Patent SG-11201909433X-A (SG)
Title: Targeted compositions
Inventor: HEYES JAMES
IPC: C12N15/113
Filed: 2018-04-10
Abstract: NH A° 0 OAc O HN OAc O H NH O O N H ODMTr (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 18 October 2018 (18.10.2018) W I PO I PCT omit Oil l II Oil DID IIV X101111111 (10) International Publication Number WO 2018/191278 A2 (51) International Patent Classification: C12N 15/113 (2010.01) A61K 48/00 (2006.01) (21) International Application Number: PCT/US2018/026918 (22) International Filing Date: 10 April 2018 (10.04.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/484,247 11 April 2017 (11.04.2017) US 62/525,071 26 June 2017 (26.06.2017) US (71) Applicant: ARBUTUS BIOPHARMA CORPO- RATION [CA/CA]; 100-8900 Glenlyon Parkway, Burna- by, British Columbia V5J 5J8 (CA). (72) Inventors; and (71) Applicants: HEYES, James [CA/CA]; 100 - 8900 Glen- lyon Parkway, Burnaby, British Columbia V5J 5J8 (CA). HOLLAND, Richard J. [CA/CA]; 100 - 8900 Glenly- on Parkway, Burnaby, British Columbia V5J 5J8 (CA). JUDGE, Adam [CA/CA]; 100 - 8900 Glenlyon Parkway, Burnaby, British Columbia V5J 5J8 (CA). LEE, Amy C. H. [CA/CA]; 100 - 8900 Glenlyon Parkway, Burnaby, British Columbia V5J 5J8 (CA). MARTIN, Alan D. [CA/CA]; 100 - 8900 Glenlyon Parkway, Burnaby, British Columbia V5J 5J8 (CA). SNEAD, Nicholas Michael [US/CA]; 100 - 8900 Glenlyon Parkway, Burnaby, British Columbia V5J 5J8 (CA). THE Emily P. [CA/CA]; 100 - 8900 Glenlyon Park- way, Burnaby, British Columbia V5J 5J8 (CA). WOOD, Mark [CA/CA]; 100 - 8900 Glenlyon Parkway, Burnaby, British Columbia V5J 5J8 (CA). YE, Xin [CN/CA]; 100 - 8900 Glenlyon Parkway, Burnaby, British Columbia V5J 5J8 (CA). (74) Agent: MALEN, Peter L. et al.; Viksnins Harris Padys Malen LLP, 7851 Metro Parkway, Suite 325, Bloomington, Minnesota 55425 (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, 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. (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, (54) Title: TARGETED COMPOSITIONS AcO OAc HN O X d = NH Ac0 0 NH Xd Figure 1: Intermediate compound of formula le, wherein a targeting ligandilinker is bound to a 00 solid phase support, and wherein Pg is the protecting group DMTr. l 1 ' N (57) : The invention provides certain nucleic acids (e.g., double stranded siRNA molecules), as well as conjugates that comprise 1-1 a targeting moiety, a double stranded siRNA, and optional linking groups. Certain embodiments also provide synthetic methods useful for preparing the conjugates. The conjugates are useful to target therapeutic double stranded siRNA to the liver and to treat liver diseases O including hepatitis (e.g. hepatitis B and hepatitis D). C [Continued on next page] WO 2018/191278 A2 MIDEDIMOHNIIMEIHOMDERHOMOIMEEDIS TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: without international search report and to be republished upon receipt of that report (Rule 48.2(g))