- Lonza to manufacture CASGEVY®, the world’s first CRISPR/Cas9 gene-edited cell therapy, for global commercial supply
- CASGEVY® will be manufactured at Lonza’s Geleen (NL) cell therapy manufacturing facility with plans to expand to Portsmouth, NH (US)
- Geleen (NL) site is now approved by the FDA, EMA and MHRA for the commercial production of CASGEVY®
Lonza, a global manufacturing partner to the pharmaceutical, biotech and nutraceutical markets, announced today the signature of a long-term commercial supply agreement for CASGEVY® (exagamglogene autotemcel).
CASGEVY® is the first cell therapy based on the CRISPR/Cas9 technology, discoverers of which were awarded the Nobel Prize in Chemistry in 2020. CASGEVY® 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, Vertex will leverage Lonza’s scientific, regulatory and manufacturing expertise, global manufacturing network, and first-hand experience in the commercial manufacture of cell therapy products. Lonza will manufacture CASGEVY® 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.
Daniel Palmacci, President, Cell & Gene, Lonza, commented: “It is a privilege to work with Vertex on bringing its innovative and cutting-edge medicines to patients suffering from life-threatening diseases. 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.”
Morrey Atkinson, Ph.D., Executive Vice President and Chief Technical Operations Officer, Head of Biopharmaceutical Sciences and Manufacturing Operations, Vertex, added: “Manufacturing a first-of-its-kind therapy like CASGEVY® is complex and requires advanced technology and capabilities. The 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.”
