Patent SG-11201809144X-A (SG)
Title: Materials and methods for treatment of hemoglobinopathies
Inventor: COWAN CHAD
IPC: C12N15/113
Filed: 2017-04-18
Abstract: targetDNA er'cif int e er s t { 3 , 11111 Him complement of PAM linker loop spacer sequence co co co N O C (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 26 October 2017 (26.10.2017) omit VIII VIII lII 0 HE (10) International Publication Number WO 2017/182881 A3 WIPO I PCT (51) International Patent Classification: A61K 38/46 (2006.01) Cl 2N 15/10 (2006.01) C12N 9/22 (2006.01) Cl 2N 15/113 (2010.01) (21) International Application Number: PCT/IB2017/000577 (22) International Filing Date: 18 April 2017 (18.04.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/324,024 18 April 2016 (18.04.2016) US 62/382,522 01 September 2016 (01.09.2016) US 62/429,428 02 December 2016 (02.12.2016) US (71) Applicant: CRISPR THERAPEUTICS AG [CH/CH]; Baarerstrasse 14, 6300 Zug (CH). (72) Inventors: COWAN, Chad, Albert; 610 Main Street, North Building, 7th Floor, Cambridge, MA 02139 (US). LUNDBERG, Ante, Sven; 610 Main Street, North Building, 7th Floor, Cambridge, MA 02139 (US). CHAKRABORTY, Tirtha; 610 Main Street, North Build- ing, 7th Floor, Cambridge, MA 02139 (US). LIN, Michelle, I-ching; 610 Main Street, North Building, 7th Floor, Cam- bridge, MA 02139 (US). MISHRA, Bibhu, Prasad; 610 Main Street, North Building, 7th Floor, Cambridge, MA 02139 (US). PAIK, Elizabeth, Jae-eun; 610 Main Street, North Building, 7th Floor, Cambridge, MA 02139 (US). KERNYTSKY, Andrew; 610 Main Street, North Build- ing, 7th Floor, Cambridge, MA 02139 (US). BORLAND, Todd, Douglass; 610 Main Street, North Building, 7th Floor, Cambridge, MA 02139 (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, 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: MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES Figure 2B protospacer crRNA tracr RNA chimera ( sg RNA) (57) : Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for deleting, modulating, or inactivating a transcriptional control sequence of a BCL11A gene in a cell by genome editing. [Continued on next page] WO 2017/182881 A3 MUNRO DOI 01011M010111311111111111111111111011111110fin 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)) with sequence listing part of description (Rule 5.2(a)) (88) Date of publication of the international search report: 30 November 2017 (30.11.2017)