Broad Institute of MIT and Harvard

01/26/2026

After training an AI model on sections of DNA that regulate gene expression, researchers at Broad and Mass General Brigham found that the new model, called DNA-Diffusion, successfully created short DNA sequences that can turn genes on or off in specific cells.

To test the technology’s therapeutic potential, the researchers targeted a gene that protects against chronic lymphocytic leukemia that is often turned off in patients with the disease. They found that many of the AI generated sequences effectively switched on the gene, even more so than their natural counterparts.

The generative AI model designed sequences that successfully reactivated a protective gene in leukemia cell lines.

01/16/2026

Over the last decade, Broad researchers have identified a variant of the CARD9 gene as protective in inflammatory bowel disease (IBD) and discovered its mechanism. Now, they have developed small-molecule drug candidates that mimic the effects of this rare gene variant and could potentially treat IBD. This human genetics-to-therapeutics pipeline can be applied to other challenging drug targets and diseases.

The molecules mimic a gene variant that protects against Crohn’s, demonstrating a roadmap for using genetics to develop therapies for inflammatory bowel disease and other chronic inflammatory disorders.

01/12/2026

New research from Broad and has uncovered a key biological pathway behind fibrosis, or tissue scarring, in inflammatory bowel disease (IBD). When inflammation goes unchecked, it can trigger scarring that leads to organ dysfunction—or even failure—yet patients currently have few treatment options.

Now, scientists have identified the molecular roots of fibrosis. They discovered a specific “crosstalk” between specific types of cells that leads to tissue scarring. The team also identified GLIS3 as the “master regulator” of this messaging.

The study points to possible new therapeutic strategies, such as targeting the GLIS3 pathway.

01/08/2026

Please note: the Broad Discovery Center will be closed on Saturday, January 10, 2026 due to building maintenance.

01/07/2026

Fibrosis, or damaging tissue scarring, occurs in many inflammatory diseases and can lead to organ dysfunction or failure. Ramnik Xavier, Dan Graham, and colleagues characterized the crosstalk between immune cells and fibroblasts that leads to fibrosis in inflammatory bowel disease. They identified a master regulator, the transcription factor GLIS3, that mediates the cell-to-cell communication. Interrupting this cellular pathway could help prevent or reduce fibrosis in patients with IBD or other diseases of chronic inflammation.

New study finds a biological pathway responsible for dangerous scarring, which could possibly be targeted by new treatments.

12/17/2025

Our immune system loses its ability to fight infection with age. To try to overcome this decline, Feng Zhang and colleagues have found a way to temporarily program cells in the liver to produce specific molecules that improve T-cell function. They used mRNA to deliver three key factors to the liver that promote T-cell survival. Treated animals showed much larger and more diverse T cell populations in response to vaccination, and also responded better to cancer immunotherapy treatments.

Stimulating the liver to produce some of the signals of the thymus can reverse age-related declines in T-cell populations and enhance response to vaccination.

12/10/2025

Friedreich’s ataxia, a rare but devastating genetic disorder, occurs due to loss of the key mitochondrial protein, frataxin. Now, scientists at Broad and Mass General Brigham, with support from the Friedreich’s Ataxia Research Alliance, have used the power of worm genetics to discover a new potential drug target for FA, suggesting a path for developing new medicines. They found that certain mutations in the mitochondrial gene FDX2 could bypass the cell’s need for frataxin. They then showed that the FDX2/frataxin balance is important, and when frataxin levels are low, simultaneously reducing FDX2 can help restore production of crucial iron-sulfur clusters. Future work could reveal the precise balance necessary and how it is regulated in humans, and whether this could be a viable therapeutic approach.

Researchers from Mass General Brigham and the Broad Institute have discovered a potential drug target for the mitochondrial disorder Friedreich’s ataxia, suggesting a path for the development of new medicines

11/24/2025

Applications are now open for our 2026 summer research programs: the Broad Summer Scholars Program (BSSP) for high school juniors in MA, and the Broad Summer Research Program (BSRP) for undergraduate students.

In both programs, students will spend the summer performing original computational- or experimental-based research in areas such as cancer biology, psychiatric disease, chemical biology, computational biology, infectious disease, and more.

Learn more and apply: broad.io/studentopps

11/24/2025

Nominations are open for the $400,000 Merkin Prize in Biomedical Technology!

Do you know a team or individual whose novel device or method has improved diagnosis, treatment, or prevention of human disease? Nominate them today for the 2026 Merkin Prize.

Deadline: December 5, 2025

Previous winners include:
- Carl June, Bruce Levine, Isabelle Rivière, and Michel Sadelain for CAR T-cell Therapy
- F. William Studier for the T-7 expression system
- Marvin Caruthers for efficient synthesis of nucleic acids

Open to teams and individuals from across the globe and in any field.

Learn more at https://merkinprize.org

11/21/2025

In a milestone for the Broad, the FDA has approved a new medicine for a type of lung cancer that previously had few treatment options. The drug from Bayer Healthcare Pharmaceuticals is the first FDA-approved cancer treatment to come from a Broad discovery. To learn more about the new drug, the challenges that nearly stalled the effort, and the future outlook for new cancer drugs, we spoke with Broad team leaders Matthew Meyerson and Heidi Greulich.

“It is an extraordinary time to make many new treatments for patients with cancer and other diseases as well,” said Meyerson. “The success of this new drug is a real proof-of-concept that we can do this again.”

Dana-Farber Cancer Institute

Broad researchers Matthew Meyerson and Heidi Greulich describe how they and Bayer scientists developed the first FDA-approved cancer drug to come from a Broad scientific discovery.

11/20/2025

The US FDA has approved the first cancer drug based on discoveries from Broad Institute scientists. This drug was developed by Bayer Healthcare Pharmaceuticals in close collaboration with the Broad, and is the first FDA-approved medicine from the long-standing Broad-Bayer oncology research alliance. The new drug is a pill approved for adult patients with lung cancer who have certain HER2 mutations in their tumors, who previously had few treatment options. This FDA approval is an important milestone for the Broad Institute, demonstrating that research by academic scientists and close collaborations with industry can lead to benefits for patients.

Dana-Farber Cancer Institute

Collaboration between Broad Institute and Bayer leads to new treatment for a hard-to-treat type of lung cancer.

11/19/2025

Researchers at Broad have developed a new genome-editing strategy that could potentially lead to a one-time treatment for multiple unrelated genetic diseases. The new technology, called PERT, uses a single prime editing agent to address a class of mutations that halt protein production too early and can cause about one-third of genetic diseases. This approach could vastly improve access to gene-editing treatments for patients.

Researchers have developed a genome-editing strategy that targets a common cause of roughly 30 percent of rare diseases and could vastly improve access to gene-editing treatments for patients.