Patent SG-11201804729R-A (SG)
Title: Methods and compositions for treating a serpinc1-associated disorder
Inventor: AKINC AKIN
IPC: C12N15/113
Filed: 2016-12-07
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) CORRECTED VERSION (19) World Intellectual Property Organization International Bureau .9 . si wiel y) (43) International Publication Date sawe 15 June 2017(15.06.2017) WIPO I PCT ID Hit (10) WO 111111111111111111111111111111 International III 2017/100236 Publication 111111111111111111111111111111t111111111111111 Number A8 (51) International Patent Classification: (81) Designated States (unless otherwise indicated, for every A61K 31/713 (2006.01) A61P 7/04 (2006.01) kind of national protection available): AE, AG, AL, AM, C12N 15/113 (2010.01) 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, (21) International Application Number: DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, PCT/US2016/065245 HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, (22) International Filing Date: KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, 7 December 2016 (07.12.2016) 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, (25) Filing Language: English SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, (26) Publication Language: English TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (30) Priority Data: 62/264,013 7 December 2015 (07.12.2015) US (84) Designated States (unless otherwise indicated, for every 62/315,228 30 March 2016 (30.03.2016) US kind of regional protection available): ARIPO (BW, GH, 62/366,304 25 July 2016 (25.07.2016) US GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, 62/429,241 2 December 2016 (02.12.2016) US TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, (71) Applicant: ALNYLAM PHARMACEUTICALS, INC. DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, [US/US]; 300 Third Street, 3rd Floor, Cambridge, MA LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, 02142 (US). TR), BJ, SM, OAPI (BF, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). (72) Inventors: AKINC, Akin; 300 Third Street, 3rd Floor, Cambridge, MA 02142 (US). SORENSEN, Benny; 300 Published: Third Street, 3rd Floor, Cambridge, MA 02142 (US). — with international search report (Art 21(3)) GARG, Pushkal; 300 Third Street, 3rd Floor, Cambridge, MA 02142 ROBBIE, Gabriel; 300 Third Street, 3rd — with sequence listing of description (Rule 5.2(a)) (US). part Floor, Cambridge, MA 02142 (US). (48) Date of publication of this corrected version: (74) Agents: ZACHARAKIS, Maria Laccotripe et al.; Mc- 20 July 2017 Carter & English, LLP, 265 Franklin Street, Boston, MA Information (15) about Correction: 02110 (US). see Notice of 20 July 2017 Ge .4 t M ei O O 1-1 it - - 1-1 0 (54) Title: METHODS AND COMPOSITIONS FOR TREATING A SERPINC1-ASSOCIATED DISORDER ei 0 (57) : The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc l ,1 . gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc 1 and to treat subjects having a Serpinc 1-associated disease, e.g., a bleeding disorder, such as a hemophilia.