Following a string of approvals in the United States and beyond, Vertex Pharmaceuticals continues to build out its manufacturing network for the world’s first CRISPR-based gene therapy, Casgevy.
Monday, Vertex’s production partner Charles River Laboratories announced that its Memphis manufacturing facility has snared regulatory approval to manufacture Vertex and CRISPR Therapeutics’ Casgevy, which picked up an FDA nod in sickle cell disease earlier this month.
The production nod follows back-to-back audits from both the U.S. FDA and the Health Products Regulatory Authority (HPRA) under the European Medicines Agency (EMA), Charles River said in a release.
As for Casgevy, the gene-editing therapy made history this year when it picked up a world-first nod in the United Kingdom in late November. Not long after that, the CRISPR-based medicine passed muster with regulators in the U.S. Meanwhile, just last week, Casgevy picked up a positive opinion from the EMA.
The European agency recommended an approval for Casgevy in transfusion-dependent beta thalassemia and severe sickle cell disease in patients ages 12 and up who are eligible for a stem cell transplant but who don’t have a suitable donor.
Sickle cell disease causes blood cells to fold into crescent shapes, which makes them more likely to collect in blood vessels, depriving the muscles of oxygen. The disorder leaves patients susceptible to a host of other complications such as anemia, jaundice, infections, gallstones and organ damage.
Manufacturing of Casgevy is no easy feat, with each batch uniquely tailored to the individual patient. But the therapy is intended to be a one-time treatment, replacing the cumbersome process of treatment via blood transfusions.
Elsewhere on the manufacturing front, Vertex has also enlisted Scottish stem cell pro RoslinCT for commercial production duties on Casgevy. The company, which is the CDMO arm of the famed Roslin Institute, boasts two manufacturing facilities—one in Edinburgh, Scotland, and the other on the outskirts of Boston.