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V O L . X X X I I N O. X I X 84 Fact Book / Doing Business in Maine I N N OVAT I O N / R & D T he Jackson Laboratory, a bio- medical research institution headquartered in Bar Harbor, has implemented a slew of partner- ships and initiatives over the past year. e launch of the Center of erapeutic Genetics is a partner- ship between JAX, the Broad Institute in Cambridge, Mass., and Boston Children's Hospital, and looks to scale individualized gene-editing therapies. e JAX-Colby Mentored Research Program recently received a $1.5 mil- lion gift from the Lunder Foundation to endow and expand the experiential learning initiative. e 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 collabora- tion with pharmaceutical giant GlaxoSmithKline could advance "human cell-based translational models" for Alzheimer's disease and other neuro- degenerative 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 e Center of erapeutic Genetics, still in its founding phase, is a collaboration that will develop patient-tailored genetic treatments for rare disease as precise, reliable and repeatable. e center is building a shared clini- cal, manufacturing and regulatory plat- form that could accelerate the develop- ment 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 extraordi- nary 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 affect- ing 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. e initiative also draws on the broader expertise and capabilities of JAX's Rare Disease Translational Center, which Lutz leads. e RDTC includes more than 75 scientists work- ing across more than 70 rare dis- eases, developing shared tools, data resources and translational frame- works that can be applied across mul- tiple diseases. A Maine lab tackles biomedical research Jackson Lab has forged partnerships and new initiatives B y L a u r i e S c h r e i b e r A B O U T J AC K S O N L A B O R AT O RY : The Jackson Laboratory is an independent, nonprofit biomedical research institution with a National Cancer Institute-designated Cancer Center. Established in Bar Harbor in 1929, JAX has nearly 3,000 employees worldwide and campuses and facilities in Maine, Connecticut, California, Florida, New York and Japan. P H O T O / C O U R T E S Y O F T H E JA C K S O N L A BO R AT O R Y JAX scientist Cat Lutz says that, for many rare diseases, "the question is no longer whether we can treat them, but whether we will build the systems to do it."

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