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1. (WO2017037719) AMINOGLYCOSIDE DERIVATIVES AND USES THEREOF IN TREATING GENETIC DISORDERS
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Pub. No.: WO/2017/037719 International Application No.: PCT/IL2016/050968
Publication Date: 09.03.2017 International Filing Date: 02.09.2016
IPC:
C07H 15/23 (2006.01) ,A61K 31/7036 (2006.01) ,A61P 7/04 (2006.01) ,A61P 21/00 (2006.01) ,A61P 21/04 (2006.01) ,C07H 15/222 (2006.01) ,C07H 15/224 (2006.01)
[IPC code unknown for C07H 15/23][IPC code unknown for A61K 31/7036][IPC code unknown for A61P 7/04][IPC code unknown for A61P 21][IPC code unknown for A61P 21/04][IPC code unknown for C07H 15/222][IPC code unknown for C07H 15/224]
Applicants:
ELOXX PHARMACEUTICALS LTD. [IL/IL]; 10 Prof. Menachem Plaut Street 7670621 Rehovot, IL
Inventors:
BAASOV, Timor; IL
TUVIA, Shmuel; IL
Agent:
EHRLICH, Gal; IL
WATERMAN, Hadassa; IL
MELNICK, Geoffrey; IL
Priority Data:
62/213,14302.09.2015US
62/213,18702.09.2015US
62/274,91505.01.2016US
Title (EN) AMINOGLYCOSIDE DERIVATIVES AND USES THEREOF IN TREATING GENETIC DISORDERS
(FR) DÉRIVÉS D'AMINOGLYCOSIDES ET LEURS UTILISATIONS DANS LE TRAITEMENT DE TROUBLES GÉNÉTIQUES
Abstract:
(EN) Novel aminoglycosides, represented by Formulae la and lb, as defined in the instant specification, designed to exhibit stop codon mutation readthrough activity, are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic diseases and disorders, such as diseases and disorders associated with stop codon mutations.
(FR) La présente invention porte sur de nouveaux aminoglycosides, représentés par les Formules la et lb, telles que définies dans la présente spécification, conçus pour faire preuve d'une activité de translecture d'une mutation du codon d'arrêt. L'invention concerne également des compositions pharmaceutiques les contenant et leurs utilisations dans le traitement de maladies et de troubles génétiques, tels que des maladies et des troubles associés à des mutations codon d'arrêt.
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, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, 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)