Patent SG-11201808981X-A (SG)
Title: Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof
Inventor: SUHY DAVID
IPC: C12N15/113
Filed: 2017-04-13
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization 111111110111101110101011111010111110111010111011111010111011111111101111011111 International Bureau 0.. .... .. ...... ... (10) International Publication Number (43) International Publication Date WO 2017/177277 Al 19 October 2017(19.10.2017) WIPO I PCT (51) International Patent Classification: (74) Agent: FB RICE; Level 23, 44 Market St, Sydney, New C12N 15/113 (2010.01) A61K 48/00 (2006.01) South Wales 2000 (AU). (21) International Application Number: (81) Designated States (unless otherwise indicated, for every PCT/AU2017/050330 kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, (22) International Filing Date: BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, 13 April 2017 (13.04.2017) DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, (25) Filing Language: English 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, (26) Publication Language: English MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, (30) Priority Data: NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, 62/322,745 14 April 2016 (14.04.2016) US 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, (71) Applicant: BENITEC BIOPHARMA LIMITED ZA, ZM, ZW. [AU/AU]; Suite 1201, 99 Mount Street North Sydney, New South Wales 2060 (AU). (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, (72) Inventors: SUHY, David; 32 Hurst Court, San Ramon, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, California 94583 (US). GRAHAM, Michael; Apt 217, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, 1125 Park Place, San Mateo, California 94403 (US). TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, TROLLET, Capucine; c/o Sorbonne Universites, UPMC DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, _ Univ Paris 06, UM76, INSERM U974, Institut de Myolo- LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, — gie, CNRS FRE3617, 47 bd de l'Hopital, 75013 Paris (FR). SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, _ MALERBA, Alberto; c/o School of Biological Sciences, GW, KM, ML, MR, NE, SN, TD, TG). Royal Holloway, University of London, Egham Surrey = TW20 OEX (GB). DICKSON, George J.; c/o School of Published: Biological Sciences, Royal Holloway, University of Lon- — with international search report (Art. 21(3)) don, Egham Surrey TW20 OEX (GB). = — with sequence listing part of description (Rule 5.2(a)) = = = = = = = = (54) Title: REAGENTS FOR TREATMENT THEREOF = = = _ Il Ir ... ' ID Ir ... ' OF OCULOPHARYNGEAL MUSCULAR Figure 1 ROM DYSTROPHY (OPMD) AND USE N 0.00 IN ,-, eJ rn , r•I en < < < Z Z Z < < < Z Z Z N Il C ui 5 5 5 5 5, Si TS -0 \"Ci \"Ci \"Ci IN Il N (57) : The present disclosure relates to RNA interference (RNAi) reagents targeting PABPN1 for treatment of oculopharyn- geal muscular dystrophy (OPMD), compositions comprising same, and use thereof to treat individuals suffering from OPMD or 0 which are predisposed thereto.