Broad Institute

Join us on Thursday, August 14 at 6 PM ET in Cambridge, MA, or online, for a Broad Discovery Series talk with @soumya-boston.bsky.social: “Arthritis, autoimmunity, and how the genome works.” Register: broadinstitute.swoogo.com/DiscoverySer...

Study finds biomarkers, drug targets, risk factors for most common form of lung cancer Large analysis of genes and proteins in lung adenocarcinoma points to possible new diagnostic and treatment approaches, and also reveals signatures of chemical exposures in patients who have never smo...

Scientists find new factors linked to poor outcomes in the most common lung cancer type cancer, hinting at how certain chemical exposures may contribute to lung cancer risk in people who have never smoked.

Collaboration aims to reduce animal use in drug safety testing OASIS consortium is evaluating omics tools in cells, including Cell Painting and transcriptomics, to predict liver toxicity of potential new medicines and agrochemicals.

The Omics for Assessing Signatures for Integrated Safety (OASIS) consortium, launched by Broad, @hesiglobal.bsky.social & scientific partners, with @malifesciences.bsky.social support, is sharing data using cell-based omics tools to reduce reliance on animals in drug & agrochemical safety testing.

Precisely defining disease variant effects in CRISPR-edited single cells - Nature A plate-based assay called CRAFTseq has been developed that uses ‘multi-omic’ single-cell RNA sequencing and direct genotyping of CRISPR edits to test the functional effects of genetic variants o...

So excited to share our latest study on defining the function of disease variants by editing single cells, and assuming them with CRAFT-seq! Special thanks to @zpmu.bsky.social and @baglaenkolab.bsky.social! @broadinstitute.org

www.nature.com/articles/s41...

Our study led by T. Bellomo and with @aniruddhpatelmd.bsky.social describes the relationship between lipoprotein(a) and incident & progressive extracoronary atherosclerotic vascular disease www.ahajournals.org/doi/10.1161/... @ahajournals.bsky.social
@broadinstitute.org @mgbresearch.bsky.social

Scientists apply optical pooled CRISPR screening to identify potential new Ebola drug targets Combining powerful imaging, perturbational screening, and machine learning, researchers uncover new human host factors that alter Ebola’s ability to infect.

Using an image-based screening method, Broad and Boston University researchers found potential new drug targets for reducing Ebola infection severity, uncovering human genes that, when silenced, impaired Ebola’s ability to replicate. broad.io/Ebola-News-0724

Prime editing treats childhood brain disease in mice Scientists use a precise form of gene editing called prime editing to correct the most common genetic mutations that cause alternating hemiplegia of childhood, a rare and severe neurological disorder ...

Partnership between David Liu, @jacksonlab.bsky.social, and rare disease group RARE Hope leads to first use of prime editing for a brain disease that reduced symptoms, extended lifespan in mice, offering hope for other rare genetic diseases. #Science #PrimeEditing

I've always found @soumya-boston.bsky.social's research to be absolutely fascinating, and I'm excited to bring him on-stage for the next talk in the @broadinstitute.org's Broad Discovery Series. Join us in-person or online on 8/14!

Seth Klarman selected Chair of the Broad Institute Board of Directors Klarman succeeds Eric Schmidt after four years as Chair; Aviv Regev joins the Board

We are thrilled to announce that Seth Klarman will become the next Chair of the Broad Institute Board of Directors, succeeding Eric Schmidt. We also welcome Aviv Regev as a new member of the Board.

talk poster

Join us on Thursday, August 14 at 6 PM ET in Cambridge, MA – or online – for a Broad Discovery Series talk with @soumya-boston.bsky.social : “Arthritis, autoimmunity, and how the genome works.” Register: broadinstitute.swoogo.com/DiscoverySer...

Scientists discover network of cells and genes involved in Crohn’s disease complication Spatial mapping reveals genes contributing to fibrosis, or scarring, in the gut, laying the foundation for better treatments.

