Priorities

Advances in AI will dramatically accelerate biological discovery, turbo-charge the identification of new therapeutic targets, and reshape medicine. But for this to happen, the research community needs to generate high-quality data needed to produce meaningful insights about human health and disease. The Broad is at the center of efforts to bring AI and the life sciences together to fully harness AI and build foundational capabilities that will allow researchers everywhere to decode biology. Among the Broad’s key efforts in AI are:

• The Eric and Wendy Schmidt Center is developing principled machine-learning approaches and partnering with experimental biologists to uncover fundamental insights into normal and disease biology. The Center is also training the next generation of leaders who are fluent in both AI and biology.

• The Machine Learning for Health (ML4H) initiative is bringing together AI researchers and clinical investigators to apply machine learning to deidentified patient data, including medical imaging and clinical records, to improve risk prediction and inform earlier diagnoses.

• A group of Broad investigators is developing agentic AI systems as scientific collaborators that can accelerate research by supporting scientists in generating hypotheses, analyzing data, and advancing discovery.

Across the Broad, scientists are also pioneering new machine learning methods and designing experiments to generate the structured, high-quality data that new AI tools need to achieve real scientific breakthroughs. These are just some examples of how the Broad is building the foundation for the fast-emerging field of AI and biology and powering the next wave of scientific discovery.

At the Broad, the Biology of Adversity Project and Broad Trauma Initiative bring together scientists across disciplines to understand how experiences such as adversity and trauma can become biologically embedded, altering how genes function and shaping health across the lifespan. By uncovering the mechanisms that connect experience to biology, Broad researchers hope to reveal new ways to predict, prevent, and treat disease.

As emerging scientific evidence points to shared genetic risk factors and biological mechanisms across different brain disorders, the Broad Program in Brain Health connects efforts such as the Stanley Center for Psychiatric Research with other initiatives such as Prions@Broad, the NeuroDevelopmental Variability Initiative, the Psychiatric Biomarkers Network, and the Center for Human Brain Variation to accelerate progress across the field of brain health. The program achieves this by breaking down the siloes between fields, linking basic neuroscience with scalable ‘omics and AI approaches as well as disease-focused research, and fostering real-time collaboration and data sharing.

Together, Broad scientists are generating foundational datasets, building better models of the human brain, and applying emerging technologies to uncover disease mechanisms and identify new and critically needed biomarkers and therapeutic targets.

The program’s ultimate goal is to improve outcomes for patients across a wide range of brain disorders. This includes Huntington’s disease, with investigators seeking to delay or prevent disease by targeting the DNA repeat expansion that a Broad team discovered actually drives disease. Broad scientists are also combining cutting-edge technologies such as the latest single-cell sequencing and spatial technologies, advanced cellular models, and computational models to understand how our immune systems may play a role in Alzheimer’s and Parkinson's disease. These are just some of the interdisciplinary, highly collaborative, and out-of-the box ways the Broad is tackling brain health.

The Broad’s Cancer Program has played a central role in the precision medicine revolution in cancer research and treatment. Because of this revolution, we can now envision a future where most cancers will be treated like chronic conditions, managed with biologically informed treatments based on a patient’s detailed genetic information.

But our work is not done. Drawing from the extraordinary Boston research ecosystem and beyond, Broad cancer researchers are now focused on overcoming the toughest challenges in cancer, including drug resistance, difficult-to-treat and rare cancers, and making cancer genomes clinically interpretable. In partnership with academia and industry, we are tackling these hurdles by developing breakthrough technologies to discover new drug targets and potential therapies—including immunotherapies—scalable strategies for predicting therapeutic combinations to combat resistance, and systematic methods to interrogate and interpret cancer genomes.

At the Gerstner Center for Cancer Diagnostics, Broad scientists are pioneering new diagnostic technologies, including less invasive techniques such as blood biopsies, with the goal of detecting cancer earlier, when it is more treatable. We work closely with the Center for Development of Therapeutics at the Broad to pioneer innovative therapeutic approaches and de-risk drug leads to the point where industry partners would be willing to take them on. These are just some ways Broad researchers continue to transform the understanding and treatment of cancer.

