Today in @NatureBiotech we report OptiPrime, a mechanism-based machine learning model for predicting prime editing (PE) efficiencies and suggesting pegRNAs to achieve a user-specified edit. OptiPrime incorporates biochemical knowledge about PE into its mathematical structure, enabling training on ~300,000 PE efficiencies collected using 40 experimental conditions in multiple labs, and outcome predictions of PE variants such as twin prime editing not used in its training. OptiPrime enabled the rapid identification of efficient PE strategies across a variety of applications in vitro and in vivo, and could outperform months or years of manual experimental optimization. Try out OptiPrime here: optipri.me PDF: drive.google.com/file/d/13JZ… 1/15

Experts in gene editing, n=1 medicine development, in vivo delivery, rare diseases, and cell & animal models of genetic disorders have founded the Center for Therapeutic Genetics (CTG), a non-profit effort involving @broadinstitute @BostonChildrens @jacksonlab, as well as others from industry, academia, and patient advocacy organizations, to bring the promise of programmable medicines including base and prime editors to patients suffering from rare genetic diseases. This article from @ginakolata @nytimes frames the compelling need: nytimes.com/2026/07/21/healt… Official announcement: broadinstitute.org/news/broa…

Today in @NatureNano, we report an all-RNA lipid nanoparticle (LNP) system for efficient in vivo prime editing (PE). We identify bottlenecks in transient prime editor delivery, develop a workflow for LNP optimization, and use it to rescue a mouse model of phenylketonuria. drive.google.com/file/d/1-rP… 1/12

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. drive.google.com/file/d/13VT… 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. drive.google.com/file/d/1Nyt… 1/11

Today in @natBME we report an in vivo base editing strategy that corrects a common disease-causing mutation and rescues pathology in a mouse model of Zellweger spectrum disorder (ZSD) and restore peroxisomal function in patient derived cells. This work highlights the promise of precision genome editing for treating peroxisomal biogenesis disorders. 1/13

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) drive.google.com/file/d/1bSv…

Science delivers medicines, clean air and water, safe food, and smarter choices—saving billions of lives. Proposed cuts to @NIH and @NSF would shutter labs, stall new cures, hurt our economy, and send future scientists elsewhere. Hear from patients saved by science: tinyurl.com/m65339fh. To share your story of how #ScienceSavedMe, email [email protected]. 1/2

A wonderful article by @DhruvKhullar on patients with progeria, the genetics behind this rare disease, and our ongoing efforts with PRF and @NIH to advance promising base editing outcomes in humanized progeria mice into a clinical trial. newyorker.com/magazine/2025/…

Today in @CellCellPress we report the use of prime editing to correct several mutations that cause alternating hemiplegia of childhood (AHC), a rare and devastating neurodevelopmental disorder, in patient-derived cells and in two mouse models. drive.google.com/file/d/1ibC… 1/10

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! youtube.com/watch?v=8YhJM6zx…

Another medical milestone! The first patient treated with a prime edited therapeutic shows positive clinical outcomes consistent with effective rescue of his chronic granulomatous disease (CGD), a debilitating and life-shortening primary immunodeficiency. At least 19 base editing and prime editing clinical trials have begun, with at least 7 trials reporting clinical outcomes (highlighted in green in the table below). They treat a wide range of diseases both in vivo and ex vivo. And while the U.S. support for science is now in crisis, that there are more base editing and prime editing clinical trials serving patients outside of the U.S. (13) than within the U.S. (9), despite the fact that these technologies were developed in our lab in the U.S. investors.primemedicine.com/…

Today in @ScienceMagazine we report the development of a laboratory-evolved CRISPR-associated transposase (evoCAST) that supports therapeutically relevant levels of RNA-programmable gene insertion in human cells, a collaboration with @SternbergLab. 1/13 drive.google.com/file/d/1I-U…