Aav variants for treatment of complement disorders
Assignee4D Molecular Therapeutics Inc.
InventorsMelissa Calton, Roxanne CROZE, Albena KANTARDZHIEVA
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Abstract
Recombinant AAV (rAAV) comprising a variant adeno-associated virus (AAV) capsid and a transgene encoding a human factor H variant are provided. Also provided are methods of delivering the transgene to the retina and methods of treating dry age-related macular degeneration and geographic atrophy secondary to age-related macular degeneration disorders by contacting retinal cells with the rAAV.
Claims44
A recombinant adeno-associated virus (rAAV) comprising (i) a variant AAV capsid protein comprising a heterologous peptide with a length of 7, 8, 9, 10 or 11 amino acids covalently inserted in the GH-loop of the capsid protein relative to a corresponding parental AAV capsid protein, wherein the peptide insertion comprises the amino acid sequence ISDQTKH (SEQ ID NO:1) and (ii) a heterologous nucleic acid comprising a nucleotide sequence encoding a complement regulator factor H (CFH) protein or a fragment thereof.
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