Throughout its nearly four-decade history, Whitehead Institute has had astonishing impact and a record of disproportionate influence on biomedical research around the world.
Whitehead Institute’s continuing accomplishments reflect both the exceptional talent it attracts and its uniquely supportive, collaborative, and collegial environment. Together, our researchers are driving major discoveries, creating important new tools and methods, and spurring the creation of dozens of new therapeutics companies. Here is a snapshot of our impact—a small sample of those discoveries, tools, and companies.
The range of Whitehead Institute researchers’ discoveries and path-breaking accomplishments is broad and deep, as represented by the following relative handful of examples. Click on the associated links for more detailed information on these achievements.
In the 1980s, Robert Weinberg discovered the first gene in humans which, in certain circumstances, could transform normal cells into tumor cells.
Member David Baltimore created the first animal model of chronic myelogenous leukemia in 1990. The model mice provided a way for scientists to test treatments for the deadly disease.
Whitehead Member Ankur Jain has discovered that certain RNAs can form aggregates, clumping together into membrane-less gels. This process, known as phase separation, has been widely studied in proteins but not in RNA. He has found that RNA gels occur in, and could contribute to, a set of neurological conditions such as amyotrophic lateral sclerosis and Huntington’s.
Fellow Olivia Corradin and her lab investigate disease-associated DNA sequence changes called genetic variants—slight differences in the same DNA sequence that vary from person to person—that are risk factors for MS. Their key discovery, published in 2020, was that immune cells are not the only cell types implicated in the development of MS; rather, genetic variants that affect cells in the central nervous system also appear to contribute to the disease.
Former Member Hazel Sive pioneered the zebrafish as an accessible model for study of brain ventricle development and function. Sive's lab identified mutants and genes that impact zebrafish brain ventricle formation, identified a series proteins required for a brain to form properly.
Institute Member Iain Cheeseman determined the structure of the centromere, a cellular component essential for chromosome replication.
Whitehead Institute Member Richard Young has made fundamental discoveries on the function and regulatory role of phase-separated condensates in cells.
Whitehead Institute Member Peter Reddien has spent his career studying regenerative flatworms called planarians. His research has revealed the role of stem cells and of muscle in regrowing body parts.
Institute Member David Sabatini discovered the mTOR protein and mTOR growth regulatory pathway -- two components of the system that guides cellular metabolism.
New knowledge often yields new kinds of questions and opportunities for discovery. But available research technologies and approaches aren’t always up to the task. In many cases, new ways must be created to enable researchers to delve more deeply into the molecular mechanisms of cellular function and dysfunction.
Whitehead Institute researchers are renowned for creating and improving tools and methods that enable investigators to drive science forward. The following examples represent just the tip of their innovation iceberg. Click on the associated links for more detailed information.
Former Director and Member Susan Lindquist introduced the use of yeast cells to study mutant genes causing neurodegeneration.
Member David Bartel and colleagues created (and are continuously enhancing) TargetScan, a globally accessible database that enables researchers to identify the targets of specific microRNAs they are studying.
Researchers in Member Richard Young's lab conceived technology to map human genome regulatory circuitry.
Member Jing-Ke Weng developed models for dissecting the biochemical synthesis of plant-derived medicinal products.
Whitehead Fellow Olivia Corradin created methods to identify and assess the impact of disease-associated genetic variants within DNA’s three-dimensional structure.
Using CRISPR technology, Whitehead Institute Member Jonathan Weissman can track the lineage of individual cancer cells as they proliferate and metastasize in real time.
Often, the most effective way to translate Whitehead Institute discoveries and technical advances is by licensing intellectual property to commercial organizations able to develop brilliant ideas into testable and marketable therapeutics and diagnostics. The following is a selection of the companies that have been created specifically to translate intellectual property developed by our researchers. (It does not include previously existing companies to which we granted licenses.)
Alnylam Pharmaceuticals was launched in 2002, based on discoveries from David Bartel and scientific collaborators. It uses the concept of RNA interference (RNAi) and designs small RNA molecules to selectively block the production of disease-causing proteins. Two of its treatments for rare diseases have received FDA approval and several others are progressing through clinical trials.
