Broad Institute of MIT and Harvard

09/03/2026

Applications are now open for Broad's next Artist-in-Residence!

Mid-career artists who are deeply interested in Broad’s scientific work and live commuting distance from Cambridge, MA are invited to apply for our unique residency program.

For more information, visit: broad.io/AiR

09/02/2026

In traditional transcriptomic methods, cells must be destroyed to access the bits of RNA within, offering only a one-time snapshot. A new cellular self-reporting approach from the Blainey lab reveals gene activity in living cells without killing them. Their method engineers cells to package their RNA into virus-like particles that can be collected from the growth medium of a cell population, which can be done repeatedly to reveal how gene activity in the same cell population changes over time as the cells mature or respond to perturbations. “It's so gratifying to see a real coming to fruition of this concept, which was complete science fiction when we started,” said Blainey. The researchers demonstrated their approach in cell lines, spheroids, co-cultures, and organ-on-a-chip devices, demonstrating its broad utility for interrogating diverse biological systems in new ways, such as uncovering how cells go awry over time in disease and how drugs affect cells.

In the new method, cells package and export their RNA, enabling researchers to sequence and analyze the RNA without killing the cells.

08/12/2026

Prime editing can fix the vast majority of genetic mutations that cause disease, but picking the right guide RNA out of hundreds or thousands of possible options has been slow and expensive.

Broad Institute scientists in David Liu's lab built OptiPrime, a machine learning model that predicts which pegRNAs will work best for a desired edit. Scientists can use OptiPrime to prioritize pegRNAs to optimize, avoiding costly, time-consuming steps in the lab. Led by Liu, Alvin Hsu, Peter Chen, and Angus Li, the team built the model around the actual biochemistry of prime editing, enabling it to predict the efficiencies of editor variants it has never seen before. The team used it to design in just 4 weeks a prime editor that corrected a pathogenic mutation in mice, a process that typically takes months. OptiPrime is free for non-commercial use, and will support efforts, such as the recently launched Center for Therapeutic Genetics, to more rapidly develop new gene-editing medicines for rare diseases.

The tool, called OptiPrime, helps researchers more easily determine the best guide RNAs for efficient prime editing, avoiding costly, time-consuming steps in the lab.

08/05/2026

The Broad’s Cancer Dependency Map (DepMap) team has expanded the resource by adding dependency data from nearly 150 three-dimensional cancer organoid and spheroid models spanning 10 cancer types, integrating this with existing data from over 1,000 traditional 2D cancer models. Published in Nature alongside related studies, the research revealed dependencies not seen in 2D models, such as a CDK6 vulnerability tied to CDKN2A loss in glioblastoma. The work also showed that 2D and 3D models are complementary, each capturing distinct biology the other misses. This integrated dataset moves DepMap closer to its goal of comprehensively mapping cancer vulnerabilities across tumor types for drug discovery.

07/23/2026

Many scientists use lab-directed evolution to design proteins with new and improved functions such as editing DNA. Broad Institute researchers led by David Liu have now discovered that starting this laboratory evolution process with more stable, AI-designed proteins leads to better results than starting with natural proteins. Their findings, in Nature, could change the way scientists engineer proteins for medicine and other applications.

Researchers found that AI-redesigned versions of natural proteins serve as superior starting points to evolve proteins with new functions.

07/21/2026

We're thrilled to announce the Center for Therapeutic Genetics (CTG), a new collaboration between Broad, Boston Children’s Hospital, and The Rare Disease Translational Center at The Jackson Laboratory — a collaboration that will develop genetic medicines and treat patients with rare disease — not as one-off breakthroughs, but as a repeatable practice.

An estimated 350–400 million people worldwide live with one of approximately 8,000 rare diseases. Fewer than 1 in 20 has an approved treatment.

Recent advances in programmable genetic medicines, including base and prime editing, are paving the way toward a new model for treating rare disease. These medicines that can be tailored to the specific mutation that causes a given condition.

Central to CTG is a platform strategy in which design tools, disease models, manufacturing processes, safety data, and clinical protocols developed for one program are shared across multiple disease programs — making genetic medicine faster, safer, less costly, and more accessible to patients.

