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LT3999643T - Spinalinės raumenų atrofijos gydymo ar prevencijos metodai - Google Patents

Spinalinės raumenų atrofijos gydymo ar prevencijos metodai

Info

Publication number
LT3999643T
LT3999643T LTEPPCT/US2020/042312T LTUS2020042312T LT3999643T LT 3999643 T LT3999643 T LT 3999643T LT US2020042312 T LTUS2020042312 T LT US2020042312T LT 3999643 T LT3999643 T LT 3999643T
Authority
LT
Lithuania
Prior art keywords
treating
methods
muscular atrophy
spinal muscular
preventing spinal
Prior art date
Application number
LTEPPCT/US2020/042312T
Other languages
English (en)
Inventor
Kenneth Swope LOVEDAY
Fengju Judy BAI
Lilly EAST
Wildon R. FARWELL
Original Assignee
Biogen Ma Inc.
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=71995097&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=LT3999643(T) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Biogen Ma Inc. filed Critical Biogen Ma Inc.
Publication of LT3999643T publication Critical patent/LT3999643T/lt

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1136Non-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 growth factors, growth regulators, cytokines, lymphokines or hormones
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0021Intradermal administration, e.g. through microneedle arrays, needleless injectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • G01N33/6896Neurological disorders, e.g. Alzheimer's disease
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing
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    • C12N2320/00Applications; Uses
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    • C12N2320/34Allele or polymorphism specific uses
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/35Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Dermatology (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
LTEPPCT/US2020/042312T 2019-07-19 2020-07-16 Spinalinės raumenų atrofijos gydymo ar prevencijos metodai LT3999643T (lt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962876360P 2019-07-19 2019-07-19
PCT/US2020/042312 WO2021016032A1 (en) 2019-07-19 2020-07-16 Methods of treating or preventing spinal muscular atrophy

Publications (1)

Publication Number Publication Date
LT3999643T true LT3999643T (lt) 2023-12-11

Family

ID=71995097

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEPPCT/US2020/042312T LT3999643T (lt) 2019-07-19 2020-07-16 Spinalinės raumenų atrofijos gydymo ar prevencijos metodai

Country Status (33)

Country Link
US (3) US20220325282A1 (lt)
EP (2) EP4335503A3 (lt)
JP (3) JP7279253B2 (lt)
KR (1) KR20220042113A (lt)
CN (1) CN114302727A (lt)
AR (1) AR119430A1 (lt)
AU (1) AU2020316997A1 (lt)
BR (1) BR112022000870A2 (lt)
CA (1) CA3149906A1 (lt)
CL (1) CL2021003602A1 (lt)
CO (1) CO2022001354A2 (lt)
CR (1) CR20220071A (lt)
DK (1) DK3999643T5 (lt)
ES (1) ES2966791T3 (lt)
FI (1) FI3999643T3 (lt)
HR (1) HRP20231515T1 (lt)
HU (1) HUE064071T2 (lt)
IL (1) IL289745A (lt)
JO (1) JOP20220007A1 (lt)
LT (1) LT3999643T (lt)
MA (1) MA62935B1 (lt)
MD (1) MD3999643T2 (lt)
MX (1) MX2022000441A (lt)
PE (1) PE20220941A1 (lt)
PH (1) PH12022550114A1 (lt)
PL (1) PL3999643T3 (lt)
PT (1) PT3999643T (lt)
RS (1) RS64879B1 (lt)
SI (1) SI3999643T1 (lt)
SM (1) SMT202300442T1 (lt)
TW (2) TW202438086A (lt)
UY (1) UY38790A (lt)
WO (1) WO2021016032A1 (lt)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA39835A (fr) 2014-04-17 2017-02-22 Biogen Ma Inc Compositions et procédés de modulation de l'épissage du smn2 chez un patient
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
FI3999643T3 (fi) 2019-07-19 2023-11-30 Biogen Ma Inc Menetelmiä spinaalisen lihasatrofian hoitamiseksi tai ehkäisemiseksi
WO2024138018A1 (en) * 2022-12-23 2024-06-27 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Treatment of gemin5-mediated disorders

