ӳ����ý

Jay Bhattacharya, director of the National Institutes of Health (NIH), visited ӳ����ý on Friday, December 5 for a tour and discussion about how ӳ����ý researchers use federal funding to drive impact for patients. The visit was organized by Massachusetts Rep. Jake Auchincloss, who joined Bhattacharya on the tour.

Institute Director Todd Golub opened the conversation in the ӳ����ý Discovery Center, a public educational space that showcases how researchers in Kendall Square and around the world work with the NIH and industry partners to better understand and treat human disease. 

“ӳ����ý’s research, much of it federally-funded, focuses on improving human health by conducting innovative, high-risk, high-reward biomedical research, and building and sharing foundational tools and datasets that accelerate discoveries everywhere,” Golub said. “The NIH is a uniquely powerful force for impact, and we were proud to show how federal funding propels the understanding of the root causes of disease and opens new pathways to treatments and cures.”

First, the group visited ӳ����ý’s Center for the Development of Therapeutics (CDoT) and learned about the Drug Repurposing Hub, one of the most comprehensive biologically-annotated collections of FDA-approved compounds in the world. These drugs have already been proven safe for humans, and may be useful against diseases they weren’t originally designed to treat. Scientists at ӳ����ý and around the world can mine this resource to find new uses for these “old” drugs.

Bhattacharya next met with David Liu, ӳ����ý core institute member, the Richard Merkin Professor, and Director of the Merkin Institute for Transformative Technologies in Healthcare at ӳ����ý. Liu described his team’s efforts to scale up life-saving gene-editing treatments and make them more accessible to the hundreds of millions of people living with genetic diseases.

“NIH support over the past 15 years made possible the development of programmable base editing and prime editing medicines,” Liu said. “Our efforts to organize and scale these medicines are energized by recent signs that the federal government appreciates the tremendous opportunity to help rare disease patients that these developments now offer.” 

Stacey Gabriel, ӳ����ý’s executive vice president of platforms and scientific execution, and Niall Lennon, chair and chief scientific officer of ӳ����ý Clinical Labs (BCL) briefed Bhattacharya and Auchincloss on BCL, which is moving high-speed, low-cost genomics into the clinic and is based in a new facility in Burlington, Mass.

Finally, the group heard from Ben Neale, co-director of the Stanley Center for Psychiatric Research at ӳ����ý, and core institute member Evan Macosko about our ongoing efforts to use genetics and other biological data to understand the biology of schizophrenia, bipolar disorder, Parkinson’s disease, and other brain disorders. This work involves assembling and analyzing massive datasets and studying single cells from human tissues to discover new biological pathways driving these conditions and create a faster and more efficient path toward effective treatments.

“NIH must deliver results that matter to the public, and I appreciate the opportunity to visit with scientists who are translating NIH-funded research into real-world discoveries and breakthroughs that will transform patient care,” Bhattacharya said.

“ӳ����ý is taking on challenges that are too big for any single lab to address alone,” Auchincloss said. “We are proud that Massachusetts is home to one of the nation’s flagship independent nonprofit research organizations, bringing together researchers from academia, government, industry, and clinical care throughout the country to address the most important challenges in biomedicine.”