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1. (WO2018225065) AMINOGLYCOSIDE DERIVATIVES AND USES THEREOF IN TREATING GENETIC DISORDERS
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Pub. No.: WO/2018/225065 International Application No.: PCT/IL2018/050612
Publication Date: 13.12.2018 International Filing Date: 05.06.2018
IPC:
C07H 15/224 (2006.01) ,C07H 15/23 (2006.01) ,C07H 15/222 (2006.01) ,A61P 21/04 (2006.01) ,A61P 21/00 (2006.01)
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
H
SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
15
Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
20
Carbocyclic rings
22
Cyclohexane rings, substituted by nitrogen atoms
222
Cyclohexane rings, substituted by at least two nitrogen atoms
224
with only one saccharide radical directly attached to the cyclohexane rings, e.g. destomycin, fortimicin, neamine
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
H
SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
15
Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
20
Carbocyclic rings
22
Cyclohexane rings, substituted by nitrogen atoms
222
Cyclohexane rings, substituted by at least two nitrogen atoms
226
with at least two saccharide radicals directly attached to the cyclohexane rings
228
attached to adjacent ring-carbon atoms of the cyclohexane rings
23
with only two saccharide radicals in the molecule, e.g. ambutyrosin, butyrosin, xylostatin, ribostamycin
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
H
SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
15
Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
20
Carbocyclic rings
22
Cyclohexane rings, substituted by nitrogen atoms
222
Cyclohexane rings, substituted by at least two nitrogen atoms
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
21
Drugs for disorders of the muscular or neuromuscular system
04
for myasthenia gravis
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
21
Drugs for disorders of the muscular or neuromuscular system
Applicants:
ELOXX PHARMACEUTICALS LTD. [IL/IL]; 10 Prof. Menachem Plaut Street 7670621 Rehovot, IL
Inventors:
BAASOV, Timor; IL
FIRZON, Vera; IL
BELAKHOV, Valery; IL
EYLON, Bat-Hen; IL
Agent:
EHRLICH, Gal; IL
WATERMAN, Hadassa; IL
MELNICK, Geoffrey, L.; IL
Priority Data:
62/515,02105.06.2017US
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 (e.g., a compound of Formula A, B, I, I*, III or III*, including compounds represented by Formula Ia, I**, I*a, I*b, IIIa, III**, III*a and, III*b), 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) L'invention concerne de nouveaux aminoglycosides, représentés par les formules (par exemple, un composé de formule A, B, I, I*, III ou III*, notamment des composés représentés par la formule Ia, I**, I*a, I*b, IIIa, III**, III*a et III*b), tels que définis dans la présente description, conçus pour présenter une activité de translecture de mutations de codons d'arrêt. L'invention concerne également des compositions pharmaceutiques contenant ceux-ci, et leurs utilisations dans le traitement de maladies et de troubles génétiques, tels que des maladies et des troubles associés aux mutations de codons d'arrêt.
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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)