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1. (WO2018007475) PRE-MRNA SPLICE SWITCHING OR MODULATING OLIGONUCLEOTIDES COMPRISING BICYCLIC SCAFFOLD MOIETIES, WITH IMPROVED CHARACTERISTICS FOR THE TREATMENT OF GENETIC DISORDERS
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Pub. No.: WO/2018/007475 International Application No.: PCT/EP2017/066845
Publication Date: 11.01.2018 International Filing Date: 05.07.2017
IPC:
C12N 15/113 (2010.01) ,A61K 31/712 (2006.01) ,A61K 31/7125 (2006.01) ,A61P 21/00 (2006.01)
Applicants: BIOMARIN TECHNOLOGIES B.V.[NL/NL]; J.H. Oortweg 21 2333 CH Leiden, NL
Inventors: VAN DEUTEKOM, Judith Christina Theodora; NL
DE VISSER, Peter Christian; NL
Agent: NEDERLANDSCH OCTROOIBUREAU; P.O. Box 29720 2502 LS The Hague, NL
Priority Data:
16177973.105.07.2016EP
Title (EN) PRE-MRNA SPLICE SWITCHING OR MODULATING OLIGONUCLEOTIDES COMPRISING BICYCLIC SCAFFOLD MOIETIES, WITH IMPROVED CHARACTERISTICS FOR THE TREATMENT OF GENETIC DISORDERS
(FR) OLIGONUCLÉOTIDES DE COMMUTATION OU DE MODULATION D'ÉPISSAGE DE PRÉ-ARNM COMPRENANT DES FRAGMENTS D'ÉCHAFAUDAGE BICYCLIQUES, PRÉSENTANT DES CARACTÉRISTIQUES AMÉLIORÉES POUR LE TRAITEMENT DES TROUBLES D'ORIGINE GÉNÉTIQUE
Abstract:
(EN) The current invention provides antisense splice-switching oligonucleotides with improved characteristics that enhance clinical applicability for treating, ameliorating, preventing, and/or delaying neuromuscular disorders, such as DMD.
(FR) La présente invention concerne des oligonucléotides antisens de commutation d'épissage présentant des caractéristiques améliorées qui renforcent leur applicabilité clinique pour le traitement, l'amélioration, la prévention et/ou le retardement des troubles neuromusculaires, tels que la DMD.
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)