An innovative new major private-public partnership led by Harvard and MIT aims to accelerate one of the hottest and most promising areas of medical research: cell and gene therapy.
“I just think it’s a huge opportunity,” said Terry McGuire, a founder of the venture capital firm Polaris Partners and a member of Massachusetts Life Sciences Strategies Group, a panel of academic, government, health-care, and industry officials. “Clearly, [it] is the next great frontier, and it’s going to be hugely important, both from a research perspective and from a clinical perspective.”
Announced Monday, the project, which will bring the universities together with leading local hospitals, major corporations and state officials, will create a new, as-yet-unnamed center for advanced biological innovation and manufacturing and remove a big impediment to research. The new facility, expected to open by the end of 2021, seeks to ensure that Massachusetts will remain a leading region globally for life sciences.
Recent years have seen dramatic breakthroughs in cell and gene therapy, with new approaches attacking genetic maladies like sickle cell disease by repairing or overriding the DNA mutations that cause them. Cancer immunotherapy, meanwhile, tunes the body’s natural defenses to attack cancer cells to which it was previously blind. Such “checkpoint inhibitors” have had nearly miraculous results, completely eradicating cancers that had shrugged off conventional treatment and spread widely through the bodies of dying patients.
“It’s an incredible thrill to see how basic science research has translated to therapy,” said Arlene Sharpe, Harvard Medical School’s George Fabyan Professor of Comparative Pathology and a pioneer in cancer immunotherapy. “This is an example of how curiosity-driven research is now being translated to therapy because the whole story of checkpoint blockade didn’t come about specifically to cure cancer. It came from understanding fundamentally how the immune system is regulated.”
Stem cells have already revolutionized laboratory science and continue their march to the bedside, while global growth in CRISPR/Cas9 gene-editing technology promises to accelerate progress in an array of fields, making widely available an accurate, relatively inexpensive way to alter the genome, change cellular behavior, and, potentially, that of an entire organism. Work is also advancing on cutting-edge therapies involving oligonucleotides, peptides, and RNA.
“Cell and gene therapies have the potential to revolutionize the global health system,” said Emmanuel Ligner, president and chief executive of GE Healthcare Life Sciences. “We have yet to realize the full potential of any of these modalities [but] there are currently more than 1,000 regenerative medicine trials taking place globally. Recently, in Sweden, the first patient received cell therapy outside of a clinical trial. It’s the start of an incredible time in the industry and in human health.”