RS61985B1 - Antisens polinukleotidi za indukovanje preskakanja egzona i postupci lečenja distrofija - Google Patents

Antisens polinukleotidi za indukovanje preskakanja egzona i postupci lečenja distrofija

Info

Publication number
RS61985B1
RS61985B1 RS20210656A RSP20210656A RS61985B1 RS 61985 B1 RS61985 B1 RS 61985B1 RS 20210656 A RS20210656 A RS 20210656A RS P20210656 A RSP20210656 A RS P20210656A RS 61985 B1 RS61985 B1 RS 61985B1
Authority
RS
Serbia
Prior art keywords
methods
exon skipping
antisense polynucleotides
induce exon
dystrophies
Prior art date
Application number
RS20210656A
Other languages
English (en)
Inventor
Elizabeth Mcnally
Original Assignee
Univ Chicago
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Chicago filed Critical Univ Chicago
Publication of RS61985B1 publication Critical patent/RS61985B1/sr

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    • C—CHEMISTRY; METALLURGY
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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
    • 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
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • 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
    • 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
    • C12N2310/10—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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3535—Nitrogen
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
RS20210656A 2012-09-06 2013-09-06 Antisens polinukleotidi za indukovanje preskakanja egzona i postupci lečenja distrofija RS61985B1 (sr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261697766P 2012-09-06 2012-09-06
EP18177365.6A EP3421602B1 (en) 2012-09-06 2013-09-06 Antisense polynucleotides to induce exon skipping and methods of treating dystrophies

Publications (1)

Publication Number Publication Date
RS61985B1 true RS61985B1 (sr) 2021-07-30

Family

ID=50237661

Family Applications (2)

Application Number Title Priority Date Filing Date
RS20181053A RS57789B1 (sr) 2012-09-06 2013-09-06 Antisens polinukleotidi za indukovanje preskakanja egzona i postupci lečenja distrofija
RS20210656A RS61985B1 (sr) 2012-09-06 2013-09-06 Antisens polinukleotidi za indukovanje preskakanja egzona i postupci lečenja distrofija

Family Applications Before (1)

Application Number Title Priority Date Filing Date
RS20181053A RS57789B1 (sr) 2012-09-06 2013-09-06 Antisens polinukleotidi za indukovanje preskakanja egzona i postupci lečenja distrofija

Country Status (17)

Country Link
US (13) US9499817B2 (sr)
EP (2) EP3421602B1 (sr)
BR (1) BR112015005091B1 (sr)
CA (1) CA2884245C (sr)
CY (2) CY1121037T1 (sr)
DK (2) DK2892617T3 (sr)
ES (2) ES2878650T3 (sr)
HK (1) HK1212272A1 (sr)
HR (2) HRP20181458T1 (sr)
HU (2) HUE039676T2 (sr)
LT (1) LT3421602T (sr)
PL (1) PL3421602T3 (sr)
PT (2) PT3421602T (sr)
RS (2) RS57789B1 (sr)
SI (2) SI2892617T1 (sr)
TN (1) TN2015000079A1 (sr)
WO (1) WO2014039916A1 (sr)

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ES2846902T3 (es) * 2015-04-08 2021-07-30 Univ Chicago Composiciones y procedimientos para corregir la distrofia muscular de cinturas tipo 2C mediante omisión de exones
US10961537B2 (en) 2017-07-18 2021-03-30 Csl Behring Gene Therapy, Inc. Compositions and methods for treating beta-hemoglobinopathies
WO2019075360A1 (en) 2017-10-13 2019-04-18 Selecta Biosciences, Inc. METHODS AND COMPOSITIONS FOR MITIGATING ANTI-VECTOR VIRAL TRANSFER IGM RESPONSES
WO2020132647A1 (en) 2018-12-21 2020-06-25 Northwestern University Use of annexins in preventing and treating muscle membrane injury
JP2022514955A (ja) 2018-12-23 2022-02-16 シーエスエル・ベーリング・エルエルシー ウィスコット・アルドリッチ症候群の造血幹細胞遺伝子治療
EP3897831A1 (en) 2018-12-23 2021-10-27 CSL Behring LLC Donor t-cells with kill switch
CA3141863A1 (en) 2019-05-28 2020-12-03 Selecta Biosciences, Inc. Methods and compositions for attenuated anti-viral transfer vector immune response
AU2021294317A1 (en) 2020-06-26 2023-02-23 Csl Behring Llc Donor T-cells with kill switch
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
US20230357437A1 (en) 2022-03-09 2023-11-09 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing

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BR112015005091A2 (pt) 2017-09-26
EP2892617A4 (en) 2016-06-22
US9499817B2 (en) 2016-11-22
US20180327743A1 (en) 2018-11-15
US20200291403A1 (en) 2020-09-17
US20160369277A1 (en) 2016-12-22
CY1124197T1 (el) 2022-05-27
DK3421602T3 (da) 2021-05-10
EP2892617B1 (en) 2018-06-13
LT3421602T (lt) 2021-05-25
US9777271B2 (en) 2017-10-03
HRP20210877T1 (hr) 2021-07-23
PT3421602T (pt) 2021-05-31
US20190194659A1 (en) 2019-06-27
US20150225718A1 (en) 2015-08-13
WO2014039916A1 (en) 2014-03-13
EP3421602A1 (en) 2019-01-02
RS57789B1 (sr) 2018-12-31
ES2878650T3 (es) 2021-11-19
EP2892617A1 (en) 2015-07-15
US20210147845A1 (en) 2021-05-20
US20210403910A1 (en) 2021-12-30
HRP20181458T1 (hr) 2018-11-02
CY1121037T1 (el) 2019-12-11
PT2892617T (pt) 2018-10-18
US20230183700A1 (en) 2023-06-15
ES2686727T3 (es) 2018-10-19
US20240076670A1 (en) 2024-03-07
CA2884245A1 (en) 2014-03-13
DK2892617T3 (en) 2018-09-03
US20180037889A1 (en) 2018-02-08
US20220251559A1 (en) 2022-08-11
US20200040340A1 (en) 2020-02-06
SI2892617T1 (sl) 2018-11-30
HUE054260T2 (hu) 2021-08-30
EP3421602B1 (en) 2021-03-03
US20240327844A1 (en) 2024-10-03
PL3421602T3 (pl) 2021-10-25
CA2884245C (en) 2023-03-14
HK1212272A1 (en) 2016-06-10
HUE039676T2 (hu) 2019-01-28
BR112015005091B1 (pt) 2021-07-06
TN2015000079A1 (en) 2016-06-29
SI3421602T1 (sl) 2021-08-31

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