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1. (WO2017180857) GENE THERAPY FOR TREATING HEMOPHILIA A
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.: WO/2017/180857 International Application No.: PCT/US2017/027396
Publication Date: 19.10.2017 International Filing Date: 13.04.2017
IPC:
A61K 48/00 (2006.01) ,C12N 15/86 (2006.01) ,C07K 14/755 (2006.01)
Applicants: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA[US/US]; 3160 Chestnut Street Suite 200 Philadelphia, PA 19104, US
Inventors: WANG, Lili; US
WILSON, James, M.; US
SIDRANE, Jenny, Agnes; US
Agent: SCHALLER, Colleen M.; US
SMITH, George, A.; US
BAK, William; US
SCHALLER, Colleen, M.; US
KODROFF, Cathy, A.; US
DUMINIAK, Robert, J.; US
Priority Data:
62/323,33615.04.2016US
62/331,80704.05.2016US
62/428,86601.12.2016US
Title (EN) GENE THERAPY FOR TREATING HEMOPHILIA A
(FR) THÉRAPIE GÉNIQUE POUR LE TRAITEMENT DE L’HÉMOPHILIE A
Abstract:
(EN) Compositions and regimens useful in treating hemophilia A are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human Factor VIII.
(FR) L'invention concerne des compositions et des posologies utiles dans le traitement de l'hémophilie A. Les compositions comprennent un virus adéno-associé recombiné (rAAV) comportant un activateur de transthyrétine et un promoteur pour diriger l'expression d'un facteur VIII humain.
Designated States: 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
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (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)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)