Patent SG-11201804064W-A (SG)
Title: Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
Inventor: Falkner Falko-Günter
IPC: C12N15/86
Filed: 2016-11-11
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau IIIIII Hit II EH DID IIIII DEEM II III 111111111111111111111111 RID IIII Human wild-type FVIII MOB rre Refacto-type BDD-FVIII (10) International Publication Number WO 2017/083764 Al WIPO I PCT (43) International Publication Date 18 May 2017 (18.05.2017) 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, 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). Declarations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.170) 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)) (51) International Patent Classification: C07K 14/755 (2006.01) C12N 15/86 (2006.01) A61K 48/00 (2006.01) (21) International Application Number: PCT/US2016/061688 (22) International Filing Date: 11 November 2016 (11.11.2016) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/255,323 13 November 2015 (13.11.2015) US (71) Applicants: BAXALTA INCORPORATED [US/US]; 1200 Lakeside Drive, Bannockburn, IL 60015 (US). BAX- ALTA GMBH [CH/CH]; Thurgauerstrasse 130, 8152 Glattpark (Opfikon) (CH). (72) Inventors: FALKNER, Falko-Giinter; Neusiedlzeile 76A, 2304 Orth/Donau (AT). HORLING, Franziska; Bauemfeldgasse 3/9, 2230 Gaenserndorf (AT). LEN- GLER, Johannes; Schrotzbergstrabe 2/23, 1020 Vienna (AT). ROTTENSTEINER, Hanspeter; Haidgasse 10/17, 1020 Vienna (AT). SCHEIFLINGER, Friedrich; Michel- beuemgasse 4/17, 1090 Vienna (AT). (74) Agents: ANTCZAK, Andrew J. et al.; Morgan, Lewis & Bockius LLP, One Market, Spear Street Tower, San Fran- cisco, CA 94105 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (54) Title: VIRAL VECTORS ENCODING RECOMBINANT FVIII VARIANTS WITH INCREASED EXPRESSION FOR GENE THERAPY OF HEMOPHILIA A W O 20 17 /0837 6 4 Al tigtgall 11; tiff Figure 1 (57) : The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and meth- ods for treating hemophilia A.