Pharma Focus Europe

RoslinCT poised to manufacture the inaugural FDA-approved CRISPR-based gene therapy, CASGEVY™ (Exagamglogene Autotemcel), in the United States

Tuesday, December 12, 2023

RoslinCT, a Contract Development and Manufacturing Organization (CDMO) specializing in cell and gene therapy, is set to produce the inaugural CRISPR-based gene therapy approved by the U.S. Food and Drug Administration (FDA).

CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy designed for the treatment of sickle cell disease (SCD) in patients aged 12 and above experiencing recurrent vaso-occlusive crises (VOCs), received FDA approval on December 8, 2023. This follows the initial authorization granted by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) on November 15, 2023.

Collaborating closely on an adaptive basis, RoslinCT and Vertex have played pivotal roles as Vertex advanced through clinical and regulatory development to introduce this groundbreaking therapy to patients. RoslinCT possesses a unique position in supporting the manufacturing of diverse autologous and allogeneic cell therapies, showcasing expertise in gene editing and leading capabilities in induced Pluripotent Stem Cell (iPSC). The organization's state-of-the-art manufacturing facilities in Edinburgh, Scotland, and Boston, USA, are purpose-built for cell therapy products.

Additionally, the FDA is currently conducting a standard review of Exa-cel for transfusion-dependent beta thalassemia, with a decision expected by March 20, 2024.

Peter Coleman, the UK Chief Executive Officer of RoslinCT, expressed delight at the approval, emphasizing the organization's leading role in developing, supporting, and manufacturing cutting-edge cell and gene therapies like CASGEVY. He underscored pride in supporting Vertex in achieving this significant milestone and anticipates further collaboration as they bring the first FDA-approved CRISPR treatment to patients in the U.S., UK, and beyond.

 

Source: globenewswire.com

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