CR20220071A - Composiciones farmacéuticas y métodos para tratar o prevenir la atrofia muscular espinal - Google Patents

Composiciones farmacéuticas y métodos para tratar o prevenir la atrofia muscular espinal

Info

Publication number
CR20220071A
CR20220071A CR20220071A CR20220071A CR20220071A CR 20220071 A CR20220071 A CR 20220071A CR 20220071 A CR20220071 A CR 20220071A CR 20220071 A CR20220071 A CR 20220071A CR 20220071 A CR20220071 A CR 20220071A
Authority
CR
Costa Rica
Prior art keywords
methods
muscular atrophy
treating
spinal muscular
preventing spinal
Prior art date
Application number
CR20220071A
Other languages
English (en)
Inventor
Wildon R Farwell
Fengju Judy Bai
Kenneth Swope Loveday
Lilly East
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 CR20220071A publication Critical patent/CR20220071A/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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • C12N15/1136—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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2310/34—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
    • 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
    • C12N2310/35—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
    • 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
    • C12N2320/34—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

Landscapes

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

Abstract

Se presentan métodos para el tratamiento o prevención de la atrofia muscular espinal. Se especifican regímenes de dosificación eficaces. También se proporcionan biomarcadores y kits.
CR20220071A 2019-07-19 2020-07-16 Composiciones farmacéuticas y métodos para tratar o prevenir la atrofia muscular espinal CR20220071A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962876360P 2019-07-19 2019-07-19
PCT/US2020/042312 WO2021016032A1 (en) 2019-07-19 2020-07-16 Methods of treating or preventing spinal muscular atrophy

Publications (1)

Publication Number Publication Date
CR20220071A true CR20220071A (es) 2022-04-22

Family

ID=71995097

Family Applications (1)

Application Number Title Priority Date Filing Date
CR20220071A CR20220071A (es) 2019-07-19 2020-07-16 Composiciones farmacéuticas y métodos para tratar o prevenir la atrofia muscular espinal

Country Status (31)

Country Link
US (2) 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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2932304T3 (es) 2014-04-17 2023-01-17 Biogen Ma Inc Composiciones y métodos para la modulación del empalme de SMN2 en un sujeto
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
DK3999643T5 (da) 2019-07-19 2024-08-05 Biogen Ma Inc Fremgangsmåder til behandling eller forebyggelse af spinal muskelatrofi
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 (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2545223T3 (es) * 2005-06-23 2015-09-09 Isis Pharmaceuticals, Inc. Composiciones y procedimientos para modular el corte y empalme de SMN2
WO2008157753A1 (en) * 2007-06-21 2008-12-24 Sma Foundation Methods of treatment for spinal muscular atrophy
EP2442816A4 (en) 2009-06-17 2013-10-02 Isis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR MODULATING SMN2 SPLICING IN A SUBJECT
WO2011032109A1 (en) 2009-09-11 2011-03-17 Sma Foundation Biomarkers for spinal muscular atrophy
CA2836364C (en) * 2011-05-25 2021-01-26 Universite Paris Descartes Erk inhibitors for use in treating spinal muscular atrophy
EP2943225A4 (en) 2013-01-09 2016-07-13 Ionis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR MODULATING SMN2 DISTRIBUTION IN THE BODY OF A PATIENT
US10436802B2 (en) * 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
BR112019000356A2 (pt) * 2016-07-15 2019-04-16 Ionis Pharmaceuticals, Inc. compostos e métodos para modulação de 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
AU2019211446A1 (en) 2018-01-25 2020-09-10 Biogen Ma Inc. Methods of treating spinal muscular atrophy
IT201800004359A1 (it) 2018-04-10 2019-10-10 IDENTIFICAZIONE DI miRNA MUSCOLARI COME BIOMARCATORI E TRATTAMENTO CO-ADIUVANTE PER L’ATROFIA MUSCOLARE SPINALE
EP3837374A4 (en) * 2018-08-15 2022-06-08 Biogen MA Inc. COMBINATION THERAPY FOR SPINAL MUSCLE ATROPHY
DK3999643T5 (da) 2019-07-19 2024-08-05 Biogen Ma Inc Fremgangsmåder til behandling eller forebyggelse af spinal muskelatrofi

Also Published As

Publication number Publication date
JP7279253B2 (ja) 2023-05-22
PL3999643T3 (pl) 2024-02-19
EP4335503A3 (en) 2024-06-05
US20220325282A1 (en) 2022-10-13
HUE064071T2 (hu) 2024-02-28
MX2022000441A (es) 2022-03-25
WO2021016032A1 (en) 2021-01-28
MA62935B1 (fr) 2023-11-30
RS64879B1 (sr) 2023-12-29
IL289745A (en) 2022-03-01
EP3999643A1 (en) 2022-05-25
HRP20231515T1 (hr) 2024-03-01
CA3149906A1 (en) 2021-01-28
SI3999643T1 (sl) 2024-01-31
MD3999643T2 (ro) 2024-02-29
DK3999643T5 (da) 2024-08-05
KR20220042113A (ko) 2022-04-04
AU2020316997A1 (en) 2022-03-10
TW202116331A (zh) 2021-05-01
JOP20220007A1 (ar) 2023-01-30
UY38790A (es) 2021-01-29
EP4335503A2 (en) 2024-03-13
DK3999643T3 (da) 2023-12-04
TWI841762B (zh) 2024-05-11
JP2023100911A (ja) 2023-07-19
EP3999643B1 (en) 2023-11-22
CO2022001354A2 (es) 2022-03-18
FI3999643T3 (fi) 2023-11-30
CN114302727A (zh) 2022-04-08
US20240318182A1 (en) 2024-09-26
ES2966791T3 (es) 2024-04-24
BR112022000870A2 (pt) 2022-03-22
AR119430A1 (es) 2021-12-15
PE20220941A1 (es) 2022-05-31
CL2021003602A1 (es) 2022-10-14
LT3999643T (lt) 2023-12-11
PT3999643T (pt) 2023-12-11
JP2022540993A (ja) 2022-09-21

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