Dr. Ramnik Xavier and Dr. Christopher Smillie from the @broadinstitute.org analyzed individual cells from the intestinal tissue of #Crohns disease patients, measuring gene activity and mapping the cells’ location: bit.ly/4nwuf0s

📃 @natgenet.nature.com: go.nature.com/45UEWDH

Single-cell and spatial transcriptomics of stricturing Crohn’s disease highlights a fibrosis-associated network - Nature Genetics Single-cell and spatial transcriptomics analysis of stricturing Crohn’s disease highlights cellular heterogeneity in fibrotic tissue and identifies neighborhoods of fibroblasts and immune cells contri...

Very excited to share our latest work in @NatureGenet with the Smillie lab: a spatial transcriptomics atlas of stricturing #Crohns disease that offers new insights into mechanisms driving fibrotic complications in chronic gut inflammation...(cont.)
www.nature.com/articles/s41...

Scientists discover network of cells and genes involved in Crohn’s disease complication Spatial mapping reveals genes contributing to fibrosis, or scarring, in the gut, laying the foundation for better treatments.

New spatial maps of a key complication of Crohn’s disease reveal the cells and genes involved and lay the foundation for better treatments.

Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases Broad core member and gene editing pioneer David Liu describes a framework that could enable the treatment of 1,000 patients with personalized gene editing therapies by 2030.

Gene editing pioneer David Liu discusses baby K.J. Muldoon, the first person treated with a customized gene editing therapy, what made K.J.'s treatment possible, and how on-demand treatments for rare genetic diseases could one day become routine. #GeneEditing #Science

#WhyIScienceQ&A: A neuroscientist advocates for stepping outside your comfort zone in research Giulia Monti reflects on how studying Alzheimer’s disease alongside researchers focused on kidney disease has helped her grow as a scientist.

Being a neuroscientist in a lab focused on protein trafficking in the kidney has given Giulia Monti a unique perspective on Alzheimer’s disease. Learn about the potential paradigm shift she sees in Alzheimer’s disease research in a new #WhyIScience Q&A: broad.io/WISGiulia

The GA4GH Genomic Knowledge Standards (GKS) Work Stream aims to define a common language for computers to describe variants — and the important biomedical kn... Genomic Knowledge Standards (GKS) Work Stream Open House (June 2025)

The GKS Work Stream held its second open house meeting this month.

Hear an overview of the newly approved Variant Annotation Specification (VA-Spec) and how GA4GH Driver Project ClinGen is implementing GKS products.

View the recording here: youtu.be/sap18DAG0aA

Realizing the promise of genome-wide association studies for effector gene prediction - Nature Genetics This Perspective argues that predicting effector genes for complex diseases is a key outcome of genetic association studies where standards are urgently needed to maximize utility, improve interoperab...

🚨Interested in which genes are the true effectors behind the scenes of the #gwas results? 🚨
This new publication in Nature Genetics, a collaboration between @broadinstitute.org @ebi.embl.org @opentargets.org focuses on approaches to predicted effector gene reporting tinyurl.com/98yxy4hf (1/3) 👇

Broad Institute and Bayer extend their cardiovascular alliance Partnership will continue with renewed focus on developing new cardiovascular therapies.

Broad and Bayer extend their 10-year cardiovascular research collaboration by 5 more years, with aims to identify joint precision cardiology targets, leverage the established human cardiomyocyte platform, and discover novel therapeutic approaches. www.broadinstitute.org/node/5558906

The 1st BIO-X Convergence Explore how agentic AI is revolutionizing healthcare—advancing precision medicine, decoding biology, and enabling next-gen clinical innovations. Join global leaders shaping the future of intelligent, ...

Neat event
(BIO-adjacent, in Boston next week)

nference.com/bio-x-conver...

Post 2: Virtual attendees can also visit the #HCA2025GM virtual Exhibition Hall here: buff.ly/OoVEkjh

Register for the Cell Biology at Scale meeting in New York City This meeting aims to spark a robust discussion that will articulate key, actionable goals toward true cell biology at scale.

