MX2020007852A - Metodos para el tratamiento de la atrofia muscular espinal. - Google Patents

Metodos para el tratamiento de la atrofia muscular espinal.

Info

Publication number
MX2020007852A
MX2020007852A MX2020007852A MX2020007852A MX2020007852A MX 2020007852 A MX2020007852 A MX 2020007852A MX 2020007852 A MX2020007852 A MX 2020007852A MX 2020007852 A MX2020007852 A MX 2020007852A MX 2020007852 A MX2020007852 A MX 2020007852A
Authority
MX
Mexico
Prior art keywords
methods
muscular atrophy
spinal muscular
treating spinal
sma
Prior art date
Application number
MX2020007852A
Other languages
English (en)
Inventor
Wildon Farwell
John Staropoli
Guolin Zhao
Alexander Mccampbell
Christopher Cody Stebbins
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
Application filed by Biogen Ma Inc filed Critical Biogen Ma Inc
Publication of MX2020007852A publication Critical patent/MX2020007852A/es

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    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical 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
    • G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/575—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of three or more carbon atoms, e.g. cholane, cholestane, ergosterol, sitosterol
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous 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
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Neurosurgery (AREA)
  • Genetics & Genomics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Food Science & Technology (AREA)
  • Analytical Chemistry (AREA)
  • Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biophysics (AREA)

Abstract

Se presentan biomarcadores para, por ejemplo, diagnosticar y pronosticar la atrofia muscular espinal (AME), así como para identificar a los respondedores del tratamiento para la AME. También se proporcionan métodos para tratar individuos con AME.
MX2020007852A 2018-01-25 2019-01-25 Metodos para el tratamiento de la atrofia muscular espinal. MX2020007852A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862622027P 2018-01-25 2018-01-25
US201862684507P 2018-06-13 2018-06-13
US201862738134P 2018-09-28 2018-09-28
PCT/US2019/015185 WO2019147960A1 (en) 2018-01-25 2019-01-25 Methods of treating spinal muscular atrophy

Publications (1)

Publication Number Publication Date
MX2020007852A true MX2020007852A (es) 2021-01-29

Family

ID=65529774

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020007852A MX2020007852A (es) 2018-01-25 2019-01-25 Metodos para el tratamiento de la atrofia muscular espinal.

Country Status (13)

Country Link
US (2) US11474113B2 (es)
EP (1) EP3743728A1 (es)
JP (2) JP7506606B2 (es)
KR (1) KR102742415B1 (es)
CN (2) CN112567246B (es)
AU (1) AU2019211446B2 (es)
BR (1) BR112020015082A2 (es)
CL (1) CL2020001936A1 (es)
IL (1) IL276180B2 (es)
MA (1) MA51678A (es)
MX (1) MX2020007852A (es)
SG (1) SG11202006677XA (es)
WO (1) WO2019147960A1 (es)

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WO2019147960A1 (en) 2018-01-25 2019-08-01 Biogen Ma Inc. Methods of treating spinal muscular atrophy
KR20210102294A (ko) 2018-12-06 2021-08-19 바이오젠 엠에이 인코포레이티드 근위축성 측삭 경화증에서 치료요법적 중재를 안내하기 위한 신경필라멘트 단백질
CN121510896A (zh) 2019-06-11 2026-02-10 库力索法荷兰有限公司 方法和系统
HUE064071T2 (hu) 2019-07-19 2024-02-28 Biogen Ma Inc Eljárások a spinális izomatrófia kezelésére vagy megelõzésére
BR112023025188A2 (pt) * 2021-06-08 2024-02-27 Alector Llc Métodos de uso de anticorpos anti-sortilina
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025115644A1 (ja) * 2023-11-27 2025-06-05 公立大学法人横浜市立大学 運動ニューロン疾患の予後及び治療効果を予測する方法

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Also Published As

Publication number Publication date
WO2019147960A1 (en) 2019-08-01
CL2020001936A1 (es) 2020-12-18
MA51678A (fr) 2020-12-02
JP2023118821A (ja) 2023-08-25
US20210041459A1 (en) 2021-02-11
KR20200112906A (ko) 2020-10-05
AU2019211446B2 (en) 2025-07-03
US20230107651A1 (en) 2023-04-06
IL276180B1 (en) 2025-03-01
KR102742415B1 (ko) 2024-12-12
IL276180A (en) 2020-09-30
CN119679944A (zh) 2025-03-25
CN112567246A (zh) 2021-03-26
IL276180B2 (en) 2025-07-01
AU2019211446A1 (en) 2020-09-10
JP7506606B2 (ja) 2024-06-26
SG11202006677XA (en) 2020-08-28
JP2021511389A (ja) 2021-05-06
EP3743728A1 (en) 2020-12-02
CN112567246B (zh) 2024-12-03
US11474113B2 (en) 2022-10-18
BR112020015082A2 (pt) 2021-01-05

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