Generating new gene and cell therapies can be as complicated as understanding the diseases themselves, often taking years and costing millions of dollars.
According to the National Institutes of Health, about 350 million people globally live with a rare disorder, 80% of which are related to genetics. Creating these kinds of treatments, which replace and alter affected genes, can be difficult due to cost, length, and complexity of the treatment, side effects, and more, per Oxford Global Resources.
Boston-based Mass General Brigham is attempting to uncomplicate processes by pooling resources for researchers through its Gene and Cell Therapy Institute (GCTI).
“The goal of the institute is to have a very focused attention on patients who can benefit from gene and cell therapy, and help investigators who have technologies for treatment of specific types of diseases take them toward that first in-human trial,” Roger Hajjar, head of the GCTI, told Healthcare Brew.
Building an institution. Across MGB, there are 6,000 investigators, 500 of whom are studying gene and cell conditions and therapies. These researchers often try to develop treatments for the conditions. For example, MGB teams have previously created therapies for lymphoma and leukemia, multiple myeloma, and spinal muscular atrophy. But bringing these products to market is an expensive and timely challenge, with one study linking the average cost to bring a gene and cell therapy to market at nearly $2 billion
“Those labs will spend many decades studying very specific diseases of the brain, of the heart, or kidney, and they’ll realize that, ultimately, the way to treat those diseases is a gene and cell therapy path,” Nathan Yozwiak, head of research at the GCTI, told Healthcare Brew. “Going from those discoveries [and then] translating them into a new gene and cell therapy is an extremely long path.”
So in late 2022, Mass General Brigham started GCTI, first by appointing Hajjar as institute head after a yearlong search. And while there are other institutes for gene and cell therapies at other academic medical centers, the GCTI is more focused on bringing the therapies to patients, rather than just studying the diseases themselves.
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The institute provides regulatory support—like seeking FDA approval for products, in manufacturing, and in clinical trial design. It’s difficult to do this within single academic labs, Yozwiak said, as the resources are “too intense,” between building a product, manufacturing it, developing a clinical trial, and finding investors.
“A really large, scalable system such as MGB can harness its power of scale to build an institute,” Yozwiak said, adding that the GCTI can build efficiencies that can bring the therapies out of academic labs and to patients.
Sparking innovation. Since late 2022, the institute’s biggest accomplishment has been launching its Spark Grant program, a new award to fund gene and cell therapy projects.
In October, the GCTI for the first time awarded six researchers $250,000 each to fund product development to focus on areas like deafness and Huntington’s disease. Award recipients have quarterly check-ins and meet with an internal advisory board of researchers with expertise in gaining external funding that can help bring the product to patients.
“We hope that in the next six months, we can have that first in-human trial,” Hajjar said.
The goal, Yozwiak said, is to get to a point where the researchers can seek external funding from the private market for their therapies. Four of the programs are already working on crafting pitch decks to raise external funds, he said.
“We view this program as a pipeline of opportunities—not only therapeutic opportunities, but also commercial opportunities,” Yozwiak said.
Financially, the GCTI is currently funded by MGB, but going forward, the health system expects the GCTI to become self-sufficient through revenue sharing.
A lot of hospitals have venture capital arms that are designed to create startups. While MBG has Mass General Brigham Ventures, the health system views the GCTI differently, considering it more of a patient-focused treatment generator rather than an organization designed to generate profit within the system.
“The long-term financial benefits of gene and cell therapies themselves have not been worked out yet and will be based on bio/pharma companies’ pricing, providers, patient acceptance, insurance/payers, and medical centers,” Hajjar said in an emailed statement.