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1. WO2022006439 - CYCLIC COMPOUNDS FOR USE IN TREATING RETINAL DEGENERATION

Publication Number WO/2022/006439
Publication Date 06.01.2022
International Application No. PCT/US2021/040157
International Filing Date 01.07.2021
IPC
A61K 31/473 2006.1
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
435having six-membered rings with one nitrogen as the only ring hetero atom
47Quinolines; Isoquinolines
473ortho- or peri-condensed with carbocyclic ring systems, e.g. acridines, phenanthridines
A61K 31/475 2006.1
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
435having six-membered rings with one nitrogen as the only ring hetero atom
47Quinolines; Isoquinolines
475having an indole ring, e.g. yohimbine, reserpine, strychnine, vinblastine
A61K 31/498 2006.1
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
498Pyrazines or piperazines ortho- or peri-condensed with carbocyclic ring systems, e.g. quinoxaline, phenazine
A61P 27/02 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
27Drugs for disorders of the senses
02Ophthalmic agents
A61K 31/137 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
13Amines, e.g. amantadine
135having aromatic rings, e.g. methadone
137Arylalkylamines, e.g. amphetamine, epinephrine, salbutamol, ephedrine
Applicants
  • THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES [US]/[US]
Inventors
  • HUANG, Wenwei
  • MONDAL, Anupam
  • ZHENG, Wei
  • CHEN, Yu Holly
  • SWAROOP, Anand
  • SWAROOP, Manju
  • TAWA, Gregory
  • PAPAL, Samantha
  • LUO, Zhiji
Agents
  • SCHWARTZ, Johanna P.
  • RYBAK, Sheree Lynn
Priority Data
63/047,85802.07.2020US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) CYCLIC COMPOUNDS FOR USE IN TREATING RETINAL DEGENERATION
(FR) MODES DE RÉALISATION DE COMPOSÉ POUR LE TRAITEMENT DE LA DÉGÉNÉRESCENCE RÉTINIENNE ET MODES DE RÉALISATION DE PROCÉDÉ DE PRÉPARATION ET DE MÉTHODES D'UTILISATION DE CELUI-CI
Abstract
(EN) Method embodiments are disclosed for treating retinal degeneration in a subject in need thereof. In some embodiments, the method comprises administering to the subject a therapeutically effective amount of compound, and/or a pharmaceutically acceptable salt, prodrug, solvate, hydrate, or tautomer thereof, selected from 3-(dibutylamino)-1-(1,3-dichloro-6-(trifluoromethyl)phenanthren-9-yl)propan-1-ol hydrochloride or a compound having a structure according to a formula selected from Formula I, II, or III, as described herein. In some non-limiting examples, the subject has retinitis pigmentosa, LCA, Stargardt's macular dystrophy, cone-rod dystrophy, choroideremia or age-related macular degeneration.
(FR) Des modes de réalisation de méthode sont divulgués pour le traitement de la dégénérescence rétinienne chez un sujet en ayant besoin. Dans certains modes de réalisation, la méthode consiste à administrer au sujet une quantité thérapeutiquement efficace de composé, et/ou d'un sel, d'un promédicament, d'un solvate, d'un hydrate ou d'un tautomère associé pharmaceutiquement acceptable, choisi parmi un chlorhydrate de 3-(dibutylamino)-1-(1,3-dichloro-6-(trifluorométhyl)phénanthrèn-9-yl)propan-1-ol ou un composé ayant une structure selon une formule choisie parmi la formule I, II, ou III, telles que décrites dans la description. Dans certains exemples non limitatifs, le sujet présente une rétinite pigmentaire, une LCA, une dystrophie maculaire de Stargardt, une dystrophie des cônes et des bâtonnets, une choroïrémie ou une dégénérescence maculaire liée à l'âge.
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