Patent SG-11202000145X-A (SG)
Title: Methods and systems for conditionally regulating gene expression
Inventor: WANG JIANBIN
IPC: A61P37/02
Filed: 2018-07-11
Abstract: International Patent Classification: A61K 35/17 (2015.01) A61P 37/02 (2006.01) A61K 38/00 (2006.01) CO7H 21/04 (2006.01) A61K 38/17 (2006.01) (21) International Application Number: PCT/US2018/041704 (22) International Filing Date: (25) Filing Language: (26) Publication Language: (30) Priority Data: 62/531,752 12 July 2017 (12.07.2017) US 62/587,668 17 November 2017 (17.11.2017) US (71) Applicant: REFUGE BIOTECHNOLOGIES, INC. [US/US]; 1505 Adams Drive, Suite D, Menlo Park, Califor- nia 94025 (US). (72) Inventors: WANG, Jianbin; 1505 Adams Drive, Suite D, Menlo Park, California 94025 (US). WANG, Bing; 1505 Adams Drive, Suite D, Menlo Park, California 94025 (US). 11 July 2018 (11.07.2018) English English LIU, Pei-Qi; 1505 Adams Drive, Suite D, Menlo Park, Cal- ifornia 94025 (US). (74) Agent: UJWAL, Rachna; Wilson Sonsini Goodrich & Rosati, 650 Page Mill Road, Palo Alto, California 94304-1050 (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, (54) Title: METHODS AND SYSTEMS FOR CONDITIONALLY REGULATING GENE EXPRESSION Gene A + .tbay. erte 8 Figure 1 (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) vitivio VIII oviolo Ennio oimIE (19) World Intellectual Property Organization International Bureau (10) International Publication Number WO 2019/014390 Al (43) International Publication Date 17 January 2019 (17.01.2019) WIPO I PCT T 'c signal pathway sgRNAa sgRNAb .;\ Trwlscri facton 70 dCas9NPR/KRAB ,Icste of a -ne dCas9 Function domain C C C (57) : The present disclosure provides systems, methods, and compositions for conditionally regulating expression of a target gene. Aspects of the present disclosure utilize intracellular signal transduction pathways to regulate the expression of a gene (e.g., transgene, exogenous gene, endogenous gene). O [Continued on next page] WO 2019/014390 Al MIDEDIMOREHIMMIEHOIROMEIMEHMEMOIS 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)) 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))