Patent SG-11201903454V-A (SG)
Title: Immunologically discernible cell surface variants for use in cell therapy
Inventor: JEKER LUKAS
IPC: C12N15/10
Filed: 2017-10-30
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 1#111011110111010101111101 110100111111111111001110111 NH III OEN Organization International Bureau (10) International Publication Number (43) International Publication Date ......0\"\"1 WO 2018/083071 Al 11 May 2018 (11.05.2018) WIPO I PCT (51) International Patent Classification: Benkenstrasse 57 D, 4102 Binningen (CH). SCHWEDE, Cl 2N 15/10 (2006.01) A61K 35/12 (2015.01) Torsten; Benkenstrasse 57 D, 4102 Binningen (CH). LEP- Cl 2N 15/90 (2006.01) CO7K 14/705 (2006.01) ORE, Rosalba; Inhere Margarethenstrasse 15, 4051 Basel C12Q 1/68 (2018.01) (CH). MATTER MARONE, Romina; Im Niederholzbo- (21) International Application Number: den 53, 4125 Riehen (CH). RECHER, Mike; Hardstrasse 75, 4052 Basel (CH). PCT/EP2017/077826 (74) Agent: HEINZ, Benjamin; Schulz Junghans Patentan- (22) International Filing Date: 30 October 2017 (30.10.2017) walte PartGmbB, GroBbeerenstraBe 71, 10963 Berlin (DE). (25) Filing Language: English (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (26) Publication Language: English AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, (30) Priority Data: 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, 16196860.7 02 November 2016 (02.11.2016) EP HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, 16196858.1 02 November 2016 (02.11.2016) EP KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, PCT/EP2017/059799 MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, 25 April 2017 (25.04.2017) EP OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, 17197820.8 23 October 2017 (23.10.2017) EP SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, (71) Applicant: UNIVERSITAT BASEL [CH/CH]; Vizerek- TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. torat Forschung, Petersgraben 35, CH-4001 Basel (CH). (84) Designated States (unless otherwise indicated, for every Inventors: JEKER, Lukas; Aeussere Baselstrasse 75, kind of regional protection available): ARIPO (BW, GH, = (72) 4125 Riehen (CH). KORNETE, Mara; Burgfelderstrasse GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, = 35, 4055 Basel (CH). BORDOLI SCHWEDE, Lorenza; UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, = (54) Title: IMMUNOLOGICALLY DISCERNIBLE CELL SURFACE VARIANTS FOR USE IN CELL THERAPY Fig. 1 Naturally occurring surface protein Engineered surface protein = = — (allele/ soforrn A) displaying minor amino acid change monoclonal (allele/isoform B) — .rnody 'r 1 \ID A) A monoclonal antibody B (b B) ,_mA = Allele editing \"N;s = = = ------- N, --------.--N N ,-..\"---....--------- Cell , e --e--\"---------- Cell = mAb A binds mAb A does not bind mAb B does not bind mAb B binds = It (57) : The invention relates to a mammalian cell, particularly a human cell, expressing a first isoform of a surface protein, ' .46 E-• wherein the first isoform is functionally indistinguishable, but immunologically distinguishable from a second isoform, for use in a Il medical treatment of a patient having cells expressing the second isoform form of the surface protein. The invention further relates to , IN an agent selected from 1) a compound comprising, or consisting of, an antibody or antibody-like molecule and 2) an immune effector e7 cell bearing an antibody-like molecule or an immune effector cell bearing a chimeric antigen receptor, for use in a method of treatment GO of a medical condition, wherein the agent is specifically reactive to either a first or a second isoform of a surface protein, wherein the 5.\"_. :, :: first isoform is functionally indistinguishable, but immunologically distinguishable from the second isoform, and wherein the agent is GC administered to ablate a cell bearing the isoform that the agent is reactive to. Il 0 N O [Continued on next page] WO 2018/083071 Al MIDEDIMOMOIDEIREIDIONIMENNWHINEVOIMIE 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 the identity of the inventor (Rule 4.170 — as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(H)) 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))