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1. (WO2018026282) ANTISENSE OLIGOMERIC COMPOUND FOR POMPE DISEASE
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Pub. No.: WO/2018/026282 International Application No.: PCT/NL2017/050525
Publication Date: 08.02.2018 International Filing Date: 04.08.2017
IPC:
C12N 15/113 (2010.01)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
Applicants:
ERASMUS UNIVERSITY MEDICAL CENTER ROTTERDAM (ERASMUS MC) [NL/NL]; Dr. Molewaterplein 50 3015 GE Rotterdam, NL
Inventors:
PIJNAPPEL, Wilhelmus Wenceslaus Matthias; NL
VAN DER PLOEG, Antje Tjitske; NL
VAN DER WAL, Erik; NL
BERGSMA, Atze Jacobus; NL
Agent:
JANSEN, C.M.; V.O. Carnegieplein 5 2517 KJ Den Haag, NL
Priority Data:
201729505.08.2016NL
Title (EN) ANTISENSE OLIGOMERIC COMPOUND FOR POMPE DISEASE
(FR) COMPOSÉ OLIGOMÈRE ANTISENS DESTINÉ AU TRAITEMENT DE LA MALADIE DE POMPE
Abstract:
(EN) The present invention comprises an antisense oligomeric compound targeting the region around cryptic splice site at c.546+ 184, that is utilized in the presence of c.546 mutations in the GAA gene. Such oligonucleotides may be used in the treatment of Pompe disease. Also claimed is a method for treating Pompe disease comprising administration of such a compound to a person in need thereof.
(FR) L'invention concerne un composé oligomère antisens ciblant la région située autour du site d'épissage cryptique à c.546+184, qui est utilisé en présence de mutations c.546 dans le gène GAA. Ces oligonucléotides peuvent être utilisés dans le traitement de la maladie de Pompe. L'invention concerne également une méthode de traitement de la maladie de Pompe, consistant à administrer ledit composé à un individu nécessitant un tel traitement.
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)