Solvias reports that it will perform analytical release testing services on the world’s first CRISPR/Cas9 genome-edited cell therapy. The company signed a long-term agreement with Vertex Pharmaceuticals for CASGEVY™ (exagamglogene autotemcel or exa-cel) which received FDA approval for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises.
Solvias officials say the company has worked with Vertex for several years to develop and validate test methods that are critical for the final release of patients’ own edited cells so that they can be delivered back to patients. The companies’ collaboration included establishing the testing methods that will be scaled for commercializing CASGEVY. Solvias also has invested significantly in preparing one of its global facilities to support the commercial release work for this transformative therapy, according to Archie Cullen, CEO.
In addition to gaining FDA approval, CASGEVY recently received a positive opinion from the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use for conditional approval of the treatment of severe SCD and transfusion-dependent beta thalassemia (TDT).