AR119430A1 - Métodos para tratar o prevenir la atrofia muscular espinal - Google Patents

Métodos para tratar o prevenir la atrofia muscular espinal

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Publication number
AR119430A1
AR119430A1 ARP200102019A ARP200102019A AR119430A1 AR 119430 A1 AR119430 A1 AR 119430A1 AR P200102019 A ARP200102019 A AR P200102019A AR P200102019 A ARP200102019 A AR P200102019A AR 119430 A1 AR119430 A1 AR 119430A1
Authority
AR
Argentina
Prior art keywords
antisense compound
muscular atrophy
spinal muscular
treat
methods
Prior art date
Application number
ARP200102019A
Other languages
English (en)
Inventor
Kenneth Swope Loveday
Fengju Judy Bai
Lilly East
Wildon R Farwell
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 AR119430A1 publication Critical patent/AR119430A1/es

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    • 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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0021—Intradermal administration, e.g. through microneedle arrays, needleless injectors
    • 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
    • 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
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    • 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
    • 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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • 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
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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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/35—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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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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

Abstract

Reivindicación 1: Un método para tratar la atrofia muscular espinal (AME) en un sujeto humano que lo necesita, el método comprende administrar al sujeto humano al menos dos dosis de carga de un compuesto antisentido que tiene la estructura de fórmula (1), o una sal de este, en donde el método comprende administrar al sujeto humano mediante inyección intratecal: (i) una primera dosis de carga del compuesto antisentido o la sal de este que es equivalente a alrededor de 50 mg del compuesto antisentido; y (ii) una segunda dosis de carga del compuesto antisentido o la sal de este que es equivalente a alrededor de 50 mg del compuesto antisentido; administrada aproximadamente 10 a 20 días luego de la administración de la primera dosis de carga.
ARP200102019A 2019-07-19 2020-07-17 Métodos para tratar o prevenir la atrofia muscular espinal AR119430A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962876360P 2019-07-19 2019-07-19

Publications (1)

Publication Number Publication Date
AR119430A1 true AR119430A1 (es) 2021-12-15

Family

ID=71995097

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP200102019A AR119430A1 (es) 2019-07-19 2020-07-17 Métodos para tratar o prevenir la atrofia muscular espinal

Country Status (31)

Country Link
US (1) US20220325282A1 (es)
EP (2) EP4335503A3 (es)
JP (2) JP7279253B2 (es)
KR (1) KR20220042113A (es)
CN (1) CN114302727A (es)
AR (1) AR119430A1 (es)
AU (1) AU2020316997A1 (es)
BR (1) BR112022000870A2 (es)
CA (1) CA3149906A1 (es)
CL (1) CL2021003602A1 (es)
CO (1) CO2022001354A2 (es)
CR (1) CR20220071A (es)
DK (1) DK3999643T5 (es)
ES (1) ES2966791T3 (es)
FI (1) FI3999643T3 (es)
HR (1) HRP20231515T1 (es)
HU (1) HUE064071T2 (es)
IL (1) IL289745A (es)
JO (1) JOP20220007A1 (es)
LT (1) LT3999643T (es)
MA (1) MA62935B1 (es)
MD (1) MD3999643T2 (es)
MX (1) MX2022000441A (es)
PE (1) PE20220941A1 (es)
PL (1) PL3999643T3 (es)
PT (1) PT3999643T (es)
RS (1) RS64879B1 (es)
SI (1) SI3999643T1 (es)
TW (1) TWI841762B (es)
UY (1) UY38790A (es)
WO (1) WO2021016032A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015161170A2 (en) 2014-04-17 2015-10-22 Isis Pharmaceuticals, 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
WO2024138018A1 (en) * 2022-12-23 2024-06-27 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Treatment of gemin5-mediated disorders

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3308788T3 (pl) 2005-06-23 2019-05-31 Biogen Ma Inc Kompozycje i sposoby modulacji splicingu smn2
WO2008157753A1 (en) * 2007-06-21 2008-12-24 Sma Foundation Methods of treatment for spinal muscular atrophy
KR20120093138A (ko) * 2009-06-17 2012-08-22 콜드스프링하버러보러토리 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법
WO2011032109A1 (en) 2009-09-11 2011-03-17 Sma Foundation Biomarkers for spinal muscular atrophy
EP2714037B1 (en) * 2011-05-25 2016-07-13 Université Paris Descartes Erk inhibitors for use in treating spinal muscular atrophy
US9926559B2 (en) * 2013-01-09 2018-03-27 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
SG11201900238UA (en) * 2016-07-15 2019-02-27 Ionis Pharmaceuticals Inc Compounds and methods for modulation of smn2
TW201927241A (zh) 2017-12-21 2019-07-16 瑞士商赫孚孟拉羅股份公司 用於肌肉失能之數位生物標記
KR20200112906A (ko) 2018-01-25 2020-10-05 바이오젠 엠에이 인코포레이티드 척추 근위축증의 치료 방법
IT201800004359A1 (it) 2018-04-10 2019-10-10 IDENTIFICAZIONE DI miRNA MUSCOLARI COME BIOMARCATORI E TRATTAMENTO CO-ADIUVANTE PER L’ATROFIA MUSCOLARE SPINALE
JP2021534154A (ja) * 2018-08-15 2021-12-09 バイオジェン・エムエイ・インコーポレイテッドBiogen MA Inc. 脊髄性筋萎縮症のための併用療法

Also Published As

Publication number Publication date
BR112022000870A2 (pt) 2022-03-22
WO2021016032A1 (en) 2021-01-28
RS64879B1 (sr) 2023-12-29
CR20220071A (es) 2022-04-22
SI3999643T1 (sl) 2024-01-31
MD3999643T2 (ro) 2024-02-29
IL289745A (en) 2022-03-01
HUE064071T2 (hu) 2024-02-28
FI3999643T3 (fi) 2023-11-30
JP2023100911A (ja) 2023-07-19
LT3999643T (lt) 2023-12-11
TW202116331A (zh) 2021-05-01
ES2966791T3 (es) 2024-04-24
JOP20220007A1 (ar) 2023-01-30
EP4335503A2 (en) 2024-03-13
AU2020316997A1 (en) 2022-03-10
HRP20231515T1 (hr) 2024-03-01
JP7279253B2 (ja) 2023-05-22
MX2022000441A (es) 2022-03-25
CL2021003602A1 (es) 2022-10-14
DK3999643T5 (da) 2024-08-05
EP3999643B1 (en) 2023-11-22
EP3999643A1 (en) 2022-05-25
JP2022540993A (ja) 2022-09-21
US20220325282A1 (en) 2022-10-13
EP4335503A3 (en) 2024-06-05
CO2022001354A2 (es) 2022-03-18
PT3999643T (pt) 2023-12-11
KR20220042113A (ko) 2022-04-04
TWI841762B (zh) 2024-05-11
CN114302727A (zh) 2022-04-08
UY38790A (es) 2021-01-29
CA3149906A1 (en) 2021-01-28
DK3999643T3 (da) 2023-12-04
PE20220941A1 (es) 2022-05-31
PL3999643T3 (pl) 2024-02-19
MA62935B1 (fr) 2023-11-30

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