CL2020001936A1 - Métodos para el tratamiento de la atrofía muscular espinal. - Google Patents

Métodos para el tratamiento de la atrofía muscular espinal.

Info

Publication number
CL2020001936A1
CL2020001936A1 CL2020001936A CL2020001936A CL2020001936A1 CL 2020001936 A1 CL2020001936 A1 CL 2020001936A1 CL 2020001936 A CL2020001936 A CL 2020001936A CL 2020001936 A CL2020001936 A CL 2020001936A CL 2020001936 A1 CL2020001936 A1 CL 2020001936A1
Authority
CL
Chile
Prior art keywords
treatment
methods
muscular atrophy
spinal muscular
sma
Prior art date
Application number
CL2020001936A
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 CL2020001936A1 publication Critical patent/CL2020001936A1/es

Links

Classifications

    • 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

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.
CL2020001936A 2018-01-25 2020-07-23 Métodos para el tratamiento de la atrofía muscular espinal. CL2020001936A1 (es)

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
CL2020001936A1 true CL2020001936A1 (es) 2020-12-18

Family

ID=65529774

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2020001936A CL2020001936A1 (es) 2018-01-25 2020-07-23 Métodos para el tratamiento de la atrofía muscular espinal.

Country Status (13)

Country Link
US (2) US11474113B2 (es)
EP (1) EP3743728A1 (es)
JP (2) JP2021511389A (es)
KR (1) KR20200112906A (es)
CN (1) CN112567246A (es)
AU (1) AU2019211446A1 (es)
BR (1) BR112020015082A2 (es)
CL (1) CL2020001936A1 (es)
IL (1) IL276180A (es)
MA (1) MA51678A (es)
MX (1) MX2020007852A (es)
SG (1) SG11202006677XA (es)
WO (1) WO2019147960A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021511389A (ja) 2018-01-25 2021-05-06 バイオジェン・エムエイ・インコーポレイテッドＢｉｏｇｅｎ ＭＡ Ｉｎｃ． 脊髄性筋萎縮症を治療する方法
EP4335503A2 (en) 2019-07-19 2024-03-13 Biogen MA Inc. Methods of treating or preventing spinal muscular atrophy
WO2022261648A2 (en) * 2021-06-08 2022-12-15 Alector Llc Methods of use of anti-sortilin antibodies
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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 (en) 1990-12-06 1992-06-25 Affymax Technologies N.V. Sequencing by hybridization of a target nucleic acid to a matrix of defined oligonucleotides
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
US6057100A (en) 1996-06-07 2000-05-02 Eos Biotechnology, Inc. Oligonucleotide arrays
US7101663B2 (en) 2001-03-02 2006-09-05 University of Pittsburgh—of the Commonwealth System of Higher Education PCR method
WO2002085926A2 (de) 2001-04-19 2002-10-31 GESELLSCHAFT FüR BIOTECHNOLOGISCHE FORSCHUNG MBH (GBF) Verfahren zur herstellung stabiler, regenerierbarer antikörper-arrays
US6812341B1 (en) 2001-05-11 2004-11-02 Ambion, Inc. High efficiency mRNA isolation methods and compositions
US20030013208A1 (en) 2001-07-13 2003-01-16 Milagen, Inc. Information enhanced antibody arrays
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
DK1910395T3 (da) 2005-06-23 2013-02-04 Isis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulation af SMN2-splejsning
US9765297B2 (en) * 2007-04-13 2017-09-19 The Trustees Of Columbia University In The City Of New York Stem cell-based culture system for drug development
WO2010129021A1 (en) * 2009-05-02 2010-11-11 Genzyme Corporation Gene therapy for neurodegenerative disorders
EP3305302B1 (en) 2009-06-17 2018-09-19 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
US8465727B2 (en) * 2009-10-06 2013-06-18 University of Pittsburgh—of the Commonwealth System of Higher Education Biomarkers for the diagnosis of ALS
EP2943225A4 (en) 2013-01-09 2016-07-13 Ionis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR MODULATING SMN2 DISTRIBUTION IN THE BODY OF A PATIENT
SG11201608109TA (en) 2014-04-01 2016-10-28 Ionis Pharmaceuticals 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
EP3797780B1 (en) 2014-04-17 2022-09-14 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
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
MX2018010109A (es) * 2016-02-23 2018-12-17 Univ Indiana Res & Tech Corp Terapias de combinacion para tratamiento de atrofia muscular espinal.
US20210172963A1 (en) * 2017-05-26 2021-06-10 University Of Miami Determining onset of amyotrophic lateral sclerosis
JP2021502060A (ja) * 2017-10-16 2021-01-28 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 筋萎縮性側索硬化症（ａｌｓ）の治療
JP2021511389A (ja) 2018-01-25 2021-05-06 バイオジェン・エムエイ・インコーポレイテッドＢｉｏｇｅｎ ＭＡ Ｉｎｃ． 脊髄性筋萎縮症を治療する方法
EA201992364A1 (ru) * 2018-04-02 2020-03-23 Кохерус Байосайенсис Инк. АГОНИСТ PPARγ ДЛЯ ЛЕЧЕНИЯ ПРОГРЕССИРУЮЩЕГО НАДЪЯДЕРНОГО ПАРАЛИЧА
WO2020061355A2 (en) 2018-09-20 2020-03-26 Biogen Ma Inc. Serum neurofilament protein for guiding therapeutic intervention in multiple sclerosis
JP2022513721A (ja) 2018-12-06 2022-02-09 バイオジェン・エムエイ・インコーポレイテッド 筋萎縮性側索硬化症における治療的介入を導くためのニューロフィラメントタンパク質
TW202035694A (zh) 2018-12-14 2020-10-01 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
WO2020167715A1 (en) 2019-02-12 2020-08-20 Biogen Ma Inc. Biomarkers of progressive multifocal leukoencephalopathy

Also Published As

Publication number Publication date
US20210041459A1 (en) 2021-02-11
MA51678A (fr) 2020-12-02
MX2020007852A (es) 2021-01-29
BR112020015082A2 (pt) 2021-01-05
KR20200112906A (ko) 2020-10-05
SG11202006677XA (en) 2020-08-28
CN112567246A (zh) 2021-03-26
JP2021511389A (ja) 2021-05-06
JP2023118821A (ja) 2023-08-25
WO2019147960A1 (en) 2019-08-01
AU2019211446A1 (en) 2020-09-10
IL276180A (en) 2020-09-30
US20230107651A1 (en) 2023-04-06
US11474113B2 (en) 2022-10-18
EP3743728A1 (en) 2020-12-02

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