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1. (WO2016085852) TMPRSS6 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
Latest bibliographic data on file with the International Bureau   

Pub. No.:    WO/2016/085852    International Application No.:    PCT/US2015/062141
Publication Date: 02.06.2016 International Filing Date: 23.11.2015
IPC:
C12N 15/113 (2010.01), A61K 31/713 (2006.01)
Applicants: ALNYLAM PHARMACEUTICALS, INC. [US/US]; 300 Third Street 3rd Floor Cambridge, MA 02142 (US)
Inventors: BUTLER, James; (US).
MAIER, Martin; (US).
FITZGERALD, Kevin; (US).
FISHMAN, Shannon; (US).
FOSTER, Donald; (US).
JADHAV, Vasant; (US).
MILSTEIN, Stuart; (US)
Agent: ZACHARAKIS, Maria, Laccotripe; (US)
Priority Data:
62/083,691 24.11.2014 US
Title (EN) TMPRSS6 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
(FR) COMPOSITIONS À BASE D'ARNI TMPRSS6 ET LEURS PROCÉDÉS D'UTILISATION
Abstract: front page image
(EN)The invention relates to RNAi agents, e.g., double- stranded RNAi agents, targeting the TMPRSS6 gene, and methods of using such RNAi agents to inhibit expression of TMPRSS6 and methods of treating subjects having a TMPRSS6 associated disorder, e.g., an iron overload associated disorder, such as β-thalassemia or hemochromatosis.
(FR)L'invention concerne des agents à base d'ARNi, par exemple des agents à base d'ARNi double brin, ciblant le gène TMPRSS6, ainsi que des procédés d'utilisation desdits agents à base d'ARNi pour inhiber l'expression du gène TMPRSS6 et des méthodes de traitement de sujets présentant une maladie liée au gène TMPRSS6, par exemple une affection liée à une surcharge en fer, telle que la β-thalassémie ou l'hémochromatose.
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, 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, 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 (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (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)