Patent SG-11201901841T-A (SG)
Title: Targeting ligands
Inventor: LI ZHEN
IPC: C12N15/87
Filed: 2017-03-07
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 08 March 2018 (08.03.2018) WIPO I PCT °mons °nolo olo Ho mum loolionio oimIE (10) International Publication Number WO 2018/044350 Al (51) International Patent Classification: A61K 31/713 (2006.01) Cl 2N 15/87 (2006.01) C12N 15/11 (2006.01) (21) International Application Number: PCT/US2017/021147 (22) International Filing Date: 07 March 2017 (07.03.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/383,221 02 September 2016 (02.09.2016) US 62/456,339 08 February 2017 (08.02.2017) US (71) Applicant: ARROWHEAD PHARMACEUTICALS, INC [US/US]; 225 South Lake Avenue, Suite 1050, Pasade- na, CA 91101 (US). (72) Inventors: LI, Zhen; Arrowhead Pharmaceuticals, Inc, 502 S. Rosa Road, Madison, WI 53719 (US). PEI, Tao; Arrow- head Pharmaceuticals, Inc, 502 S. Rosa Road, Madison, WI 53719 (US). GLEBOCKA, Agnieska; Arrowhead Phar- maceuticals, Inc, 502 S. Rosa Road, Madison, WI 53719 (US). LAWLER, Michael; Arrowhead Pharmaceuticals, Inc, 502 S. Rosa Road, Madison, WI 53719 (US). FLEITZ, Fred; Arrowhead Pharmaceuticals, Inc, 502 S. Rosa Road, Madison, WI 53719 (US). ALTENHOFER, Erich; Arrow- head Pharmaceuticals, Inc, 502 S. Rosa Road, Madison, WI 53719 (US). KUMAR, Pankaj; Arrowhead Pharmaceuti- cals, Inc, 502 S. Rosa Road, Madison, WI 53719 (US). (84) 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). Declarations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(0) Published: — with international search report (Art. 21(3)) (74) Agent: TEIGEN, Robert, Michael; Arrowhead Pharma- ceuticals, Inc, 502 S. Rosa Road, Madison, WI 53719 (US). = (81) 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, 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. W O 20 18/04435 0 Al (54) Title: TARGETING LIGANDS (57) : Described are novel targeting ligands that may be linked to compounds, such therapeutic compounds that are useful in directing the compounds to the in vivo target. The targeting ligands disclosed herein can serve to target expression-inhibiting oligomeric compounds, such as RNAi agents, to liver cells to modulate gene expression. The targeting ligands disclosed herein, when conjugated to a therapeutic compound, may be used in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Compositions including the targeting ligands disclosed herein when linked to expression- inhibiting oligomeric compounds are capable of mediating expression of target nucleic acid sequences in liver cells, such as hepatocytes, which may be useful in the treatment of diseases or conditions that respond to inhibition of gene expression or activity in a cell, tissue, or organism.