# US 2026/0062701 A1 · Compositions and methods for treating meningioma

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- Number: US 2026/0062701 A1
- Document ID: US20260062701A1
- Status: Published application
- Kind: A1
- Application number: 19/304,919
- Filed: 2025-08-20
- Published: 2026-03-05
- Earliest claimed: 2024-08-21
- Assignees: The Jackson Laboratory
- Applicants: The Jackson Laboratory
- Inventors: Olga Anczuków-Camarda; Nathan Leclair
- Web page: https://patentgrep.com/patent/US20260062701A1

## CPC

- C12N 15/113: Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants C12N15/8218)}
- A61K 31/7115: Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
- A61K 31/712: Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
- A61K 31/7125: Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
- C12N 2310/11: Antisense
- C12N 2310/315: Phosphorothioates
- C12N 2310/321: 2'-O-R Modification
- C12N 2310/33: of the base
- C12N 2310/346: having a combination of backbone and sugar modifications
- C12N 2320/33: Alteration of splicing
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- 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

The disclosure relates to methods and compositions that regulate splicing in NASP transcripts.

## Claims

### Claim 1

An engineered splice-switching antisense oligonucleotide that binds to the nucleotide sequence of SEQ ID NO: 1.

### Claim 2 (depends on 1)

The engineered splice-switching antisense oligonucleotide of claim 1 that binds to the nucleotide sequence of SEQ ID NO: 2.

### Claim 3

An engineered splice-switching antisense oligonucleotide comprising the nucleotide sequence of SEQ ID NO: 3.

### Claim 4 (depends on 1)

The engineered splice-switching antisense oligonucleotide of claim 1 comprising the nucleotide sequence of SEQ ID NO: 4 or a nucleotide sequence having at least 90% identity to the nucleotide sequence of SEQ ID NO: 4.

### Claim 5 (depends on 4)

The engineered splice-switching antisense oligonucleotide of claim 4 comprising a nucleotide sequence having at least 95% identity to the nucleotide sequence of SEQ ID NO: 4.

### Claim 6 (depends on 5)

The engineered splice-switching antisense oligonucleotide of claim 5 comprising the nucleotide sequence of SEQ ID NO: 4.

### Claim 7 (depends on 1)

The engineered splice-switching antisense oligonucleotide of claim 1 comprising a chemical modification.

### Claim 8 (depends on 7)

The engineered splice-switching antisense oligonucleotide of claim 7, wherein the chemical modification is selected from backbone modifications, sugar modifications, and base modifications.

### Claim 9 (depends on 8)

The engineered splice-switching antisense oligonucleotide of claim 8, wherein the chemical modification is a backbone modification.

### Claim 10 (depends on 9)

The engineered splice-switching antisense oligonucleotide of claim 9, wherein the backbone modification is a phosphorothioate (PS) modification.

### Claim 11 (depends on 8)

The engineered splice-switching antisense oligonucleotide of claim 8, wherein the chemical modification is a sugar modification.

### Claim 12 (depends on 11)

The engineered splice-switching antisense oligonucleotide of claim 11, wherein the sugar modification is 2′-O-Methyl (2′-OMe) or 2′-O-Methoxyethyl (2′-MOE).

### Claim 13 (depends on 1)

A composition comprising the engineered splice-switching antisense oligonucleotide of claim 1 and an excipient.

### Claim 14 (depends on 1)

A composition comprising the engineered splice-switching antisense oligonucleotide claim 1 associated with a delivery vehicle.

### Claim 15 (depends on 14)

The composition of claim 14, wherein the delivery vehicle is selected from lipid nanoparticles, liposomes, polymeric nanoparticles, gold nanoparticles, peptide-based delivery systems, aptamer-based delivery systems, exosomes, viral vectors, and molecules capable of crossing the blood-brain barrier.

### Claim 16 (depends on 13)

The composition of claim 13, wherein the concentration of the engineered splice-switching antisense oligonucleotide in the composition is about 2 mg/ml to about 200 mg/ml.

### Claim 17 (depends on 1)

A method comprising administering the engineered splice-switching antisense oligonucleotide of claim 1 to a subject, wherein the subject has a meningioma.

### Claim 18 (depends on 17)

The method of claim 17, wherein the engineered splice-switching antisense oligonucleotide is administered via intrathecal injection, intracranial injection, or intravenous infusion.

### Claim 19 (depends on 17)

The method of claim 17, wherein the meningioma is classified as Hypermitotic meningioma.

### Claim 20

(canceled)

### Claim 21 (depends on 1)

A method comprising administering the engineered splice-switching antisense oligonucleotide of claim 1 to a cell, optionally a meningioma cell, in an amount effective to modify splicing of NASP transcript.

### Claim 22

(canceled)