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AssigneeShape Therapeutics Inc.
InventorsThomas PACKARD, Adrian Wrangham BRIGGS, David Jeffrey HUSS, Francois VIGNEAULT, Ronald James HAUSE, JR., Yue JIANG, Kevin Christopher STEIN, Bora BANJANIN
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Disclosed herein are engineered AAV VP capsid polypeptides with the ability to assemble into virus particles and having improved tissue tropism to eye tissues, for example, retinal tissues, retinal pigment epithelial cells, or photoreceptor cells. The capsids are engineered using the high throughput discovery system described herein. In certain embodiments, provided herein are recombinant adeno-associated virus (AAV) VP capsid polypeptides having at least one mutation in a 581 to 589 region of the VP capsid polypeptide, corresponding to residues 581 to residue 589 of a VP1 polypeptide of SEQ ID NO: 1.
A viral protein (VP) capsid polypeptide comprising a 581 to 589 region corresponding to residues 581 to 589 of an AAV5 VP1 polypeptide,
A viral protein (VP) capsid polypeptide comprising a 581 to 589 region corresponding to residues 581 to 589 of an AAV5 VP1 polypeptide, wherein the 581-589 region of the VP capsid polypeptide comprises a sequence of SEQ ID NO:
A viral protein (VP) capsid polypeptide comprising a 581 to 589 region corresponding to residues 581 to 589 of an AAV5 VP1 polypeptide, wherein the 581 to 589 region of the VP capsid polypeptide comprises at least two basic amino acid residues and no more than one acidic amino acid residue.
A viral protein (VP) capsid polypeptide comprising a 581 to 589 region corresponding to residues 581 to 589 of an AAV5 VP1 polypeptide, wherein the 581 to 589 region comprises a sequence having at least 85% sequence identity to any one of SEQ ID NO:
A viral protein (VP) capsid polypeptide comprising a 581 to 589 region corresponding to residues 581 to 589 of an AAV5 VP1 polypeptide, wherein the 581 to 589 region comprises a sequence having at least 85% sequence identity to any one of SEQ ID NO:
A method of delivering a payload to a retina tissue of a subject, the method comprising administering a recombinant adeno-associated virus (rAAV) to the subject via intravitreal administration, wherein the rAAV encapsidates the payload, and wherein the rAAV comprises a VP capsid polypeptide comprising a 581 to 589 region comprising at least one substitution relative to SEQ ID NO:
A method of delivering a payload to a retina tissue of a subject, the method comprising administering a recombinant adeno-associated virus (rAAV) to the subject via intravitreal administration, wherein the rAAV encapsidates the payload, and wherein the rAAV comprises a VP capsid polypeptide comprising a 581 to 589 region corresponding to residues 581 to 589 of an AAV5 VP1 polypeptide, wherein the 581 to 589 region comprises a sequence having at least 85% sequence identity to any one of SEQ ID NO:
A method of delivering a payload to a retina tissue of a subject, the method comprising:
A method of delivering a payload to a retina tissue of a subject, the method comprising:
A method of treating a condition in a subject, the method comprising administering a recombinant adeno-associated virus (rAAV) to the subject via intravitreal administration, wherein the rAAV encapsidates a payload, and wherein the rAAV comprises a VP capsid polypeptide comprising a 581 to 589 region comprising at least one substitution relative to SEQ ID NO:
A method of treating a condition in a subject, the method comprising administering a recombinant adeno-associated virus (rAAV) to the subject via intravitreal administration, wherein the rAAV encapsidates a payload, and wherein the rAAV comprises a VP capsid polypeptide comprising a 581 to 589 region corresponding to residues 581 to 589 of an AAV5 VP1 polypeptide having at least 85% sequence identity to any one of SEQ ID NO:
A method of treating a condition in a subject, the method comprising:
A method of treating a condition in a subject, the method comprising:
A viral protein (VP) capsid polypeptide comprising a 581 to 589 region corresponding to residues 581 to 589 of an AAV5 VP1 polypeptide, wherein the 581 to 589 region comprises a sequence having at least 85% sequence identity to any one of SEQ ID NO:
A viral protein (VP) capsid polypeptide comprising a 581 to 589 region corresponding to residues 581 to 589 of an AAV5 VP1 polypeptide, wherein the 581 to 589 region comprises a sequence having at least 85% sequence identity to any one of SEQ ID NO:
A method of delivering a payload to a retinal pigment epithelium tissue of a subject, the method comprising administering a recombinant adeno-associated virus (rAAV) to the subject via intravitreal administration, wherein the rAAV encapsidates the payload, and wherein the rAAV comprises a VP capsid polypeptide comprising a 581 to 589 region corresponding to residues 581 to 589 of an AAV5 VP1 polypeptide having at least 85% sequence identity to any one of SEQ ID NO:
A method of delivering a payload to a retinal pigment epithelium tissue of a subject, the method comprising administering a recombinant adeno-associated virus (rAAV) to the subject via intravitreal administration, wherein the rAAV encapsidates the payload, and wherein the rAAV comprises a VP capsid polypeptide comprising a 581 to 589 region corresponding to residues 581 to 589 of an AAV5 VP1 polypeptide having at least 85% sequence identity to any one of SEQ ID NO:
A method of delivering a payload to a choroid tissue of a subject, the method comprising:
A method of delivering a payload to a retinal pigment epithelium tissue of a subject, the method comprising:
A method of treating a condition in a subject, the method comprising administering a recombinant adeno-associated virus (rAAV) to the subject via intravitreal administration, wherein the rAAV encapsidates a payload, and wherein the rAAV comprises a VP capsid polypeptide comprising a 581 to 589 region corresponding to residues 581 to 589 of an AAV5 VP1 polypeptide having at least 85% sequence identity to any one of SEQ ID NO:
A method of treating a condition in a subject, the method comprising:
Dependencies are inferred from the claim text.