IP Library › Granted Patent US 12,734,252
Granted Patent B2
US 12,734,252 · App. 18/491,365 · Granted Sep 15, 2026

Adeno-associated viral vector variants

Inventors: Beverly Davidson (Philadelphia, PA); Yonghong Chen (Philadelphia, PA); Paul T. Ranum (Philadelphia, PA); Xueyuan Liu (Philadelphia, PA)
Assignee: The Children's Hospital of Philadelphia
A61K48/0075A61P25/00C07K7/06C12N15/86C12N2750/14122C12N2750/14143C12N2750/14171
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Quick Facts
Patent No.
US 12,734,252
App. No.
18/491,365
Granted
Sep 15, 2026
Kind
B2
Abstract

Provided herein are targeting peptides and vectors containing a sequence that encodes the targeting peptides that deliver agents to specific substructures in the brain.

Claims (19)

1 . A modified adeno-associated virus (AAV) capsid protein comprising a targeting peptide that targets a viral vector comprising the modified AAV capsid protein to a distinct organ or brain structure, wherein the targeting peptide comprises the amino acid sequence of SEQ ID NO: 21, wherein the modified AAV capsid protein is a modified AAV1 capsid protein having an amino acid sequence at least 95% identical to the amino acid sequence of SEQ ID NO: 138 and the targeting peptide is inserted after residue 590 of the amino acid sequence of SEQ ID NO: 138.

2 . The modified AAV capsid protein of claim 1 , wherein the distinct brain structure is the brainstem, caudate, cerebellar, cochlea (ear), cortex, cerebral cortex, deep cerebellar nuclei, ependyma, globus pallidus, hippocampus, meninges, motor cortex, optic nerve, prefrontal cortex, putamen, spinal cord, substantia nigra, subthalamic nuclei, temporal cortex, thalamus, or visual cortex.

3 . The modified AAV capsid protein of claim 1 , wherein the modified AAV capsid protein comprises an AAV1 capsid protein comprising the amino acid sequence of SEQ ID NO: 138.

4 . The modified AAV capsid protein of claim 3 , wherein the targeting peptide is flanked by linker sequences.

5 . The modified AAV capsid protein of claim 4 , wherein the linker sequences are SSA on the N-terminal side of the targeting peptide and AS on the C-terminal side of the targeting peptide.

6 . The modified AAV capsid protein of claim 5 , wherein the modified AAV1 capsid protein has an amino acid sequence at least 95% identical to the amino acid sequence of SEQ ID NO: 141.

7 . The modified AAV capsid protein of claim 1 , wherein the targeting peptide consists of the amino acid sequence of SEQ ID NO: 21.

8 . The modified AAV capsid protein of claim 1 , wherein the distinct brain structure is the brainstem, caudate, cerebellar cortex, cerebral cortex, ependyma, globus pallidus, hippocampus, meninges, optic nerve, putamen, spinal cord, substantia nigra, subthalamic nuclei, or thalamus.

9 . The modified AAV capsid protein of claim 4 , wherein the linker sequences on each side of the targeting peptide are two or three amino acids long.

10 . A recombinant adeno-associated virus (rAAV) comprising the modified AAV1 capsid protein of claim 6 .

11 . The rAAV of claim 10 , wherein the modified AAV1 capsid protein has an amino acid sequence at least 96% identical to the amino acid sequence of SEQ ID NO: 141.

12 . The rAAV of claim 10 , wherein the modified AAV1 capsid protein has an amino acid sequence at least 97% identical to the amino acid sequence of SEQ ID NO: 141.

13 . The rAAV of claim 10 , wherein the modified AAV1 capsid protein has an amino acid sequence at least 98% identical to the amino acid sequence of SEQ ID NO: 141.

14 . The rAAV of claim 10 , wherein the modified AAV1 capsid protein has an amino acid sequence at least 99% identical to the amino acid sequence of SEQ ID NO: 141.

15 . A recombinant adeno-associated virus (rAAV) comprising the modified AAV1 capsid protein of claim 7 .

16 . A pharmaceutical composition comprising the rAAV of any one of claims 10-15 and a pharmaceutically acceptable carrier.

17 . An in vitro cell comprising the rAAV of any one of claims 10-15 .

18 . The cell of claim 17 , wherein the cell is a mammalian cell.

19 . The cell of claim 18 , wherein the mammalian cell is a human cell.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jan 4, 2024
From: DAVIDSON, BEVERLY; RANUM, PAUL; CHEN, YONGHONG; LIU, XUEYUAN
To: THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Reel/Frame 066019/0147 →
Continuity (4)
Continuation 17778783 · Nov 20, 2020
Provisional Application 63084709 · Sep 29, 2020
Provisional Application 62939315 · Nov 22, 2019
Related Publication 20240100194A1 · Mar 28, 2024
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