The identity and structural integrity of voretigene neparvovec have been confirmed using orthogonal analytical methods:
- DNA Sequencing: The entire vector genome has been sequenced to confirm the identity and integrity of the ITRs, promoter, transgene, and polyA signal.
- Peptide Mapping and Mass Spectrometry: The identity and ratio of the three AAV2 capsid proteins (VP1, VP2, VP3) have been confirmed.
- Transmission Electron Microscopy (TEM): Used to confirm the expected morphology and size of the viral particles and to assess the ratio of full to empty capsids.