Cardiometabolic syndrome—a cluster of risk factors that increases one’s susceptibility to heart disease, diabetes, and obesity—has reached epidemic levels worldwide. While lifestyle factors like diet and exercise contribute to these disorders, they don’t fully explain why some populations are at a higher risk than others. Researchers at the Broad seek to understand how genetic variants contribute to disease risk in order to uncover new biologically informed, therapeutic targets. (While GLP-1 medications have had a profound impact for many people living with diabetes or obesity, others do not respond to them or cannot tolerate their side effects, underscoring the need for alternative treatments.)

Our researchers in cardiometabolic disease are pioneering new tools and methods to identify risk genes and convert them into biological mechanisms at scale. Through the Novo Nordisk Foundation Center for Genomic Mechanisms of Disease, Broad scientists are building powerful experimental and computational platforms to systematically uncover how genetic variation influences disease biology, accelerating the discovery of new therapeutic opportunities. This work can lead to the identification of new disease subtypes, which not only reveals new potential drug targets but can eventually help clinicians stratify patients, ensuring they get the right drugs. Ultimately, researchers at the Broad seek to translate genetic and biological discoveries into clinical practice and address urgent unmet clinical needs.

Infectious diseases rank among the world’s leading killers. The Broad pioneers and leverages the most advanced tools in genomics, the data sciences, and other fields to better understand pathogen biology, the interplay between host cells and pathogens, and the mechanisms driving infectious diseases and antibiotic resistance. We are also creating pandemic prevention and surveillance systems, developing advanced AI tools to accelerate diagnostic design, and pioneering new approaches to identifying potential new antimicrobial treatments.

In 2020, the Broad partnered with the Institute of Genomics and Global Health in Nigeria to launch Sentinel, a pandemic prevention system that empowers local and regional public health workers to detect and monitor disease threats by integrating genomic data and advanced analytics. This model has helped frontline healthcare workers and public health leaders in Nigeria and Sierra Leone combat COVID-19, mpox, and Lassa fever. Elsewhere in Africa, Sentinel has supported efforts to tackle outbreaks of Ebola, Marburg virus, and other infectious diseases. Designed as a flexible, scalable system, Sentinel is now being expanded to other countries in Africa.

In parallel, Broad scientists are investigating the complex interactions between pathogens and the human hosts that determine why people become infected, how disease progresses, and ultimately patient outcomes. The Center for Integrated Solutions to Infectious Diseases (CISID) brings together a community of physician-scientists and investigators from the Broad and our partner hospitals to answer the fundamental questions about infectious disease by learning directly from patients and develop powerful new weapons—laboratory tools, rapid diagnostics, and promising drug leads—to ensure that these research efforts have an immediate impact on patients.

Investing in talented scientists is central to the Broad’s mission of improving the understanding and treatment of human disease. The biggest challenges in biomedicine can only be conquered if promising scientists are nurtured at the most crucial stages of their careers. This requires not only mentoring and training, but also providing resources that allow them to pursue their most ambitious ideas.

We have worked with philanthropic partners to launch fellowships and awards that provide support to our most exceptional investigators. Below are examples of philanthropic partnerships supporting the next generation of scientific leaders.

BroadIgnite

BroadIgnite is an exciting initiative to support the bold ideas of early-career scientists—who often have difficulty obtaining funding from traditional sources—by connecting them with philanthropists.

Broad Summer Research Program

The Broad Summer Research Program (BSRP) is an intensive nine-week summer research opportunity designed for undergraduates with a commitment to biomedical research and an interest in genomics. BSRP has a strong record of success in helping students nurture their passion for research and succeed in graduate school and scientific careers. Students spend the summer performing original research in labs across the Broad's research areas.

Broad Summer Scholars Program

The Broad Summer Scholars Program (BSSP) provides motivated, curious high school students from the Greater Boston area the opportunity to participate in world-class scientific research.

