Patent SG-11201901531T-A (SG)
Title: Regulation of gene expression using engineered nucleases
Inventor: MILLER JEFFREY C
IPC: C12N15/85
Filed: 2017-08-24
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 01 March 2018 (01.03.2018) W I PO I PCT limionommiollmonolooliiiimilomomolioluovoimill (10) International Publication Number WO 2018/039440 Al (51) International Patent Classification: C07K 14/47 (2006.01) A61K 35/12 (2015.01) C12N 9/16 (2006.01) A61K 38/46 (2006.01) C12N 15/85 (2006.01) (21) International Application Number: PCT/US2017/048397 (22) International Filing Date: 24 August 2017 (24.08.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/378,978 24 August 2016 (24.08.2016) US 62/443,981 09 January 2017 (09.01.2017) US 62/545,778 15 August 2017 (15.08.2017) US (71) Applicant: SANGAMO THERAPEUTICS, INC. [US/US]; Point Richmond Tech Center, 501 Canal Blvd., Suite A100, Richmond, California 94804 (US). (72) Inventors: MILLER, Jeffrey C.; c/o Sangamo Therapeu- tics, Inc., Point Richmond Tech Center, 501 Canal Blvd., Suite A100, Richmond, California 94804 (US). REBAR, Edward J.; c/o Sangamo Therapeutics, Inc., Point Rich- mond Tech Center, 501 Canal Blvd., Suite A100, Rich- mond, California 94804 (US). (74) Agent: PASTERNAK, Dahna S.; Pasternak Patent Law, 1900 Embarcadero Road, Suite 211, Palo Alto, California 94303 (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, 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, (54) Title: REGULATION OF GENE EXPRESSION USING ENGINEERED NUCLEASES 25.00 20.00 15.00 10.00 % 7 Glo bin Pro te in 0.00 U I 5.00 0 untransfected 63014/65722 untransfected 63014/65722 PB-M R-003 PB-M R-004 7/ % (Left) 7/Total 13 % (Right) FIG. 6 1-1 0 (57) : The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a \" hematopoietic cell. C [Continued on next page] WO 2018/039440 Al 1#110figinigE101010111001MOHOHOEMEEM 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))