(INNS)
BOTARETIGEN SPAROPARVOVEK(INNC)
RECOMBINANT, NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 5 (RAAV5) VECTOR, ENCODING A TRUNCATED FORM OF HUMAN RETINITIS PIGMENTOSA GTPASE REGULATOR (RPGR) ISOFORM 6 (RPGR-ORF15), CONTAINING AN INFRAME DELETION OF 378 BASE PAIRS (BP) IN THE HIGHLY REPETITIVE PURINE-RICH REGION, PRECEDED BY A SIMIAN VIRUS 40 (SV40) INTRON, UNDER CONTROL OF A HUMAN RHODOPSIN KINASE (RK) PROMOTER AND TERMINATED WITH A POLYADENYLATION SIGNAL; FLANKED BY AAV2 INVERTED TERMINAL REPEATS (ITRS)(WHO)
BOTARETIGENUM SPAROPARVOVECUM(INN)