Zunibergene Rocparvovec

Adeno-associated virus vector 4D-150 DNA (recombinant replication-defective adeno-associated virus vector 4D-150 cytomegalovirus immediate-early enhancer element plus chicken β-actin exon 1 plus intron 1 plus vascular endothelial growth factor C-specific microRNA gene plus chicken β-actin intron 1 fragment plus rabbit β-globin splice acceptor plus synthetic human VEGFR-1-VEGFR-2-IgG1-Fc fusion protein monomer precursor codon-optimized gene plus simian virus 40 polyadenylation signal plus adeno-associated virus 2 inverted terminal repeat flanks-specifying) recombinant non-replicating adeno-associated virus (AAV) vector with a retina-specific capsid variant, encoding a microRNA (miRNA) targeting expression of vascular endothelial growth factor C (VEGF-C) and a codon-optimised fusion protein that consists of VEGF-binding regions derived from the extracellular domains of human VEGF receptors 1 and 2 fused to the Fc portion of human immunoglobulin G1 (the mature expressed protein is equivalent to aflibercept), under control of a synthetic CAG promoter and the simian virus 40 polyadenylation sequence, flanked by AAV2 inverted terminal repeats. The VEGF-C targeting miRNA sequence is located within the chicken beta-actin intron of the CAG promoter zunibergene rocparvovec [INN]