JP2025514978A - 脊髄性筋萎縮症を治療するためのミオスタチン阻害薬の使用 - Google Patents

脊髄性筋萎縮症を治療するためのミオスタチン阻害薬の使用 Download PDF

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JP2025514978A
JP2025514978A JP2024563648A JP2024563648A JP2025514978A JP 2025514978 A JP2025514978 A JP 2025514978A JP 2024563648 A JP2024563648 A JP 2024563648A JP 2024563648 A JP2024563648 A JP 2024563648A JP 2025514978 A JP2025514978 A JP 2025514978A
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sma
apitegromab
patient
therapy
subject
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JP2025514978A5 (enExample
Inventor
ジョージ ノミコス
グオチェン ソン
ヤン チョン
ミカ ティー. ウェブスター
ナタリー ケルテス
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Scholar Rock Inc
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Scholar Rock Inc
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • AHUMAN NECESSITIES
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    • 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
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    • C12Q1/34Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/70Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving creatine or creatinine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K2039/54Medicinal preparations containing antigens or antibodies characterised by the route of administration
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
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    • A61K2039/55Medicinal preparations containing antigens or antibodies characterised by the host/recipient, e.g. newborn with maternal antibodies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/20Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
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    • C07K2317/00Immunoglobulins specific features
    • C07K2317/70Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/76Antagonist effect on antigen, e.g. neutralization or inhibition of binding
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    • C12N2310/11Antisense
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/28Neurological disorders
    • G01N2800/2878Muscular dystrophy
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/52Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

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  • General Physics & Mathematics (AREA)
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JP2024563648A 2022-05-04 2023-05-03 脊髄性筋萎縮症を治療するためのミオスタチン阻害薬の使用 Pending JP2025514978A (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202263364188P 2022-05-04 2022-05-04
US63/364,188 2022-05-04
US202263366447P 2022-06-15 2022-06-15
US63/366,447 2022-06-15
US202263366662P 2022-06-20 2022-06-20
US63/366,662 2022-06-20
US202263378987P 2022-10-10 2022-10-10
US63/378,987 2022-10-10
PCT/US2023/020843 WO2023215384A2 (en) 2022-05-04 2023-05-03 Use of myostatin inhibitor for treating spinal muscular atrophy

Publications (2)

Publication Number Publication Date
JP2025514978A true JP2025514978A (ja) 2025-05-13
JP2025514978A5 JP2025514978A5 (enExample) 2026-05-11

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JP2024563648A Pending JP2025514978A (ja) 2022-05-04 2023-05-03 脊髄性筋萎縮症を治療するためのミオスタチン阻害薬の使用

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Country Link
US (1) US20250326827A1 (enExample)
EP (1) EP4518970B1 (enExample)
JP (1) JP2025514978A (enExample)
KR (1) KR20250021401A (enExample)
CN (1) CN119451696A (enExample)
AU (1) AU2023265846A1 (enExample)
CA (1) CA3251441A1 (enExample)
DK (1) DK4518970T3 (enExample)
FI (1) FI4518970T3 (enExample)
IL (1) IL316463A (enExample)
LT (1) LT4518970T (enExample)
MX (1) MX2024013313A (enExample)
WO (1) WO2023215384A2 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3922645T (pt) 2015-09-15 2025-05-29 Scholar Rock Inc ¿anticorpos anti-pró-miostatina/miostatina latente e suas utilizações
AU2023413166A1 (en) 2022-12-22 2025-07-03 Scholar Rock, Inc. Selective and potent inhibitory antibodies of myostatin activation
WO2026076383A1 (en) * 2024-10-06 2026-04-09 Scholar Rock, Inc. Use of myostatin inhibitor for treating spinal muscular atrophy

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040044063A1 (en) 2002-05-31 2004-03-04 Brent Stockwell SMA therapy and cell based assay for identifying therapies
WO2008127722A1 (en) 2007-04-12 2008-10-23 Paratek Pharmaceuticals, Inc. Methods for treating spinal muscular atrophy using tetracycline compounds
NZ730607A (en) 2014-12-19 2022-07-01 Chugai Pharmaceutical Co Ltd Anti-myostatin antibodies, polypeptides containing variant fc regions, and methods of use
PT3922645T (pt) 2015-09-15 2025-05-29 Scholar Rock Inc ¿anticorpos anti-pró-miostatina/miostatina latente e suas utilizações
KR20210082548A (ko) * 2016-06-13 2021-07-05 스칼러 락, 인크. 미오스타틴 억제제의 용도 및 조합 요법
PL4232151T3 (pl) * 2020-10-26 2026-02-23 Scholar Rock, Inc. Zastosowanie przeciwciała przeciwko pro/latentnej miostatynie w leczeniu rdzeniowego zaniku mięśni
BR112023015177A2 (pt) 2021-01-29 2023-11-14 Biocad Joint Stock Co Ácido nucleico compreendendo smn1 e mir-23a para tratamento da atrofia muscular espinhal, cassete de expressão, vetor de expressão, vírus recombinante, composição farmacêutica, uso e método relacionados

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Publication number Publication date
WO2023215384A2 (en) 2023-11-09
AU2023265846A1 (en) 2024-12-12
IL316463A (en) 2024-12-01
EP4518970A2 (en) 2025-03-12
CA3251441A1 (en) 2023-11-09
MX2024013313A (es) 2025-02-10
LT4518970T (lt) 2026-04-27
CN119451696A (zh) 2025-02-14
WO2023215384A3 (en) 2023-12-07
EP4518970B1 (en) 2026-03-25
US20250326827A1 (en) 2025-10-23
KR20250021401A (ko) 2025-02-12
DK4518970T3 (da) 2026-04-07
FI4518970T3 (fi) 2026-05-08

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