Some content of this application is unavailable at the moment.
If this situation persist, please contact us atFeedback&Contact
1. (WO2017040271) MODIFIED ANTISENSE OLIGOMERS FOR EXON INCLUSION IN SPINAL MUSCULAR ATROPHY
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.: WO/2017/040271 International Application No.: PCT/US2016/048965
Publication Date: 09.03.2017 International Filing Date: 26.08.2016
IPC:
C12N 15/11 (2006.01) ,A61K 31/7088 (2006.01)
[IPC code unknown for C12N 15/11][IPC code unknown for A61K 31/7088]
Applicants:
SAREPTA THERAPEUTICS, INC. [US/US]; 215 First Street Cambridge, MA 02142, US
Inventors:
PASSINI, Marco, A.; US
HANSON, Gunnar, J.; US
Agent:
MCDONELL, Leslie, A.; US
Priority Data:
62/211,67828.08.2015US
62/379,69625.08.2016US
Title (EN) MODIFIED ANTISENSE OLIGOMERS FOR EXON INCLUSION IN SPINAL MUSCULAR ATROPHY
(FR) OLIGOMÈRES ANTISENS MODIFIÉS POUR L'INCLUSION D'EXONS DANS L'ATROPHIE MUSCULAIRE SPINALE
Abstract:
(EN) The present disclosure relates to modified antisense oligomers and related compositions and methods for increasing the expression of functional SMN protein and methods for treating spinal muscular atrophy and relates to inducing inclusion of exon 7 in SMN2 mRNA.
(FR) La présente invention concerne des oligomères antisens modifiés ainsi que des compositions et des méthodes associées pour augmenter l'expression d'une protéine SMN fonctionnelle et des méthodes de traitement de l'atrophie musculaire spinale et concerne l'induction d'inclusion de l'exon 7 dans l'ARNm de SMN2.
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)