Patent SG-11201803951Q-A (SG)
Title: Oligonucleotides for inducing paternal ube3a expression
Inventor: COSTA VERONICA
IPC: C12N15/113
Filed: 2016-11-11
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property - Organization International Bureau (43) International Publication Date .... .....r„..1 18 May 2017 (18.05.2017) WIPO I PCT ID Hit (10) WO International 2017/081223 1111111111111111111111111111111111111111111111111111111111111t111111111111111 Publication Number Al (51) International Patent Classification: zacherstrasse 124, 4070 Basel (CH). PEDERSEN, Lykke; C12N 15/113 (2010.01) C12N 15/11 (2006.01) Roche Innovation Center Copenhagen A/S, Fremtidsvej 3, 2970 Horsholm (DK). RASMUSSEN, Soren Vester- (21) International Application Number: gaard; Roche Innovation Center Copenhagen A/S, Fremt- PCT/EP2016/077383 idsvej 3, 2970 Horsholm (DK). (22) International Filing Date: (74) Agent: RUDOLPH HANSEN, Marianne; Fremtidsvej 3, 11 November 2016 (11.11.2016) 2970 Horsholm (DK). (25) Filing Language: English (81) Designated States (unless otherwise indicated, for every (26) Publication Language: English kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, (30) Priority Data: BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, 15194367.7 12 November 2015 (12.11.2015) EP DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, 16189502.4 19 September 2016 (19.09.2016) EP HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, (71) Applicant: ROCHE INNOVATION CENTER COPEN- KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, HAGEN A/S [DK/DK]; Fremtidsvej 3, 2970 Horsholm MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, (DK). 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, (71) Applicant (for all designated States except US): F. HOFF- TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, MANN-LA ROCHE AG [CH/CH]; Grenzacherstrasse ZW. 124, 4070 Basel (CH). (84) Designated States (unless otherwise indicated, for every (71) Applicant (for US only): HOFFMANN-LA ROCHE kind of regional available): ARIPO (BW, GH, protection INC. [US/US]; Overlook at Great Notch, 150 Clove Road, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, 8th Floor, Suite 8 - Legal Department, Little Falls, New TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, Jersey 07424 (US). TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, (72) Inventors: COSTA, Veronica; F. Hoffmann-La Roche LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, AG, Grenzacherstrasse 124, 4070 Basel (CH). TR), BJ, SM, OAPI (BF, CF, CG, CI, CM, GA, GN, GQ, HEDTJARN, Maj; Roche Innovation Center Copenhagen GW, KM, ML, MR, NE, SN, TD, TG). A/S, Fremtidsvej 3, 2970 Horsholm HOENER, (DK). Marius; F. Hoffmann-La Roche AG, Grenzacherstrasse Published: 124, 4070 Basel (CH). JAGASIA, Ravi; F. Hoffmann-La — with international search report (Art 21(3)) Roche AG, Grenzacherstrasse 124, 4070 Basel (CH). JENSEN, Mads Aaboe; Roche Innovation Center Copen- — with sequence listing part of description (Rule 5.2(a)) hagen A/S, Fremtidsvej 3, 2970 Horsholm (DK). PATSCH, Christoph; F. Hoffmann-La Roche AG, Gren- 1-1 .4t M ei ei 1-1 ot O N 1-1 (54) Title: OLIGONUCLEOTIDES FOR INDUCING PATERNAL UBE3A EXPRESSION 0 ei (57) : The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase ea ) E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A pa- ternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD109B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.