Patent SG-11201908844Y-A (SG)
Title: Vectors and compositions for treating hemoglobinopathies
Inventor: VERES GABOR
IPC: C12N5/10
Filed: 2018-03-29
Abstract: RU9a9 RRE cPPT CoIEI Kanamycin r— f1 Ori bGH poly A -miR223 downsaeam miRNA223 upstream Venus cDNA B-Globin promoter' A-'C HS2 sin 18 R US HS3 Synthetic pA miR223 downstream miRNA223 upstream Exon1 betaGp HS2 W O 2018/ 18369 2 A8 (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) CORRECTED VERSION (19) World Intellectual Property Organization International Bureau (43) International Publication Date 11111111111110111111111111111111111110 1 1 111 11111 11111 11111 11111 1111 1 11 111111 1111 111 1111 (10) International Publication Number WO 2018/183692 A8 04 October 2018 (04.10.2018) WIP0 I PCT (51) International Patent Classification: A 61K 48/00 (2006.01) Cl 2N 15/867 (2006.01) A 61K 31/7088 (2006.01) Cl 2N 5/10 (2006.01) A61K 35/28 (2015.01) C07H 21/04 (2006.01) C12N 15/86 (2006.01) (21) International Application Number: PCT/US2018/025165 (22) International Filing Date: 29 March 2018 (29.03.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/478,375 29 March 2017 (29.03.2017) US 62/489,149 24 April 2017 (24.04.2017) US (71) Applicants: BLUEBIRD BIO, INC. [US/US]; 60 Bin- ney Street, Cambridge, Massachusetts 02142 (US). CHILDREN'S MEDICAL CENTER CORPORATION [IJS/US]; 55 Shattuck St., Boston, Massachusetts 02115 (US). (72) Inventors: VERES, Gabor; c/o bluebird bio. Inc., 60 Binney St., Cambridge, Massachusetts 02142 (US). WIL- LIAMS, David A.; 168 Centre St., Dover, Massachusetts 02030 (US). (74) Agent: ROGEL, Mark E. et al.; Cooley LLP, 1299 Penn- sylvania Avenue, NW, Suite 700, Washington, District of Columbia 20004 (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, (54) Title: VECTORS AND COMPOSITIONS FOR TREATING HEMOGLOBINOPATHIES (57) : The invention provides improved gene therapy vectors, compo- FIGURE 1 sitions, and methods. RSV [Continued on next page] WO 2018/183692 A8 I VIII 111111110111111 VIII 11111 OM MI 1 II 111 OM Mil Dill VIII 111110111111101111 I1II 1111 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). Declarations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(i1)) as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) Published: with international search report (Art. 21(3)) with sequence listing part of description (Rule 5.2(a)) (48) Date of publication of this corrected version: 02 May 2019 (02.05.2019) (15) Information about Correction: see Notice of 02 May 2019 (02.05.2019)