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The first regulatory submission for a CRISPR-based therapy has been validated by the European Medicines Agency, to treat sickle cell disease and transfusion-dependent beta thalassemia.
Reblozyl, the first erythroid maturation agent showed increased haemoglobin in transfusion-dependant patients with myelodysplastic syndromes.
In this article, experts from Charles River Associates discuss how the introduction of cell and gene therapies will impact upon various aspects of the healthcare system, from provision of care to delivery and supply, and pricing and market access.
Adam Pearson, Senior Oncology Analyst at GlobalData, comments on the development of CRISPR-based therapies and outlines potential opportunities and challenges in the space.
After regulatory approval, Bristol-Myers Squibb has announced its successful acquisition of Celgene, completing the merger.
