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1. (WO2019032607) OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
Latest bibliographic data on file with the International BureauSubmit observation

Pub. No.: WO/2019/032607 International Application No.: PCT/US2018/045653
Publication Date: 14.02.2019 International Filing Date: 07.08.2018
IPC:
A61K 31/7115 (2006.01) ,A61K 31/712 (2006.01) ,A61K 31/7125 (2006.01) ,A61K 48/00 (2006.01) ,C07H 21/02 (2006.01) ,C07H 21/04 (2006.01) ,C12N 15/11 (2006.01) ,C12N 15/113 (2010.01)
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
7115
Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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
712
Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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
7125
Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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
02
with ribosyl as saccharide radical
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
04
with deoxyribosyl as saccharide radical
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
Applicants:
WAVE LIFE SCIENCES LTD. [SG/SG]; 7 Straits View #12-00 Marina One East Tower Singapore 018936, SG
DODART, Jean-Cosme [US/US]; US (US)
LIU, Yuanjing [CN/US]; US (US)
VARGEESE, Chandra [US/US]; US (US)
ZHONG, Zhong [US/US]; US (US)
IWAMOTO, Naoki [JP/US]; US (US)
ZHANG, Jason Jingxin [US/US]; US (US)
KANDASAMY, Pachamuthu [CA/US]; US (US)
DIVAKARAMENON, Sethumadhavan [IN/US]; US (US)
LU, Genliang [US/US]; US (US)
MARAPPAN, Subramanian [US/US]; US (US)
Inventors:
DODART, Jean-Cosme; US
LIU, Yuanjing; US
VARGEESE, Chandra; US
ZHONG, Zhong; US
IWAMOTO, Naoki; US
ZHANG, Jason Jingxin; US
KANDASAMY, Pachamuthu; US
DIVAKARAMENON, Sethumadhavan; US
LU, Genliang; US
MARAPPAN, Subramanian; US
Agent:
LI, Xiaodong; US
ADATO, Ronen; US
AUGST, Alexander D.; US
BUTEAU, Kristen C.; US
BYCHOWSKI, Meaghan E.; US
FORCIER, John V.; 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
PACE, Nicholas J.; US
PYSHER, Paul A.; US
REARICK, John P.; US
REESE, Brian E.; US
SAHR, Robert N.; US
SCHMITT, Michael; 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
TSE, Janet M.; US
VETTER, Michael L.; US
VRABLIK, Tracy L.; US
Priority Data:
62/542,77808.08.2017US
Title (EN) OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
(FR) COMPOSITIONS OLIGONUCLÉOTIDIQUES ET PROCÉDÉS ASSOCIÉS
Abstract:
(EN) Among other things, the present disclosure provides oligonucleotides, compositions, and methods thereof. 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., knockdown ability, stability, delivery, etc. In some embodiments, the oligonucleotides decrease the expression, activity and/or level of a C9orf72 gene, including but not limited to, one comprising a repeat expansion, or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in treatment of C9orf72-related disorders.
(FR) La présente invention concerne, entre autres, des oligonucléotides, des compositions et des procédés associés. Entre autres, la présente invention concerne la reconnaissance du fait que des éléments structuraux d'oligonucléotides, tels qu'une séquence de base, des modifications chimiques (par exemple, des modifications de sucre, de base et/ou de liaisons internucléotidiques) ou des motifs de ceux-ci, la conjugaison avec des fragments chimiques supplémentaires, et/ou la stéréochimie [par exemple, la stéréochimie de centres chiraux de squelette (liaisons internucléotidiques chirales)], et/ou des motifs de ceux-ci, peuvent avoir un impact significatif sur les propriétés et les activités d'oligonucléotides, par exemple, la capacité d'inactivation, la stabilité, la distribution, etc. Dans certains modes de réalisation, les oligonucléotides diminuent l'expression, l'activité et/ou le taux d'un gène C9orf72, comprenant, mais non limité à, un gène comprenant une expansion répétée, ou un produit génique de celui-ci. Dans certains modes de réalisation, la présente invention concerne des procédés de traitement de maladies au moyen de compositions oligonucléotidiques selon l'invention, par exemple, dans le traitement de troubles liés à C9orf72.
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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 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)