The drug substance release specification includes tests for:
- Appearance: Colorless to slightly opalescent liquid, free of visible particulates.
- Identity: Confirmed by qPCR using primers specific to the RPE65 gene.
- Purity: Assessed by SDS-PAGE to confirm the VP1/VP2/VP3 protein profile.
- Potency: An in-vitro cell-based assay measuring the expression of the RPE65 protein in a target cell line.
- Vector Genome Titer: Measured by validated qPCR to ensure the correct concentration of viral vector.
- Safety: Tests for Sterility (USP <71>), Endotoxin (USP <85>), and Mycoplasma.
- Impurities: Limits for host cell protein and DNA.