Patent SG-11201900028V-A (SG)
Title: Crispr/cas9-based compositions and methods for treating cancer
Inventor: JASKULA-RANGA VINOD
IPC: C12N15/85
Filed: 2017-07-05
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 1111111111111111111111111111111111111111111111111111111111111111111111111110111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\"\" WO 2018/009525 Al 11 January 2018 (11.01.2018) W I PO I PCT (51) International Patent Classification: TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, A61K 48/00 (2006.01) C12N 9/16 (2006.01) KM, ML, MR, NE, SN, TD, TG). A61K 38/46 (2006.01) Cl 2N 15/63 (2006.01) A61P 35/00 (2006.01) Cl 2N 15/85 (2006.01) Published: C12N 15/113 (2010.01) — with international search report (Art. 21(3)) (21) International Application Number: PCT/US2017/040696 (22) International Filing Date: 05 July 2017 (05.07.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/358,339 05 July 2016 (05.07.2016) US (71) Applicant: THE JOHNSON HOPKINS UNIVERSITY [US/US]; 3400 North Charles Street, Baltimore, MD 21218 (US). (72) Inventors: JASKULA-RANGA, Vinod; 1201 West Mount Royal Avenue, Unit 506, Baltimore, MD 21217 (US). ZACK, Donald; 3 Ruby Field Ct., Baltimore, MD 21209 (US). BUNZ, Fred; 10 Roland Mews, Baltimore, MD 21210 (US). Agent: RUSSELL, Hathaway, P. et al.; Foley Hoag (74) = LLP, Seaport West, 155 Seaport Boulevard, Boston, MA 02210-2600 (US). 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, 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. (81) 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, (84) 1-1 In N (54) Title: CRISPR/CAS9-BASED COMPOSITIONS AND METHODS FOR TREATING CANCER ir) CT (57) : Described herein are methods for preventing, inhibiting, or treating cancer in a subject. Also provided herein are methods of altering expression of one or more gene products in a cell, such as a cancer cell. Such methods may comprise utilizing a modified 0 ---- nuclease system, such as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR associated (Cas) 9 (CRISPR- GC , 1 Cas9) comprising a bidirectional HI promoter and gRNAs directed to oncogenes (rAAV-Onco-CRISPR) or tumor suppressor genes © (rAAV-TSG) packaged in a compact adeno-associated virus (AAV) particle. Such methods may comprise co-administering or concur- f'1 rently providing a recombinant adeno-associated virus-packaging adenovirus (Ad-rAAVpack) with the nuclease system. C