JP2023549455A - 脊髄性筋萎縮症を治療するための抗プロミオスタチン／抗潜在型ミオスタチン抗体の使用 - Google Patents

脊髄性筋萎縮症を治療するための抗プロミオスタチン／抗潜在型ミオスタチン抗体の使用 Download PDF

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JP2023549455A
JP2023549455A JP2023524333A JP2023524333A JP2023549455A JP 2023549455 A JP2023549455 A JP 2023549455A JP 2023524333 A JP2023524333 A JP 2023524333A JP 2023524333 A JP2023524333 A JP 2023524333A JP 2023549455 A JP2023549455 A JP 2023549455A
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sma
apitegromab
patient
therapy
smn
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JPWO2022093724A5 (https=
JP2023549455A5 (https=
Inventor
ジョージ ノミコス
グオチェン ソン
ライアン イアロビノ
ユン チョン
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Scholar Rock Inc
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Scholar Rock Inc
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily
    • C07K16/2809—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily against the T-cell receptor (TcR)-CD3 complex
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily
    • C07K16/2833—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily against MHC-molecules, e.g. HLA-molecules
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/545—Medicinal preparations containing antigens or antibodies characterised by the dose, timing or administration schedule
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/32—Immunoglobulins specific features characterized by aspects of specificity or valency specific for a neo-epitope on a complex, e.g. antibody-antigen or ligand-receptor
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/33—Crossreactivity, e.g. for species or epitope, or lack of said crossreactivity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622—Single chain antibody (scFv)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/73—Inducing cell death, e.g. apoptosis, necrosis or inhibition of cell proliferation
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/76—Antagonist effect on antigen, e.g. neutralization or inhibition of binding

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023524333A 2020-10-26 2021-10-25 脊髄性筋萎縮症を治療するための抗プロミオスタチン／抗潜在型ミオスタチン抗体の使用 Pending JP2023549455A (ja)

Applications Claiming Priority (19)

Application Number Priority Date Filing Date Title
US202063105850P 2020-10-26 2020-10-26
US63/105,850 2020-10-26
US202063106172P 2020-10-27 2020-10-27
US63/106,172 2020-10-27
US202163200955P 2021-04-05 2021-04-05
US63/200,955 2021-04-05
US202163201157P 2021-04-15 2021-04-15
US63/201,157 2021-04-15
US202163202317P 2021-06-06 2021-06-06
US63/202,317 2021-06-06
US202163202372P 2021-06-08 2021-06-08
US63/202,372 2021-06-08
US202163202900P 2021-06-29 2021-06-29
US63/202,900 2021-06-29
US202163260725P 2021-08-30 2021-08-30
US63/260,725 2021-08-30
US202163261398P 2021-09-20 2021-09-20
US63/261,398 2021-09-20
PCT/US2021/056517 WO2022093724A1 (en) 2020-10-26 2021-10-25 Use of anti-pro/latent myostatin antibody for treating spinal muscular atrophy

Publications (3)

Publication Number Publication Date
JP2023549455A true JP2023549455A (ja) 2023-11-27
JPWO2022093724A5 JPWO2022093724A5 (https=) 2024-10-10
JP2023549455A5 JP2023549455A5 (https=) 2024-10-10

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JP2023524333A Pending JP2023549455A (ja) 2020-10-26 2021-10-25 脊髄性筋萎縮症を治療するための抗プロミオスタチン／抗潜在型ミオスタチン抗体の使用

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US (1) US20240002490A1 (https=)
EP (2) EP4729069A1 (https=)
JP (1) JP2023549455A (https=)
KR (1) KR20230095998A (https=)
CN (1) CN116390756A (https=)
AU (1) AU2021369471A1 (https=)
CA (1) CA3193909A1 (https=)
ES (1) ES3058087T3 (https=)
IL (1) IL302132A (https=)
MX (1) MX2023004763A (https=)
PL (1) PL4232151T3 (https=)
TW (1) TW202233234A (https=)
WO (1) WO2022093724A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
LT3350220T (lt) 2015-09-15 2021-09-27 Scholar Rock, Inc. Anti pro/latentinio miostatino antikūnai ir jų panaudojimas
DK4518970T3 (da) 2022-05-04 2026-04-07 Scholar Rock Inc Anvendelse af myostatinhæmmer til behandling af spinal muskelatrofi
IL321628A (en) 2022-12-22 2025-08-01 Scholar Rock Inc Selective and potent inhibitory antibodies for myostatin activation
WO2026076383A1 (en) 2024-10-06 2026-04-09 Scholar Rock, Inc. Use of myostatin inhibitor for treating spinal muscular atrophy

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019523295A (ja) * 2016-06-13 2019-08-22 スカラー ロック インコーポレイテッドＳｃｈｏｌａｒ Ｒｏｃｋ，Ｉｎｃ． ミオスタチン阻害剤の使用および併用療法

Family Cites Families (3)

* 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
AU2008239606A1 (en) 2007-04-12 2008-10-23 Paratek Pharmaceuticals, Inc. Methods for treating spinal muscular atrophy using tetracycline compounds
LT3350220T (lt) 2015-09-15 2021-09-27 Scholar Rock, Inc. Anti pro/latentinio miostatino antikūnai ir jų panaudojimas

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019523295A (ja) * 2016-06-13 2019-08-22 スカラー ロック インコーポレイテッドＳｃｈｏｌａｒ Ｒｏｃｋ，Ｉｎｃ． ミオスタチン阻害剤の使用および併用療法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
PLACE, A. ET AL.: "A Phase 2 Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal M", EUROPEAN JOURNAL OF NEUROLOGY, vol. Vol.27, suppl.1, JPN6025041119, May 2020 (2020-05-01), pages 103 - 522, ISSN: 0005833041 *

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Publication number Publication date
EP4232151B1 (en) 2025-10-22
MX2023004763A (es) 2023-05-09
CN116390756A (zh) 2023-07-04
ES3058087T3 (en) 2026-03-06
PL4232151T3 (pl) 2026-02-23
AU2021369471A1 (en) 2023-06-15
EP4232151A1 (en) 2023-08-30
TW202233234A (zh) 2022-09-01
AU2021369471A9 (en) 2024-02-08
CA3193909A1 (en) 2022-05-05
US20240002490A1 (en) 2024-01-04
KR20230095998A (ko) 2023-06-29
IL302132A (en) 2023-06-01
WO2022093724A1 (en) 2022-05-05
EP4729069A1 (en) 2026-04-22

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