Patent SG-11201907814S-A (SG)
Title: Methods and compositions for transducing and expanding lymphocytes and regulating the activity thereof
Inventor: FROST GREGORY IAN
IPC: C12N15/867
Filed: 2018-03-03
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 07 September 2018 (07.09.2018) WIP0 I PCT onion °nolo mu oloiloilooimmo oimIE (10) International Publication Number WO 2018/161064 Al (71) Applicant: Fl ONCOLOGY, INC. [US/US]; 625 N. Fla- gler Dr., Suite 625, West Palm Beach, Florida 33401 (US). (72) Inventors: FROST, Gregory Ian; 625 N. Flagler Dr., Suite 625, West Palm Beach, Florida 33401 (US). ONUFFER, James Joseph; 625 N. Flagler Dr., Suite 625, West Palm Beach, Florida 33401 (US). GUIBINGA, Ghiabe H.; 1613 Fort Stockton Drive, San Diego, California 92103 (US). HAERIZADEH, Farzad; 13954 Royal Dornoch Square, SAN DIEGO, California 92128 (US). (74) Agent: VACCHIANO, Emanuel J.; 8110 Woodside Lane, Burr Ridge, Illinois 60527 (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, (51) International Patent Classification: C12N 15/10 (2006.01) CO7K 14/705 (2006.01) C12N 15/867 (2006.01) CO7K 14/715 (2006.01) C12N 5/0783 (2010.01) A61K 48/00 (2006.01) C12N 5/10 (2006.01) C12N 15/86 (2006.01) CO7K 14/725 (2006.01) (21) International Application Number: PCT/US2018/020818 (22) International Filing Date: 03 March 2018 (03.03.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/467,039 03 March 2017 (03.03.2017) US 15/462,855 19 March 2017 (19.03.2017) US PCT/US2017/023112 19 March 2017 (19.03.2017) US 15/644,778 08 July 2017 (08.07.2017) US PCT/US2017/041277 08 July 2017 (08.07.2017) US 62/560,176 18 September 2017 (18.09.2017) US 62/564,253 27 September 2017 (27.09.2017) US 62/564,991 28 September 2017 (28.09.2017) US — (54) Title: METHODS AND COMPOSITIONS FOR TRANSDUCING AND EXPANDING LYMPHOCYTES AND REGULATING = THE ACTIVITY THEREOF W O 20 18/ 16 106 4 Al (57) : The present disclosure provides methods for genetically modify- ing lymphocytes and methods for performing adoptive cellular therapy that include transducing T cells and/or NK cells. The methods can include inhibitory RNA molecule(s) and/or engineered signaling polypeptides that can include a lymphopro- liferative element, and/or a chimeric antigen receptor (CAR), for example a microen- vironment restricted biologic CAR (MRB-CAR). Additional elements of such engi- neered signaling polypeptides are provided herein, such as those that drive prolifer- ation and regulatory elements therefor, as well as replication incompetent recombi- nant retroviral particles and packaging cell lines and methods of making the same. Numerous elements and methods for regulating transduced and/or genetically mod- ified T cells and/or NK cells are provided, such as, for example, those including ri- boswitches, MRB-CARs, recognition domains, and/or pH-modulating agents. [Continued on next page] WO 2018/161064 Al MIDEDIM01101 DIV IIMOU1111111111111111111111111111101111111110111111111111 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)) 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))