# US 2026/0077005 A1 · Lentiviral vector formulations

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- Number: US 2026/0077005 A1
- Document ID: US20260077005A1
- Status: Published application
- Kind: A1
- Application number: 19/284,911
- Filed: 2025-07-30
- Published: 2026-03-19
- Earliest claimed: 2019-09-30
- Assignees: Bioverativ Therapeutics Inc.; Fondazione Telethon; Ospedale San Raffaele S.R.L.
- Applicants: Bioverativ Therapeutics Inc.; Fondazione Telethon; Ospedale San Raffaele S.R.L.
- Inventors: Andrew Kroetsch; Isidro Zarraga
- Web page: https://patentgrep.com/patent/US20260077005A1

## CPC

- A61K 35/76: Viruses; Subviral particles; Bacteriophages
- C12N 7/00: Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof (preparing medicinal viral antigen or antibody compositions, e.g. virus vaccines, A61K39/00)
- A61K 47/26: Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
- A61P 7/04: Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
- C07K 14/755: Factors VIII {, e.g. factor VIII C (AHF), factor VIII Ag (VWF)}
- C12N 9/644: {Coagulation factor IXa (3.4.21.22)}
- C12N 15/86: Viral vectors
- C12N 2740/15051: Methods of production or purification of viral material
- C12N 2740/16043: viral genome or elements thereof as genetic vector
- C12N 2740/16051: Methods of production or purification of viral material
- 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)
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- 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

Lentiviral vector (LV) formulations, and pharmaceutical compositions comprising such LV formulations, with improved stability and suitable for systemic administration are provided. Methods for treating disorders, especially blood disorders, using systemic administration of LV formulations are also provided.

## Claims

### Claim at position 1

1-48. (canceled)

### Claim 49

A reduced in-vivo toxicity recombinant lentiviral particle preparation comprising:
(a) a therapeutically effective dose of a lentiviral particle comprising a recombinant lentiviral vector;
(b) a TRIS-free buffer system comprising a phosphate buffer;
(c) a salt;
(d) a poloxamer 188 (P188);
(e) a carbohydrate;
wherein the lentiviral particle further comprises an envelope comprising a vesicular stomatitis virus G (VSV-G) protein or a fragment thereof;
wherein the recombinant lentiviral vector comprises a nucleic acid comprising a nucleotide sequence that is at least 80% identical to a Factor IX (FIX) coding sequence set forth in SEQ ID NO: 3;
wherein the recombinant lentiviral particle preparation is suitable for systemic administration to a human patient; and
wherein systemic administration of the recombinant lentiviral particle preparation to the patient has reduced in-vivo toxicity relative to a recombinant lentiviral particle preparation comprising a TRIS buffer.

### Claim 50 (depends on 49)

The preparation of claim 49, wherein the pH of the buffer system or of the preparation is from about from about 7.0 to about 8.0.

### Claim 51 (depends on 49)

The preparation of claim 49, wherein the concentration of the phosphate buffer is from about 5 mM to about 30 mM.

### Claim 52 (depends on 49)

The preparation of claim 49, wherein the concentration of the phosphate buffer is from about 10 mM to about 20 mM.

### Claim 53 (depends on 49)

The preparation of claim 49, wherein the concentration of the salt is from about 80 mM to about 150 mM.

### Claim 54 (depends on 49)

The preparation of claim 49, wherein the concentration of the poloxamer 188 is from about 0.01% (w/v) to about 0.1% (w/v), about 0.03% (w/v), about 0.05% (w/v), about 0.07% (w/v), or about 0.09% (w/v).

### Claim 55 (depends on 49)

The preparation of claim 49, wherein the concentration of the carbohydrate is from about 0.50% (w/v) to about 50% (w/v), about 1% (w/v), about 2% (w/v), about 30% (w/v), or about 4% (w/v), optionally wherein the carbohydrate is sucrose.

