Patent SG-11201809753Y-A (SG)
Title: Reagents for treatment of hepatitis b virus (hbv) infection and use thereof
Inventor: MAO TIN
IPC: C12N15/113
Filed: 2017-05-05
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 1111111011110111111111111110101110111011111011111101111101111111111011110111111 Organization - - - ' International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2017/190197 Al 09 November 2017 (09.11.2017) W I P0 I P C T (51) (21) International Application Number: (22) International Filing Date: (25) Filing Language: (26) Publication Language: (30) (71) Applicant: BENITEC BIOPHARMA LIMITED (72) International Patent Classification: (81) Designated States (unless otherwise indicated, for every C12N 15/113 (2010.01) A61P 31/20 (2006.01) kind of national protection available): AE, AG, AL, AM, A61K 31/713 (2006.01) 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, DO, PCT/AU2017/050413 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, 05 May 2017 (05.05.2017) 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, English SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, English TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. Priority Data: (84) Designated States (unless otherwise indicated, for every 62/332,245 05 May 2016 (05.05.2016) US 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, [AU/AU]; Suite 1201, 99 Mount Street, North Sydney, New TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, South Wales 2060 (AU). EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, Inventors: MAO, Tin; 3831 Madera Way, San Bruno, Cal- MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, ifornia 94066 (US). SUHY, David; 32 Hurst Court, San Ra- TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, mon, California 94583 (US). KM, ML, MR, NE, SN, TD, TG). Agent: FB RICE; Level 23, 44 Market St, Sydney, New South Wales 2000 (AU). (74) = = (54) Title: REAGENTS FOR TREATMENT OF HEPATITIS B VIRUS (HBV) INFECTION AND USE THEREOF : This comprising or in combination with additional shmiRNA or shRNA. (57) = positions Figure 5 • Single stranded AAV8 • Permuted promoters to =I reduce shRNA expression (10- 1.00X) vs. wt promoters • Low Fidelity: pool of siliNAs Self complementary AAV8 Faster onset of sh RNA expression than vs single stranded ARV Permuted promoters to reduce sh RNA expression (10•180X) vs. wt promoters Low Fidefity: pool of siRNAs Single stranded AAV8 • Wild type Doi ill promoters for high expression Modell into miRNA backbone • High fidelity: 1-2 predominate species of siRNA • Reduce potential toxicity of hepatitis B virus (HBV) infection, cam- are artificial miRNA (shmiRNA) used = • t. ,_____.. = Ur:4 , P_ 1 - ARNA,12 ssAAV8 HBV-shRNAx3-v1 • • — = = = = = = U. - 644 , ac,smINA6 as t•rfiRNA•12. scAAV8 HBV-shRNAx3-v1 = Il .4 IN ll © 0\ 1-1 IN N alone i1 LiMienraiiiii6 : • : iRii.IS I ' shmik.a. ssAAV8 HBV-shmiRx3-v1 disclosure relates to RNA interference (RNAi) reagents for treatment same, and use thereof to treat individuals infected with HBV. The reagents C [Continued on next page] WO 2017/190197 Al MIDEDIMOMMIDIRMEMEEMIERMIEHMEMOIMIE Published: — with international search report (Art. 21(3)) — with sequence listing part of description (Rule 5.2(a))