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1. (WO2018223056) OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
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Pub. No.: WO/2018/223056 International Application No.: PCT/US2018/035687
Publication Date: 06.12.2018 International Filing Date: 01.06.2018
IPC:
A61K 48/00 (2006.01) ,C07H 21/00 (2006.01) ,C07H 21/02 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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
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:
VARGEESE, Chandra [US/US]; US (US)
IWAMOTO, Naoki [JP/US]; US (US)
BUTLER, David Charles Donnell [GB/US]; US (US)
MARAPPAN, Subramanian [US/US]; US (US)
LU, Genliang [US/US]; US (US)
ZHANG, Jason Jingxin [US/US]; US (US)
VATHIPADIEKAL, Vinod [IN/US]; US (US)
FRANK-KAMENETSKY, Maria David [US/US]; US (US)
APPONI, Luciano Henrique [BR/US]; US (US)
WAVE LIFE SCIENCES LTD. [SG/SG]; 7 Straits View #12-00 Marina One East Tower Singapore 018936, SG
Inventors:
VARGEESE, Chandra; US
IWAMOTO, Naoki; US
BUTLER, David Charles Donnell; US
MARAPPAN, Subramanian; US
LU, Genliang; US
ZHANG, Jason Jingxin; US
VATHIPADIEKAL, Vinod; US
FRANK-KAMENETSKY, Maria David; US
APPONI, Luciano Henrique; US
Agent:
LI, Xiaodong; US
ADATO, Ronen; US
AUGST, Alexander D.; US
BUTEAU, Kristen C.; US
BYCHOWSKI, Meaghan E.; US
HAULBROOK, William R.; US
JARRELL, Brenda Herschbach; US
KLEIN, Daniel A.; US
LI, Bin; US
LYON, Charles E.; US
MEDINA, Rolando; US
MONROE, Margo R.; US
NGUYEN, Suzanne P.; US
PACE, Nicholas J.; US
PYSHER, Paul A.; US
REARICK, John P.; US
REESE, Brian E.; US
SAHR, Robert N.; US
SCHONEWALD, Stephanie L.; US
SHAIKH, Nishat A.; US
SHINALL, Michael A.; US
SHORE, David E.; US
SMITH, Maria C.; US
SUH, Su Kyung; US
VETTER, Michael L.; US
VRABLIK, Tracy L.; US
Priority Data:
62/514,76902.06.2017US
62/514,77102.06.2017US
62/656,94912.04.2018US
62/670,68611.05.2018US
62/670,70911.05.2018US
Title (EN) OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
(FR) COMPOSITIONS D'OLIGONUCLÉOTIDES ET LEURS PROCÉDÉS D'UTILISATION
Abstract:
(EN) Among other things, the present disclosure provides designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown.
(FR) La présente invention concerne, entre autres, des oligonucléotides fabriqués, des compositions et des procédés associés. Selon certains modes de réalisation, des compositions d'oligonucléotides selon l'invention fournissent un ARN interférent simple brin amélioré et/ou un inactivation induite par RNase H. La présente invention concerne, entre autres, la reconnaissance que des éléments structuraux d'oligonucléotides, tels que la séquence de base, des modifications chimiques (par exemple, des modifications de liaisons de sucre, de base et/ou internucléotidiques) ou des motifs de celles-ci, la conjugaison avec des fractions chimiques supplémentaires, et/ou la stéréochimie [par exemple, la stéréochimie de centres chiraux de squelette (liaisons entre nucléotides chiraux)], et/ou des motifs de ceux-ci, peuvent avoir un impact significatif sur des propriétés et des activités d'oligonucléotides, par exemple, l'activité, la stabilité, l'administration d'ARN interférent (ARNi), et analogues. Selon certains modes de réalisation, la présente invention concerne des procédés de traitement de maladies au moyen de compositions d'oligonucléotides selon la présente invention, par exemple, en interférence ARN et/ou l'inactivation induite par RNase H.
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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)