Patent SG-11201901140Q-A (SG)
Title: Compounds and methods for reducing tau expression
Inventor: KORDASIEWICZ HOLLY
IPC: C12N15/113
Filed: 2017-09-29
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 11111111111111111111111111111111111111111111111111111111111111111111111111111111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2018/064593 Al 05 April 2018 (05.04.2018) WIP0 I PCT (51) International Patent Classification: C12N 15/113 (2010.01) (21) International Application Number: PCT/US2017/054540 (22) International Filing Date: 29 September 2017 (29.09.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/401,723 29 September 2016 (29.09.2016) US 62/450,469 25 January 2017 (25.01.2017) US (71) Applicant: IONIS PHARMACEUTICALS, INC. [US/US]; 2855 Gazelle Court, Carlsbad, CA 92010 (US). (72) Inventor: KORDASIEWICZ, Holly; 2855 Gazelle Ct., Carlsbad, CA 92010 (US). (74) Agent: SCARR, Rebecca, B. et al.; McNeill Baur PLLC, 125 Cambridge Park Drive, Suite 301, Cambridge, MA 02140 (US). Designated States (unless otherwise indicated, for every kind of national protection available): 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. (81) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, 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, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). (84) Published: with international search report (Art. 21(3)) — — with sequence listing part of description (Rule 5.2(a)) M ON (54) Title: COMPOUNDS AND METHODS FOR REDUCING TAU EXPRESSION in 71' (57) : Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of Tau mRNA in a cell or animal, and in certain instances reducing the amount of Tau protein in a cell or animal. Such compounds, methods, and ---- pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include loss GC of memory, loss of motor function, and increase in the number and/or volume of neurofibrillary inclusions. Such neurodegenerative 1-1 © diseases include tauopathies, Alzheimer's Disease, Fronto-temporal Dementia (FTD), FTDP-17, Progressive Supranuclear Palsy (PSP), f'1 Chronic Traumatic Encephalopathy (CTE), Corticobasal Ganglionic Degeneration (CBD), Epilepsy, and Dravet's Syndrome. C