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1. (WO2015168172) LINKAGE MODIFIED OLIGOMERIC COMPOUNDS
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/2015/168172 International Application No.: PCT/US2015/028076
Publication Date: 05.11.2015 International Filing Date: 28.04.2015
IPC:
C07H 19/067 (2006.01) ,C07H 19/167 (2006.01) ,C07H 21/02 (2006.01) ,C12N 15/11 (2006.01)
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
H
SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
19
Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro derivatives thereof
02
sharing nitrogen
04
Heterocyclic radicals containing only nitrogen as ring hetero atom
06
Pyrimidine radicals
067
with ribosyl as the saccharide radical
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
H
SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
19
Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro derivatives thereof
02
sharing nitrogen
04
Heterocyclic radicals containing only nitrogen as ring hetero atom
16
Purine radicals
167
with ribosyl as the 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
02
with ribosyl 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
Applicants:
IONIS PHARMACEUTICALS, INC. [US/US]; 2855 Gazelle Court Carlsbad, CA 92010, US
Inventors:
WAN, W., Brad; US
MIGAWA, Michael, T.; US
OESTERGAARD, Michael; US
SWAYZE, Eric, E.; US
SETH, Punit, P.; US
Agent:
STRAHER, Michael; US
Priority Data:
61/985,19628.04.2014US
62/114,15310.02.2015US
Title (EN) LINKAGE MODIFIED OLIGOMERIC COMPOUNDS
(FR) COMPOSÉS OLIGOMÈRES MODIFIÉS PAR LIAISON
Abstract:
(EN) The present invention provides oligomeric compounds comprising at least one neutral methoxypropyl phosphonate modified internucleoside linkage. Such oligomeric compounds have one or more improved properties such as selectivity, potency, improved toxicity profile and or improved proinflammatory profile. Such oligomeric compounds have enhanced stability to exposure to base during synthesis. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.
(FR) La présente invention concerne des composés oligomères comprenant au moins une liaison internucléosidique modifiée par un méthoxypropyl phosphonate neutre. De tels composés oligomères présentent une ou plusieurs propriétés améliorées, telles que la sélectivité, la puissance, un profil de toxicité amélioré et/ou un profil pro-inflammatoire amélioré. De tels composés oligomères possèdent une stabilité améliorée à l'exposition à une base pendant la synthèse. Certains tels composés oligomères sont utiles pour l'hybridation à un acide nucléique complémentaire, notamment, mais sans y être limité, à des acides nucléiques dans une cellule. Dans certains modes de réalisation, l'hybridation conduit à une modulation de la quantité d'activité ou d'expression de l'acide nucléique cible dans une cellule.
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 (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)