RoslinCT to manufacture world’s-first CRISPR-based therapy

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, announces that it will manufacture the world’s first CRISPR-based therapy, exa-cel, across the world as part of the global manufacturing network for Vertex Pharmaceuticals Inc.

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 has a rich history of innovation in stem cell technology, dating back to its genesis at the Roslin Institute in Edinburgh.

Exa-cel is a new treatment for sickle cell disease and transfusion-dependent beta thalassemia, authorised by the UK Medicines and Healthcare products Regulatory Agency (‘MHRA’) on 15 November 2023. The MHRA granted RoslinCT a Manufacturer’s Authorisation License (‘MIA’) for commercial manufacturing of cell therapy products in March 2023.

RoslinCT has maintained its position at the forefront of healthcare and scientific advancements and has extensive process development expertise and cGMP manufacturing capability across a broad range of autologous and allogeneic cell therapies, with unparalleled expertise in gene editing and industry-leading induced Pluripotent Stem Cell (‘iPSC’) capabilities, giving it the ability to deliver this unique therapy. Its state-of-the-art manufacturing facilities in Edinburgh, Scotland and Boston, US are purpose-built for cell therapy products.

Peter Coleman, Chief Executive Officer of RoslinCT said: “RoslinCT prides itself in being a thoughtful and practical partner to the world’s most innovative companies, helping them bring cutting-edge and often highly complex therapies to patients. Being one of the manufacturing sites for the world’s first therapy using the Nobel Prize winning technology, CRISPR, is another example of this skillset.

“RoslinCT has deep roots in stem cell technology and decades of expertise and excellence in process and cGMP capability making us a perfect partner to support a highly complex project like this. We feel privileged to be working alongside Vertex to bring this ground-breaking therapy to patients and are excited about the broader potential to deepen and enhance our skillset.”

Steve Bates, Chief Executive Officer of the UK Bioindustry Association said: “As the first country to authorise a CRISPR-based therapy and home to one of the key manufacturing sites, the UK is well positioned to lead on providing access to gene editing treatments. We also already have in place the NHS innovative medicine fund as an explicit policy route to enable rapid adoption of innovation.

“RoslinCT is an exemplar of the tremendous innovation being built in the UK and is at the heart of the world leading, medicine manufacturing community, not only making exa-cel for Vertex, but supporting companies across the world to translate innovation into impactful medicines.”

Exa-cel is a new treatment for sickle cell disease and transfusion-dependent beta thalassemia, authorised by the MHRA on November 15, 2023. It is the first medicine to be licensed that uses the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020. Exa-cel is designed to work by editing a patient’s blood stem cells so that the body produces functioning haemoglobin. To do this, stem cells are taken out of bone marrow, edited in a laboratory and then infused back into the patient after which the results have the potential to be life-long.


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