Jackson Lab takes new approaches to biomedical research

The Jackson Laboratory, a biomedical research institution headquartered in Bar Harbor, has implemented a slew of partnerships and initiatives over the past year. The launch of the Center of Therapeutic Genetics is a partnership between JAX, the Broad Institute in Cambridge, Mass., and Boston Children’s Hospital, and looks to scale individualized gene-editing therapies. The JAX-Colby Mentored Research Program recently received a $1.5 million gift from the Lunder Foundation to endow and expand the experiential learning initiative. The lab has a $5.4 million plan underway to modernize and increase production of “humanized” mouse models for immunology, oncology and infectious diseases. A five-year research collaboration with pharmaceutical giant GlaxoSmithKline could advance “human cell-based translational models” for Alzheimer’s disease and other neurodegenerative conditions. Late last year, JAX completed its acquisition of the New York Stem Cell Foundation, a nonprofit that developed an artificial intelligence platform that supports large amounts of data related to stem cell research. Therapeutic genetics The Center of Therapeutic Genetics, still in its founding phase, is a collaboration that will develop patient-tailored genetic treatments for rare disease as precise, reliable and repeatable. The center is building a shared clinical, manufacturing and regulatory platform that could accelerate the development of treatments for thousands of rare diseases, rather than developing every therapy from scratch. As many as 400 million people worldwide live with a rare disease. Fewer than 5% receive an approved treatment. CTG asks: Can personalized gene therapies that emerge from extraordinary scientific breakthroughs evolve into a repeatable model of care? 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 are paving the way toward medicines that can be tailored to the specific mutation that causes a given rare disease and have already been used to treat more than 200 patients. Leaders of the effort include Cat Lutz, a JAX leader in rare disease model development and translational science. “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,” said Lutz. The initiative also draws on the broader expertise and capabilities of JAX’s Rare Disease Translational Center, which Lutz leads. The RDTC includes more than 75 scientists working across more than 70 rare diseases, developing shared tools, data resources and translational frameworks that can be applied across multiple diseases. The center’s programs are supported by a recent award of up to $34.5 million from the ARPA-H THRIVE program. JAX-Colby Launched as a pilot in 2021, the JAX-Colby Mentored Research Program pairs Colby undergraduates in biomedical research training with JAX faculty mentors. Lunder’s $1.5 million endowment allows JAX to offer academic-year and summertime research opportunities. Over the past five years, 15 students have participated. The JAX-Colby program is one in a series of training initiatives overseen by JAX Genomic Education, which offers educational programming for a variety of cohorts, including aK-12 teachers bringing genetics and genomics into their classrooms, students and clinicians who want to apply genomics to patient care. Philanthropy helps fund the programs. ‘Humanized’ mouse models Earlier in 2026, the lab received a $2 million grant through Maine Technology Institute’s Maine Technology Asset Fund program toward the cost of renovating an existing 3,500-square-foot facility on the Bar Harbor campus, expand capacity and meet growing market demand for advanced research mice. The goal is to create “a state-of-the-art humanized mouse model production facility.” The balance of the cost will come from institutional capital improvement funds. A humanized mouse refers to a mouse that carries some aspect of human biology, which makes them models for studying human disease. The mice are often engrafted with human tissue, such as immune cells, meaning that the mice have “more human” immune systems. That allows mice to match human biology, allowing researchers to better understand human disease and more accurately test potential new treatments. The combination of engineered strains with human-relevant biological systems is expected to help researchers evaluate therapies in models that better reflect human disease. Completion is targeted for December 2027. The project is expected to create eight positions to operate the mouse production and distribution facility. GlaxoSmithKline collaboration A five-year research collaboration with pharmaceutical giant GlaxoSmithKline could advance “human cell-based translational models” for Alzheimer’s disease and other neurodegenerative conditions. “Human cell-based translational models” use cells from the human body to develop therapies. GlaxoSmithKline (NYSE: GSK) has its global headquarters in London and U.S headquarters in Philadelphia. The company had 2025 sales of $44 billion, driven by strong specialty medicines performance, according to the firm. The collaboration brings together JAX’s expertise in genetics, disease biology and stem cell research with GSK’s in neurodegeneration and drug discovery. The goal is to help close a gap between scientific discovery and the development of new medicines. Disease-relevant cellular models can enable the scientific community to study the underlying human biology of neurodegenerative conditions and accelerate the translation of those insights into potential therapies. “This collaboration is a concrete example of where early stage biomedical research is heading,” Lon Cardon, the lab’s president and CEO, has said. Certain types of models developed from a patient’s stem cells can more accurately capture the complexity of human biology at scale and accelerate therapies, Cardon said. The collaboration includes the New York Stem Cell Foundation, which JAX acquired late last year. Stem Cell Foundation Jackson Lab acquired the New York Stem Cell Foundation to leverage biomedical data, marking “a turning point for biomedical research” that combines the lab’s expertise in genetics and mouse models with the foundation’s stem cell and automation technologies, Cardon has said. The foundation is a nonprofit that developed an artificial intelligence platform that supports large amounts of data related to stem cell research. Together, the organizations’ capabilities are expected to create a platform that accelerates early-stage biomedical research that could uncover disease mechanisms earlier and advance progress toward therapies for diseases such as Alzheimer’s, cancer, diabetes, Parkinson’s and rare diseases. The deal unites the lab’s strengths in mouse genetics and disease research with the foundation’s stem cell technologies and advanced automation, plus their shared expertise in data science and AI, which together could speed the discovery of new treatments and cures. The deal was planned for quite some time, with financing built into JAX’s long-term strategy. As part of the process, 77 foundation employees join the lab’s 3,000 employees at campuses and facilities in Maine, Connecticut, California, Florida, Japan and now New York. The foundation, established in 2005, is at 619 Wes 54th St. in Manhattan. Its platform is called the “global stem cell array. The technology makes it possible to investigate biology at the level of individual patients. The lab’s genetically diverse mouse models and the foundation’s automated array give researchers a platform to predict treatment responses, test the predictions on different human cells and validate results in whole organisms. The integrated organization will operate as a nonprofit. The foundation will remain in Manhattan.