Vertex Enlists Lonza for Gene Therapy Production

Lonza has entered into a long-term contract with Vertex to produce Casgevy (exagamglogene autotemcel), the pioneering CRISPR/Cas9 gene-edited cell therapy. This collaboration will utilize Lonza's Geleen, Netherlands facility, with future plans to extend production to their Portsmouth, New Hampshire site. Casgevy serves as a one-time treatment for patients diagnosed with transfusion-dependent beta-thalassemia or sickle cell disease and will be manufactured at Lonza’s advanced cGMP cell therapy center.

The Geleen facility has now received approval from key regulatory bodies, including the FDA, EMA, and MHRA, for the commercial production of Casgevy, signifying a significant step in the therapy’s global distribution efforts. The drug initially gained approval in December 2023 for treating sickle cell disease and has subsequently been authorized by the FDA for use in managing transfusion-dependent beta thalassemia (TDT) in patients aged 12 years and older.

Casgevy employs CRISPR/Cas9 technology to alter patients' genes, directly addressing the underlying cause of these blood disorders by modifying stem cells to generate healthy red blood cells. This single-dose treatment offers a potentially curative solution for those suffering from chronic anemia due to TDT.