Vertex enlists Lonza for global production of CRISPR therapy Casgevy

Vertex Pharmaceuticals is strengthening its gene-editing therapy supply chain with a significant new partnership. The company has signed a long-term agreement with Lonza, a major Swiss manufacturer, to produce the global commercial supply of Casgevy. This announcement, made on Tuesday, highlights Lonza's role in manufacturing the therapy at its cell therapy facilities in Geleen, Netherlands. Plans are also in motion to expand production to Lonza's U.S. facility in Portsmouth, New Hampshire, by next year.

Currently, the Geleen facility has obtained good manufacturing practices (GMP) licenses from the FDA, the European Medicines Agency (EMA), and the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA). However, the Portsmouth site is not expected to begin operations until the following year. Financial specifics of this agreement remain undisclosed.

Dr. Morrey Atkinson, Executive Vice President and Chief Technical Operations Officer at Vertex, emphasized the complexity of manufacturing a pioneering therapy like Casgevy. He praised Lonza for their excellent partnership and advanced technological capabilities, which are crucial for ensuring that Casgevy reaches the patients who need it.

Casgevy, a shared venture between Vertex and CRISPR Therapeutics, marked a significant milestone late last year by securing approvals in both the U.K. and the U.S. as a potential one-time treatment for sickle cell disease (SCD), a severe blood disorder. Shortly after this achievement, the therapy also gained FDA approval for treating transfusion-dependent beta thalassemia. Notably, Casgevy is the first approved medicine utilizing the CRISPR gene-editing system, a breakthrough that earned its inventors a Nobel Prize in 2020.

To maintain a steady supply of Casgevy, Vertex has also joined forces with leading contract development and manufacturing organizations (CDMOs) Charles River Laboratories and RoslinCT. RoslinCT is affiliated with the Roslin Institute, renowned for cloning Dolly the Sheep in 1996.

Although Casgevy's launch is still in its early stages, Vertex expressed satisfaction with the drug's commercial progress when it reported its second-quarter earnings in August. At that point, approximately 20 patients had started their treatment journey through the cell collection process, indicating promising initial uptake for what Vertex describes as a "potential multi-billion opportunity." The one-time therapy is priced at $2.2 million.

In summary, with the support of Lonza and other prominent CDMOs, Vertex is well-positioned to ensure the global availability of Casgevy. This groundbreaking therapy not only showcases the potential of CRISPR gene-editing technology but also offers new hope for patients suffering from debilitating blood disorders.