Patent SG-11201807593T-A (SG)
Title: Compositions and methods to program therapeutic cells using targeted nucleic acid nanocarriers
Inventor: STEPHAN MATTHIAS
IPC: C12N15/62
Filed: 2017-04-14
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 19 October 2017 (19.10.2017) WIPO I PCT (10) International Publication Number WO 2017/181110 Al 111111111111110111011111111111010111110 0111H 011101110101111111111111110111111 (51) International Patent Classification: A61K 47/48 (2006.01) C07K 16/28 (2006.01) C07K 14/705 (2006.01) C12N 15/62 (2006.01) C07K 14/725 (2006.01) (21) International Application Number: PCT/US2017/027767 (22) International Filing Date: 14 April 2017 (14.04.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/322,581 14 April 2016 (14.04.2016) US 62/442,890 5 January 2017 (05.01.2017) US (71) Applicant: FRED HUTCHINSON CANCER RE- SEARCH CENTER [US/US]; 1100 Fairview Avenue North, Seattle, Washington 98109 (US). 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, 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, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). (72) Inventors: STEFAN, Matthias; 1100 Fairview Avenue Decl North, Seattle, Washington 98109 (US). MOFFETT, Howell F.; 1100 Fairview Avenue North, Seattle, Wash- ington 98109 (US). (74) Agents: WINGER, C. Rachal et al.; Lee & Hayes PLLC, 601 W. Riverside Ave., Suite 1400, Spokane, Washington 99201 (US). Published: (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, arations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.1700) as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.1 7 (iii)) — with international search report (Art. 21(3)) W O 20 17 / 18 1110 Al (54) Title: COMPOSITIONS AND METHODS TO PROGRAM THERAPEUTIC CELLS USING TARGETED NUCLEIC ACID NANOCARRIERS (57) : Compositions and methods that rapidly and selectively modify hematopoietic stem cells (or cells derived therefrom) to achieve therapeutic objectives by providing for transient expression of nucleic acids are described. The transient expression leads to permanent therapeutic changes in the modified cells, referred to herein as \"hit and run\" effects.