RoslinCT, a cell and gene therapy contract development and manufacturing organization (CDMO), will manufacture the first ever U.S. FDA approved CRISPR-based gene therapy.

CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy, was approved by the FDA in December 2023 for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). 

RoslinCT and Vertex have worked closely on an adaptive basis as Vertex progressed with its clinical and regulatory development to advance this therapeutic.

Peter Coleman, UK CEO 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.”