Following the historic approval of the world’s first CRISPR-based gene-editing therapy in the United Kingdom this month, Vertex Pharmaceuticals has tapped its long-time manufacturing partner RoslinCT for commercial production duties near and far.
RoslinCT, the CDMO arm of the Roslin Institute—which famously cloned Dolly the Sheep back in 1996—is set to manufacture Vertex and CRISPR Therapeutics’ exa-cel “across the world” as part of Vertex’s global production network, the company said in a press release Tuesday.
Exa-cel was recently approved in the U.K. under the name Casgevy to treat patients with severe forms of sickle cell disease and transfusion-dependent beta thalassemia. Analysts at Leerink Partners noted in mid-November that the green light represents a “historic milestone for gene editing.”
While several unknowns still surround the drug’s launch, especially concerning the gene-editing therapy’s price, Vertex has said its drug could potentially open treatment up to 2,000 patients in the U.K.
RoslinCT, for its part, has been ready to roll since the spring. Back in March, the company picked up a Manufacturer’s Authorization License for commercial production of cell therapies from the U.K.’s biopharma regulatory body.
Vertex’s manufacturing deal with RoslinCT marks the "exciting" culmination of a seven-year journey that began with CRISPR Therapeutics, the Scottish CDMO’s chief executive officer, Peter Coleman, said in an interview. The deal advances Roslin, which specializes in production of cell and gene therapies, to the “brink of commercial manufacturing” for Vertex and CRISPR’s Casgevy.
“I’ve been in this industry for 20 years and I’ve seen lots of products come and go,” Coleman said, “but to see a process start from the very early beginnings and see Roslin grow with the product to where we are now—it’s extremely exciting.”
Currently, RoslinCT boasts two manufacturing facilities—one in Edinburgh, Scotland, and the other on the outskirts of Boston. When it comes to cranking out exa-cel, RoslinCT has a commercial license to manufacture the product from its “BioCube” in Edinburgh, Coleman explained.
Should Vertex and CRISPR snare further approvals for exa-cel, RoslinCT is in line to “deliver products all over the world,” the CEO added.
As for how exa-cel is made, each batch is uniquely tailored to the individual patient, Coleman explained. First, blood is drawn from the patient and shipped to Roslin’s facility in Scotland, where, over the course of six-to-seven days, the CDMO extracts the patient’s bone marrow stem cells for the subsequent gene-editing phase. Once the patient’s faulty genes are corrected, vials of exa-cel are frozen before several weeks of testing. After that step is complete, the bespoke exa-cel dose is ready to be delivered back to the patient.
Set up as the production arm of the Institute, RoslinCT snared a manufacturing license in 2012 and set out as a contract development and manufacturing organization (CDMO) in 2014, Coleman said.
Over the past five years, the organization has “grown substantially,” swelling from an original 30 people to more than 350 employees today, he added.
RoslinCT operates sites in Scotland, as well as the United States, where the company last year acquired a facility in Hopkinton on the edge of Boston. Across both facilities, Roslyn boasts 22 manufacturing suites—14 in the U.S. and eight in U.K.—Coleman explained.