# US 2026/0077019 A1 · Trizepatide and other glp1ra as a novel treatment for patients with lipodystrophy

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- Number: US 2026/0077019 A1
- Document ID: US20260077019A1
- Status: Published application
- Kind: A1
- Application number: 19/332,597
- Filed: 2025-09-18
- Published: 2026-03-19
- Earliest claimed: 2024-09-18
- Assignees: Rutgers, The State University of New Jersey
- Applicants: Rutgers, The State University of New Jersey
- Inventors: Christopher Buettner; Svetlana Ten
- Web page: https://patentgrep.com/patent/US20260077019A1

## CPC

- A61K 38/26: Glucagons
- A61K 38/28: Insulins
- A61P 3/00: Drugs for disorders of the metabolism (of the blood or the extracellular fluid A61P7/00)
- 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

The present disclosure provides methods of treating congenital generalized lipodystrophy using tirzepatide. The disclosure also provides methods for treating a disease in a patient characterized by leptin and leptin signaling deficiency.

## Claims

### Claim 1

A method of treating generalized lipodystrophy in a patient in need thereof, the method comprising administering a therapeutically effective amount of a GLP-1 receptor agonist to the patient.

### Claim 2 (depends on 1)

The method of claim 1, wherein the GLP-1 receptor agonist is selected from the group consisting of tirzepatide, semaglutide and retatrutide.

### Claim 3 (depends on 1)

The method of claim 1, wherein the patient has a 1-acylglycerol-3-phosphate O-acyltransferase 2 (AGPAT2) gene mutation, a Berardinelli-Seip congenital lipodystrophy type 2 protein (BSCL2) gene mutation, a caveolin-1 protein (CAV1) gene mutation, or A polymerase I and transcript release factor (PTRF) gene mutation.

### Claim 4 (depends on 3)

The method of claim 3, wherein the AGPAT2 gene mutation selected from the group consisting of: c.IVS4-2A>G, rs104894093 (ARG68TER), rs116807569 (c.589-2A>G), rs387906355 (1-BP INS 377T), rs104894100 (LEU228PRO), rs387906356 (3-BP DEL, 418TTC), rs121908925 (LYS215TER), rs606231168 (IVS3 A-G, -1), rs121908926 (PHE189TER), rs1255380257 (GLU229TER), and rs748157664 (GLU172LYS).

### Claim 5 (depends on 3)

The method of claim 3, wherein the BSCL2 gene mutation selected from the group consisting of: rs786205068 (2-BP DEL3-BP INS, NT536), rs786205069 (2-BP INS, 645AA), rs786205070 (2-BP DEL, 659GT), rs587777608 (5-BP DEL, NT659), rs786205071, (1-BP INS, 669A), rs137852970 (ARG138TER), rs1945400235 (IVS4, G-A, +1), rs13785297 (ALA212PRO), rs758843908 (1-BP DEL, 980C), rs786205072 (IVS6, G-A, +5), and rs786205073 (IVS6, C-G, -3).

### Claim 6 (depends on 3)

The method of claim 3, wherein the CAV1 gene mutation selected from the group consisting of: rs121434501 (GLU38TER) and 2-BP DEL, NT237.

### Claim 7 (depends on 3)

The method of claim 3, wherein the PTRF gene mutation selected from the group consisting of: 696insC and 525delG.

### Claim 8 (depends on 1)

The method of claim 1, wherein the patient does not have adipose tissue.

### Claim 9 (depends on 1)

The method of claim 1, wherein the patient has a deficiency in leptin.

### Claim 10 (depends on 1)

The method of claim 1, wherein the patient has a blood plasma concentration of leptin of 0.5 ng/mL or less.

### Claim 11 (depends on 1)

The method of claim 1, wherein the patient is diabetic or not diabetic.

### Claim 12 (depends on 1)

The method of claim 1, wherein the patient has severe insulin resistance resulting from generalized lipodystrophy.

### Claim 13 (depends on 12)

The method of claim 12, wherein the GLP-1 receptor agonist is co-administered with insulin, wherein after the patient's glucose levels normalize, the GLP-1 receptor agonist is administered without insulin.

### Claim 14 (depends on 1)

The method of any one of claim 1, wherein the therapeutically effective dose of the GLP-1 receptor to be administered is about 2.5 mg per 0.5 mL, about 5 mg per 0.5 mL, about 7.5 mg per 0.5 mL, about 10 mg per 0.5 mL, about 12.5 mg per 0.5 mL, or about 15 mg per 0.5 mL.

### Claim 15

A method of treating a disease in a patient characterized by leptin and leptin signaling deficiency, the method comprising administering a therapeutically effective amount of a GLP-1 receptor agonist to the patient.

### Claim 16 (depends on 15)

The method of claim 15, wherein the disease is selected from the group consisting of: CGL, CGL1, CGL2, CGL3, CGL4, familial partial lipodystrophy, acquired generalized lipodystrophy (AGL), acquired partial lipodystrophy, high active antiretroviral therapy-induced lipodystrophy, localized lipodystrophy, congenital leptin deficiency, and acquired hypoleptinemia.

### Claim 17 (depends on 15)

The method of claim 15, wherein the GLP-1 receptor agonist is selected from the group consisting of tirzepatide, semaglutide and retatrutide.

### Claim 18 (depends on 15)

The method of claim 15, wherein the leptin and leptin signaling deficiency results from mutations in the leptin (LEP) gene or leptin receptor (LEPR) gene.

### Claim 19 (depends on 18)

The method of claim 18, wherein the patient has a LEP gene mutation selected from the group consisting of: rs104894023 (ARG105TRP), rs200575914 (GLY59SER), and 1-BP DEL, FS147TER.

### Claim 20 (depends on 18)

The method of claim 18, wherein the patient has a LEPR gene mutation selected from the group consisting of: rs1474810899 (IVS16DS, G-A, +1), rs1557670950 (TYR155TER).