July 31, virtual and in person in NYC:
Cell Biology at Scale.
See what's next for the data generators that will power the next phase of bio discoveries!
www.czbiohub.org/events/cell-...

Delighted to have our work on Polygenic Modifiers of #TelomereBiologyDisorders, led by Michael Poeschla, @sashagusevposts.bsky.social, @mitchiela.bsky.social, @sharonsavage.bsky.social, @tummalalab.bsky.social, + co, published in @jclinical-invest.bsky.social today: www.jci.org/articles/vie...

A new combination technique enables DNA sequencing and high-resolution imaging in intact cells — offering new insights into progeria and aging. broad.io/expansion-in-situ @jbuenrostro.bsky.social

Making antibiotics more potent against drug-resistant bacteria Scientists find combinations of known antibiotics and other molecules that, together, have greater bacteria-killing power.

A new pipeline could help scientists discover unique combinations of molecules that increase the effectiveness of antibiotics against drug-resistant bacteria. broad.io/CITADel-news @mit.edu @tuftsmedschool.bsky.social

Gene editing disrupts Huntington’s mutation in mice Making single-letter edits in stretches of repeated DNA stopped or reversed the genetic change that causes Huntington’s disease and Friedreich’s ataxia.

Researchers have developed a way to edit the genetic sequences at the root of Huntington’s disease and Friedreich’s ataxia.

"BRAIN Armamentarium Consortium" overlayed on a background of brain scans of various colors. Cell Press branding on the bottom right.

This collection describes a range of methods allowing access to neuronal and non-neuronal cell types, opening the door to targeting the nervous system in ways that were previously unimaginable 🧠

Check out our BRAIN Armamentarium Consortium🧵
www.cell.com/consortium/b...

Type 2 cytokines act on enteric sensory neurons to regulate neuropeptide-driven host defense Enteric nervous system (ENS)–derived neuropeptides modulate immune cell function, yet our understanding of how inflammatory cues directly influence enteric neuron responses during infection is conside...

🚨New paper with @thexavierlab.bsky.social @science.org on the critical role of 🔄neuroimmune crosstalk in immune defense in the gut🪱🪱.
🧪@genelayinstitute.bsky.social @mgbresearch.bsky.social @harvardmed.bsky.social @broadinstitute.org
www.science.org/doi/10.1126/...

2025 Merkin Prize in Biomedical Technology awarded to pioneers of CAR T-cell therapy The winners are Carl June, Bruce Levine, Isabelle Rivière, and Michel Sadelain, who developed technology that reprograms patients’ immune systems to target cancer, leading to several FDA-approved trea...

I am deeply honored and humbled to have received the 2025 Merkin Prize in Biomedical Technology along with my long term mentor and colleague Carl June, and Isabelle Rivière, and Michel Sadelain, w/ collaborative work of many scientists, clinicians, & patients. www.broadinstitute.org/news/2025-me...

2025 Merkin Prize in Biomedical Technology awarded to pioneers of CAR T-cell therapy The winners are Carl June, Bruce Levine, Isabelle Rivière, and Michel Sadelain, who developed technology that reprograms patients’ immune systems to target cancer, leading to several FDA-approved trea...

Carl June (@upenn.edu), Bruce Levine (Penn), Isabelle Rivière (Takeda), Michel Sadelain (@columbiauniversity.bsky.social) have been awarded the 2025 Richard N. Merkin Prize in Biomedical Technology for developing CAR T-cell therapy for cancer.

Scientists build cell line atlas of deadly cancers of the bile ducts and gallbladder Researchers reveal new subtypes and potential therapeutic targets for biliary tract cancers, which lack effective treatments.

A new cell line atlas of biliary tract cancers uncovers new subtypes and potential drug targets for better treatment of these deadly diseases.

@mgbresearch.bsky.social @targetcancer.bsky.social