# US 12,213,956 B2 · Method of treating fatty acid oxidation disorders using omega3/omega6 unsaturated or branched chain fatty acids

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- Number: US 12,213,956 B2
- Document ID: US12213956B2
- Status: Granted
- Kind: B2
- Application number: 17/387,299
- Filed: 2021-07-28
- Published: 2021-11-18
- Granted: 2025-02-04
- Earliest claimed: 2018-07-11
- Assignees: University of Pittsburgh—Of the Commonwealth System of Higher Education
- Applicants: University of Pittsburgh—Of the Commonwealth System of Higher Education
- Inventors: Al-Walid A. Mohsen
- Web page: https://patentgrep.com/patent/US12213956B2

## CPC

- A61K 31/221: with compounds having an amino group, e.g. acetylcholine, acetylcarnitine
- A61K 9/0053: {Mouth and digestive tract, i.e. intraoral and peroral administration}
- A61K 31/23: of acids having a carboxyl group bound to a chain of seven or more carbon atoms
- A61K 31/232: having three or more double bonds, e.g. etretinate
- A61P 3/00: Drugs for disorders of the metabolism (of the blood or the extracellular fluid A61P7/00)
- C07C 229/22: the carbon skeleton being further substituted by oxygen atoms
- 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
- C07C: ACYCLIC OR CARBOCYCLIC COMPOUNDS

## Abstract

A method of treating medium chain acyl-CoA dehydrogenase deficiency, very long chain acyl-CoA dehydrogenase deficiency, long chain hydroxyacyl-CoA dehydrogenase deficiency, trifunctional protein deficiency, or CPT II deficiency in a patient is provided comprising administering to the patient a therapeutic amount of a triglyceride or other conjugated fatty acid that bypasses or leads to an intermediate that bypasses the deficient enzyme. Amino acid-conjugated fatty acids also are provided as well as compositions comprising the amino acid-conjugated fatty acids.

## Claims

### Claim 1

A method of treating a patient having medium chain acyl-CoA dehydrogenase deficiency (MCADD) comprising administering to a patient an effective amount of an active agent triglyceride comprising one to three fatty acid moieties selected from the group consisting of heptanoyl, 2-methylheptanoyl, 2,6-dimethylheptanoyl, 4,8-dimethylnonanoyl, 6-amino-2,4-dimethylheptanoyl, linolenoyl, docosahexaenoyl, and eicosapentaenoyl in any combination, or a pharmaceutically-acceptable salt thereof, effective to treat the MCADD in a patient.

### Claim 2 (depends on 1)

The method of claim 1, wherein the triglyceride comprises one or more heptanoyl moieties.

### Claim 3 (depends on 1)

The method of claim 1, wherein the triglyceride comprises two or three heptanoyl moieties.

### Claim 4 (depends on 1)

The method of claim 1, wherein the triglyceride comprises a heptanoyl moiety and an @3/06 unsaturated fatty acid moiety.

### Claim 5 (depends on 1)

The method of claim 1, wherein the triglyceride comprises a heptanoyl moiety and a C18:3, C20:5, or C22:6 fatty acid moiety having double bonds at positions ω3 and ω6 of the C18:3, C20:5, or C22:6 fatty acid moiety.

### Claim 6 (depends on 1)

The method of claim 1, wherein the triglyceride further comprises one or two threoninyl, serinyl, or succinyl moieties attached by an ester bond to the triglyceride.

### Claim 7 (depends on 6)

The method of claim 6, wherein the triglyceride comprises at least one heptanoyl moiety.

### Claim 8 (depends on 7)

The method of claim 7, wherein the triglyceride comprises two α-linolenoyl moieties and one heptanoyl moiety.

### Claim 9 (depends on 7)

The method of claim 7, wherein the triglyceride comprises one α-linolenoyl moiety and two heptanoyl moieties.

### Claim 10 (depends on 1)

The method of claim 1, wherein the triglyceride is 1,2,3-triheptanoyl glycerol.

### Claim 11 (depends on 1)

The method of claim 1, wherein the patient is further administered a therapeutically-effective amount of a triglyceride comprising an α-linolenic acid fatty acid moiety, such as an α-linolenic acid fatty acid moiety provided in an oil of a plant rich in ω3/ω6 unsaturated fatty acids.

### Claim 12 (depends on 11)

The method of claim 11, wherein the triglyceride comprising an α-linolenic acid fatty acid is provided in a form that comprises a fatty acid content of at least 45% α-linolenic acid.

### Claim 13 (depends on 1)

The method of claim 1, wherein a diet of the patient suffering from medium chain acyl-CoA dehydrogenase deficiency is substantially free of triglycerides able to produce C8-CoA.