CRISPR Therapeutics AG announced that On December 15, 2023, it was announced that the European Medicines Agency?s Committee for Medicinal Products?for Human Use has adopted a positive opinion for the conditional approval of CASGEVY? (exagamglogene autotemcel), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease and transfusion-dependent beta thalassemia being jointly developed by CRISPR Therapeutics AG and its partner, Vertex Pharmaceuticals Incorporated. The use of CASGEVY in the European Union remains investigational.

An approval decision by the European Commission is expected in February 2024. Conditional marketing authorizations ("CMAs") are for medicines that fulfil a significant unmet medical need such as being for serious and life-threatening diseases, where no satisfactory treatment methods are available or where the medicine offers a major therapeutic advantage. A CMA is granted where comprehensive clinical data is not yet complete, but the benefit of the medicine to address a significant unmet need outweighs the need for data that will become available in the future.

CMAs are valid for one year and renewable annually with ongoing regulatory review of data.