When Mass General Brigham launched its Gene and Cell Therapy Institute last year, the hospital giant staked a claim in the biotech research ecosystem.
One year in, the institute and its selected research projects say they're seeing success toward its goal of shortening the timeline from research project to patients care.
The institute is working with selected researchers to make their way to first-in-human trials, with a system in place that is designed to address the so-called “translational canyon” in the development process, said Nathan Yozwiak, head of research at the GCTI.
The institute was launched in December 2022 to centralize the work from hundreds of researchers at MGB's five academic medical centers, but the official research kickoff in October marked the start of the 12-month research and funding period. Six projects were selected to receive an even cut of the $1.5 million in Spark grants doled out by the institute.
The six selected projects spanned three of MGB's centers. The awardees are:
- Dr. Artur Indzhykulian, Mass Eye and Ear, researching ways to restore hearing
- Casey Maguire, Massachusetts General Hospital, researching ways to cross the blood-brain barrier
- Robert Manguso, Massachusetts General Hospital, researching antigen receptors
- Dr. Ricardo Mouro, Massachusetts General Hospital, researching new CRISPR editing therapeutics
- Dr. William Renthal, Brigham and Women’s, researching gene therapy for chronic pain management
- Nicholas Todd, Brigham and Women’s, researching use of focused ultrasound to cross the BBB
For Todd, the 12-month period is an opportunity to get enough data to use as a springboard for what’s next.
Todd’s research is addressing one potential way to get through the blood-brain barrier, an area of medicine that is a major unsolved challenge, to better treat neurological disorders. Typically, an invasive neurosurgical procedure is necessary, said Todd. Finding a way to deliver a drug systemically and have it reach the brain would be an innovation, and Todd is using a method involving focused ultrasound to temporarily make the BBB more permeable. The platform technology could be applied to a number of diseases.
The gene and cell therapy institute funding has made it possible to quickly turn the idea into an active project, he said. Currently, Todd is combining the platform with a specific vector developed by researcher Feng Feng Bei at Brigham and Women’s. Within the year, Todd says they’ll be able to have the data they need to get more money, and they’ll be able to be published academically along the way to spur advancement in the field among other researchers.
The first investments targeted projects that showed most promise in getting to trials. The selection process involved determining which projects were almost there, but needed a boost.
“We’re focused on getting our patients the most advanced and most cutting-edge care. Commercialization sometimes enhances the first human trials and getting treatment to patients,” said Dr. Roger Hajjar, the head of the GCTI.
First-in-human trials are where patients can finally access treatments that are often years in the making. That’s where the GCTI wants to be making its investments, said Yozwiak.
The projects are being advised by a group at MGB that acts like a board of directors. By the time the 12-month period ends, they will know what the next step is for their project, whether it be seeking more external funding or spinning off into a new company.
The board-like advisors help researchers build manufacturing relationships and meet with the U.S. Food and Drug Administration.
“We want to bring expertise from the biotech industry to share what it takes to bring an idea to the next stage,” said Yozwiak.
The institute will follow a similar model for the next year, he said.
