AAV-mediated delivery of therapeutic antibodies to the inner ear
AssigneeAkouos, Inc.
InventorsEmmanuel John Simons, Robert Ng, Michael McKenna
Life of this patent
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Abstract
Provided herein are methods that include introducing into an inner ear of a mammal a therapeutically effective amount of an adeno-associated virus (AAV) vector that includes a nucleotide sequence encoding (a) a polypeptide including an antibody heavy chain variable domain operably linked to a signal peptide and a polypeptide including an antibody light chain variable domain operably linked to a signal peptide; (b) a polypeptide including an antigen-binding antibody fragment operably linked to a signal peptide; or (c) a soluble vascular endothelial growth factor receptor operably linked to a signal peptide.
Claims25
An adeno-associated virus (AAV) vector comprising a nucleotide sequence which comprises:
- (i) a first coding sequence that encodes a first polypeptide comprising an antibody heavy chain variable domain operably linked to a first signal peptide, and a second coding sequence that encodes a second polypeptide comprising an antibody light chain variable domain operably linked to a second signal peptide,
- wherein the sequence encoding the antibody heavy chain variable domain consists of SEQ ID NO:
- 52, and the sequence encoding the antibody light chain variable domain consists of SEQ ID NO:
- 53, and
- (ii) a sequence encoding a thosea asigna virus 2A (T2A) peptide that is present between the first coding sequence and the second coding sequence,
- wherein the first and second polypeptides together specifically bind to one or more mammalian VEGF proteins.
A composition comprising an adeno-associated virus (AAV) vector and a pharmaceutically acceptable carrier,
- wherein the AAV vector comprises a nucleotide sequence comprising:
- (i) a first coding sequence that encodes a first polypeptide comprising an antibody heavy chain variable domain operably linked to a first signal peptide, and a second coding sequence that encodes a second polypeptide comprising an antibody light chain variable domain operably linked to a second signal peptide,
- wherein the sequence encoding the antibody heavy chain variable domain consists of SEQ ID NO:
- 52, and the sequence encoding the antibody light chain variable domain consists of SEQ ID NO:
- 53, and
- (ii) a sequence encoding a thosea asigna virus 2A (T2A) peptide that is present between the first coding sequence and the second coding sequence,
- wherein the first and second polypeptides together specifically bind to one or more mammalian VEGF proteins, and
- wherein the composition is formulated for intra-cochlear administration.
Dependencies are inferred from the claim text.