Biomay Announces its Successful Support of Approval of First CRISPR/Cas9-based Therapy

Biomay, a biotech contract development and manufacturing organization based in Vienna, Austria, announces successful completion of an FDA inspection qualifying Biomay as a cGMP manufacturer and supplier of recombinant nuclease Cas9 for use in gene editing therapies.

As part of an ongoing cooperation initially dating back to 2017, Biomay has manufactured and delivered Cas9 in the course of Vertex´and CRISPR Therapeutics’ clinical development of exagamglogene autotemcel (CASGEVY®), a gene edited therapy for the treatment of sickle cell disease (SCD) and transfusion dependent beta-thalassemia (TDT). CASGEVY® is the first CRISPR/Cas9-based therapy to receive marketing approval from the FDA, MHRA and positive CHMP opinion from the EMA.

Biomay has developed and validated a Cas9 manufacturing process together with the associated analytical quality control methods. This included the construction of an E. coli expression system, GMP cell banking, upstream and downstream development, as well as the establishment of a comprehensive set of analytical quality control methods. An in-depth product and process characterization program, analytical method validation, as well as full manufacturing process validation have been performed. Recently, the U.S. FDA completed a Pre-License Inspection of Biomay´s facilities and systems in the context of Vertex’s Biologics License Applications (BLAs) for exagamglogene autotemcel (CASGEVY®).

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