Patent SG-11201908418R-A (SG)
Title: Compositions and methods for selective elimination and replacement of hematopoietic stem cells
Inventor: OSTERTAG ERIC
IPC: A61K35/17
Filed: 2018-03-13
Abstract: Mature Blood Cells ® Progenitors ( ) ( 1 54 HSC Niche • •+s +.s •ks• : : FIGURE 3 e* S 4 ' \"•••• Total Body Irradiation or Busuffan Ablation of Progenitors + HSCs HSC Engraftment with Transplantation Myelosuppression CAR-T Cells Specific Ablation Clearance of . . . P : r\") Targeted to HSCs of HSCs CAR-T Cells ffi 4.1 0 Zt 1 O c e t , * 00 Antigen Targets 1 e 2 us 3 CAR-T Cells Selective to 1 — `s. Combination of 411 I HSC Antigens 1+2+3 CAR-T Cells Un reactive to Other Tissue Antigens 2 3 s 3 \ (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 20 September 2018 (20.09.2018) WIPOIPCT ill 1111u°11111DIVIIl °nolo om IIIII o!moIIHE111110 DIIIIII (10) International Publication Number WO 2018/169948 Al (51) International Patent Classification: A61K 35/17 (2015.01) C12N 5/0783 (2010.01) A61K 35/28 (2015.01) C12N 15/09 (2006.01) A61K 48/00 (2006.01) (21) International Application Number: PCT/US2018/022169 (22) International Filing Date: 13 March 2018 (13.03.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/470,814 13 March 2017 (13.03.2017) US 62/596,062 07 December 2017 (07.12.2017) US (71) Applicant: POSEIDA THERAPEUTICS, INC. [US/US]; 4242 Campus Point Court, Suite 700, San Diego, California 92121 (US). (72) Inventors: OSTERTAG, Eric; 4242 Campus Point Court, Suite 700, San Diego, California 92121 (US). SHED- LOCK, Devon; 4242 Campus Point Court, Suite 700, San Diego, California 92121 (US). DOWN, Julian David; 4242 Campus Pont Drive, San Diego, California 92121 (US). (74) Agent: MILLER, Katherine J. et al.; Cooley LLP, 1299 Pennsylvania Avenue, NW, 7th Floor, Washington, District of Columbia 20004 (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, (54) Title: COMPOSITIONS AND METHODS FOR SELECTIVE ELIMINATION AND REPLACEMENT OF HEMATOPOIETIC = STEM CELLS HSC Transplantation \ \ ,4km ‘If Engraftment with Maintenance of Blood Cell Production q) 2 • •,,e40,,,Ckr-. ... : GC 71- 00 O O (57) : Disclosed are methods of eliminating at least one target cell in a subject, comprising administering to the subject an effective amount of a composition comprising a plurality of immune cells, wherein each immune cell of the plurality expresses one or more chimeric ligand receptor(s) (CLR(s)) that each specifically bind to a target ligand on the at least one target cell, wherein specifically binding of the one or more CLR(s) to the target activates the immune cell, and wherein the activated immune cell induces death of the target cell. Exemplary target cells include, but are not limited to, hematopoietic stem cells (HSCs). [Continued on next page] WO 2018/169948 Al MIDEDIMOMOIDEIREEMOMOHIMEIMOIEHIMEN 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)) — with sequence listing part of description (Rule 5.2(a))