SG11201908719QA - Biomarkers and car t cell therapies with enhanced efficacy - Google Patents

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 27 September 2018 (27.09.2018) WIPO I PCT onion °nolo oloinomll n ono oimIE (10) International Publication Number WO 2018/175733 Al (51) International Patent Classification: A61K 39/00 (2006.01) C07K 16/28 (2006.01) (21) International Application Number: PCT/US2018/023785 (22) International Filing Date: 22 March 2018 (22.03.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/474,991 22 March 2017 (22.03.2017) US 62/621,356 24 January 2018 (24.01.2018) US (71) Applicants: NOVARTIS AG [CH/CH]; Lichtstrasse 35, 4056 Basel (CH). THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA [US/US]; 3160 Chestnut Street, Suite 200, Philadelphia, PA 19104 (US). (72) Inventors: FRAIETTA, Joseph, A.; 18 Saddle Lane, Cherry Hill, NJ 08002 (US). MELENHORST, Jan, J.; 11 Exton Circle, Cherry Hill, NJ 08003 (US). (74) Agent: COLLAZO, Diana M. et al.; Lando & Anastasi, LLP, Riverfront Office Park, One Main Street, Suite 1100, Cambridge, MA 02142 (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, 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). Declarations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(ii)) as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) Published: with international search report (Art. 21(3)) before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) with sequence listing part of description (Rule 5.2(a)) 1-1 (54) Title: BIOMARKERS AND CAR T CELL THERAPIES WITH ENHANCED EFFICACY (57) : The invention provides compositions and methods improved CART cell therapies. Specifically, the invention provides 0 cells with altered expression and/or function of one or more genes, e.g., associated with Tet2, and methods of use therefore. The \" invention further provides inhibitors of the one or more genes and methods of use therefore in connection with CART cells.