Vertex adds Lonza to the Casgevy sales network

Autologous, ex vivo CRISPR/Cas9 Gene Therapy Casgevy Receives Approval From US Food and Drug Administration (FDA) in December 2023 to treat sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).

However, similar to other advanced therapy deploymentsthe rollout has been slow, with Vertex and its partner CRISPR Therapeutics not treating any patients in the six months following launch. But now that the therapy is gaining momentum (about 20 patients have had cells collected during the second quarter of 2024), Vertex announced a long-term supply agreement for production of the therapy with contract development and manufacturing organization (CDMO) Lonza.

Under the terms of the agreement, Lonza will manufacture Casgevy from its facilities in Geleenin the Netherlands, with a second location in Portsmouth, New Hampshire, expected to come online in 2025.

The news broke when Ciaran Brady, vice president of manufacturing science and technology (MS&T) at Vertex Pharmaceuticals, took the plenary stage at BPI East – part of Biotech Week Boston (BWB) – the last week.

Although he did not specifically reference Lonza, he told delegates that Vertex’s manufacturing strategy was based on a centralized model. “We have sites in the United States and Europe where we send these cells to complete the manufacturing process. We are currently expanding our network to have more sites around the world (…) close to where patients are.

Brady admitted that although Casgevy has been approved for about nine months, “we’ve only really gotten to the starting line.” This has given us the opportunity to reach patients, but we still have a long way to go because it is a very nascent platform and technology.

Beyond Lonza (with whom Vertex already has a partnership to support its portfolio of cellular therapies against type 1 diabetes), Vertex is work with Charles River Laboratories for the production of Casgevy from a plant in Memphis, Tennessee.

By using an outsourced model, he said Vertex was adding capacity through a “very flexible network”, which is key to avoiding overcapacity issues that would significantly reduce potential profits. “We need to find the right size of capacity to meet demand so we don’t leave these large factories idle and pass those costs onto the product. This is a journey we are currently undertaking.

Additionally, Brady emphasized the need to scale up its processes “to get as many batches as possible from as small a facility as possible and in a very controlled manner,” citing that improved and maximized yields are essential to balancing manufacturing costs. ‘such a product.

Talk with Introduced to bioprocesses After the presentation, Brady spoke in more detail about the challenges of balancing relatively unknown demand while not incurring the enormous overhead costs of overcapacity. “We are still in the early stages. We have a lot to do, but so far so good.