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Thursday, September 21, 2017

Fall River lab working on cutting-edge gene therapy

September 18, 2017   Jennette Barnes





FALL RIVER — A gene therapy treatment manufactured at MassBiologics in Fall River could soon improve quality of life for people with advanced Parkinson’s disease.
The therapy is the first product to come out of the biological medicine laboratory’s Fall River location, according to Dr. Mark Klempner, executive vice chancellor for the nonprofit MassBiologics, which UMass Medical School oversees. The lab collaborates on the Parkinson’s project with Voyager Therapeutics, a gene therapy company in Cambridge.
He spoke to legislators and others who visited the lab Monday for an invitation-only tour.
“We’re making the medicine in here, right now, for the big clinical trial that is going to go on in the next year,” said Klempner, an infectious disease physician.
Existing Parkinson’s treatment typically works well for four to seven years, after which it becomes less effective, he said. At first, an enzyme in the brain converts L-dopa, the substance used to treat Parkinson’s, into dopamine. But over time, patients lose that enzyme and their symptoms get worse and worse.
The new therapy puts the information for that enzyme in a virus, and places that virus in the brain.
“They then take their dopa pills again, and voilĂ , they’re dramatically better,” he said.
The lab, located near Amazon in the SouthCoast Life Science and Technology Park, was originally slated to be a biomanufacturing incubator, but with financial support from the state, MassBiologics upgraded the facility to production standards. The Fall River location has about 30 employees. The lab’s main location is in Mattapan.
Visitors on the tour peered into a variety of rooms used to grow cells, purify viruses, and fill vials. They saw “clean rooms,” each with its own separate air supply designed to ensure that air is filtered not only on the way in, but also on the way out, since it can contain viruses.
Upstairs, visitors saw the row of massive ventilation systems necessary to keep the clean air flowing.
Sen. Michael Rodrigues, D-Westport, hosted the tour.
“It’s pretty cool, isn’t it?” he said. “Just fascinating. All here in Fall River.”
One of the city’s selling points for biomanufacturing is the availability of water, he said.
“We have excess capacity of water. A lot of places can’t say that, and they’d die to have water because it’s an important component for biomanufacturing,” Rodrigues said.
Dr. Michael Collins, a physician and chancellor of UMass Medical School, told visitors the school’s research work receives substantial funding from the federal government, and especially from the National Institutes of Health. State funding for the medical school has stayed stagnant for about 30 years, he said.
http://www.southcoasttoday.com/news/20170918/fall-river-lab-working-on-cutting-edge-gene-therapy

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