# US 12,180,500 B2 · Chimeric capsids

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- Number: US 12,180,500 B2
- Document ID: US12180500B2
- Status: Granted
- Kind: B2
- Application number: 16/745,873
- Filed: 2020-01-17
- Published: 2020-05-14
- Granted: 2024-12-31
- Earliest claimed: 2014-06-09
- Assignees: Voyager Therapeutics, Inc.
- Applicants: Voyager Therapeutics, Inc.
- Inventors: Robert Kotin; Jinzhao Hou; James McLaughlin
- Web page: https://patentgrep.com/patent/US12180500B2

## CPC

- C12N 15/86: Viral vectors
- C07K 14/005: from viruses
- C12N 2750/14122: New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
- C12N 2750/14143: viral genome or elements thereof as genetic vector
- C12N 2750/14145: Special targeting system for viral vectors
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00)

## Abstract

The present invention relates to viral vectors and methods of their production and use.

## Claims

### Claim 1

An adeno-associated virus (AAV) capsid protein, said AAV capsid protein comprising a chimeric polypeptide which has at least 95% identity with a reference sequence comprising SEQ ID NO: 436; wherein the chimeric polypeptide is a substitutional variant of SEQ ID NO: 436; and
wherein the VRVIII-CNS domain of SEQ ID NO: 436 is replaced with a donor sequence selected from any one of SEQ ID NOs: 547 or 549-553.

### Claim 2 (depends on 1)

The AAV capsid protein of claim 1, wherein the donor sequence is SEQ ID NO: 547.

### Claim 3 (depends on 1)

The AAV capsid protein of claim 1, wherein the donor sequence is SEQ ID NO: 549.

### Claim 4 (depends on 1)

The AAV capsid protein of claim 1, wherein the donor sequence is SEQ ID NO: 550.

### Claim 5 (depends on 1)

The AAV capsid protein of claim 1, wherein the donor sequence is SEQ ID NO: 551.

### Claim 6 (depends on 1)

The AAV capsid protein of claim 1, wherein the donor sequence is SEQ ID NO: 552.

### Claim 7 (depends on 1)

The AAV capsid protein of claim 1, wherein the donor sequence is SEQ ID NO: 553.

### Claim 8 (depends on 1)

An adeno-associated virus (AAV) particle comprising the AAV capsid protein of claim 1 and a vector genome which comprises a payload construct.

### Claim 9 (depends on 8)

The AAV particle of claim 8, wherein the payload construct comprises a polynucleotide sequence encoding a therapeutic protein.

### Claim 10 (depends on 8)

The AAV particle of claim 8, wherein the payload construct comprises a polynucleotide sequence encoding an siRNA, an shRNA, an miRNA, or a precursor thereof.