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1. WO2022006550 - 2-(INDAZOL-5-YL)-6-(PIPERIDIN-4-YL)-1,7-NAPHTHYRIDINE DERIVATIVES AND RELATED COMPOUNDS AS MODULATORS FOR SPLICING NUCLEIC ACIDS AND FOR THE TREATMENT OF PROLIFERATIVE DISEASES

Publication Number WO/2022/006550
Publication Date 06.01.2022
International Application No. PCT/US2021/040364
International Filing Date 02.07.2021
IPC
C07D 471/04 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
471Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/-C07D463/251
02in which the condensed system contains two hetero rings
04Ortho-condensed systems
C07D 401/14 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
401Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
14containing three or more hetero rings
C07D 413/14 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
413Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
14containing three or more hetero rings
C07D 417/14 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
417Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/196
14containing three or more hetero rings
C07D 487/04 2006.1
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
C07D 487/10 2006.1
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
10Spiro-condensed systems
CPC
A61P 11/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
11Drugs for disorders of the respiratory system
A61P 13/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
13Drugs for disorders of the urinary system
A61P 13/12
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
13Drugs for disorders of the urinary system
12of the kidneys
A61P 25/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
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 3/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
Applicants
  • REMIX THERAPEUTICS INC. [US]/[US]
Inventors
  • REYNOLDS, Dominic
  • SEILER, Michael, W.
  • AGRAWAL, Anant, A.
  • VAILLANCOURT, Frederic
  • SMITH, Peter
  • HOPPER, Allen, T.
  • PRAJAPATI, Sudeep
  • VYSKOCIL, Stepan
Agents
  • LU, David
  • JUDA, Cristin, E.
  • LARKIN, Angelyn
Priority Data
63/047,89802.07.2020US
63/072,92131.08.2020US
63/126,49516.12.2020US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) 2-(INDAZOL-5-YL)-6-(PIPERIDIN-4-YL)-1,7-NAPHTHYRIDINE DERIVATIVES AND RELATED COMPOUNDS AS MODULATORS FOR SPLICING NUCLEIC ACIDS AND FOR THE TREATMENT OF PROLIFERATIVE DISEASES
(FR) DÉRIVÉS DE 2-(INDAZOL-5-YL)-6-(PIPERIDIN-4-YL)-1,7-NAPHTHYRIDINE ET COMPOSÉS Y RELATIFS EN TANT QUE MODULATEURS POUR L'ÉPISSAGE D'ACIDES NUCLÉIQUES ET POUR LE TRAITEMENT DE MALADIES PROLIFÉRANTES
Abstract
(EN) The present disclosure features compounds of formula (l-a) and of formula (II) and pharmaceutical compositions thereof. The present disclosure further discloses the compounds of formula (I- a) and (II) and their compositions for use in methods of modulating nucleic acid splicing, e.g., splicing of a pre-mRNA, as well as said compounds for use in methods of treating e.g.: • proliferative diseases, such as e.g. cancer, benign neoplasms or angiogenesis, • neurological diseases or disorders, such as e.g. Huntington's disease, • autoimmune diseases or disorders, immunodeficiency diseases or disorders, lysosomal storage disease or disorder, cardiovascular diseases or disorders, metabolic diseases or disorders, respiratory diseases or disorders, renal diseases or disorders, or infectious diseases. Exemplary compounds are e.g. 2-(indazol-5-yl)-6-(piperidin-4-yl)-l,7- naphthyridine derivatives and similar compounds.
(FR) La présente invention concerne des composés de formule (I-a) et de formule (II) et des compositions pharmaceutiques de ces dernières. La présente invention concerne les composés de formule (I- a) et (II) et leurs compositions aux fins d'utilisation dans des méthodes de modulation d'épissage d'acide nucléique, p.ex., épissage d'un pré-mRNA, ainsi que ces composés aux fins d'utilisation dans des méthodes de traitement p.ex.: • maladies proliférantes, telles que p.ex. le cancer, des néoplasmes bénins ou l'angiogénèse, • des maladies ou troubles neurologiques, tels que p.ex. la maladie de Huntington, • les maladies ou troubles autoimmuns, les maladies ou troubles immunodéficients, la maladie ou le trouble lysosomique de surcharge, les maladies ou troubles cardiovasculaires, les maladies ou troubles métaboliques, les maladies ou troubles respiratoires, les maladies ou troubles rénaux ou maladies infectieuses. Des composés cités à titre d'exemples sont p.ex. des dérivés de 2-(indazol-5-yl)-6-(pipéridin-4-yl)-l,7- naphthyridine et des composés similaires.
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