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Wednesday, November 27, 2019

Vertex plans major Boston expansion to support gene, cell therapy ambitions

Vertex Pharmaceuticals is preparing to grow even bigger—another 256,000 square feet bigger, to be exact.
The drugmaker is in advanced talks to lease a building in Innovation Square, a research campus in Boston’s Raymond Flynn Marine Industrial Park, The Boston Globe reported.
The target is the entire second phase of the new R&D hub that developer Related Beal is building on the South Boston waterfront. It’s close to Vertex’s existing 1.1 million-square-feet Fan Pier headquarters and would serve as a research and manufacturing facility for gene and cell therapies, according to the newspaper.
Vertex scoured the greater Boston region for a new foothold, including sites in Cambridge, Waltham and Watertown, but picked the Innovation Square because it’s one of the most advanced projects in the neighborhood, on track to open in 2021, Vertex CEO Jeffrey Leiden reportedly said.
Expansion at the Innovation Square comes as the biotech giant diversifies beyond its fundamental cystic fibrosis business and into the burgeoning gene and cell therapy arena.
“The question became how are we going to grow those programs if we’re running out of space at Fan Pier?” said Leiden, as quoted by the Globe. “The answer is a new building.”
Leiden is transitioning to executive chairman, handing the baton to Chief Medical Officer Reshma Kewalramani. But before he moves up, a blueprint for Vertex’s future growth has been laid out.

In June, Vertex put down $245 million upfront to acquire Exonics and its gene editing technology, which uses CRISPR to repair dystrophin, the protein missing in patients with Duchenne muscular dystrophy (DMD). At the same time, it shelled out $175 million upfront to deepen its ties with CRISPR Therapeutics, also for using CRISPR-Cas9 to develop DMD and myotonic dystrophy Type 1 therapies.
The first project coming out of the CRISPR-Vertex partnership has just shown promise. CTX001, a CRISPR-based therapy for severe blood disorders marked by abnormal hemoglobin, helped a beta thalassemia patient live without transfusions for nine months, and a sickle cell patient was free of the painful vaso-occlusive crises after four months, the pair unveiled last week.
Vertex also agreed to pay $950 million to snatch up Semma Therapeutics and its stem cell treatment for Type 1 diabetes.

The new building Vertex plans to lease will house 300 to 400 people, including employees from Exonics and Semma, as well as new hires, Leiden said, according to the Globe. Besides the lab and office space at its Fan Pier HQ, Vertex also has a lease for about 100,000 square feet of space in the Marine Industrial Park for “certain logistical and laboratory operations and manufacturing equipment,” the company’s annual securities filing shows.
On the company’s third-quarter earnings call in October, Leiden said the company will continue to do deals on early-stage assets, especially bolt-on deals to further its gene editing strategy.
Meanwhile, the cystic fibrosis franchise will continue to provide Vertex’s revenue backbone for some time. Last month, the company won FDA approval for Trikafta, a triple combo designed to treat cystic fibrosis patients with a mutated delF508 gene, which is found in 90% of the U.S. cystic fibrosis population.

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