KR20220042113A - 척수성 근위축증의 치료 또는 예방 방법 - Google Patents

척수성 근위축증의 치료 또는 예방 방법 Download PDF

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KR20220042113A
KR20220042113A KR1020227001151A KR20227001151A KR20220042113A KR 20220042113 A KR20220042113 A KR 20220042113A KR 1020227001151 A KR1020227001151 A KR 1020227001151A KR 20227001151 A KR20227001151 A KR 20227001151A KR 20220042113 A KR20220042113 A KR 20220042113A
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antisense compound
salt
dose
administered
human subject
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케네스 스워프 러브데이
펭주 주디 바이
릴리 이스트
윌든 알 파웰
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바이오젠 엠에이 인코포레이티드
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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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 or needleless injectors
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    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • C12N15/09—Recombinant DNA-technology
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    • 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
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    • 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
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KR1020227001151A 2019-07-19 2020-07-16 척수성 근위축증의 치료 또는 예방 방법 Pending KR20220042113A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962876360P 2019-07-19 2019-07-19
US62/876,360 2019-07-19
PCT/US2020/042312 WO2021016032A1 (en) 2019-07-19 2020-07-16 Methods of treating or preventing spinal muscular atrophy

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US (3) US20220325282A1 (https=)
EP (2) EP4335503A3 (https=)
JP (3) JP7279253B2 (https=)
KR (1) KR20220042113A (https=)
CN (1) CN114302727A (https=)
AR (1) AR119430A1 (https=)
AU (1) AU2020316997A1 (https=)
BR (1) BR112022000870A2 (https=)
CA (1) CA3149906A1 (https=)
CL (1) CL2021003602A1 (https=)
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CR (1) CR20220071A (https=)
DK (1) DK3999643T5 (https=)
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TW (2) TW202438086A (https=)
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Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3797780B1 (en) 2014-04-17 2022-09-14 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
PT3999643T (pt) 2019-07-19 2023-12-11 Biogen Ma Inc Métodos de tratamento da atrofia muscular espinhal
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
EP3470072A1 (en) * 2005-06-23 2019-04-17 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing
WO2008157753A1 (en) * 2007-06-21 2008-12-24 Sma Foundation Methods of treatment for spinal muscular atrophy
ES2699827T3 (es) 2009-06-17 2019-02-13 Biogen Ma Inc Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto
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
WO2014110291A1 (en) 2013-01-09 2014-07-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
EP3484524B1 (en) * 2016-07-15 2022-11-09 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
CN119679944A (zh) 2018-01-25 2025-03-25 渤健马萨诸塞州股份有限公司 治疗脊髓性肌萎缩症的方法
IT201800004359A1 (it) 2018-04-10 2019-10-10 IDENTIFICAZIONE DI miRNA MUSCOLARI COME BIOMARCATORI E TRATTAMENTO CO-ADIUVANTE PER L’ATROFIA MUSCOLARE SPINALE
US20210308281A1 (en) * 2018-08-15 2021-10-07 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
PT3999643T (pt) 2019-07-19 2023-12-11 Biogen Ma Inc Métodos de tratamento da atrofia muscular espinhal

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US20250230440A1 (en) 2025-07-17
PE20220941A1 (es) 2022-05-31
TWI841762B (zh) 2024-05-11
ES2966791T3 (es) 2024-04-24
US20220325282A1 (en) 2022-10-13
EP4335503A3 (en) 2024-06-05
JOP20220007A1 (ar) 2023-01-30
HRP20231515T1 (hr) 2024-03-01
TW202116331A (zh) 2021-05-01
US20240318182A1 (en) 2024-09-26
TW202438086A (zh) 2024-10-01
FI3999643T3 (fi) 2023-11-30
BR112022000870A2 (pt) 2022-03-22
JOP20250103A1 (ar) 2025-05-05
EP3999643B1 (en) 2023-11-22
DK3999643T5 (da) 2024-08-05
JP2025096486A (ja) 2025-06-26
IL289745A (en) 2022-03-01
LT3999643T (lt) 2023-12-11
CN114302727A (zh) 2022-04-08
SMT202300442T1 (it) 2024-01-10
AU2020316997A1 (en) 2022-03-10
CL2021003602A1 (es) 2022-10-14
MD3999643T2 (ro) 2024-02-29
AR119430A1 (es) 2021-12-15
DK3999643T3 (da) 2023-12-04
PT3999643T (pt) 2023-12-11
JP7279253B2 (ja) 2023-05-22
EP4335503A2 (en) 2024-03-13
HUE064071T2 (hu) 2024-02-28
JP2022540993A (ja) 2022-09-21
MA62935B1 (fr) 2023-11-30
CA3149906A1 (en) 2021-01-28
RS64879B1 (sr) 2023-12-29
UY38790A (es) 2021-01-29
PH12022550114A1 (en) 2023-04-17
PL3999643T3 (pl) 2024-02-19
SI3999643T1 (sl) 2024-01-31
CO2022001354A2 (es) 2022-03-18
WO2021016032A1 (en) 2021-01-28
JP2023100911A (ja) 2023-07-19
CR20220071A (es) 2022-04-22
MX2022000441A (es) 2022-03-25
JP7665678B2 (ja) 2025-04-21
EP3999643A1 (en) 2022-05-25

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