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US 2023/0399657 A1Published application
Liver de-targeted recombinant aav capsid proteins
AssigneeHomology Medicines, Inc.
InventorsLaura Jane SMITH, Laura Van Lieshout, Lindsay Schulman, Omar Francone
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Abstract
Provided herein are recombinant adeno-associated virus (AAV) capsid proteins, compositions (e.g., rAAV) comprising the capsid proteins, nucleic acids encoding the capsid proteins, and methods of making and using the capsid proteins.
Claims25
(canceled)
A recombinant adeno-associated virus (AAV) capsid protein, wherein:
- the amino acid in the capsid protein corresponding to amino acid position 346 of SEQ ID NO:
- 14 is alanine;
- the amino acid in the capsid protein corresponding to amino acid position 501 of SEQ ID NO:
- 14 is isoleucine;
- the amino acid in the capsid protein corresponding to amino acid position 505 of SEQ ID NO:
- 14 is arginine;
- and/or the amino acid in the capsid protein corresponding to amino acid position 706 of SEQ ID NO:
- 14 is cysteine, wherein the capsid protein does not comprise the amino acid sequence of amino acids 203-736 of SEQ ID NOs:
- 11-15;
- or
- the amino acid in the capsid protein corresponding to amino acid positions 346, 501, 505, and/or 706 of SEQ ID NO:
- 14 is not the wild type amino acid.
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Dependencies are inferred from the claim text.