SI3421602T1 - Protismiselni polinukleotidi za sprožanje preskakovanja eksonov in postopki zdravljenja distrofij - Google Patents

Protismiselni polinukleotidi za sprožanje preskakovanja eksonov in postopki zdravljenja distrofij

Info

Publication number
SI3421602T1
SI3421602T1 SI201331887T SI201331887T SI3421602T1 SI 3421602 T1 SI3421602 T1 SI 3421602T1 SI 201331887 T SI201331887 T SI 201331887T SI 201331887 T SI201331887 T SI 201331887T SI 3421602 T1 SI3421602 T1 SI 3421602T1
Authority
SI
Slovenia
Prior art keywords
methods
exon skipping
antisense polynucleotides
induce exon
dystrophies
Prior art date
Application number
SI201331887T
Other languages
English (en)
Inventor
Elizabeth Mcnally
Original Assignee
The University Of 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 The University Of Chicago filed Critical The University Of Chicago
Publication of SI3421602T1 publication Critical patent/SI3421602T1/sl

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    • 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
    • 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
    • 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
    • C12N2310/351—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)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
SI201331887T 2012-09-06 2013-09-06 Protismiselni polinukleotidi za sprožanje preskakovanja eksonov in postopki zdravljenja distrofij SI3421602T1 (sl)

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
SI3421602T1 true SI3421602T1 (sl) 2021-08-31

Family

ID=50237661

Family Applications (2)

Application Number Title Priority Date Filing Date
SI201331142T SI2892617T1 (sl) 2012-09-06 2013-09-06 Protismiselni polinukleotidi za zagotavljanje preskakovanja eksonov in postopki zdravljenja distrofij
SI201331887T SI3421602T1 (sl) 2012-09-06 2013-09-06 Protismiselni polinukleotidi za sprožanje preskakovanja eksonov in postopki zdravljenja distrofij

Family Applications Before (1)

Application Number Title Priority Date Filing Date
SI201331142T SI2892617T1 (sl) 2012-09-06 2013-09-06 Protismiselni polinukleotidi za zagotavljanje preskakovanja eksonov in postopki zdravljenja distrofij

Country Status (17)

Country Link
US (12) US9499817B2 (sl)
EP (2) EP3421602B1 (sl)
BR (1) BR112015005091B1 (sl)
CA (1) CA2884245C (sl)
CY (2) CY1121037T1 (sl)
DK (2) DK2892617T3 (sl)
ES (2) ES2686727T3 (sl)
HK (1) HK1212272A1 (sl)
HR (2) HRP20181458T1 (sl)
HU (2) HUE054260T2 (sl)
LT (1) LT3421602T (sl)
PL (1) PL3421602T3 (sl)
PT (2) PT3421602T (sl)
RS (2) RS61985B1 (sl)
SI (2) SI2892617T1 (sl)
TN (1) TN2015000079A1 (sl)
WO (1) WO2014039916A1 (sl)

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CN106714826A (zh) 2014-09-07 2017-05-24 西莱克塔生物科技公司 用于减弱基因表达调节抗病毒转移载体免疫应答的方法和组合物
SI3283500T1 (sl) 2015-04-08 2020-12-31 The University Of Chicago Sestavki in postopki za popravilo medenično ramenske distrofije tipa 2C z uporabo preskakovanja eksonov
EP3655534A1 (en) 2017-07-18 2020-05-27 Calimmune, Inc. Compositions and methods for treating beta-hemoglobinopathies
MX2020003838A (es) 2017-10-13 2020-08-06 Selecta Biosciences Inc Metodos y composiciones para atenuar las respuestas de inmunoglobulina m del vector de transferencia antiviral.
WO2020132647A1 (en) 2018-12-21 2020-06-25 Northwestern University Use of annexins in preventing and treating muscle membrane injury
AU2019417700A1 (en) 2018-12-23 2021-07-08 Csl Behring L.L.C. Donor T-cells with kill switch
CN113518825A (zh) 2018-12-23 2021-10-19 美国杰特贝林生物制品有限公司 Wiskott-aldrich综合征的造血干细胞基因疗法
MX2021014566A (es) 2019-05-28 2022-03-22 Selecta Biosciences Inc Métodos y composiciones para la respuesta inmune atenuada anti-vector de transferencia viral.
CN115997015A (zh) 2020-06-26 2023-04-21 美国杰特贝林生物制品有限公司 具有杀伤开关的供体t细胞
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Publication number Publication date
US20180037889A1 (en) 2018-02-08
US20190194659A1 (en) 2019-06-27
EP2892617B1 (en) 2018-06-13
CY1121037T1 (el) 2019-12-11
US20160369277A1 (en) 2016-12-22
PL3421602T3 (pl) 2021-10-25
PT3421602T (pt) 2021-05-31
ES2686727T3 (es) 2018-10-19
EP2892617A4 (en) 2016-06-22
US20180327743A1 (en) 2018-11-15
HK1212272A1 (en) 2016-06-10
LT3421602T (lt) 2021-05-25
HRP20210877T1 (hr) 2021-07-23
HUE039676T2 (hu) 2019-01-28
SI2892617T1 (sl) 2018-11-30
HRP20181458T1 (hr) 2018-11-02
BR112015005091A2 (pt) 2017-09-26
DK3421602T3 (da) 2021-05-10
US20200040340A1 (en) 2020-02-06
CY1124197T1 (el) 2022-05-27
EP3421602B1 (en) 2021-03-03
ES2878650T3 (es) 2021-11-19
US20220251559A1 (en) 2022-08-11
HUE054260T2 (hu) 2021-08-30
PT2892617T (pt) 2018-10-18
RS61985B1 (sr) 2021-07-30
US20150225718A1 (en) 2015-08-13
DK2892617T3 (en) 2018-09-03
BR112015005091B1 (pt) 2021-07-06
US20210403910A1 (en) 2021-12-30
US20200291403A1 (en) 2020-09-17
CA2884245A1 (en) 2014-03-13
EP2892617A1 (en) 2015-07-15
EP3421602A1 (en) 2019-01-02
US9499817B2 (en) 2016-11-22
WO2014039916A1 (en) 2014-03-13
TN2015000079A1 (en) 2016-06-29
US20210147845A1 (en) 2021-05-20
US9777271B2 (en) 2017-10-03
US20230183700A1 (en) 2023-06-15
RS57789B1 (sr) 2018-12-31
CA2884245C (en) 2023-03-14
US20240076670A1 (en) 2024-03-07

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