Patent SG-11201805217X-A (SG)
Title: Compositions and methods for the treatment of hemoglobinopathies
Inventor: BOITANO ANTHONY EDWARD
IPC: C12N15/113
Filed: 2016-12-26
Abstract: 12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization 11111111011110111010101111101011111011101110111110101111011101111101111011111 International Bureau ... ..... ..)d (10) International Publication Number International Publication Date .... ...rr,' WO 2017/115268 Al 6 July 2017 (06.07.2017) WI P0 I P CT (51) (21) (22) (25) Filing Language: (26) Publication Language: (30) (71) (72) Inventors: BOITANO, Anthony Edward; 13037 Calvary International Patent Classification: Reddy; 110 Francis Wyman Road, Burlington, MA 01803 C12N 15/113 (2010.01) (US). SNEAD, Jennifer; c/o Novartis Institute for Func- tional Genomics, Inc., dba Genomics Institute of the No- International Application Number: yards Research Foundation, 10675 John Jay Hopkins PCT/IB2016/058007 Drive, San Diego, CA 92121 (US). STEVENSON, Susan International Filing Date: C.; c/o Novartis Institutes for Biomedical Research Inc., 26 December 2016 (26.12.2016) 250 Massachusetts Avenue, Cambridge, MA 02139 (US). STEWART, Morag; c/o Intellia Therapeutics, Inc., 40 English Erie Street, Suite 130, Cambridge, MA 02139 (US). English YANG, Yi; c/o Novartis Institutes for Biomedical Re- search Inc., 250 Massachusetts Avenue, Cambridge, MA Priority Data: 02139 (US). 62/271,968 28 December 2015 (28.12.2015) US 62/347,484 8 June 2016 (08.06.2016) US (74) Common Representative: NOVARTIS AG; Lichtstrasse 35, 4056 Basel (CH). Applicants: NOVARTIS AG [CH/CH]; Lichtstrasse 35, 4056 Basel (CH). INTELLIA THERAPEUTICS, INC. (81) Designated States (unless otherwise indicated, for every [US/US]; 40 Erie Street, Suite 130, Cambridge, MA 02139 kind of national protection available): AE, AG, AL, AM, (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, Ct., San Diego, CA 92129 (US). COOKE, Michael; 4265 HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, Ingleside Avenue, San Diego, CA 92103 (US). KLICK- KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, STEIN, Lloyd B.; c/o Novartis Institutes for Biomedical MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, Research Inc., 250 Massachusetts Avenue, Cambridge, MA NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, 02139 (US). LESCARBEAU, Reynald; c/o Intellia Thera- RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, peutics, Inc., 40 Erie Street, Suite 130, Cambridge, MA TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, 02139 (US). MICKANIN, Craig Stephen; c/o Novartis ZA, ZM, ZW. Institutes for Biomedical Research Inc., 250 Massachusetts Avenue, Cambridge, MA 02139 (US). MULUMBA, Ka- (84) Designated States (unless otherwise indicated, for every bungo; c/o Intellia Therapeutics, Inc., 40 Erie Street, Suite kind of regional protection available): ARIPO (BW, GH, 130, Cambridge, MA 02139 (US). POLICE, Seshidhar GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, [Continued on next page] (54) Title: COMPOSITIONS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES O pathies. Figure 1 458 Editing Efficiency by Location . , to , 0 Ot: , .4. ' t 2', .. 9. 1r 4 ..) for the treatment of hemoglobino - Il GC V; ei 11 - 1--1 1-1 (57) ei kr) 6449440 0 11 89494600 60494804 mama Generns C42101044 ph , : The present invention is directed to genome editing systems, 50495209 60483400 2) reagents and methods O WO 2017/115268 Al 1#1101MMIMIIMME3111010111IIMEHOIS TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, Published: TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, — DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, with international search report (Art. 21(3)) LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, — before the expiration of the time limit for amending the SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). claims and to be republished in amendments (Rule 48.2(h)) the event of receipt of Declarations under Rule 4.17: — as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(ii))