STEM Education and Inclusion

The Broad runs several educational programs to increase access to research for K-12 students, including Scientists in the Classroom, which teaches eighth grade students about genetics and evolution research, and an after-school program called the Bio-Coding Club.

Technological breakthroughs are ushering in the era of programmable therapeutics—a new class of modular, gene- and cell-based medicines that can be designed, adapted, and redeployed to address a wide range of conditions. Rather than simply treating disease symptoms, programmable therapeutics aim to correct disease-causing changes in DNA, direct cells to produce therapeutic proteins, or rewire cellular pathways that drive disease.

Many of the technologies enabling this new era were developed by Broad scientists, including Feng Zhang, a pioneer in CRISPR technologies, and David Liu, who developed base editing and prime editing. They and other Broad researchers, including Xiao Wang, are continuing to expand the toolbox of gene- and cell-based technologies to improve scalability, precision, and accessibility across a wide range of conditions, ranging from genetic disorders to aging-related illnesses. Broad researchers are also pioneering new systems to deliver these medicines—one of the major hurdles for these therapeutics, especially in regards to overcoming the blood–brain barrier.

Psychiatric disorders exact an enormous human and economic toll, yet patients still have inadequate treatment options. The high heritabilities of psychiatric disorders mean that if specific genetic risk factors could be identified, they would provide important scientific clues and suggest approaches to therapeutics. Founded in 2007, the Stanley Center for Psychiatric Research brings state-of-the-art scientific tools and methods to bear on serious psychiatric disorders, including schizophrenia and bipolar disorder, in order to find new treatments and ease the burden for patients and their families.

Stanley Center researchers and their international collaborators have led the identification of significant genetic risk factors for schizophrenia and bipolar disorder—valuable clues to the underlying biology of these conditions and thus, potential drug targets. To do so, they have assembled the world’s largest collection of DNA samples of psychiatric disease, and have developed cutting-edge tools and methods, including new models of the human brain and new analytical approaches to translate genetic findings.

Ultimately, the Center seeks not only to unravel the biology of severe psychiatric disorders and develop therapeutic approaches, but also develop genetic risk prediction and biologically based diagnostic tools—transforming psychiatric treatment.

Nearly one in 10 people globally are affected by a rare genetic disease. Patients with rare diseases are often severely ill, but they face limited treatment options. The Broad is committed to leveraging the latest advances in biomedicine to rapidly accelerate the discovery and development of treatments and cures for rare diseases. To fulfill this mission, we bring together state-of-the-art technologies, scientific expertise, clinical experience, the biopharma and regulatory community, and patients and advocacy groups.

These efforts include the Center for Therapeutic Genetics, a collaboration between the Broad, The Jackson Laboratory, and Boston Children’s Hospital. While new genetic medicines have shown incredible potential in treating genetic disorders, the vast majority of rare disease patients do not have access to them. That’s because for each disease, there are too few patients to justify the high cost of commercial drug development. In order to overcome this challenge, the Center for Therapeutic Genetics aims to democratize genetic treatments for rare disease patients by derisking, making them scalable, helping to streamline the regulatory process, and sharing protocols.

The Ladders to Cure (L2C) Scientific Accelerator at the Broad also convenes scientists, clinicians, and patient advocates to catalyze progress across the rare disease research ecosystem and accelerate advances leading to treatments and cures. L2C focuses on uncovering “nodal biology”—common biological pathways across diseases—as well as technological innovations that speed biological investigations and therapeutic development.

The Broad is also advancing treatments for specific rare diseases such as Friedreich's ataxia, a rare inherited neurodegenerative disorder. The Friedreich's Ataxia Accelerator (FAA) brings together researchers across the Broad to develop new therapeutic strategies by leveraging expertise in genomics, genome editing, drug discovery, and disease biology. In addition to accelerating the search for treatments, the Accelerator is deepening our understanding of the biological mechanisms that drive the disease.