RoslinCT, a renowned cell and gene therapy Contract Development and Manufacturing Organisation (CDMO), has announced a significant advancement in the field of medical science.
They will be manufacturing the world’s first CRISPR-based therapy, exa-cel, for Vertex Pharmaceuticals Inc. This development follows an extensive collaboration between the two entities.
Key Highlights:
- Innovative CRISPR-Based Therapy: Exa-cel, developed by RoslinCT, represents a groundbreaking step in medical treatment. It is the first therapy to use the Nobel Prize-winning CRISPR technology. This gene-editing tool has been instrumental in the development of exa-cel.
- Focus on Serious Diseases: Exa-cel is a new treatment for sickle cell disease and transfusion-dependent beta thalassemia. It was authorized by the UK Medicines and Healthcare products Regulatory Agency (MHRA) on November 15, 2023.
- Manufacturing Excellence: RoslinCT received a Manufacturer’s Authorisation License for commercial manufacturing of cell therapy products in March 2023. Their facilities in Edinburgh, Scotland, and Boston, US, are equipped with state-of-the-art technology for cell therapy product manufacturing.
- Expertise in Stem Cell Technology: RoslinCT, with its roots in the Roslin Institute, is known for its innovation in stem cell technology. They have extensive experience in gene editing and industry-leading induced Pluripotent Stem Cell (iPSC) capabilities.
- Impact on Patients: Peter Coleman, CEO of RoslinCT, emphasized their commitment to partnering with innovative companies to bring complex therapies to patients. Exa-cel is expected to have a significant impact on those suffering from sickle cell disease and beta thalassemia.
- UK’s Leading Role in Gene Editing Treatments: Steve Bates, CEO of the UK Bioindustry Association, highlighted the UK’s pioneering role in authorizing CRISPR-based therapies and its capacity to lead in providing access to gene editing treatments.
About RoslinCT:
RoslinCT has a rich history of innovation, tracing back to the cloning of Dolly the Sheep at the Roslin Institute in 1997.
They have been at the forefront of developing human medicines, particularly in the field of cell and gene therapies. Their facilities are designed to support the development and commercialization of these advanced therapies.
About Exagamglogene Autotemcel (Exa-cel):
Exa-cel works by editing a patient’s blood stem cells to produce functioning haemoglobin. This process involves extracting stem cells from bone marrow, editing them in a laboratory, and then infusing them back into the patient. The results of this treatment have the potential to be life-long.
This collaboration between RoslinCT and Vertex Pharmaceuticals marks a significant milestone in the field of gene therapy and showcases the potential of CRISPR technology in treating serious diseases.