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI3308788T1 (sl) 2005-06-23 2019-01-31 Biogen Ma Inc. Sestave in metode za modulacijo združevanja SMN2
WO2008157753A1 (en) * 2007-06-21 2008-12-24 Sma Foundation Methods of treatment for spinal muscular atrophy
CN115227710A (zh) 2009-06-17 2022-10-25 冷泉港实验室 用于在对象中调节smn2剪接的组合物和方法
WO2011032109A1 (en) 2009-09-11 2011-03-17 Sma Foundation Biomarkers for spinal muscular atrophy
ES2597052T3 (es) * 2011-05-25 2017-01-13 Université Paris Descartes Inhibidores de ERK para su uso en el tratamiento de atrofia muscular espinal
EP2943225A4 (en) * 2013-01-09 2016-07-13 Ionis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR MODULATING SMN2 DISTRIBUTION IN THE BODY OF A PATIENT
BR112017004056A2 (pt) * 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
CA3030864A1 (en) * 2016-07-15 2018-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of smn2
TW201927241A (zh) 2017-12-21 2019-07-16 瑞士商赫孚孟拉羅股份公司 用於肌肉失能之數位生物標記
EP3743728A1 (en) 2018-01-25 2020-12-02 Biogen MA Inc. Methods of treating spinal muscular atrophy
IT201800004359A1 (it) 2018-04-10 2019-10-10 IDENTIFICAZIONE DI miRNA MUSCOLARI COME BIOMARCATORI E TRATTAMENTO CO-ADIUVANTE PER L’ATROFIA MUSCOLARE SPINALE
US20210308281A1 (en) * 2018-08-15 2021-10-07 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
FI3999643T3 (fi) 2019-07-19 2023-11-30 Biogen Ma Inc Menetelmiä spinaalisen lihasatrofian hoitamiseksi tai ehkäisemiseksi

Also Published As

Publication number Publication date
MA62935B1 (fr) 2023-11-30
UY38790A (es) 2021-01-29
JP2025096486A (ja) 2025-06-26
MD3999643T2 (ro) 2024-02-29
MX2022000441A (es) 2022-03-25
CL2021003602A1 (es) 2022-10-14
ES2966791T3 (es) 2024-04-24
CN114302727A (zh) 2022-04-08
IL289745A (en) 2022-03-01
SI3999643T1 (sl) 2024-01-31
JP2022540993A (ja) 2022-09-21
EP4335503A3 (en) 2024-06-05
US20220325282A1 (en) 2022-10-13
AR119430A1 (es) 2021-12-15
FI3999643T3 (fi) 2023-11-30
SMT202300442T1 (it) 2024-01-10
TW202438086A (zh) 2024-10-01
JP7665678B2 (ja) 2025-04-21
PT3999643T (pt) 2023-12-11
US20240318182A1 (en) 2024-09-26
WO2021016032A1 (en) 2021-01-28
PL3999643T3 (pl) 2024-02-19
EP4335503A2 (en) 2024-03-13
DK3999643T3 (da) 2023-12-04
CA3149906A1 (en) 2021-01-28
KR20220042113A (ko) 2022-04-04
BR112022000870A2 (pt) 2022-03-22
TWI841762B (zh) 2024-05-11
PE20220941A1 (es) 2022-05-31
CR20220071A (es) 2022-04-22
EP3999643B1 (en) 2023-11-22
JP2023100911A (ja) 2023-07-19
EP3999643A1 (en) 2022-05-25
TW202116331A (zh) 2021-05-01
DK3999643T5 (da) 2024-08-05
HUE064071T2 (hu) 2024-02-28
HRP20231515T1 (hr) 2024-03-01
JOP20220007A1 (ar) 2023-01-30
CO2022001354A2 (es) 2022-03-18
PH12022550114A1 (en) 2023-04-17
JP7279253B2 (ja) 2023-05-22
AU2020316997A1 (en) 2022-03-10
RS64879B1 (sr) 2023-12-29
US20250230440A1 (en) 2025-07-17

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