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1. (WO2018144709) GENE THERAPY FOR TREATING CITRULLENEMIA
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Pub. No.: WO/2018/144709 International Application No.: PCT/US2018/016413
Publication Date: 09.08.2018 International Filing Date: 01.02.2018
IPC:
A61K 35/76 (2015.01) ,A61K 38/16 (2006.01)
[IPC code unknown for A61K 35/76][IPC code unknown for A61K 38/16]
Applicants:
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA [US/US]; 3160 Chestnut Street Suite 200 Philadelphia, PA 19104, US
Inventors:
WILSON, James, M.; US
SIDRANE, Jenny, Agnes; US
WANG, Lili; US
Agent:
SCHALLER, Collen, M.; US
BAK, Mary, E.; US
BAK, William; US
SMITH, George, A.; US
KODROFF, Cathy, A.; US
Priority Data:
62/453,42401.02.2017US
62/469,65010.03.2017US
Title (EN) GENE THERAPY FOR TREATING CITRULLENEMIA
(FR) THÉRAPIE GÉNIQUE POUR LE TRAITEMENT DE CITRULLINÉMIE
Abstract:
(EN) Compositions and regimens useful in treating type I citrullenemia are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human Argininosuccinate Synthase 1 (ASS1).
(FR) L'invention concerne des compositions et des posologies utiles dans le traitement de citrullinémie de type 1. Les compositions comprennent un virus adéno-associé recombinant (rAAV) avec un activateur de transthyrétine et un promoteur entraînant l'expression d'une Argininosuccinate synthase 1 (ASS1) humaine.
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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, 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
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)