Cell and gene therapy CDMO RoslinCT will commercially produce Vertex and CRISPR Therapeutics’ CRISPR/Cas9 gene-edited therapy exa-cel.
Vertex Pharmaceuticals has picked UK-based contract development and manufacturing organization (CDMO) RoslinCT – a spinout from the Roslin Institute – for commercial manufacturing of the world’s first CRISPR-based gene-edited therapy, Casgevy (exagamglogene autotemcel [exa-cel]).
“We have been working with Vertex […] on exa-cel for seven years on a very collaborative and open basis, and this relationship significantly predates the MHRA’s approval of exa-cel. Our expertise and capabilities with advanced therapies were, I believe, more significant here than the fact we are headquartered in the UK,” Peter Coleman, CEO of RoslinCT, told BioProcess Insider.
Casgevy won conditional marketing authorization for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) earlier this week.
“That said, the MHRA has shown real leadership here in the approval of the world’s first ever CRISPR-edited gene therapy, which is obviously positive for the UK.”
The CDMO will use its MHRA commercially approved facility in Edinburgh, Scotland to manufacture Casgevy and the site has already produced over 100 batches of the therapy. Coleman said it is “ready to go with this product” but, if necessary, it “will grow our capacity to meet expected demand.”
Vertex and CRISPR Therapeutics have collaborated for a number of years, but first partnered on exa-cel in April 2021, with Vertex paying CRISPR $900 million upfront for the commercialization rights. Exa-cel is an autologous, ex vivo CRISPR/Cas9 gene-edited therapy in which a patient’s hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin.
“Although the GMP production process is relatively short, the QA/QC testing is extensive and takes longer to ensure the product is safe before being reinfused back into the patient,” Coleman said.
Exa-cel is under review by the European Medicines Agency, the Saudi Food and Drug Authority, and the US Food and Drug Administration (FDA). The latter has given a Prescription Drug User Fee Act (PDUFA) target action date of December 8, 2023 for the sickle cell application and March 30, 2024 for the beta thalassemia application.
In August 2022, RoslinCT merged with Massachusetts-based Lykan Bioscience to bring customers a transatlantic footprint and combined expertise in cell and gene therapies (CGTs).
The entity will offer clients combined process and analytical development laboratories and cGMP manufacturing facilities. RoslinCT’s Edinburgh facility and a 64,000 square-foot facility in Hopkinton, Massachusetts, operated by Lykan, offers cell therapy manufacturing and development laboratories with a total of 16 cGMP processing suites following an expansion in 2021.