Pharma Focus Europe

Roslinct and Vertex Pharmaceuticals Inc to Collaborate on Manufacturing Exagamglogene Autotemcel (Exa-cel) Following Extensive Development Partnership

Wednesday, November 29, 2023

RoslinCT, a Contract Development and Manufacturing Organization (CDMO) specializing in cell and gene therapy, has announced its involvement in manufacturing the world's first CRISPR-based therapy, exa-cel, as part of Vertex Pharmaceuticals Inc.'s global manufacturing network.

Collaborating closely with Vertex, RoslinCT has played a crucial role in the adaptive development of exa-cel, a groundbreaking treatment for sickle cell disease and transfusion-dependent beta thalassemia. The therapy received authorization from the UK Medicines and Healthcare products Regulatory Agency (MHRA) on November 15, 2023. In March 2023, RoslinCT obtained a Manufacturer's Authorization License (MIA) from MHRA for the commercial manufacturing of cell therapy products.

With a history rooted in stem cell technology dating back to its origin at the Roslin Institute in Edinburgh, RoslinCT has maintained a leading position in healthcare and scientific advancements. The organization possesses extensive expertise in process development, cGMP manufacturing, gene editing, and induced Pluripotent Stem Cell (iPSC) capabilities. Its state-of-the-art manufacturing facilities in Edinburgh, Scotland, and Boston, US, are designed specifically for cell therapy products.

RoslinCT, emphasized the organization's commitment to being a strategic partner for innovative companies, facilitating the delivery of cutting-edge therapies. He highlighted the significance of RoslinCT's role in manufacturing the world's first CRISPR-based therapy and expressed excitement about the broader potential to enhance their capabilities through collaboration with Vertex.

Steve Bates, CEO of the UK Bioindustry Association, recognized the UK's leading position in authorizing a CRISPR-based therapy and acknowledged RoslinCT as a prime example of innovation in the country. He emphasized the UK's ability to lead in providing access to gene editing treatments and praised RoslinCT for contributing to the global advancement of impactful medicines.

RoslinCT's collaboration with Vertex in manufacturing exa-cel marks a significant milestone in the field of cell and gene therapy, showcasing the organization's expertise and commitment to advancing innovative treatments for serious and life-threatening diseases.



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