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.”