Patent SG-11201803701Y-A (SG)
Title: Methods and compositions for gene editing in hematopoietic stem cells
Inventor: GILL SAAR
IPC: A61P35/02
Filed: 2016-11-03
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -' Organization International Bureau ires.. 00) (43) International Publication Date ..... ..r ,„,„01 11 May 2017(11.05.2017) WIPO I PCT (10) WO International Publication Number 111111111111311111111111111111111111111111111111111111111111111111111311111111111111111 2017/079400 Al (51) International Patent Classification: AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, A61K 35/15 (2015.01) A61K 48/00 (2006.01) BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, A61K 35/17 (2015.01) A61P 35/00 (2006.01) DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, A61K 45/06 (2006.01) A61P 35/02 (2006.01) HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, (21) International Application Number: MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, PCT/US2016/060273 OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, (22) International Filing Date: SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, 3 November 2016 (03.11.2016) TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (25) Filing Language: English (84) Designated States (unless otherwise indicated, for every (26) Publication Language: English kind of regional protection available): ARIPO (BW, GH, (30) Priority Data: GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, 62/250,561 4 November 2015 (04.11.2015) US TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, (71) Applicant . THE TRUSTEES OF THE UNIVERSITY DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, OF PENNSYLVANIA [US/US]; 3160 Chestnut Street, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, Suite 200, Philadelphia, Pennsylvania 19104 (US). SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, (72) Inventors: GILL, Saar; 30 North Third Street, Apt. 4A, GW, KM, ML, MR, NE, SN, TD, TG). Philadelphia, Pennsylvania 19106 (US). KIM, Miriam; Declarations under Rule 4.17: 2400 Chestnut Street, Apt. 2604, Philadelphia, of inventorship (Rule 4.17(iv)) Pennsylvania 19103 (US). Published: (74) Agents: DOYLE, Kathryn et al.; Saul Ewing LLP, Centre Square West, 1500 Market Street, 38th Floor, Philadelphia, — with international search report (Art 21(3)) Pennsylvania 19102 (US). — with sequence listing part of description (Rule 5.2(a)) Designated (81) States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (54) Title: METHODS AND COMPOSITIONS FOR GENE EDITING IN HEMATOPOIETIC STEM CELLS A. trunc-gRNAvI trunc-gRNAv2 hum- RNAv3 trunc-gR NAv5 1-1 . .4t i I i CD33n ;40.3 S r x :310 1 0 / 0 it Cr\ IN O ir.... (57) : The present invention relates to compositions and 1-1 c:::, cells. One aspect of the invention includes a modified hematopoietic stem or progenitor cell comprising a nucleic acid capable of de N creasing expression of an endogenous gene or a portion thereof, wherein 0 antigen domain targeted by a chimeric antigen receptor (CAR). Another ,1 . modified hematopoietic stem or progenitor cell. Also included are fled cell for adoptive therapy and treating a condition, such as an autoimmune 1)33neg 364 ^890 , 1 t Figures 1 methods of generating modified hematopoietic stem or progenitor - the endogenous gene encodes a polypeptide comprising an aspect of the invention includes a method for generating a methods and pharmaceutical compositions comprising the modi - disease or cancer.