HUE027321T2 - Géntranszfer nukleinsav - Google Patents

Géntranszfer nukleinsav Download PDF

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Publication number
HUE027321T2
HUE027321T2 HUE11821996A HUE11821996A HUE027321T2 HU E027321 T2 HUE027321 T2 HU E027321T2 HU E11821996 A HUE11821996 A HU E11821996A HU E11821996 A HUE11821996 A HU E11821996A HU E027321 T2 HUE027321 T2 HU E027321T2
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nucleic acid
synthetic nucleic
artificial
exon
gene
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HUE11821996A
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English (en)
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Naoki Watanabe
Youhei Satou
Shin'ichi Takeda
Tetsuya Nagata
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Nippon Shinyaku Co Ltd
Nat Center Neurology & Psychiatry
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Application filed by Nippon Shinyaku Co Ltd, Nat Center Neurology & Psychiatry filed Critical Nippon Shinyaku Co Ltd
Publication of HUE027321T2 publication Critical patent/HUE027321T2/hu

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
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  • Medicinal Chemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Claims (7)

  1. imBAii«MríGiHYFc»x:m 1,0¾ mely komán disztrofm génben az S3, exon átugrását okozza, mély a humán dísztrofm gén 53. exo® '5’-végéről a 32.-56, vagy 36,-56. ankkotfp# állé Szekvenciák bármelyikével komplementer nuklcotid szekvenciát tartalma
  2. 2. Az 1. igénypont szerinti géatranszfer-oügomer* mely egy oHgotmkleoid,
  3. 3. A 2. igénypont szerinti géntmmzíer-oUgomer^ahol. legilább egy, m olpnukleotidot képező nukleotid cokorkomponease IsAagy rlgi§a. méissltva van,
  4. 4. A 3. igénypont szerinti génínmszier-öligomér, siti legalább egy, ál oligonukkaotidol képező nukleotid cukorkomponense rifeőiz, ábol a 2’-OH-csoport valamely, az OR, R, R'QR, SM, SR, NH>, NBR, NR?> N3, CR 1, Cl, Br és I (ahol R egy api- vagy anlesoptnt és i’ alkíiéncsopot) csoportéi ||vála^tei.:helye«eslté^f| Van.M^tÉítve*
  5. 5. A 3, vágy 4. igénypont ftNÉ&tti· génl^izfér-o^^í#*gAíl lipatofe egy»; m-oligonukleoüdot képező nukleoiid tlxsziátképao régiőfa a .toszínritoát kőtés^ ;i>sz.forpioit kötés, alkillbszfbnát kötés, foszforamidát kötés, ét egy boranolbszíat kötés; esoporiml van kiválasztva,
  6. 6. Az I. igénypont szerinti,géntensll«r»oipmér, ||^:^í^(^Iin€i|^|Ígcs*^
  7. 7. A 6. Ig^ypont: sgerinit porfoli no-oli gom er. B. A 6, vágy 7, igénypont szerinti géntransztér-oligomer, ahol az 54-vég az alábbi tl> (3) kémiai képletek valamelyike szerinti;
    í§) dl %-Az !"B. szerlbi gtera»^fe^ilg»mers mély a II vagy BS azonosMsl mámé;&Aieoild «gd^^lsiol áll, I% Győgys^erkéíiacliíséíw, mely hstóepfegkéne¥«lsmely, «Μ igénypontok bármelyike szeximi géaimszfér-oligomerts vagy annak gyógyászadlag elíogadlmii sojli vagy hidráíját t&i1a!mazxa, izomsorvadás kezelésében való alkalmazásra. IL A I0, igénypont szerimi alkalmazásra szolgáié gyógyszerkészítmény, mely a Doeiapge izpgBorvadás kezelésÉíe adMdifeg.
HUE11821996A 2010-09-01 2011-08-31 Géntranszfer nukleinsav HUE027321T2 (hu)

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JP2010196032 2010-09-01

Publications (1)

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ID=45773054

Family Applications (2)

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HUE15199455A HUE046364T2 (hu) 2010-09-01 2011-08-31 Antiszensz nukleinsavak
HUE11821996A HUE027321T2 (hu) 2010-09-01 2011-08-31 Géntranszfer nukleinsav

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US (15) US9079934B2 (hu)
EP (4) EP3543341A1 (hu)
JP (11) JP5363655B2 (hu)
KR (1) KR101310569B1 (hu)
CN (1) CN103154245B (hu)
AU (1) AU2011296882B2 (hu)
CA (1) CA2809637C (hu)
CY (2) CY1117367T1 (hu)
DK (2) DK2612917T3 (hu)
ES (2) ES2567411T3 (hu)
HR (2) HRP20160336T1 (hu)
HU (2) HUE046364T2 (hu)
LT (1) LT3018211T (hu)
PL (2) PL3018211T3 (hu)
PT (1) PT3018211T (hu)
RS (2) RS54649B1 (hu)
RU (1) RU2567664C2 (hu)
SI (2) SI2612917T1 (hu)
SM (1) SMT201600111B (hu)
TW (1) TWI541024B (hu)
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