Casgevy is the first cell therapy based on the CRISPR/Cas9 technology, discoverers of which were awarded the Nobel Prize in Chemistry in 2020. It offers the potential of a one-time treatment for eligible patients with transfusion-dependent beta-thalassemia or sickle cell disease.

Under the terms of the agreement, Lonza will manufacture casgevy for Vertex at the state-of-the-art cGMP cell therapy manufacturing facilities in Geleen (NL), with plans to expand to its Portsmouth (US) facility.

The Geleen (NL) cell manufacturing facility was recently granted a GMP license by the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), and Medicines and Healthcare Products Regulatory Agency (MHRA). The Portsmouth (US) site is expected to begin GMP operations in 2025.

“It is a privilege to work with Vertex on bringing its innovative and cutting-edge medicines to patients suffering from life-threatening diseases,” commented Daniel Palmacci, President, Cell & Gene, Lonza. “We are also pleased to reach a significant milestone towards supporting the commercial manufacture of casgevy by receiving the regulatory approval at our state-of-the-art cell therapy manufacturing site in Geleen.” 

“Manufacturing a first-of-its-kind therapy like casgevy is complex and requires advanced technology and capabilities,” added Morrey Atkinson, Executive Vice President and Chief Technical Operations Officer, Head of Biopharmaceutical Sciences and Manufacturing Operations, Vertex. “Lonza team have been excellent partners as we have invested in our global manufacturing network to ensure casgevy will be available for the patients who need it. We look forward to our continued collaboration.”