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Scientists unveil more than 600 new tissue models of Human Cancer. August 10, 2026 Derived from patient tumor samples and available to researchers around the world, the cells will aid the development of new cancer treatments. To develop new targeted treatments for cancer, scientists need tissue models that accurately represent the genetic and molecular traits of the cancer they're studying. An international team led by researchers at MIT's Koch Institute, the Broad Institute, the Dana-Farber Cancer Institute, the National Cancer Institute, and numerous other partnering institutions has developed nearly 700 new cancer models, derived from patient tumors, which they hope will aid in drug development. These cells, which represent 25 different types of cancer, are now available for cancer researchers around the world to use. The project is described in a new paper appearing August 5, 2026 in Nature, with contributors from more than two dozen institutions. The open-access article is titled "A Compendium of Next-Generation Patient-Derived Models for Diverse Cancers." The models are the result of a 10-year initiative, funded by the National Cancer Institute, to expand the number of patient-derived tissue models available. For most of these models, the researchers converted tumor cells into organoids - 3D cell cultures that can survive indefinitely and mimic the genetic and molecular features of the tumors that they originally came from. This type of model could help researchers identify new drug targets and test potential new treatments for many more types of cancer. "Since the sequencing of the human genome and the analysis of cancer genomes over the last 20 years, we have had many ideas about cancer targets, but we need experimental systems in the lab to validate those targets and launch drug discovery projects," says Jesse Boehm, a research scientist at MIT's Koch Institute and one of the senior authors of the study. From Tumors to Organoids The Human Cancer Models Initiative (HCMI) was launched in 2016, following the completion of the Cancer Genome Atlas, an effort to catalog the genomic alterations responsible for cancer growth. For the atlas project, researchers sequenced cancer cell samples from thousands of patients. That work revealed that the diversity of tumor genetic profiles was not fully captured by the roughly 1,000 patient-derived cancer cell lines that existed at the time. "We realized that a thousand wasn't enough, that the international community needed to invest in many more thousands to represent all cancers, all genotypes, all ethnicities," Boehm says. "Most existing models come from European and Southeast Asian patients, and many rare cancers are missing." Funded by the National Cancer Institute and the United Kingdom's Wellcome Trust, hundreds of scientists across dozens of institutions participated in obtaining patient samples and developing them into cell lines that could be used for research. "It's been an enormous initiative, and this Nature paper is the culmination of that 10-year swath of activity," Boehm says. More than 2,700 tumor samples were obtained from hospitals participating in the study, from patients who gave their permission for their cells to be used for research. These samples were collected by hospitals in the United States, the United Kingdom, and the Netherlands. "A resource of this scale depends on the kind of systematic effort that often happens behind the scenes," says Mushriq Al-Jazrawe, scientific director of the High Throughput Sciences (HTS) platform at the Koch Institute and one of the lead authors of the study. "I'm especially grateful to the technical and scientific teams across the participating institutes whose careful, expert work turns patient tumor samples into well-characterized models and data that researchers everywhere can use with confidence." Most of these samples came from commonly seen cancers such as lung, liver, and pancreatic, but they also included about 150 rare types including tumors of the gallbladder and the small intestine. To convert these samples into cells that can survive indefinitely in the lab, the researchers developed techniques for culturing the cells in specialized growth media with a scaffold that helps them grow into a 3D structure. Overall, the researchers were able to successfully convert about one-third of the patient samples that they received. Most of these new models consist of organoids, which in some cases more closely mimic the structure of the tissue that the cells came from. Traditional cancer cell lines, which were developed beginning in the 1950s, exist