Struggling gene therapy pioneer uniQure brings out the ax, chopping programs and staffers in overhaul

UniQure was able to snare the world’s first approval for a gene therapy. But it wasn’t able to cash in. And with its top pipeline programs struggling against heavyweight competition, the Dutch/US biotech $QURE today took out the ax and started to chop away at its R&D structure in search of some big cost savings.

The ax is being aimed at R&D in The Netherlands as uniQure plans to consolidate manufacturing in the US, where it has facilities in Lexington, MA. Up to 60 jobs, a quarter of its workforce, will be cut in the reorganization.

UniQure is looking to drop AMT-110 for Sanfilippo B, axing its focus on that disease along with Parkinson’s as it reviews its options. The top priorities now: hemophilia B, Huntington’s disease and its collaboration with Bristol-Myers Squib in cardiovascular disease.

As part of the company’s plan, Deya Corzo, M.D., senior vice president of the liver and metabolic therapeutic area, and Charles Richard, M.D., Ph.D., senior vice president of the central nervous system therapeutic area, are on the way out of uniQure and a shrunken R&D group in Amsterdam will move into new digs next year.

The retrenching comes after uniQure has faced off against some tough competition in hemophilia B, with Spark Therapeutics and others advancing rival programs.

Trouble has been brewing at uniQure for some time. It has one gene therapy approved in Europe, but no market for it. In September, the biotech’s new CEO, Dan Soland, left abruptly after only 9 months on the job. And that changeup came a few months after uniQure fared poorly in the latest beauty contest among the gene therapies being developed for hemophilia B. A crowded field, Spark Therapeutics drew praise for achieving 27% to 35% Factor IX (FIX) activity among 4 patients, while uniQure had to settle for a 2% to 6.3% range for a single infusion of AMT-060 — though Spark recently experienced a setback of its own. Two months later Shire opted to drop its rival gene therapy in the field, the one-time leader BAX 335, in favor of a preclinical effort after analysts panned the variable results the program had posted.

Matthew Kapusta, interim chief executive officer of uniQure, had this to say:

“Along with our investigators and collaboration partner, we are enthusiastic about the interim data from our ongoing Phase I/II study of AMT-060 in hemophilia B and will allocate necessary resources to expedite bringing AMT-060 to market, with commercial manufacturing capabilities in our state-of-the-art U.S. facility which is already in place.”

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Recursion Pharmaceuticals Salt Lake City, UT
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Atlas Venture Cambridge, MA

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