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Thursday, 11/30/2017 10:34:10 AM

Thursday, November 30, 2017 10:34:10 AM

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As the CAR-T pioneers know only too well, working with patient cells in creating a personalized therapy is no easy task. In gene therapy you have to extract the cells, include a viral vector and corrective gene and get it back into the veins of patients in a matter of weeks. Any interruption along that process — or loss of quality — can be fatal for a gene therapy company like bluebird. And manufacturing issues in general remain one of the most significant threats to any biotech looking to take breakthrough products into the market, as the FDA has highlighted time and again in recent years.

Given the stakes, and the complexity, I asked Adams what keeps him up at night.

“The thing that keeps me up at night,” says Adams, “is that the overall supply chain for gene therapy products is so complex” it can be hard to make sure all the pieces are under control.

“I suspect that Derek is sleeping a little better now that we closed the deal,” says Walsh.

HTTPS://endpts.com/bluebird-bio-orchestrates-a-series-of-deals-and-a-buyout-to-guarantee-gene-therapy-manufacturing/
marcusl230 Nov '17 - 12:05 - 27708 of 27726 2 0 Edit
"This is really an important time in gene therapy," says Dr. David Williams, professor of pediatrics at Harvard Medical School and chief scientific officer at Boston Children's Hospital

HTTPS://www.npr.org/sections/health-shots/2017/11/29/565728869/gene-therapy-shows-promise-for-a-growing-list-of-diseases