as single layers of cells grown in a lab dish, while organoids consist of three-dimensional balls of cells embedded in a gelatin-like structure. Once the organoids and cell lines were established, which can take up to a year, the researchers analyzed them to make sure that their genomic sequences, RNA expression, and epigenomic modifications closely matched those of the tumor cells that they were derived from. Cancer Vulnerabilities All of the models developed as part of the HCMI were deposited at the American Type Culture Collection (ATCC), a nonprofit distributor of cell lines. Each model also has extensive data from the patient whose cells were used to start the cell line, including mutations that the patient inherited from their parents (germline mutations), and information on the cancer treatments they received. Using these models, scientists should be able to perform much larger scale screens that could aid in drug development efforts. In another paper also appearing in Nature on August 5, 2026, Broad Institute researchers reported that they were able to profile more than 300 of the new models using high-throughput genome-sequencing, RNA sequencing, and more than 100 with CRISPR loss-of-function screens. This enabled them to identify vulnerabilities in each model that could be targeted with new drugs. These findings have been added to a resource known as the Cancer Dependency Map (DepMap), which now includes information on more than 2,000 types of cancer. In another Nature companion paper, researchers at the Sanger Institute led an effort to characterize an additional 256 organoids developed through the HCMI project. Additionally, even though most aspects of the formal HCMI project are currently winding down, researchers hope to continue developing models derived from additional patient tumor samples, including more pediatric cancers and rare cancers. "We now have about 2,000, but if we really want to represent all humans with cancer in our preclinical research, more work is needed. We have to invite patients to donate tissue to make research tools that the whole world can use," Boehm says. "I think this will hopefully be not the end, but the beginning." "A major opportunity now is to carry the lessons of HCMI forward, so we can generate as much insight as possible from these precious tissue donations," says Al-Jazrawe, who is also a researcher in the Broad Institute's Cancer Program. "Here at HTS, we are continuing the work by developing methods to study patient-derived samples and models reproducibly and at scale, and by providing a platform for close collaboration with clinical and research teams." Other senior authors of the HCMI paper are Mathew Garnett of the Wellcome Sanger Institute, David Tuveson of Cold Spring Harbor Laboratory, Andrea Califano of Columbia University Vagelos College of Physicians and Surgeons, Paul Spellman of the University of California at Los Angeles, Keith Ligon of Dana-Farber Cancer Institute, Daniela Gerhard of the NCI Center for Cancer Genomics, and Louis Staudt of the NCI Center for Cancer Research. In addition to Al-Jazrawe, the paper's lead authors are Dina El-Harouni of the Broad Institute and Dana Farber, Seongmin Choi of Memorial Sloan Kettering Cancer Center, Merve Dede of the University of Texas MD Anderson Cancer Center, Toshinori Hinoue of the Van Andel Institute, Sean Misek of the Broad Institute and Dana-Farber, Heeju Hoh of the Institute of Systems Biology and the Columbia University Vagelos College of Physicians and Surgeons, and Luca Zanella of the Columbia University Vagelos College of Physicians and Surgeons. The research was funded primarily by the National Cancer Institute and the Wellcome Trust. This posting is based on release authored by Anne Trafton, MIT News.

A $160 million 'moonshot' backs Boston effort to revolutionize rare disease treatment. By Allyson Chiu Globe Staff, Updated July 21, 2026, 8:00 a.m. The Broad Institute, Boston Children's Hospital, and Maine's Jackson Laboratory announced a collaboration Tuesday to develop gene therapies for people with rare diseases and to make the life-saving therapies cheaper and more accessible to patients. With relatively few gene therapies on the market, families of children with rare genetic disorders have few options but to partner individually with researchers and raise millions of dollars to develop treatments for their conditions. The newly formed nonprofit Center for Therapeutic Genetics will create precision medicines and share those methods, data, and training with other clinicians so they can do the same. The goal is to make these personalized treatments more like clinical procedures, such