# US 11,897,938 B2 · Factor VIII proteins having ancestral sequences, expression vectors, and uses related thereto

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- Number: US 11,897,938 B2
- Document ID: US11897938B2
- Status: Granted
- Kind: B2
- Application number: 17/355,656
- Filed: 2021-06-23
- Published: 2021-12-16
- Granted: 2024-02-13
- Earliest claimed: 2015-01-30
- Assignees: Emory University; Georgia Tech Research Corporation
- Applicants: Emory University; Georgia Tech Research Corporation
- Inventors: H. Trent Spencer; Christopher B. Doering; Philip M. Zakas; Eric Gaucher
- Web page: https://patentgrep.com/patent/US11897938B2

## CPC

- C07K 14/755: Factors VIII {, e.g. factor VIII C (AHF), factor VIII Ag (VWF)}
- A61K 38/00: Medicinal preparations containing peptides (peptides containing beta-lactam rings A61K31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K31/00; ergot alkaloids of the cyclic peptide type A61K31/48; containing macromolecular compounds having statistically distributed amino acid units A61K31/74; medicinal preparations containing antigens or antibodies A61K39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K47/00)
- 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)
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)

## Abstract

This disclosure relates to recombinant or chimeric FVIII proteins, variants, and vectors encoding the proteins containing one or more ancestral mutations. In certain embodiments, one or more protein domains comprise amino acid sequences that are derived from ancestrally reconstructed amino acid sequences. In certain embodiments, the disclosure relates to pharmaceutical compositions comprising the proteins or vectors and related methods of inducing blood clotting.

## Claims

### Claim 1

A vector comprising a promotor nucleic acid sequence in operable combination with a heterologous nucleic acid sequence encoding a polypeptide selected from the group consisting of SEQ ID NO: 20, SEQ ID NO: 2, SEQ ID NO: 30 and SEQ ID NO: 11.

### Claim 2 (depends on 1)

The vector of claim 1, wherein the promoter is a liver-specific promotor.

### Claim 3 (depends on 1)

The vector of claim 1, further comprising 5′ and 3′ AAV inverted terminal repeats (ITRs).

### Claim 4 (depends on 1)

The vector of claim 1, wherein the vector is an adeno-associated viral vector.

### Claim 5 (depends on 1)

The vector of claim 1, wherein the vector is a lentiviral vector.

### Claim 6 (depends on 1)

An expression system comprising the vector of claim 1.

### Claim 7 (depends on 1)

A viral particle or capsid comprising the vector of claim 1.

### Claim 8 (depends on 1)

A pharmaceutical composition comprising the vector of claim 1.

### Claim 9 (depends on 6)

A pharmaceutical composition comprising the viral particle or capsid of claim 6.