Aav vector encoding diamine oxidase and uses thereof
AssigneeOyster Point Pharma, Inc.
InventorsEric C. CARLSON, Jeffrey Alan NAU
Life of this patent
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Abstract
Provided are recombinant adeno-associated virus (rAAV) vectors, methods of treating a condition (e.g., an ocular condition), pharmaceutical compositions, and other compositions and methods, in which the rAAV vector comprises a polynucleotide encoding diamine oxidase (DAO1). Methods of treatment may include administration to the lacrimal gland.
Claims95
A recombinant adeno-associated virus (rAAV) vector comprising an AAV capsid and an expression cassette, the expression cassette comprising a polynucleotide encoding diamine oxidase (DAO1), operatively linked to a promoter.
A composition comprising an rAAV vector, wherein the rAAV vector comprises:
- (a) an AAV capsid, and
- (b) an expression cassette, wherein the expression cassette comprises a polynucleotide comprising a nucleotide sequence sharing at least 95% identity to a nucleotide sequence selected from SEQ ID NOs:
- 29-31, and wherein the polynucleotide is linked to a promoter.
A composition comprising an rAAV vector, wherein the rAAV vector comprises:
- (a) an AAV2, AAV5, AAV8, or AAV9 capsid, and
- (b) an expression cassette, wherein the expression cassette comprises a polynucleotide comprising a nucleotide sequence sharing at least 95% identity to a nucleotide sequence selected from SEQ ID NOs:
- 29-31, and wherein the polynucleotide is linked to a promoter.
A composition comprising an rAAV vector, wherein the rAAV vector comprises:
- (a) an AAV2, AAV5, AAV8, or AAV9 capsid, and
- (b) an expression cassette, wherein the expression cassette comprises a polynucleotide sequence sharing at least 95% identity to SEQ ID NO:
- 23.
A pharmaceutical composition, comprising
- a) a polypeptide comprising a diamine oxidase (“DAO1”) enzyme, or a fragment thereof, optionally wherein the polypeptide has an amino acid sequence having at least 90% identity to an amino acid sequence selected from SEQ ID NO:
- 1 and 2;
- and
- b) a pharmaceutically-acceptable carrier suitable for administration to an eye of a human subject.
A pharmaceutical composition, comprising
- a) a vector comprising a polynucleotide that encodes a polypeptide comprising a diamine oxidase (“DAO1”) enzyme, or a fragment thereof, optionally wherein the polypeptide has an amino acid sequence having at least 90% identity to an amino acid sequence selected from SEQ ID NO:
- 1 and 2;
- and
- b) a pharmaceutically-acceptable carrier suitable for administration to an eye of a human subject.
Dependencies are inferred from the claim text.