Patent SG-11201407239S-A (SG)
Title: BI-FUNCTIONAL SHORT-HAIRPIN RNA (BI-SHRNA) SPECIFIC FOR SINGLE-NUCLEOTIDE <i>KRAS</i> MUTATIONS
Inventor: RAO DONALD
IPC: C12N15/85
Filed: 2013-05-09
Abstract: (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 14 November 2013 (14.11.2013) WIPOIPCT (10) International Publication Number WO 2013/170071 A1 (51) International Patent Classification: C12N15/113 (2010.01) C12N15/85 (2006.01) A61K 48/00 (2006.01) A ft IK 9/127 (2006.01) A61P 35/00 (2006.01) B82Y 5/00 (2011.01) (21) International Application Number: (22) International Filing Date: (25) Filing Language: (26) Publication Language: PCT/US2013/040407 9 May 2013 (09.05.2013) English (30) Priority Data: 61/644,875 13/890,957 9 May 2012 (09.05.2012) 9 May 2013 (09.05.2013) English US US (71) Applicant: GRADALIS, INC. [US/US]; 2545 Golden Bear Drive, Suite 110, Carollton, TX 75006 (US). (72) Inventors: RAO, Donald; 3355 Blackburn Street, Dallas, TX 75204 (US). WANG, Zhaohui; 4210 Valleywood Drive, Grapevine, TX 76051 (US). NEMUNAITIS, John J.; 1136 Mallard Pointe Drive, Cedar Hill, TX 75104-6911 (US). SENZER, Neil; 8 Milford Place, Dallas, TX 75230 (US). (74) Agents: CHALKER, Daniel J. et al.; Chalker Flores, LLP, 14951 North Dallas Parkway, Suite 400, Dallas, TX 75254 (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, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IS, JP, KE, KG, KM, KN, KP, KR, KZ, LA, LC, LK, LR, LS, LT, 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, 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, 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, ML, MR, NE, SN, TD, TG). Published: — with international search report (Art. 21(3)) — with sequence listing part of description (Rule 5.2(a)) (54) Title: BI-FUNCTIONAL SHORT-HAIRPIN RNA (BI-SHRNA) SPECIFIC FOR SINGLE-NUCLEOTIDE KRAS MUTA­ TIONS SamHS 4A5-1 SViKHt-S sxiyfA) l> o o l> i-H en HSV-TK W?4 Prrsii! A / \ SgSi / v FIG. 1 o CJ (57) Abstract: The present invention includes compositions and methods for making and using a bifunctional shRNAs capable of reducing an expression of K-ras a gene, e.g., a mutated K-ras gene, wherein at least one target site sequence of the bifunctional RNA Q molecule located within is the K-ras gene and wherein the bifunctional RNA molecule is capable of activating cleavage-dependent a and cleavage-independent RNA-induced silencing a complex for reducing the expression level of K-ras.