# US 11,298,343 B2 · Method for treating thalassemia

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- Number: US 11,298,343 B2
- Document ID: US11298343B2
- Status: Granted
- Kind: B2
- Application number: 16/758,986
- Filed: 2018-10-26
- Published: 2020-11-05
- Granted: 2022-04-12
- Earliest claimed: 2017-10-27
- Assignees: St. Jude Children's Research Hospital
- Applicants: St. Jude Children's Research Hospital
- Inventors: Mitchell J. Weiss; Mondira Kundu; Christophe Lechauve
- Web page: https://patentgrep.com/patent/US11298343B2

## CPC

- A61K 31/436: the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
- A61P 7/06: Antianaemics
- A61K 45/06: Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
- 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

Methods for removing excess free α-globin in erythroid cells and treating a thalassemia using an agent that activates Unc-51 Like autophagy activating Kinase (ULK) are described.

## Claims

### Claim 1

A method for removing excess free α-globin in reticulocytes or erythrocytes comprising contacting reticulocytes or erythrocytes with an effective amount of an agonist that directly activates Unc-51 Like autophagy activating Kinase (ULK), AMP-Activated Protein Kinase (AMPK), or Glycogen synthase kinase 3 (GSK3) thereby removing excess free α-globin in the reticulocytes or erythrocytes.

### Claim 2

A method for removing excess free, insoluble α-globin in reticulocytes or erythrocytes comprising contacting reticulocytes or erythrocytes with an effective amount of an agent that activates Unc-51 Like autophagy activating Kinase (ULK) thereby removing excess free, insoluble α-globin in the reticulocytes or erythrocytes, wherein the agent inhibits Mechanistic Target Of Rapamycin (mTOR).

### Claim 3

A method for treating a thalassemia comprising administering to a subject in need thereof an effective amount of an agonist that directly activates Unc-51 Like autophagy activating Kinase (ULK), AMP-Activated Protein Kinase (AMPK), or Glycogen synthase kinase 3 (GSK3) and stimulates ULK1-dependent autophagy thereby treating the subject's thalassemia.

### Claim 4

A method for treating a thalassemia comprising administering to a subject in need thereof an effective amount of an agent that activates Unc-51 Like autophagy activating Kinase (ULK), wherein the agent inhibits Mechanistic Target Of Rapamycin (mTOR) and is not an ATP-competitive mTOR kinase inhibitor.

### Claim 5 (depends on 3)

The method of claim 3, wherein the thalassemia is β-thalassemia.

### Claim 6 (depends on 4)

The method of claim 4, wherein the thalassemia is β-thalassemia.

### Claim 7 (depends on 2)

The method of claim 2, wherein the agent that inhibits mTOR is not an ATP-competitive mTOR kinase inhibitor.