MX2021006548A - Proteina de neurofilamento para guiar la intervencion terapeutica en la esclerosis lateral amiotrofica. - Google Patents

Proteina de neurofilamento para guiar la intervencion terapeutica en la esclerosis lateral amiotrofica.

Info

Publication number
MX2021006548A
MX2021006548A MX2021006548A MX2021006548A MX2021006548A MX 2021006548 A MX2021006548 A MX 2021006548A MX 2021006548 A MX2021006548 A MX 2021006548A MX 2021006548 A MX2021006548 A MX 2021006548A MX 2021006548 A MX2021006548 A MX 2021006548A
Authority
MX
Mexico
Prior art keywords
lateral sclerosis
amyotrophic lateral
patient
neurofilament protein
therapeutic intervention
Prior art date
Application number
MX2021006548A
Other languages
English (en)
Inventor
Toby Arlo Ferguson
Danielle Leezetta Graham
Steve Sang-Woo Han
Alexander Reid Vincent Mccampbell
Giulio Srubek-Tomassy
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 MX2021006548A publication Critical patent/MX2021006548A/es

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    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • 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
    • C12N15/1137—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 against enzymes
    • C—CHEMISTRY; METALLURGY
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    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • 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
    • G01N33/6887—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids from muscle, cartilage or connective tissue
    • C—CHEMISTRY; METALLURGY
    • 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
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    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
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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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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)
    • 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

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

Abstract

En un aspecto, la invención proporciona un método para aliviar un síntoma en un paciente con esclerosis lateral amiotrófica, que comprende la administración de un producto terapéutico eficaz que es eficaz para el paciente, el producto terapéutico eficaz, cuando se le administra al paciente, tiene como resultado un nivel de proteína de neurofilamento en una muestra biológica del paciente que es inferior que un nivel de proteína de neurofilamento en un paciente con esclerosis lateral amiotrófica al que no se le administró el producto terapéutico. En algunas modalidades, al paciente se le está administrando actualmente o se le ha administrado anteriormente un producto terapéutico no eficaz o un producto terapéutico que es diferente al producto terapéutico eficaz.
MX2021006548A 2018-12-06 2019-12-03 Proteina de neurofilamento para guiar la intervencion terapeutica en la esclerosis lateral amiotrofica. MX2021006548A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862776253P 2018-12-06 2018-12-06
US201962840431P 2019-04-30 2019-04-30
US201962842063P 2019-05-02 2019-05-02
PCT/US2019/064190 WO2020117772A1 (en) 2018-12-06 2019-12-03 Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis

Publications (1)

Publication Number Publication Date
MX2021006548A true MX2021006548A (es) 2021-09-21

Family

ID=68966114

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021006548A MX2021006548A (es) 2018-12-06 2019-12-03 Proteina de neurofilamento para guiar la intervencion terapeutica en la esclerosis lateral amiotrofica.

Country Status (10)

Country Link
US (1) US20220034907A1 (es)
EP (1) EP3890752A1 (es)
JP (1) JP2022513721A (es)
KR (1) KR20210102294A (es)
CN (1) CN113365640A (es)
BR (1) BR112021010660A2 (es)
CA (1) CA3121749A1 (es)
MA (1) MA54383A (es)
MX (1) MX2021006548A (es)
WO (1) WO2020117772A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112020015082A2 (pt) 2018-01-25 2021-01-05 Biogen Ma, Inc. Métodos de tratamento da atrofia muscular espinhal

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US20050019915A1 (en) * 2001-06-21 2005-01-27 Bennett C. Frank Antisense modulation of superoxide dismutase 1, soluble expression
WO2003000707A2 (en) 2001-06-21 2003-01-03 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
DK3404027T3 (da) 2012-05-09 2020-06-02 Biogen Ma Inc Cellekernetransportmodulatorer og anvendelser deraf
DK2906696T4 (da) 2012-10-15 2023-02-27 Ionis Pharmaceuticals Inc Fremgangsmåder til modulering af c9orf72-ekspression
RU2730677C2 (ru) 2012-10-15 2020-08-24 Ионис Фармасьютикалз, Инк. Соединение для модуляции экспрессии гена c9orf72 и его применение
WO2014062736A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
IN2015DN04172A (es) * 2012-10-24 2015-10-16 Daiichi Sankyo Companyltd
SG11201602597YA (en) 2013-10-11 2016-05-30 Ionis Pharmaceuticals Inc Compositions for modulating c9orf72 expression
ES2747260T3 (es) 2013-10-14 2020-03-10 Ionis Pharmaceuticals Inc Métodos para modular la expresión de transcrito antisentido C9ORF72
WO2015057727A1 (en) 2013-10-14 2015-04-23 Isis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
ES2890677T3 (es) 2014-03-19 2022-01-21 Ionis Pharmaceuticals Inc Composiciones para modular la expresión de ataxina 2
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MX2017006216A (es) * 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
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JP2021521896A (ja) 2018-04-27 2021-08-30 バイオジェン・エムエイ・インコーポレイテッドＢｉｏｇｅｎ ＭＡ Ｉｎｃ． ヒト由来の抗（ポリｇａ）ジペプチドリピート（ｄｐｒ）抗体

Also Published As

Publication number Publication date
BR112021010660A2 (pt) 2021-08-24
MA54383A (fr) 2021-10-13
CA3121749A1 (en) 2020-06-11
KR20210102294A (ko) 2021-08-19
EP3890752A1 (en) 2021-10-13
US20220034907A1 (en) 2022-02-03
WO2020117772A1 (en) 2020-06-11
JP2022513721A (ja) 2022-02-09
CN113365640A (zh) 2021-09-07

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