolata@nytimes, was the second patient treated with a prime-edited therapeutic.
https://t.co/ZiVq1SmYsn
Today in @Nature we report a new prime editing strategy that can rescue a common cause of many genetic diseases in a disease-agnostic manner. This approach converts a redundant endogenous tRNA into an optimized suppressor tRNA, enabling a single prime edit to rescue premature stop codons across different diseases.
(1/15)
https://t.co/zs0qu5bhXx
The new Plausible Mechanism Pathway will bring the power of personalized therapies — like we saw with Baby KJ — to more patients with rare diseases. Our piece in this week’s New England Journal of Medicine:
https://t.co/1BMeLSfyAl
I discuss the urgent crisis from the loss of federal support of science in the US and recent clinical gene editing breakthroughs with @WalterIsaacson & @amanpour on @AmanpourCoPBS, airing on @PBS tonight at 11 pm ET, and on @cnni earlier today. Pls share!
https://t.co/cyVZpAwJiq
🚨 The full collection of #ASGCT2025 abstracts is available NOW! 🚨
Check out the most innovative science in the #celltherapy and #genetherapy field, with 2,200 pieces of original research—over 10% more than last year!
Read and download. https://t.co/MR4S6C2GoF
It’s been a fantastic 1st CRISPRMED24 conference! Rivaling the top CRISPR conferences! Special thanks to Jens-Ole Bock, quite a special person 😊, and the entire @CrisprMedicine team to pull off the difficult task of assembling a conference. Looking forward to next year!
A nice video rendering of the molecular steps of prime editing precisely correcting a mutation based on real structures of the component molecules and domains (when known). See the previous video here for the earlier steps: https://t.co/Paljviy238
Delighted to try out the world’s first CRISPR-edited salad from Conscious Foods and @PairwisePL. Mustard greens were edited with Cpf1/Cas12a to reduce their spiciness while retaining their nutritional content (which is 2X better than romaine lettuce). 🙏 @zhangf for the invite
📣 We are proud that we achieved another milestone. More than 1,000 instruments of our digital PCR system QIAcuity have been placed since its launch.
✅ The success story continues as we address biopharmaceutical customers' specific needs with our new solutions.
#QIAcuity#dpcr
Congratulations to the scientists at @VerveTx and @BeamTx for achieving this milestone. Two base editing clinical trials have begun dosing now (the @ucl leukemia trial and this heart disease trial), with a third cleared by @US_FDA to begin soon (Beam’s sickle-cell disease trial).
The first human patients have been treated with base edited cells in an academic clinical trial in the UK led by Professor Waseem Qasim @ucl. Allogeneic CAR-T cells containing multiple base edits are infused to treat relapsed or refractory T-cell leukemia.
https://t.co/MRxG74wfTl