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1. EP2911671 - PYRAZOLOPYRIDAZINES AND METHODS FOR TREATING RETINAL-DEGENERATIVE DISEASES AND HEARING LOSS ASSOCIATED WITH USHER SYNDROME

Office
European Patent Office
Application Number 13848995
Application Date 25.10.2013
Publication Number 2911671
Publication Date 02.09.2015
Publication Kind B1
IPC
A61K 31/5025
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
495having six-membered rings with two nitrogen atoms as the only ring hetero atoms, e.g. piperazine
50Pyridazines; Hydrogenated pyridazines
5025ortho- or peri-condensed with heterocyclic ring systems
C07D 487/04
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
487Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/-C07D477/183
02in which the condensed system contains two hetero rings
04Ortho-condensed systems
CPC
A61P 25/28
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
28for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A61P 27/02
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
27Drugs for disorders of the senses
02Ophthalmic agents
A61P 27/16
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
27Drugs for disorders of the senses
16Otologicals
C07D 487/04
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
487Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by C07D451/00 - C07D477/00
02in which the condensed system contains two hetero rings
04Ortho-condensed systems
Applicants USHER III INITIATIVE INC
Inventors BURLI ROLAND WERNER
ESMIEU WILLIAM RAMESHCHANDRA KRISHNA
LOCK CHRISTOPHER JAMES
MALAGU KARINE FABIENNE
OWENS ANDREW PATE
HARTE WILLIAM EDWARD
Designated States
Priority Data 201261718611 25.10.2012 US
201361775402 08.03.2013 US
Title
(DE) PYRAZOLOPYRIDAZINE UND VERFAHREN ZUR BEHANDLUNG VON DEGENERATIVEN NETZHAUTERKRANKUNGEN UND HÖRVERLUST MIT USHER-SYNDROM
(EN) PYRAZOLOPYRIDAZINES AND METHODS FOR TREATING RETINAL-DEGENERATIVE DISEASES AND HEARING LOSS ASSOCIATED WITH USHER SYNDROME
(FR) PYRAZOLOPYRIDAZINES ET MÉTHODES DE TRAITEMENT DE MALADIES DÉGÉNÉRATIVES DE LA RÉTINE ET DE LA PERTE D'AUDITION ASSOCIÉE AU SYNDROME D'USHER
Abstract
(EN) 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. Usher Syndrome, a rare genetic disorder and a leading cause of deafness and blindness, is associated with a mutation in any one of ten genes.
(FR) La présente invention concerne des composés, des compositions et des méthodes pour le traitement de maladies dégénératives de la rétine, telles que la rétinite pigmentaire, l'amaurose congénitale de Leber, des dégénérescences rétiniennes syndromiques, la dégénérescence maculaire liée à l'âge et le syndrome d'Usher, ainsi que la perte d'audition associée au syndrome d'Usher. Le syndrome d'Usher qui est une maladie génétique et une cause majeure de surdité et de cécité, est associé à une mutation dans un gène quelconque parmi dix gènes.