Patent SG-11201907870V-A (SG)
Title: Compositions and methods for inhibition of lineage specific proteins
Inventor: BOLEN JOSEPH
IPC: C07K16/28
Filed: 2018-02-28
Abstract: Modifiedor deleted epitope No Lysis -1 cc 00 O C N (12) 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) WIPO I PCT omit VIII °nolo omioolimmoiflo oimIE (10) International Publication Number WO 2018/160768 Al AML Cell Lysis (51) International Patent Classification: A61K 35/12 (2015.01) CO7K 14/705 (2006.01) A61K 35/14 (2015.01) CO7K 16/28 (2006.01) A61K 35/17 (2015.01) C12N 5/078 (2010.01) A61K 35/38 (2015.01) C12N 5/0789 (2010.01) (21) International Application Number: PCT/US2018/020327 (22) International Filing Date: 28 February 2018 (28.02.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/464,975 28 February 2017 (28.02.2017) US (71) Applicant: VOR BIOPHARMA, INC. [US/US]; 501 Boylston Street, Suite 6102, Boston, MA 02116 (US). (72) Inventors: BOLEN, Joseph; 501 Boylston Street, Suite 6102, Boston, MA 02116 (US). RADOVIC-MORENO, Aleksandar, Filip; 501 Boylston Street, Suite 6102, Boston, MA 02116 (US). LYDEARD, John; 501 Boylston Street, Suite 6102, Boston, MA 02116 (US). (74) Agent: WITTE-GARCIA, Chelsea, E.; Wolf, Green- field & Sacks, P.C., 600 Atlantic Avenue, Boston, MA 02210-2206 (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, (54) Title: COMPOSITIONS AND METHODS FOR INHIBITION LINEAGE SPECIFIC PROTEINS FIGURE 3 B Targeting CD33+AML Cells No Binding to Edited Donor Graft Cells (57) : Disclosed herein are compositions, methods, and kits for use in treating hematopoietic malignancies, the compositions, methods, and kits comprise a cytotoxic agent targeting cells expressing a lineage-specific cell-surface protein and a population of hematopoietic cells that express the lineage-specific cell-surface protein, the hematopoietic cells being manipulated such that they do not bind the cytotoxic agent. [Continued on next page] WO 2018/160768 Al MIDEDIMOMMIDIREIDIDEMIEHEIHISIONVOIMIE 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))