MX2023011214A - Tratamiento de la esclerosis lateral amiotrófica. - Google Patents

Tratamiento de la esclerosis lateral amiotrófica.

Info

Publication number
MX2023011214A
MX2023011214A MX2023011214A MX2023011214A MX2023011214A MX 2023011214 A MX2023011214 A MX 2023011214A MX 2023011214 A MX2023011214 A MX 2023011214A MX 2023011214 A MX2023011214 A MX 2023011214A MX 2023011214 A MX2023011214 A MX 2023011214A
Authority
MX
Mexico
Prior art keywords
treatment
lateral sclerosis
amyotrophic lateral
sod1
presymptomatic
Prior art date
Application number
MX2023011214A
Other languages
English (en)
Spanish (es)
Inventor
Peng Sun
Danielle Leezetta Graham
Yingying Liu
Stephanie Melillo Fradette
Janice Chun Yee Wong
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 MX2023011214A publication Critical patent/MX2023011214A/es

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
    • C12N15/1137Non-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
    • 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
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y115/00Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001Superoxide dismutase (1.15.1.1)
    • 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
    • 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
    • C12N2310/3222'-R Modification
    • 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
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • 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
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy
    • 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
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3531Hydrogen
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
MX2023011214A 2021-03-31 2022-03-30 Tratamiento de la esclerosis lateral amiotrófica. MX2023011214A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163168972P 2021-03-31 2021-03-31
PCT/US2022/022485 WO2022212459A1 (en) 2021-03-31 2022-03-30 Treatment of amyotrophic lateral sclerosis

Publications (1)

Publication Number Publication Date
MX2023011214A true MX2023011214A (es) 2023-12-08

Family

ID=81346346

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023011214A MX2023011214A (es) 2021-03-31 2022-03-30 Tratamiento de la esclerosis lateral amiotrófica.

Country Status (11)

Country Link
US (1) US20240182903A1 (cg-RX-API-DMAC7.html)
EP (1) EP4314294A1 (cg-RX-API-DMAC7.html)
JP (1) JP2024511767A (cg-RX-API-DMAC7.html)
KR (1) KR20230172502A (cg-RX-API-DMAC7.html)
CN (1) CN117062911A (cg-RX-API-DMAC7.html)
AU (1) AU2022252236A1 (cg-RX-API-DMAC7.html)
BR (1) BR112023019877A2 (cg-RX-API-DMAC7.html)
CA (1) CA3214585A1 (cg-RX-API-DMAC7.html)
MX (1) MX2023011214A (cg-RX-API-DMAC7.html)
TW (1) TW202304471A (cg-RX-API-DMAC7.html)
WO (1) WO2022212459A1 (cg-RX-API-DMAC7.html)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN120350010B (zh) * 2025-06-24 2025-09-09 羿美诚健(上海)生物医药有限公司 靶向PIKfyve基因的小核酸及其药物组合物和用途

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2452458A1 (en) 2001-07-03 2003-01-16 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
HUE050704T2 (hu) 2014-04-01 2020-12-28 Biogen Ma Inc Összetételek a SOD-1 expressziójának modulálására
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

Also Published As

Publication number Publication date
KR20230172502A (ko) 2023-12-22
WO2022212459A1 (en) 2022-10-06
US20240182903A1 (en) 2024-06-06
AU2022252236A1 (en) 2023-10-12
TW202304471A (zh) 2023-02-01
CN117062911A (zh) 2023-11-14
EP4314294A1 (en) 2024-02-07
AU2022252236A9 (en) 2023-10-19
BR112023019877A2 (pt) 2023-11-07
JP2024511767A (ja) 2024-03-15
CA3214585A1 (en) 2022-10-06

Similar Documents

Publication Publication Date Title
WO2006133052A3 (en) A cellular therapy for ocular degeneration
WO2021071788A3 (en) Oligonucleotide compositions and methods of use thereof
WO2020227691A3 (en) Oligonucleotide compositions and methods of use thereof
MY146937A (en) Hydrocarbon-soluble molybdenum catalyst precursors and methods of making same
BRPI0506713A (pt) composições de ácido peroxicarboxìlico de cadeia média
WO2011153553A3 (en) Methods and compositions for kinase inhibition
MX2023011214A (es) Tratamiento de la esclerosis lateral amiotrófica.
CO6400140A2 (es) Derivados de benzotiazol como agentes anticancer
WO2020176654A8 (en) Use of caseinolytic protease p function as a biomarker of drug response to imipridone-like agents
IN2014DN10996A (cg-RX-API-DMAC7.html)
MX2011005162A (es) Proceso para el tratamiento de glicerol.
NZ706745A (en) Methods of treatment of pancreatic cancer
BR112013003002A2 (pt) composição contendo aditivo polar e agente quelante útil no hidroprocessamento de suprimentos hidrocarbonetos e método de sua produção e utilização
MY153871A (en) Isoprene synthase variants for improved microbial production of isoprene
PT1938839E (pt) Composições oftálmicas de estimulantes parassimpáticos e anti-inflamatórios para utilização no tratamento de presbiopia
MX2009013649A (es) Variantes geneticas en el cromosoma 15q24 como marcadores para usarse en diagnostico, prognosis y tratamiento de sindrome de exfoliacion y glaucoma.
TW200726778A (en) Compositions and methods for producing silicone hydrogel contact lenses
AR060779A1 (es) Un metodo para disenar una lente de contacto multifocal que hacen uso de l a apodizacion de la pupila
MX2008010801A (es) Indolopiridinas como moduladores de quinesina eg5.
CL2022000825A1 (es) Modificaciones químicas de arn pequeño de interferencia con contenido mínimo de flúor
MX2025004102A (es) Metodos de identificacion de pacientes con posibilidad de beneficiarse de un tratamiento con un inhibidor de la telomerasa
NZ588999A (en) Compositions for enhancing the antibacterial activity of myeloperoxidase and methods of use thereof
PH12022550124A1 (en) Enzyme inhibitors
WO2010063630A3 (en) Pancreatic tumour treatment
MX354827B (es) Mezcla de policarbonato y metodo de produccion de la misma.