# US 11,077,116 B2 · Isoxazole derivatives as inducers of fetal hemoglobin in erythroid precursor cells from beta-thalassemic patients

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

- Number: US 11,077,116 B2
- Document ID: US11077116B2
- Status: Granted
- Kind: B2
- Application number: 16/631,295
- Filed: 2018-07-13
- Published: 2020-07-02
- Granted: 2021-08-03
- Earliest claimed: 2017-07-18
- Assignees: Rare Partners Srl
- Applicants: Rare Partners Srl
- Inventors: Ilaria Lampronti; Roberto Gambari; Daniele Simoni
- Web page: https://patentgrep.com/patent/US11077116B2

## CPC

- A61K 31/422: not condensed and containing further heterocyclic rings
- A61K 31/5377: not condensed and containing further heterocyclic rings, e.g. timolol
- A61K 31/42: Oxazoles
- A61P 7/06: Antianaemics
- 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 invention refers to the use of isoxazole derivatives to prepare medicament able to induce fetal hemoglobin (HbF) synthesis in β-thalassemia and sickle cell disease (SCD) patients.

## Claims

### Claim 1

A method to induce fetal hemoglobin for therapeutic treatment of thalassemia and sickle cell disease in patients in need thereof with compounds of formula I
wherein R1 is C1-C3 alkyl or chlorine, fluorine, bromine;
R2 is hydroxy or methyl;
X is a C1-C4 alkylamino group or C1-C4 fluoroalkylamino group;
Y is a C2-C4 acyl group, a benzoyl group, optionally substituted with one or more methoxy, heteroaryl moieties, or a group with the following formula:

### Claim 2 (depends on 1)

The method of claim 1 wherein R1 is chlorine or isopropyl.

### Claim 3 (depends on 1)

The method of claim 1 wherein X is ethylamino or 2,2,2-trifluoroethylamino.

### Claim 4 (depends on 1)

The method of claim 1 wherein Y is acetyl, tert-butylcarbonyl, para-methoxybenzoyl, 3,4-dimethoxy-benzoyl, 3-methyl-tienyl, 5-acetyl-isoxazolyl.

### Claim 5 (depends on 1)

The method of claim 1, wherein said compounds are selected from the group consisting of