Recombinant aavs with improved tropism and specificity
AssigneeAffinia Therapeutics Inc.
InventorsCharles Francis ALBRIGHT, Kevin C. OLIVIERI, Xiaohong CAO, Jie TAN
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Abstract
The present disclosure provides a modified AAV capsid protein comprising a targeting peptide in variable region VIII (VR, VIII) and/or a peptide segment in variable region I (VR I). The modified AAV capsid protein can form an rAAV, which has a preferred tropism, specificity or biodistribution in vivo or in vitro. The rAAV of the present disclosure can be used for gene therapies targeted at a specific tissue.
Claims211
A modified adeno-associated virus (AAV) capsid protein, comprising:
- (i) a targeting peptide within VR VIII;
- or
- (ii) a peptide segment within VR I,
- wherein the targeting peptide has a sequence of X1X2X3RGDX7X8X9X10, wherein X1, X2, X3, X7, X8, X9 and X10 are independently selected from any amino acid residue, and wherein the peptide segment has an amino acid sequence of P1P2P3P4P5P6P7P8NDNP12 and P1, P2, P3, P4, P5, P6, P7, P8, and P12 are independently selected from any amino acid residue.
The modified AAV capsid protein of any one of 6-12, wherein the reference AAV capsid protein is a capsid protein having a sequence of SEQ ID NO:
- 61 or a fragment thereof.
A modified adeno-associated virus (AAV) capsid protein, comprising:
- (i) a targeting peptide at a site within VR VIII, wherein the targeting peptide has a sequence selected from SEQ ID NOs:
- 44864-44867, 44879-44883, 44911, 44912, 44913, and 44918-44919;
- and
- (ii) a peptide segment within VR I, wherein the peptide segment has a sequence selected from SEQ ID NOs:
- 46026, 46029, 46031, 46035, 46073, 46076, 46079, 46082, 46505, 46508, 46511, 46514, 46554, 46560, 46609, 46600, 46603, 46606, 46650, 46653, 46656, 46659, 47128, 47131, 47134, and 47137.
Dependencies are inferred from the claim text.