HUE046966T2 - Készítmények és módszerek az SMN2 splicing egy alanynál történõ modulálására - Google Patents

Készítmények és módszerek az SMN2 splicing egy alanynál történõ modulálására

Info

Publication number
HUE046966T2
HUE046966T2 HUE18185820A HUE18185820A HUE046966T2 HU E046966 T2 HUE046966 T2 HU E046966T2 HU E18185820 A HUE18185820 A HU E18185820A HU E18185820 A HUE18185820 A HU E18185820A HU E046966 T2 HUE046966 T2 HU E046966T2
Authority
HU
Hungary
Prior art keywords
compositions
subject
methods
smn2 splicing
modulating smn2
Prior art date
Application number
HUE18185820A
Other languages
English (en)
Hungarian (hu)
Inventor
C Frank Bennett
Gene Hung
Frank Rigo
Adrian R Krainer
Yimin Hua
Marco A Passini
Lamya Shihabuddin
Seng H Cheng
Katherine W Klinger
Original Assignee
Biogen Ma Inc
Cold Spring Harbor Laboratory
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=43356760&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HUE046966(T2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Biogen Ma Inc, Cold Spring Harbor Laboratory filed Critical Biogen Ma Inc
Publication of HUE046966T2 publication Critical patent/HUE046966T2/hu

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    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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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
    • A—HUMAN NECESSITIES
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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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    • 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
    • C12N2310/352—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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Physics & Mathematics (AREA)
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  • Physical Education & Sports Medicine (AREA)
  • Dermatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Pain & Pain Management (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
HUE18185820A 2009-06-17 2010-06-17 Készítmények és módszerek az SMN2 splicing egy alanynál történõ modulálására HUE046966T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US21803109P 2009-06-17 2009-06-17

Publications (1)

Publication Number Publication Date
HUE046966T2 true HUE046966T2 (hu) 2020-04-28

Family

ID=43356760

Family Applications (2)

Application Number Title Priority Date Filing Date
HUE18185820A HUE046966T2 (hu) 2009-06-17 2010-06-17 Készítmények és módszerek az SMN2 splicing egy alanynál történõ modulálására
HUE17203913A HUE039741T2 (hu) 2009-06-17 2010-06-17 Készítmények és módszerek az SMN2 splicing módosítására egy alanynál

Family Applications After (1)

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HUE17203913A HUE039741T2 (hu) 2009-06-17 2010-06-17 Készítmények és módszerek az SMN2 splicing módosítására egy alanynál

Country Status (25)

Country Link
US (5) US8980853B2 (enExample)
EP (4) EP2442816A4 (enExample)
JP (7) JP5707396B2 (enExample)
KR (8) KR20200047790A (enExample)
CN (5) CN115227710A (enExample)
AU (2) AU2010262862C1 (enExample)
CA (1) CA2765396C (enExample)
CY (2) CY1120807T1 (enExample)
DK (2) DK3449926T3 (enExample)
ES (2) ES2761614T3 (enExample)
HK (1) HK1246648B (enExample)
HR (2) HRP20181712T1 (enExample)
HU (2) HUE046966T2 (enExample)
IL (2) IL217014A (enExample)
LT (2) LT3449926T (enExample)
MX (3) MX2011014004A (enExample)
NO (1) NO2020008I1 (enExample)
NZ (2) NZ597071A (enExample)
PL (2) PL3449926T3 (enExample)
PT (2) PT3449926T (enExample)
RU (2) RU2566724C9 (enExample)
SI (2) SI3305302T1 (enExample)
SM (2) SMT202000020T1 (enExample)
TR (1) TR201816256T4 (enExample)
WO (1) WO2010148249A1 (enExample)

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