Non-replicating recombinant vector derived from adeno-associated virus AAV6 serotype, containing a codon-optimised version of the human Factor VIII gene
Non-replicating recombinant vector derived from adeno-associated virus AAV9, lacking all AAV viral genes and containing a miniaturized version of the human dystrophin gene
The nOPV2 candidate strains include different combinations of 5 distinct modified regions of the Sabin-2 genome, including changes to the RNA sequence in the 5’ untranslated region of polio genome (5’ UTR), the capsid protein coding region (P1), the non-structural protein 2C, and the polymerase 3D. Of these modifications, only the changes to polymerase 3D result in a change in the amino acid sequence. The rest of the modifications aim to stabilize the genetic sequence against reversion in either the 5’ UTR or capsid regions.