Memphis facility to make gene-editing treatment for sickle cell

By Aisling Mäki – Daily Memphian

January 14, 2024

A Southeast Memphis facility will manufacture the first FDA-approved gene-editing treatment for sickle cell disease.

Boston-based Charles River Laboratories operates a cell-therapy contract development and manufacturing organization (CDMO) at 4600 E. Shelby Drive, where it produces clinical and commercial cell and gene therapies.

In late December, the FDA cleared Charles River to begin manufacturing Vertex Pharmaceuticals’ Casgevy at the Memphis site. Vertex developed the therapy in collaboration with CRISPR Therapeutics.

Casgevy uses CRISPR gene-editing technology, which allows scientists to locate and modify specific DNA sequences within cells. The technology was recognized with the 2020 Nobel Prize in Chemistry.

The treatment targets severe sickle cell disease, a life-threatening blood disorder affecting about 100,000 people in the U.S. The condition causes red blood cells to become misshapen, leading to early cell death and reduced oxygen delivery throughout the body.

Casgevy works by editing a patient’s blood stem cells, which are then reinfused through a one-time stem cell transplant.

The therapy has a cost of approximately $2.2 million. Many patients with sickle cell disease rely on Medicaid, and questions remain about how broadly the treatment will be accessible.

Casgevy is the second commercially approved therapy produced at the Memphis facility, which dates back to the early 2000s. The site was originally developed by Cell Genesys, later became Cognate Bioservices in 2007, and was acquired by Charles River in 2021.

In 2022, the facility expanded its cleanroom manufacturing capacity by more than 50%. That same year, it became the first North American contract manufacturer approved by the European Medicines Agency to produce a commercial allogeneic T-cell therapy.

Charles River employs more than 350 people in Memphis across manufacturing, science, engineering, and regulatory roles, with plans for continued growth.