CAMP4 Therapeutics was launched in 2016, based on discoveries by Richard Young and scientific collaborators. CAMP4 is mapping how signaling pathways control genes implicated in specific diseases; and is using this capability to identify new therapeutic targets and develop novel therapeutics.
Dewpoint Therapeutics was launched in 2019, based on discoveries by Richard Young and scientific collaborators. It is working to identify specifically how membrane-less biomolecular condensates in cells contribute to a vast range of diseases—including cancer, neurodegeneration, cardiovascular disease, and metabolic disease—and to develop drugs that target those processes.
Fate Therapeutics was launched in 2007, based on discoveries by Rudolf Jaenisch and scientific collaborators. It is developing cell-based cancer immunotherapies using induced pluripotent stem cells, which serve as a renewable source for the mass production of engineered NK cell and CAR T-cell therapies that can be delivered off-the-shelf to patients.
Fulcrum Therapeutics was launched in 2016, based on discoveries by Rudolf Jaenisch and scientific collaborators. It is developing treatments for genetically defined diseases, using patient-derived cell models. Its drug for FSHD—a rare, disabling disorder affecting skeletal muscle—is in clinical trials; and its drugs for Sickle Cell Disease and b-Thalassemia are on the cusp of clinical trials.
Genzyme (now Sanofi Genzyme) was founded in 1981, based on discoveries by Harvey Lodish and scientific collaborators that led to treatments for Gaucher’s disease and, ultimately, many other rare genetic disorders. Sanofi Genzyme has become one of the world’s most respected companies, recognized for innovations in medical science and biomanufacturing.
Ironwood Pharmaceuticals was founded in 1998, based on discoveries by scientists in the lab of Gerald Fink. It develops treatments for gastrointestinal diseases—including the drug linaclotide (Linzess®), now marketed for adults with irritable bowel syndrome with constipation (IBS-C) or chronic idiopathic constipation; and drugs now in clinical trial for esophageal reflux and abdominal pain associated with IBS.
KSQ Therapeutics was founded in 2015, based on discoveries by David Sabatini, Jonathan Weissman, and scientific collaborators. KSQ employs CRISPR-enabled functional genomics and a variety of cell types and screening methods to identify opportunities for new treatments for cancer, autoimmune disease, and select rare diseases.
Navitor Pharmaceuticals was founded in 2014, based on discoveries by David Sabatini and scientific collaborators. It develops therapeutics targeting mTORC1, the gatekeeper of cellular metabolism and renewal. The company’s initial development efforts address treatment-resistant depression and chronic kidney disease, and have potential application for age-related diseases.
Omega Therapeutics was founded in 2017, based in part on discoveries by Richard Young and Rudolf Jaenisch. By targeting the human genome’s fundamental regulators, the Insulated Genomic Domains (the combination of genes and their regulatory machinery), the company’s engineered therapeutics tune genes to their native correct state to treat disease.
Rubius Therapeutics was founded in 2014, based on discoveries by Harvey Lodish and scientific collaborators. Its innovative genetic engineering and cell culture processes enable it to use red blood cells as a foundation for an entirely new class of cellular treatments. The company is currently developing therapeutics for cancer and autoimmune disease.
Syros Pharmaceuticals was founded in 2013, based on discoveries by Richard Young and scientific collaborators. Syros is developing small molecules to control the expression of genes with the aim of treating patients with cancer and genetic diseases. The company has investigational therapies in ongoing clinical trials for acute myeloid leukemia, and breast, colorectal, lung, and ovarian tumors.
Verastem Oncology was founded in 2010, based on discoveries by Robert Weinberg and scientific collaborators. The company develops treatments that inhibit signaling pathways that promote cancer cell survival and tumor growth. Its first FDA approved product treats indolent non-Hodgkin’s lymphoma and its pipeline includes treatments for follicular lymphoma and ovarian and lung tumors.
Yumanity Therapeutics was founded in 2014, based on discoveries by the late Susan Lindquist and scientific collaborators. It develops drugs that target protein misfolding in neurodegenerative conditions such as Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis. The company recently began clinical trials of a drug for Parkinson’s.