Patent SG-11201808831T-A (SG)
Title: Transgenic t cell and chimeric antigen receptor t cell compositions and related methods
Inventor: SADELAIN MICHEL
IPC: C12N15/63
Filed: 2017-04-14
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 11111 11111 0 11101 010 11111 010 1111 1 0 I 311101101111111101110 11110 1111 OM Organization International Bureau (10) International Publication Number (43) International Publication Date ......0\"\"1 WO 2017/180989 A3 19 October 2017 (19.10.2017) WIPO I PCT (51) International Patent Classification: (74) Agent: ANTLER, Adriane, M. et al.; Jones Day, 250 C12N 5/0783 (2010.01) C12N 9/22 (2006.01) Vesey Street, New York, NY 10281-1047 (US). C07K 14/725 (2006.01) C12N 15/63 (2006.01) (81) Designated States (unless otherwise indicated, for every (21) International Application Number: kind of national protection available): AE, AG, AL, AM, PCT/US2017/027601 AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, (22) International Filing Date: CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, 14 April 2017 (14.04.2017) 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, (25) Filing Language: English KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, (26) Publication Language: English 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, (30) Priority Data: SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, 62/323,623 15 April 2016 (15.04.2016) US TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. 62/323,675 16 April 2016 (16.04.2016) US (84) Designated States (unless otherwise indicated, for every 62/461,677 21 February 2017 (21.02.2017) US kind of regional protection available): ARIPO (BW, GH, 62/462,243 22 February 2017 (22.02.2017) US GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, (71) Applicant: MEMORIAL SLOAN KETTERING CAN- UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, CER CENTER [US/US]; 1275 York Avenue, New York, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, NY 10065 (US). 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, (72) Inventors: SADELAIN, Michel, W., J.; 444 Central Park = TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, = West, #8h, New York, NY 10025 (US). EYQUEM, Justin, KM, ML, MR, NE, SN, TD, TG). Gabriel, Andre, Francois; 310 East 66th Street, Apt. 2A, = New York, NY 10065 (US). MANSILLA-SOTO, Jorge; 72-72 112st Street, Apt. 3A, Forest Hills, NY 11375 (US). = = — (54) Title: TRANSGENIC T CELL AND CHIMERIC ANTIGEN RECEPTOR T CELL COMPOSITIONS AND RELATED METHODS ,as (57) : The invention provides a T cell wherein one or more therapeutic ,tt — transgenes is integrated at a within the genome of the cell such that expression = H 4 _ of the transgene is under control of an endogenous promoter of the T cell. The invention additional provides methods of making and using such cells to treat = 1 [1.1 , ... Im1 , 1 I a subject with T cell therapy. The invention also provides a T cell wherein a = w Eb. 9 282 ATI recombinantnucleic acid sequence encoding a chimeric antigen receptor (CAR) 1A---fri—rq = is integrated at a first site within the genome of the cell such that the CAR is _ FIG lA expressed by the cell at the surface of the cell, and wherein integration of the = nucleic acid encoding the CAR at the first site reduces or prevents expression of a = „... . Id 0 '' . functional T cell receptor (TCR) complex at the surface of the cell. The invention = — I* WI v additional provides methods of making and using such cells to treat a subject with CAR therapy. A en qqqq C:r 1 c. i FIG. 1 ES 7 It —T— C:r a 11 © «aas i A i GC FIG. 1C i FIG. 10 11 H IN 11 1 © ei Fin 1F C [Continued on next page] WO 2017/180989 A3 MIDEDIMOMONIIMMEMOMOHMO 1101HEHOMMIE 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 amendments (Rule 48.2(h)) of — with sequence listing part of description (Rule 5.2(a)) (88) Date of publication of the international search report: 14 December 2017 (14.12.2017)