Lonza has inked a long-term agreement with Vertex to supply Casgevy (exagamglogene autotemcel), the world’s first CRISPR/Cas9 gene-edited cell therapy.
The partnership will see support from Lonza to manufacture Casgevy at its Geleen, Netherlands facility, with plans to expand production to its Portsmouth, New Hampshire site. Casgevy, a one-time treatment for eligible patients with transfusion-dependent beta-thalassemia or sickle cell disease, will be produced at Lonza’s state-of-the-art cGMP cell therapy facility.
The Geleen site is now approved by the FDA, EMA, and MHRA for the commercial manufacture of the therapy, marking a milestone in the product’s global rollout.
The drug was first approved in December 2023 for treating sickle cell disease, and it has now received FDA approval for transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.
Casgevy uses CRISPR/Cas9 technology to edit patients' genes, targeting the root cause of these blood disorders by modifying stem cells to produce healthy red blood cells. This one-time treatment offers a potentially curative approach for individuals suffering from chronic anemia caused by TDT.