### Claim 56 (depends on 49)

The preparation of claim 49, comprising:
(a) a therapeutically effective dose of a recombinant lentiviral vector;
(b) about 10 mM phosphate buffer;
(c) about 100 mM sodium chloride;
(d) about 0.05% (w/v) P188; and
(e) about 3% (w/v) sucrose,
wherein the pH of the preparation is about 7.3, and wherein the preparation is suitable for systemic administration to a human patient.

### Claim 57 (depends on 49)

The preparation of claim 49, comprising:
(a) a therapeutically effective dose of a recombinant lentiviral vector;
(b) about 10 mM phosphate buffer;
(c) about 130 mM sodium chloride;
(d) about 0.05% (w/v) P188; and
(e) about 1% (w/v) sucrose,
wherein the pH of the preparation is about 7.3, and wherein the preparation is suitable for systemic administration to a human patient.

### Claim 58 (depends on 49)

The preparation of claim 49, comprising:
(a) a therapeutically effective dose of a recombinant lentiviral vector;
(b) about 10 mM phosphate buffer;
(c) about 100 mM sodium chloride;
(d) about 0.05% (w/v) P188; and
(e) about 3% (w/v) sucrose,
wherein the pH of the preparation is about 7.0, and wherein the preparation is suitable for systemic administration to a human patient.

### Claim 59 (depends on 49)

The preparation of claim 49, wherein the recombinant lentiviral vector comprises:
an enhanced transthyretin (ET) promoter; and/or
a nucleotide sequence at least 90% identical to the target sequence for miR-142 set forth in SEQ ID NO: 7.

### Claim 60 (depends on 49)

The preparation of claim 49, wherein the recombinant lentiviral vector is isolated from a transfected host cell selected from the group consisting of a CHO cell, a HEK293 cell, a BHK21 cell, a PER.C6 cell, an NSO cell, and a CAP cell.

### Claim 61 (depends on 49)

The preparation of claim 49, wherein the recombinant lentiviral vector comprises a nucleic acid comprising the FIX coding sequence set forth in SEQ ID NO: 3.

### Claim 62

A reduced in-vivo toxicity recombinant lentiviral particle preparation, comprising:
(a) a therapeutically effective dose of a lentiviral particle comprising a recombinant lentiviral vector;
(b) a TRIS-free buffer system comprising about 10 mM phosphate buffer;
(c) about 100 mM or about 130 mM sodium chloride;
(d) about 0.05% (w/v) P188;
(e) about 3% (w/v) sucrose;
wherein the pH of the preparation is about 7.3;
wherein the lentiviral particle preparation is suitable for systemic administration to a human patient;
wherein the recombinant lentiviral particle further comprises an envelope comprising a vesicular stomatitis virus G (VSV-G) protein or a fragment thereof;
wherein the recombinant lentiviral vector comprises a nucleic acid comprising a nucleotide sequence that is at least 80% identical to a Factor IX (FIX) coding sequence set forth in SEQ ID NO: 3; and
wherein administration of the lentiviral particle preparation to a patient has reduced in-vivo toxicity relative to a recombinant lentiviral particle preparation comprising a TRIS buffer.

### Claim 63

A reduced in-vivo toxicity recombinant lentiviral particle preparation, comprising:
(a) a therapeutically effective dose of a lentiviral particle comprising a recombinant lentiviral vector;
(b) a TRIS-free buffer system comprising about 10 mM phosphate buffer;
(c) about 100 mM or about 130 mM sodium chloride;
(d) about 0.05% (w/v) P188;
(e) about 3% (w/v) sucrose;
wherein the pH of the preparation is about 7.0; and wherein the lentiviral particle preparation is suitable for systemic administration to a human patient;
wherein the recombinant lentiviral particle further comprises an envelope comprising a vesicular stomatitis virus G (VSV-G) protein or a fragment thereof;
wherein the recombinant lentiviral vector comprises a nucleic acid comprising a nucleotide sequence that is at least 80% identical to a Factor IX (FIX) coding sequence set forth in SEQ ID NO: 3; and
wherein administration of the lentiviral particle preparation to a patient has reduced in-vivo toxicity relative to a recombinant lentiviral particle preparation comprising a TRIS buffer.