RoslinCT To Manufacture First-Ever US FDA Approved CRISPR-Based Gene Therapy CASGEVY (Exagamglogene Autotemcel)

RoslinCT to manufacture this ground-breaking treatment for patients with Sickle Cell Disease (SCD) in the US and in the UK for SCD and Transfusion-Dependent Beta Thalassemia

RoslinCT, a cell and gene therapy Contract Development and Manufacturing Organisation (CDMO) dedicated to developing therapies for patients who live with serious and life-threatening diseases, will manufacture the first ever U.S. Food and Drug Administration (FDA) approved CRISPR-based gene therapy.

CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy, was approved by the U.S. FDA on 8 December 2023 for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). This builds on the world’s first authorisation of the treatment by UK’s Medicines and Healthcare products Regulatory Agency (MHRA) on 15 November 2023.

RoslinCT and Vertex have worked closely on an adaptive basis as Vertex progressed with its clinical and regulatory development to advance this world first therapeutic to patients. RoslinCT is uniquely positioned to support the manufacturing of a broad range of highly complex autologous and allogeneic cell therapies, with unparalleled expertise in gene editing and industry-leading induced Pluripotent Stem Cell (iPSC) capabilities. Its state-of-the-art manufacturing facilities in Edinburgh, Scotland and Boston, US are purpose-built for cell therapy products.

The FDA is also undertaking a standard review of Exa-cel for transfusion-dependent beta thalassemia due by 20 March 2024.

Peter Coleman, UK Chief Executive Officer of RoslinCT said: “We are delighted that patients in both the US and UK are now able to access this transformational medicine. This approval by the FDA further demonstrates our world leading capabilities in development, support and manufacture cutting-edge 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.”

About CASGEVY (exagamglogene autotemcel [exa-cel])
CASGEVY is a genome-edited cellular therapy consisting of autologous CD34+ hematopoietic stem cells (HSCs) edited by CRISPR/Cas9 technology at the erythroid-specific enhancer region of the BCL11A gene. CASGEVY is intended for one time administration via a hematopoietic stem cell transplant procedure where the patient’s own CD34+ cells are modified to reduce BCL11A expression in erythroid lineage cells, leading to increased fetal hemoglobin (HbF) production. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate vaso-occlusive crises for patients with SCD.

CASGEVY was granted a conditional marketing authorization in Great Britain by the U.K. Medicines and Healthcare products Regulatory Agency and by the National Health Regulatory Authority in Bahrain for patients 12 years of age and older with SCD characterized by recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia (TDT), for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related hematopoietic stem cell donor is not available. CASGEVY is currently under review by the European Medicines Agency and the Saudi Food and Drug Agency for both SCD and TDT.

The use of CASGEVY for the treatment of TDT in the U.S. remains investigational. Vertex has submitted a BLA to the U.S. FDA for the potential use of CASGEVY for patients 12 years and older with TDT and has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of March 30, 2024. About exagamglogene autotemcel (exa-cel).