MA51678A - Méthodes de traitement de l'amyotrophie musculaire - Google Patents

Méthodes de traitement de l'amyotrophie musculaire

Info

Publication number
MA51678A
MA51678A MA051678A MA51678A MA51678A MA 51678 A MA51678 A MA 51678A MA 051678 A MA051678 A MA 051678A MA 51678 A MA51678 A MA 51678A MA 51678 A MA51678 A MA 51678A
Authority
MA
Morocco
Prior art keywords
amyotrophy
muscle
treatment methods
treatment
methods
Prior art date
Application number
MA051678A
Other languages
English (en)
Inventor
Wildon Farwell
Alexander Mccampbell
John Staropoli
Christopher Cody Stebbins
Guolin Zhao
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 MA51678A publication Critical patent/MA51678A/fr

Links

Classifications

    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/56Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/575Compounds 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
    • 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
    • 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
    • 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
    • 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
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/28Neurological disorders
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/52Predicting 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)
MA051678A 2018-01-25 2019-01-25 Méthodes de traitement de l'amyotrophie musculaire MA51678A (fr)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
MA51678A true MA51678A (fr) 2020-12-02

Family

ID=65529774

Family Applications (1)

Application Number Title Priority Date Filing Date
MA051678A MA51678A (fr) 2018-01-25 2019-01-25 Méthodes de traitement de l'amyotrophie musculaire

Country Status (13)

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

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019147960A1 (fr) 2018-01-25 2019-08-01 Biogen Ma Inc. Méthodes de traitement de l'amyotrophie musculaire
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 (fr) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions et procédés pour le traitement de troubles liés à l'amyotrophie spinale
WO2025035143A1 (fr) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions et méthodes de traitement de l'amyotrophie spinale
WO2025115644A1 (fr) * 2023-11-27 2025-06-05 公立大学法人横浜市立大学 Méthode de prédiction de pronostic et d'effet thérapeutique sur une maladie des motoneurones

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US5156840A (en) 1982-03-09 1992-10-20 Cytogen Corporation Amine-containing porphyrin derivatives
US4582789A (en) 1984-03-21 1986-04-15 Cetus Corporation Process for labeling nucleic acids using psoralen derivatives
US4563417A (en) 1984-08-31 1986-01-07 Miles Laboratories, Inc. Nucleic acid hybridization assay employing antibodies to intercalation complexes
US5057313A (en) 1986-02-25 1991-10-15 The Center For Molecular Medicine And Immunology Diagnostic and therapeutic antibody conjugates
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5210015A (en) 1990-08-06 1993-05-11 Hoffman-La Roche Inc. Homogeneous assay system using the nuclease activity of a nucleic acid polymerase
WO1992010588A1 (fr) 1990-12-06 1992-06-25 Affymax Technologies N.V. Mise en sequence par hybridation d'un acide nucleique cible en une matrice d'oligonucleotides determines
US6027880A (en) 1995-08-02 2000-02-22 Affymetrix, Inc. Arrays of nucleic acid probes and methods of using the same for detecting cystic fibrosis
US6156501A (en) 1993-10-26 2000-12-05 Affymetrix, Inc. Arrays of modified nucleic acid probes and methods of use
AU3568897A (en) 1996-06-07 1998-01-05 Eos Biotechnology, Inc. Immobilised linear oligonucleotide arrays
WO2002070751A1 (fr) 2001-03-02 2002-09-12 University Of Pittsburgh Of The Commonwealth System Of Higher Education Procede de reaction en chaine de la polymerase
EP1379545A2 (fr) 2001-04-19 2004-01-14 Gesellschaft für biotechnologische Forschung mbH (GBF) Procede de production de series d'anticorps stables regenerables
US6812341B1 (en) 2001-05-11 2004-11-02 Ambion, Inc. High efficiency mRNA isolation methods and compositions
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US7252976B2 (en) 2002-08-28 2007-08-07 Board Of Regents The University Of Texas System Quantitative RT-PCR to AC133 to diagnose cancer and monitor angiogenic activity in a cell sample
ES2397113T3 (es) 2005-06-23 2013-03-04 Isis Pharmaceuticals, Inc. Composiciones y procedimientos para modular el corte y empalme de SMN2
WO2008016973A1 (fr) 2006-08-01 2008-02-07 Families Of Spinal Muscular Atrophy 2,4-diaminoquinazolines contre l'amyotrophie spinale
WO2008127974A1 (fr) * 2007-04-13 2008-10-23 The Trustees Of Columbia University In The City Of New York Système de culture à base de cellules souches pour la mise au point de médicaments
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
CN115227709A (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
US8465727B2 (en) 2009-10-06 2013-06-18 University of Pittsburgh—of the Commonwealth System of Higher Education Biomarkers for the diagnosis of ALS
WO2014110291A1 (fr) 2013-01-09 2014-07-17 Isis Pharmaceuticals, Inc. Compositions et procédés pour la modulation de l'épissage de smn2 chez un sujet
MY192634A (en) 2014-04-01 2022-08-29 Biogen Ma Inc Compositions for modulating sod-1 expression
US10024870B2 (en) * 2014-04-02 2018-07-17 Wisconsin Alumni Research Foundation Therapeutic and diagnostic methods and compositions for neurodegenerative diseases
MA39835A (fr) 2014-04-17 2017-02-22 Biogen Ma Inc Compositions et procédés de modulation de l'épissage du smn2 chez un patient
WO2016040748A1 (fr) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions et procédés de détection d'une protéine smn chez un patient et traitement d'un patient
US9895358B2 (en) * 2016-02-23 2018-02-20 Indiana University Research And Technology Corporation Combination therapies for treatment of spinal muscular atrophy
WO2017207600A1 (fr) 2016-06-03 2017-12-07 F. Hoffmann-La Roche Ag Nouveau traitement de la sma
EP3631470B1 (fr) * 2017-05-26 2025-12-31 University of Miami Détermination de l'apparition de la sclérose latérale amyotrophique
US11434502B2 (en) * 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
WO2019147960A1 (fr) 2018-01-25 2019-08-01 Biogen Ma Inc. Méthodes de traitement de l'amyotrophie musculaire
EA201992364A1 (ru) * 2018-04-02 2020-03-23 Кохерус Байосайенсис Инк. АГОНИСТ PPARγ ДЛЯ ЛЕЧЕНИЯ ПРОГРЕССИРУЮЩЕГО НАДЪЯДЕРНОГО ПАРАЛИЧА
WO2020061355A2 (fr) 2018-09-20 2020-03-26 Biogen Ma Inc. Protéine neurofilamenteuse sérique pour guider une intervention thérapeutique dans la sclérose en plaques
KR20210102294A (ko) 2018-12-06 2021-08-19 바이오젠 엠에이 인코포레이티드 근위축성 측삭 경화증에서 치료요법적 중재를 안내하기 위한 신경필라멘트 단백질
TWI906204B (zh) 2018-12-14 2025-12-01 美商百健Ma公司 用於治療及預防肌肉萎縮性脊髓側索硬化症之組合物及方法
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Also Published As

Publication number Publication date
WO2019147960A1 (fr) 2019-08-01
CL2020001936A1 (es) 2020-12-18
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
MX2020007852A (es) 2021-01-29
JP2021511389A (ja) 2021-05-06
EP3743728A1 (fr) 2020-12-02
CN112567246B (zh) 2024-12-03
US11474113B2 (en) 2022-10-18
BR112020015082A2 (pt) 2021-01-05

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