# US 12,180,474 B2 · Methods of treating porphyria

> Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt

- Number: US 12,180,474 B2
- Document ID: US12180474B2
- Status: Granted
- Kind: B2
- Application number: 17/194,706
- Filed: 2021-03-08
- Published: 2021-07-22
- Granted: 2024-12-31
- Earliest claimed: 2018-07-13
- Assignees: University of Pittsburgh—Of the Commonwealth System of Higher Education
- Applicants: University of Pittsburgh—Of the Commonwealth System of Higher Education
- Inventors: Satdarshan Monga; Kari Nejak-Bowen
- Web page: https://patentgrep.com/patent/US12180474B2

## 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 31/444: containing a six-membered ring with nitrogen as a ring heteroatom, e.g. amrinone
- A61K 31/519: ortho- or peri-condensed with heterocyclic rings
- A61P 1/16: for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
- C12N 2310/14: interfering nucleic acids [NA]
- C12N 2320/30: Special therapeutic applications
- 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)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

A method of treating porphyria in a patient is provided comprising knocking down or reducing expression or activity of β-catenin in the patient. e.g., in the liver of a patient, to an extent effective to treat porphyria in a patient.

## Claims

### Claim 1

A method of treating porphyria in a patient in need thereof, comprising: decreasing porphyrin production and/or accumulation in the patient by administering to the patient an amount of a therapeutic agent effective to decrease porphyrin production and/or accumulation in the patient, wherein the therapeutic agent is selected from the group consisting of Wnt-C59, PRI724, ICG001, an isomer, stereoisomer, or enantiomer of ICG001, and combinations thereof.

### Claim 2 (depends on 1)

The method of claim 1, wherein the therapeutic agent is Wnt-C59.

### Claim 3 (depends on 1)

The method of claim 1, wherein the therapeutic agent is ICG001.

### Claim 4 (depends on 3)

The method of claim 3, wherein the therapeutic agent is PRI724.

### Claim 5 (depends on 1)

The method of claim 1, wherein activity of β-catenin is decreased by use of the therapeutic agent.

### Claim 6 (depends on 1)

The method of claim 1, wherein expression or activity of β-catenin in a liver of the patient is decreased.

### Claim 7 (depends on 1)

The method of claim 1, wherein the patient's liver is targeted for decreasing porphyrin production and/or accumulation.

### Claim 8 (depends on 1)

The method of claim 1, wherein the patient has one or more symptoms of porphyria.

### Claim 9 (depends on 8)

The method of claim 8, wherein the one or more symptoms of porphyria is liver injury.

### Claim 10

A method of decreasing porphyrin production in a patient, comprising: administering to the patient an amount of a therapeutic agent effective to decrease porphyrin production in the patient, wherein the therapeutic agent is selected from the group consisting of Wnt-C59, PRI724, ICG001 an isomer, stereoisomer, or enantiomer of ICG001, and combinations thereof.

### Claim 11 (depends on 10)

The method of claim 10, wherein the therapeutic agent is Wnt-C59.

### Claim 12 (depends on 10)

The method of claim 10, wherein the therapeutic agent is ICG001.

### Claim 13 (depends on 10)

The method of claim 10, wherein the therapeutic agent is PRI724.

### Claim 14 (depends on 10)

The method of claim 10, wherein the patient's liver is targeted for decreasing porphyrin production.

### Claim 15

A method of decreasing porphyrin accumulation in a patient, comprising: administering to the patient an amount of a therapeutic agent effective to decrease porphyrin accumulation in the patient, wherein the therapeutic agent is selected from the group consisting of Wnt-C59, PRI724, ICG001 an isomer, stereoisomer, or enantiomer of ICG001, and combinations thereof.

### Claim 16 (depends on 15)

The method of claim 15, wherein the therapeutic agent is Wnt-C59.

### Claim 17 (depends on 15)

The method of claim 15, wherein the therapeutic agent is ICG001.

### Claim 18 (depends on 17)

The method of claim 17, wherein the therapeutic agent is PRI724.

### Claim 19 (depends on 15)

The method of claim 15, wherein the patient's liver is targeted for decreasing porphyrin accumulation.