# US 10,307,422 B2 · Pyrazolopyridazines and methods for treating retinal-degenerative diseases and hearing loss associated with usher syndrome

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- Number: US 10,307,422 B2
- Document ID: US10307422B2
- Status: Granted
- Kind: B2
- Application number: 15/924,831
- Filed: 2018-03-19
- Published: 2018-07-26
- Granted: 2019-06-04
- Earliest claimed: 2011-04-25
- Assignees: Usher III Initiative, Inc.
- Applicants: Usher III Initiative, Inc.
- Inventors: Roland Werner Bürli; William Rameshchandra Krishna Esmieu; Christopher James Lock; Karine Fabienne Malagu; Andrew Pate Owens; William E. Harte
- Web page: https://patentgrep.com/patent/US10307422B2

## CPC

- A61K 31/5025: ortho- or peri-condensed with heterocyclic ring systems
- A61K 31/165: having aromatic rings, e.g. colchicine, atenolol, progabide
- A61K 31/167: having the nitrogen of a carboxamide group directly attached to the aromatic ring, e.g. lidocaine, paracetamol
- A61K 31/40: having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
- A61P 27/00: Drugs for disorders of the senses
- A61P 27/02: Ophthalmic agents
- A61P 27/16: Otologicals
- C07C 243/28: to hydrogen atoms or to carbon atoms of a saturated carbon skeleton
- C07C 251/86: having doubly-bound carbon atoms of hydrazone groups bound to carbon atoms of six-membered aromatic rings
- C07D 487/04: Ortho-condensed systems
- C07D 495/04: Ortho-condensed systems
- C07D 519/00: Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00
- G01N 33/502: {for testing non-proliferative effects}
- G01N 33/6848: {Methods of protein analysis involving mass spectrometry}
- G01N 33/6893: {related to diseases not provided for elsewhere}
- C07C 2602/08: the other ring being five-membered, e.g. indane
- G01N 2333/4703: Regulators; Modulating activity
- G01N 2400/40: Glycosaminoglycans, i.e. GAG or mucopolysaccharides, e.g. chondroitin sulfate, dermatan sulfate, hyaluronic acid, heparin, heparan sulfate, and related sulfated polysaccharides
- G01N 2500/04: Screening involving studying the effect of compounds C directly on molecule A (e.g. C are potential ligands for a receptor A, or potential substrates for an enzyme A)
- G01N 2500/10: involving cells
- 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
- C07D: HETEROCYCLIC COMPOUNDS (macromolecular compounds C08)
- G01N: INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES (measuring or testing apparatus or processes other than immunoassay, involving enzymes or microorganisms C12M, C12Q)

## Abstract

Compounds, compositions and methods for the treatment of retinal degenerative diseases, such as retinitis pigmentosa, Leber's congenital Amaurosis, Syndromic retinal degenerations, age-related macular degeneration and Usher Syndrome, and hearing loss associated with Usher Syndrome are described herein.

## Claims

### Claim 1

A composition comprising an effective amount of
or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier or vehicle.

### Claim 2

A composition comprising an effective amount of
and a pharmaceutically acceptable carrier or vehicle.