Patent SG-11201809002R-A (SG)
Title: G-quadruplex-containing antisense oligonucleotides
Inventor: PHAN ANH TUAN
IPC: C12N15/113
Filed: 2017-05-02
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property MD HIM 01E101011M HO 11111 0 I 011111111111111101111111101110111 OEN Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2017/188898 Al 02 November 2017 (02.11.2017) WIP0 I PCT (51) International Patent Classification: (84) Designated States (unless otherwise indicated, for every Cl 2N 15/113 (2010.01) A61K 31/7088 (2006.01) kind of regional protection available): ARIPO (BW, GH, (21) International Application Number: GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, PCT/SG2017/050231 UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, (22) International Filing Date: EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, 02 May 2017 (02.05.2017) 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, (25) Filing Language: English KM, ML, MR, NE, SN, TD, TG). (26) Publication Language: English Published: (30) Priority Data: — with international search report (Art. 21(3)) 10201603451U 29 April 2016 (29.04.2016) SG — with sequence listing part of description (Rule 5.2(a)) (71) Applicant: NANYANG TECHNOLOGICAL UNIVERSITY [SG/SG]; 50 Nanyang Avenue, Singapore 639798 (SG). (72) Inventors: PHAN, Anh Tuan; c/o Nanyang Technological University, 50 Nanyang Avenue, Singapore 639798 (SG). LIM, Kah Wai; c/o Nanyang Technological University, 50 _ Nanyang Avenue, Singapore 639798 (SG). CHEONG, Vee Vee; c/o Nanyang Technological University, 50 Nanyang Avenue, Singapore 639798 (SG). LECH, Christopher BA- = _ an Henry Jacques; c/o Nanyang Technological Universi- _ ty, 50 Nanyang Avenue, Singapore 639798 (SG). HED- = DI, Brahim; c/o Nanyang Technological University, 50 - = Nanyang Avenue, Singapore 639798 (SG). (74) Agent: VIERING, JENTSCHURA & PARTNER LLP; P.O Box 1088, Rochor Post Office, Rochor Road, Singapore = 911833 (SG). (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. _ = = = = = = — = = (54) Title: G-QUADRUPLEX-CONTAINING ANTISENSE OLIGONUCLEOTIDES 1-1 (57) : The present invention relates to conjugates comprising (a) an anti- sense oligonucleotide (ASO) and (b) at least one G-quadruplex structure, wherein the Linker GC ASO and the at least one G-quadruplex structure are heterologous to each other. The G-quadruplex of the conjugate may be further conjugated with ligands or function- 0\ GC GC GC IN 1-1 mismaimisminarv\", al moieties for addressable delivery and enhanced properties including potency and .?; therapeutic index. Further encompassed are such conjugates for use as a medicament, Antisense 0-quadruplex a method of modulating the stability, translation, splicing, cleavage, or activity of a oligortucleotide target nucleic acid molecule using such conjugates, and use of G-quadruplex in sta- © ei bilizing antisense oligonucleotides. O