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1. (WO2018193428) MODIFIED OLIGOMERIC COMPOUNDS COMPRISING TRICYCLO-DNA NUCLEOSIDES AND USES THEREOF
Latest bibliographic data on file with the International BureauSubmit observation

Pub. No.: WO/2018/193428 International Application No.: PCT/IB2018/052781
Publication Date: 25.10.2018 International Filing Date: 20.04.2018
IPC:
C07H 21/02 (2006.01) ,C07H 21/04 (2006.01) ,A61K 31/712 (2006.01) ,A61K 31/7125 (2006.01) ,A61P 25/00 (2006.01)
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
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
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25
Drugs for disorders of the nervous system
Applicants:
SYNTHENA AG [CH/CH]; Freiestrasse 3 CH-3012 Bern, CH
Inventors:
RENNER, Wolfgang Andreas; CH
DUGOVIC, Branislav; CH
BERTOLINI, Reto; CH
Priority Data:
17167427.820.04.2017EP
62/562,12422.09.2017US
Title (EN) MODIFIED OLIGOMERIC COMPOUNDS COMPRISING TRICYCLO-DNA NUCLEOSIDES AND USES THEREOF
(FR) COMPOSÉS OLIGOMÈRES MODIFIÉS COMPRENANT DES NUCLÉOSIDES TRICYCLO-ADN ET UTILISATIONS CORRESPONDANTES
Abstract:
(EN) The present invention relates to a composition comprising an oligomeric compound comprising one or more tricyclo-deoxyribonucleic acid (tc-DNA) nucleosides and one or more lipid moiety, wherein said one or more lipid moiety is covalently linked to said oligomeric compound either directly or via a spacer, and wherein preferably said oligomeric compound comprises from 5 to 40 monomer subunits, as well as pharmaceutical compositions thereof and their uses in the prevention or treatment of neuromuscular or musculoskeletal diseases such as Duchenne muscular dystrophy or Steinert disease.
(FR) La présente invention concerne une composition comprenant un composé oligomère comprenant un ou plusieurs nucléosides de type acide tricyclo-désoxyribonucléique (tc-ADN) et un ou plusieurs fragments lipidiques, ledit un ou lesdits plusieurs fragments lipidiques étant lié(s) par covalence audit composé oligomère, soit directement, soit par l'intermédiaire d'un espaceur et, de préférence, ledit composé oligomère comprenant 5 à 40 sous-motifs monomères, ainsi que des compositions pharmaceutiques correspondantes et leurs utilisations dans la prévention ou le traitement de maladies neuromusculaires ou musculo-squelettiques, telles que la dystrophie musculaire de Duchenne ou la maladie de Steinert.
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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)