The center is founded by pioneers in genetic medicine and long-standing scientific collaborators, including David R. Liu, Core Institute Member and Merkin Professor at the Broad Institute; Cat Lutz, Vice President, Rare Disease Translational Center, The Jackson Laboratory; Timothy Yu, Staff Physician, Division of Genetics and Genomics, Boston Children’s Hospital; Wendy Chung, Chief, Department of Pediatrics, Boston Children’s Hospital; and Winston Yan, Director of CTG.

The new collaboration aims to develop precision medicines, including base and prime editing, to treat patients with rare diseases, using infrastructure and repeatable practices the center will share with others. div.hero-section__author { display:none;}

07/16/2026

When Broad core member and epigenomic tool-maker Jason Buenrostro won a prestigious “genius grant” from the MacArthur Foundation in 2023, it came at an opportune time. He’d been reflecting on stressful times from his youth and wondered how he’d achieved professional and personal success, while so many in his community and larger family struggled with poverty or addiction. With a confidence boost from the award and support from his family, Buenrostro shifted his research focus entirely. In partnership with the Treehouse Family Foundation, which shares the same passion and desire to make headway on the health impacts of life experiences, he launched the Biology of Adversity Project, a pioneering effort to study the problem at scale and with scientific rigor.

“Fixing the damage of stress is something that keeps me up at night,” Buenrostro said. Learn more about his life, his path to a career in research, and his new purpose to better understand the roots of resilience and response to adversity.

Shaped by a tumultuous childhood, Jason Buenrostro pivots from technology development to studying the biology of stress and adversity and finding new ways to boost resilience.

07/09/2026

We’re thrilled to announce that a Broad-led consortium of 12 organizations has been selected to receive funding from Advanced Research Projects Agency for Health under its THRIVE program to launch the Pediatric Epilepsies and Rare CNS (PERC) Gene Editing Platform — a collaboration aimed at developing gene-editing treatments for children with rare, devastating forms of epilepsy.

Developmental and epileptic encephalopathies affect more than 3 million children worldwide, yet there are few therapeutic options. PERC is designed to change that, starting with two severe conditions — Alternating Hemiplegia of Childhood and Dravet syndrome — using precision gene editors delivered via a Broad-developed AAV vector engineered to cross the blood-brain barrier without neurosurgery.

The collaboration brings together: Broad Institute, Boston Children’s Hospital, The Jackson Laboratory, Children’s Hospital Colorado, Children's Hospital of Philadelphia, Apertura Gene Therapy, Viralgen, Rare Epilepsy Network, RARE Hope, and the Dravet Syndrome Foundation.

PERC is also part of a broader coalition that includes Critical Path Institute, N=1 Collaborative, Global Genes, Worldwide Clinical Trials, Beam Therapeutics, Prime Medicine, and Mahzi Therapeutics.

We are honored to collaborate with these partners and look forward to the promising work ahead!

Twelve organizations spanning gene editing science, clinical medicine, manufacturing, regulatory strategy, and patient advocacy aim to deliver first-in-human trial within three years. div.hero-section__author { display:none;}

07/08/2026

Scientists in the Broad’s Stanley Center and Broad Clinical Labs have developed a low-cost sequencing approach that lowers the cost of sequencing by 75 percent over gold-standard methods, while delivering high-quality, unbiased data. The Blended Genome Exome (BGE) is becoming one of the most commonly used sequencing methods at the Broad and can enable large-scale studies to uncover the genetic roots of human traits and illnesses, especially in underrepresented populations. In a new paper, the researchers shared how they built BGE and used it to examine 53,000 samples for the PUMAS project, which aims to study the genetic causes of mental illness in an ancestrally diverse population.

“We’ve shown that the BGE technology works and it works at scale, and now the entire field can benefit from the method,” said study leader Alicia Martin.

The Blended Genome Exome approach delivers high-quality, unbiased genetic data at a quarter of the cost of the leading sequencing method.

04/09/2026

Leukemia is adept at dodging the immune system, making it resistant to many of the newest generation of cancer immunotherapies. Now Jooho Chung, Mounica Vallurupalli, Todd Golub, Rob Manguso, and colleagues have found a new way that this and possibly other cancers hide from immune cells: leukemia cells have a protein called CD43 on their surface that is coated so heavily in sugar molecules that it forms a physical barrier, shielding the cells from immune attack. CD43 could be a potential target for new cancer immunotherapies.

Targeting this protein, called CD43, could offer a new path to treatment for leukemia and other cancers.