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RoslinCT will manufacture Vertex Pharmaceuticals’ CRISPR-based gene therapy, Casgevy, recently approved for treating sickle cell disease and β thalassemia.
On Dec. 11, 2023, RoslinCT, a UK-based cell and gene therapy contract development and manufacturing organization (CDMO), announced that it will manufacture Vertex Pharmaceuticals’ Casgevy (exagamglogene autotemcel [exa-cel]), a recently approved clustered regularly interspaced short palindromic repeats (CRISPR)-based gene therapy for treating sickle cell disease (SCD) and β thalassemia.
FDA approved Casgevy on Dec. 8, 2023 for treating SCD in patients 12 years and older in the United States, and the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) approved it for treating SCD and transfusion-dependent β thalassemia (TDT) in the UK on Nov. 16, 2023 (1). The gene therapy is also under review by FDA for treating TDT in the US, with a decision expected by March 20, 2024. In its press release, FDA stated that Casgevy is “the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy.” CRISPR technology, being so new, led to quite a stir when the MHRA approved Casgevy in November.
RoslinCT has been working with Vertex on an adaptive basis while Vertex moved through clinical and regulatory development of Casgevy. The collaboration has positioned RoslinRT in a place to support the manufacturing of a broad range of highly complex autologous and allogeneic cell therapies beyond Casgevy. The company runs manufacturing facilities in Edinburgh, Scotland and Boston, Mass., which are purpose-built for cell therapy products.
“We are delighted that patients in both the US and UK are now able to access this transformational medicine. This approval by [FDA] further demonstrates our … capabilities in development, support, and manufactur[ing] … cell and gene therapies such as Casgevy. We are proud to have supported Vertex in achieving this significant milestone and look forward to supporting their team as they bring the first approved CRISPR treatment to patients in the US, UK, and beyond.”
In addition to Casgevy, FDA also approved bluebird bio’s Lyfgenia (lovotibeglogene autotemcel) for treating SCD in patients 12 years and older on Dec. 8, 2023. Lyfgenia uses a lentiviral vector for genetic modification.
“Sickle cell disease is a rare, debilitating, and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Nicole Verdun, director of the Office of Therapeutic Products within FDA’s Center for Biologics Evaluation and Research (CBER), in the agency’s press release. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”
“These approvals represent an important medical advance with the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health,” said Peter Marks, director of CBER, in the release. “Today’s actions follow rigorous evaluations of the scientific and clinical data needed to support approval, reflecting [FDA’s] commitment to facilitating development of safe and effective treatments for conditions with severe impacts on human health.”
FDA granted both Casgevy and Lyfgenia priority review, orphan drug, fast track, and regenerative medicine advanced therapy designations.
1. Vertex Pharmaceuticals. Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia. Press Release, Nov. 16, 2023.