CN119451696A - 肌生成抑制蛋白抑制剂用于治疗脊髓性肌肉萎缩的用途 - Google Patents

肌生成抑制蛋白抑制剂用于治疗脊髓性肌肉萎缩的用途 Download PDF

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Publication number
CN119451696A
CN119451696A CN202380045672.1A CN202380045672A CN119451696A CN 119451696 A CN119451696 A CN 119451696A CN 202380045672 A CN202380045672 A CN 202380045672A CN 119451696 A CN119451696 A CN 119451696A
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patient
sma
therapy
apilimumab
subject
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CN202380045672.1A
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Chinese (zh)
Inventor
G·诺米科斯
宋国臣
Y·奇恩格
M·T·韦伯斯特
N·科特兹
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Stone Supply Co
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Stone Supply Co
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    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic 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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
    • 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
    • G01N33/70—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving creatine or creatinine
    • 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
    • A61K2039/54—Medicinal preparations containing antigens or antibodies characterised by the route of administration
    • 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
    • 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
    • A61K2039/55—Medicinal preparations containing antigens or antibodies characterised by the host/recipient, e.g. newborn with maternal antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • 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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders
    • G01N2800/2878—Muscular dystrophy
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

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  • Medicinal Chemistry (AREA)
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  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Analytical Chemistry (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Endocrinology (AREA)
  • Mycology (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Food Science & Technology (AREA)
CN202380045672.1A 2022-05-04 2023-05-03 肌生成抑制蛋白抑制剂用于治疗脊髓性肌肉萎缩的用途 Pending CN119451696A (zh)

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 (1)

Publication Number Publication Date
CN119451696A true CN119451696A (zh) 2025-02-14

Family

ID=86732326

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202380045672.1A Pending CN119451696A (zh) 2022-05-04 2023-05-03 肌生成抑制蛋白抑制剂用于治疗脊髓性肌肉萎缩的用途

Country Status (20)

Country Link
US (1) US20250326827A1 (enExample)
EP (2) 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)
ES (1) ES3072446T3 (enExample)
FI (1) FI4518970T3 (enExample)
HR (1) HRP20260506T1 (enExample)
IL (1) IL316463A (enExample)
LT (1) LT4518970T (enExample)
MX (1) MX2024013313A (enExample)
PL (1) PL4518970T3 (enExample)
PT (1) PT4518970T (enExample)
RS (1) RS68105B1 (enExample)
SI (1) SI4518970T1 (enExample)
SM (1) SMT202600209T1 (enExample)
WO (1) WO2023215384A2 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016073853A1 (en) 2014-11-06 2016-05-12 Scholar Rock, Inc. Anti-pro/latent-myostatin antibodies and uses thereof
EA038146B1 (ru) 2015-09-15 2021-07-13 Сколар Рок, Инк. Антитела к про-/латентному миостатину и их применения
CN120677175A (zh) 2022-12-22 2025-09-19 供石公司 肌生成抑制蛋白活化的选择性和强效抑制剂
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
EP2144614A1 (en) 2007-04-12 2010-01-20 Paratek Pharmaceuticals, Inc. Methods for treating spinal muscular atrophy using tetracycline compounds
MX2017005774A (es) 2014-12-19 2017-07-28 Chugai Pharmaceutical Co Ltd Anticuerpos antimiostatina, polipeptidos que contienen regiones fc variantes, y metodos de uso.
EA038146B1 (ru) 2015-09-15 2021-07-13 Сколар Рок, Инк. Антитела к про-/латентному миостатину и их применения
PL4218804T3 (pl) * 2016-06-13 2026-05-25 Scholar Rock, Inc. Stosowanie inhibitorów miostatyny i terapii skojarzonych
EP4232151B1 (en) 2020-10-26 2025-10-22 Scholar Rock, Inc. Use of anti-pro/latent myostatin antibody for treating spinal muscular atrophy
CN117545842A (zh) 2021-01-29 2024-02-09 拜奥卡德联合股份公司 SMN1和miR-23a在治疗脊髓性肌萎缩中的协同效应

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Publication number Publication date
MX2024013313A (es) 2025-02-10
HRP20260506T1 (hr) 2026-05-22
EP4778536A2 (en) 2026-07-22
AU2023265846A1 (en) 2024-12-12
EP4518970A2 (en) 2025-03-12
LT4518970T (lt) 2026-04-27
ES3072446T3 (en) 2026-07-01
PL4518970T3 (pl) 2026-07-27
KR20250021401A (ko) 2025-02-12
US20250326827A1 (en) 2025-10-23
FI4518970T3 (fi) 2026-05-08
CA3251441A1 (en) 2023-11-09
DK4518970T3 (da) 2026-04-07
SMT202600209T1 (it) 2026-07-16
SI4518970T1 (sl) 2026-06-30
PT4518970T (pt) 2026-06-08
WO2023215384A3 (en) 2023-12-07
EP4518970B1 (en) 2026-03-25
RS68105B1 (sr) 2026-06-30
JP2025514978A (ja) 2025-05-13
IL316463A (en) 2024-12-01
WO2023215384A2 (en) 2023-11-09

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