as organ transplants, so doctors don't have to seek separate regulatory approvals for each use, said Dr. Winston Yan, founding director of the new center. "Rare disease families, they carry too much on their shoulders today, and it just feels so unfair," Yan said. "If we achieve our vision, we think there's a world in which those families can just show up to such a center and have this be the standard of care. You get a genetic diagnosis? Well, here is a procedure that will get you your genetic treatment, and that feels really exciting to me." One in 10 Americans lives with a rare disease, half of whom are children, according to the National Organization for Rare Disorders. Less than 5 percent of known rare diseases, of which there are more than 10,000, have approved treatments. Developing treatments for these diseases has been a challenge because of a lack of scientific tools and investment. But the science now exists to create transformative treatments, largely because of breakthroughs in gene editing and gene therapy. Getting treatments to patients, however, remains a problem, Yan said. "There's a lot of headwinds for biotech," Yan said, noting that many rare diseases often don't affect enough people to drive commercial investment in developing treatments. "That means that patients fundamentally don't have access to life-saving technologies, even though in many cases the science actually works. You can actually treat their diseases meaningfully with these technologies." The center's goal, he said, is to create treatment platforms, including disease models, manufacturing processes, safety data, and clinical programs that can be shared and replicated by others. The center will initially focus on developing precision gene-editing treatments for children with rare forms of genetic epilepsies, an effort supported by a federal grant through ARPA-H, the country's "moonshot" agency for health research. Earlier this month, the agency announced that it will spend up to $160 million to advance custom gene editing treatments for a number of rare diseases. "Our model is not to be family-funded for particular diseases," said Timothy Yu, one of the center's founding partners and an attending physician in the division of genetics and genomics at Boston Children's Hospital. "Our model is to try to raise money philanthropically to support the field, to pick the conditions that will advance the field the furthest, the quickest, and with the most confidence." "I don't want to set the expectation that people should be calling us to put their name on a waiting list," Yu added. The center is also currently in its "founding phase" and not yet accepting patient referrals or providing clinical care, according to Tuesday's announcement. Pamela Gavin, chief executive of the National Organization for Rare Disorders, said the center's work could address a "phenomenal need." "We're excited about what they're building because it addresses one of the greatest unmet needs in rare disease," Gavin said. "They're not only advancing the science of genetic medicine, they're also developing repeatable approaches that could make these therapies more scalable and ultimately more accessible for patients with very small populations." The new collaboration would help make critical resources available to patients and advocacy groups as well as pharmaceutical and biotechnology companies, said Patricia Musolino, a critical care and vascular neurologist at Mass General Brigham, who has been involved in gene therapy for 15 years. "We want to change the way we're thinking about genetic medicines," said Musolino, who also recently received a grant from ARPA-H to work on genomic medicines to treat rare genetic vascular diseases. In the future, gene therapy would ideally be treated more like surgical procedures, she said. "The surgeon doesn't have to go to the FDA to get approved for exactly what he's going to be doing to fix your gallbladder or your lungs," she said. "He's going to use the approved resources." But expanding access to treatment for rare diseases is a complex challenge, Gavin said. She noted that her organization and others around the country have been working to address similar issues. The National Organization for Rare Disorders runs a network of nearly 50 leading US medical and research institutions that are dedicated to advancing rare disease diagnosis, care, and research. "The science is advancing remarkably quickly," Gavin said. "The harder challenge now is building the systems that allow those scientific advances to reach patients safely, efficiently, and at scale. That's not something any one institution can solve alone."

