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1. (WO2019028440) METHODS AND COMPOSITIONS FOR MODULATING SPLICING
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Pub. No.: WO/2019/028440 International Application No.: PCT/US2018/045282
Publication Date: 07.02.2019 International Filing Date: 03.08.2018
IPC:
A61K 31/439 (2006.01) ,A61P 25/00 (2006.01) ,C07D 471/08 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
33
Heterocyclic compounds
395
having nitrogen as a ring hetero atom, e.g. guanethidine, rifamycins
435
having six-membered rings with one nitrogen as the only ring hetero atom
439
the ring forming part of a bridged ring system, e.g. quinuclidine
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25
Drugs for disorders of the nervous system
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
471
Heterocyclic 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
02
in which the condensed system contains two hetero rings
08
Bridged systems
Applicants:
SKYHAWK THERAPEUTICS, INC. [US/US]; 35 Gatehouse Drive Waltham, Massachusetts 02451, US
Inventors:
LUZZIO, Michael; US
MCCARTHY, Kathleen; US
HANEY, William; US
Agent:
KUZNETSOV, ALEXEY; US
Priority Data:
62/541,20204.08.2017US
62/562,92725.09.2017US
62/562,94825.09.2017US
Title (EN) METHODS AND COMPOSITIONS FOR MODULATING SPLICING
(FR) MÉTHODES ET COMPOSITIONS PERMETTANT DE MODULER L’ÉPISSAGEÉ
Abstract:
(EN) Described herein are small molecule splicing modulator compounds that modulate splicing of mRNA, such as pre-mRNA, encoded by genes, and methods of use of the small molecule splicing modulator compounds for modulating splicing and treating diseases and conditions.
(FR) La présente invention concerne des composés modulant l’épissage de petites molécules qui modulent l’épissage d’ARNm, tel que le pré-ARNm codé par les gènes, et des méthodes d’utilisation des composés modulant l’épissage de petites molécules pour moduler l’épissage et traiter des maladies et des pathologies.
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Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)