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1. (WO2011123468) SIRNA THERAPY FOR TRANSTHYRETIN (TTR) RELATED OCULAR AMYLOIDOSIS
Latest bibliographic data on file with the International Bureau   

Pub. No.: WO/2011/123468 International Application No.: PCT/US2011/030392
Publication Date: 06.10.2011 International Filing Date: 29.03.2011
IPC:
A61K 31/7088 (2006.01) ,C07H 21/02 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
H
SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21
Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
02
with ribosyl as saccharide radical
Applicants:
ANDO, Yukio [US/US]; US
JONO, Hirofumi [US/US]; US
ALVAREZ, Rene [US/US]; US (UsOnly)
SAH, Dinah, Wen-Yee [US/US]; US (UsOnly)
ALNYLAM PHARMACEUTICALS, INC. [US/US]; 300 Third Street, Third Floor Cambridge, MA 02142, US (AllExceptUS)
KUMAMOTO UNIVERSITY [JP/JP]; 2-39-1 Kurokami Kumamoto, 860-8555, JP (AllExceptUS)
Inventors:
ANDO, Yukio; US
JONO, Hirofumi; US
ALVAREZ, Rene; US
SAH, Dinah, Wen-Yee; US
Agent:
SHUSTER, Michael, J.; Fenwick & West LLP Silicon Valley Center 801 California Street Mountain View, CA 94041, US
Priority Data:
61/318,70229.03.2010US
61/318,70429.03.2010US
Title (EN) SIRNA THERAPY FOR TRANSTHYRETIN (TTR) RELATED OCULAR AMYLOIDOSIS
(FR) THÉRAPIE SIARN POUR AMYLOSE OCULAIRE LIÉE À LA TRANSTHYRÉTINE (TTR)
Abstract:
(EN) The invention relates to a method of treating ocular amyloidosis by reducing TTR expression in a subject by administering a double-stranded ribonucleic acid (dsRNA) that targets a TTR gene to the retinal pigment epithelium of the subject.
(FR) La présente invention concerne un procédé de traitement de l'amylose oculaire par réduction de l'expression de la TTR chez un sujet, par l'administration d'un acide ribonucléique à double brins (dsARN) qui dirige un gène TTR vers l'épithélium pigmentaire rétinien du sujet.
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Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, 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, IS, JP, KE, KG, KM, KN, KP, KR, KZ, LA, LC, LK, LR, LS, LT, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PE, PG, PH, PL, PT, RO, RS, RU, 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, SD, SL, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, MD, 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, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)