Broad Institute, Boston Children's Hospital, and The Jackson Laboratory launch the Center for Therapeutic Genetics, a non-profit effort to develop genetic medicines for rare and ultra-rare diseases. 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. July 21, 2026 Broad Institute, Boston Children's Hospital, and The Jackson Laboratory today announced the Center for Therapeutic Genetics (CTG), a collaboration that will develop genetic medicines and treat patients for rare disease not as one-off breakthroughs but as a repeatable practice, and share the methods, data, and training so others can do the same. An estimated 350-400 million people worldwide live with one of approximately 8,000 rare diseases. Children are disproportionately affected, and many of these conditions are progressive, life-threatening, or debilitating, yet fewer than one in 20 has an approved treatment. Traditional drug development typically takes many years and costs hundreds of millions of dollars - a model designed for diseases affecting large populations, not for rare and ultra-rare diseases that may each affect only a few people. 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 can be tailored to the specific mutation that causes a given rare disease and have already been used to treat more than 200 patients. For example, in 2018, a team led by CTG co-founder Timothy Yu at Boston Children's Hospital developed a custom antisense drug for a child with a progressive neurodegenerative disease - the first ever drug tailored for a single patient's specific disease-causing mutation. More recently, the University of Pennsylvania and Children's Hospital of Philadelphia treated an infant with a severe metabolic disorder, using a gene-editing medicine built for that child's specific mutation. This treatment used base-editing technology developed by CTG co-founder David Liu and his laboratory at the Broad Institute. CTG aims to make these patient-tailored genetic treatments for rare disease as precise, reliable, and repeatable as today's most complex medical procedures, and to make them available at scale to patients in a sustainable way. The center is founded by pioneers in genetic medicine and long-standing scientific collaborators, including David Liu, Core Institute Member and Merkin Professor at the Broad Institute and inventor of base and prime editing; Cat Lutz, Vice President, Rare Disease Translational Center, The Jackson Laboratory and a leader in rare disease model development and translational science; Timothy Yu, Staff Physician, Division of Genetics and Genomics, Boston Children's Hospital and a pioneer in individualized genetic therapies; Wendy Chung, Chief, Department of Pediatrics, Boston Children's Hospital and a leader in the clinical implementation of genomic medicine; and Winston Yan, Director of CTG, who has expertise building both nonprofit and industry biotech organizations and has brought a gene-editing therapy to the clinic. Central to the CTG model 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. The center also aims to expand by welcoming additional institutions and partners as it continues to develop. Over time, the result is a scientific and regulatory infrastructure that makes genetic medicine faster, safer, less costly, and more accessible to patients. Bringing these treatments to patients will require regulatory and reimbursement frameworks suited to medicines that are, by design, made for one or a few patients. CTG members will continue to work alongside the FDA, HHS, CMS, and other authorities to generate rigorous evidence to inform the evolution of such frameworks. CTG programs will include the development of precision gene-editing treatments for children with rare forms of genetic epilepsies, an effort supported by a recent award of up to $34.5 million from the ARPA-H THRIVE program. "Scaling and sustaining treatment for ultra-rare disease is a hard problem that many scientists, clinicians, patients, and drug developers are working on. What we have here is a group of leaders who believe that by approaching genetic medicines not as products, but as a standardized clinical procedure, and by sharing what we learn openly across institutions, we can make precision genetic medicine faster, safer, less expensive, and more accessible to patients and families in need." - Winston Yan, Founding Director, Center for Therapeutic Genetics "We receive messages every week from parents asking for help: Can we do for their child what has been done for other children who have received treatments? The honest answer today is usually 'not yet', often not because the science doesn't exist, but because we don't yet have the infrastructure to bring these treatments to many patients. CTG is our commitment to closing that gap, so that eventually every family who needs this kind of treatment has a path forward." - David Liu, Broad Institute "What we are building together is scalable treatment, something our institutions will learn to do well and repeat again and again. At Boston Children's, we see children every day for whom a diagnosis is only the beginning of a much longer journey. CTG exists to close a major gap in care and ensure that what we learn from one disease accelerates treatment for the next." - Wendy Chung, Boston Children's Hospital "We are at a moment in genomic medicine where, for many rare diseases, the question is no longer whether we can treat them, but whether we will build the systems to do it. CTG answers that call." - Cat Lutz, The Jackson Laboratory "We used to see precisely tailored therapies for children with genetic diseases as remarkable exceptions. CTG is built on the conviction that they don't have to be - that the methods we develop for one rare disease will carry to the next, and eventually to the many." - Timothy Yu, Boston Children's Hospital CTG updates and information. The Center for Therapeutic Genetics (CTG) is currently in its founding phase and is not yet accepting patient referrals or providing clinical care. CTG is committed to communicating openly as the center evolves. Individuals interested in CTG's progress, potential collaborations, research opportunities, or future clinical programs are encouraged to visit centerfortherapeuticgenetics.org.