Patent SG-11201908771Y-A (SG)
Title: Modified oligonucleotides and therapeutic uses thereof
Inventor: ROLOFF ALEXANDER
IPC: A61K48/00
Filed: 2018-03-21
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization O V SID o Ho SID Em VII IE International Bureau (10) International Publication Number WO 2018/175592 Al (43) International Publication Date 27 September 2018 (27.09.2018) WIPO I PCT (51) International Patent Classification: A61K 47/54 (2017.01) A61K 9/00 (2006.01) A61K 48/00 (2006.01) A61P 35/00 (2006.01) A61K 47/42 (2006.01) (21) International Application Number: PCT/US2018/023578 (22) International Filing Date: 21 March 2018 (21.03.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/475,185 22 March 2017 (22.03.2017) US (71) Applicants: THE REGENTS OF THE UNIVERSI- TY OF CALIFORNIA [US/US]; 1111 Franklin Street, 5th Floor, Oakland, California 94607 (US). VY- BYL HOLDINGS, INC. [US/US]; 2501 Davey Road, Woodridge, Illinois 60517 (US). (72) Inventors: ROLOFF, Alexander; 10300 North Torrey Pines Road, San Diego, California 92037 (US). GIAN- NESCHI, Nathan C.; 10300 North Torrey Pines Road, San Diego, California 92037 (US). CALLMANN, Cassandra E.; 10300 North Torrey Pines Road, San Diego, Califor- nia 92037 (US). THOMPSPN, Matthew P.; 10300 North Torrey Pines Road, San Diego, California 92037 (US). BERTIN, Paul A.; 2501 Davey Road, Woodridge, Illinois 60517 (US). (74) Agent: DAILEY, Robert S.; Kilpatrick Townsend & Stockton LLP, 1001 West Fourth Street, Winston Salem, North Carolina 27101 (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, (54) Title: MODIFIED OLIGONUCLEOTIDES AND THERAPEUTIC USES THEREOF 0 OH oligonucleotide 8 0 FIG. 1 (57) : The present disclosure generally provides nucleotide-based compounds useful for treating various diseases, including cancer. In some aspects, the disclosure provides oligonucleotides that are chemically modified to include an engineered fatty-acid residue, for example, to assist with improving the half-life of such compounds or assisting with cell penetration (e.g., penetration into tumor cells). In some aspects, the disclosure provides compositions that include such modified nucleotides and a protein, such as albumin or mimetics thereof. The disclosure provides various uses of the compounds and compositions. [Continued on next page] WO 2018/175592 Al MIDEDIM01101 DIDIREIDIOMOMIIII111111111111111111111111111111111 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, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: — with international search report (Art. 21(3))