JP6475639B2 - デュシェンヌ筋ジストロフィーの治療におけるフォリスタチン - Google Patents

デュシェンヌ筋ジストロフィーの治療におけるフォリスタチン Download PDF

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JP6475639B2
JP6475639B2 JP2015555353A JP2015555353A JP6475639B2 JP 6475639 B2 JP6475639 B2 JP 6475639B2 JP 2015555353 A JP2015555353 A JP 2015555353A JP 2015555353 A JP2015555353 A JP 2015555353A JP 6475639 B2 JP6475639 B2 JP 6475639B2
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follistatin
muscle
days
protein
amino acid
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JP2016511751A (ja
JP2016511751A5 (enExample
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ロシェル ミネアウ
ロシェル ミネアウ
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シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド
シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/04—Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/10—Drugs for disorders of the urinary system of the bladder
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/06—Anabolic agents
    • 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
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Neurosurgery (AREA)
  • Rheumatology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Cardiology (AREA)
  • Psychiatry (AREA)
JP2015555353A 2013-01-25 2014-01-24 デュシェンヌ筋ジストロフィーの治療におけるフォリスタチン Expired - Fee Related JP6475639B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361756996P 2013-01-25 2013-01-25
US61/756,996 2013-01-25
US201361915733P 2013-12-13 2013-12-13
US61/915,733 2013-12-13
PCT/US2014/012996 WO2014116981A1 (en) 2013-01-25 2014-01-24 Follistatin in treating duchenne muscular dystrophy

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JP2018203613A Division JP2019059730A (ja) 2013-01-25 2018-10-30 デュシェンヌ筋ジストロフィーの治療におけるフォリスタチン

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JP2016511751A JP2016511751A (ja) 2016-04-21
JP2016511751A5 JP2016511751A5 (enExample) 2017-02-23
JP6475639B2 true JP6475639B2 (ja) 2019-02-27

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JP2018203613A Pending JP2019059730A (ja) 2013-01-25 2018-10-30 デュシェンヌ筋ジストロフィーの治療におけるフォリスタチン

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US (2) US9957309B2 (enExample)
EP (1) EP2948161B1 (enExample)
JP (2) JP6475639B2 (enExample)
KR (1) KR102253597B1 (enExample)
CN (1) CN104902915B (enExample)
AU (2) AU2014209178B2 (enExample)
CA (1) CA2898121A1 (enExample)
EA (1) EA201590719A1 (enExample)
ES (1) ES2715710T3 (enExample)
IL (1) IL240024B (enExample)
MX (1) MX362105B (enExample)
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JP2019059730A (ja) * 2013-01-25 2019-04-18 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド デュシェンヌ筋ジストロフィーの治療におけるフォリスタチン

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TWI782836B (zh) 2007-02-02 2022-11-01 美商艾瑟勒朗法瑪公司 衍生自ActRIIB的變體與其用途
US20100008918A1 (en) 2008-06-26 2010-01-14 Acceleron Pharma Inc. Methods for dosing an actriib antagonist and monitoring of treated patients
US8216997B2 (en) 2008-08-14 2012-07-10 Acceleron Pharma, Inc. Methods for increasing red blood cell levels and treating anemia using a combination of GDF traps and erythropoietin receptor activators
EP2971039B1 (en) 2013-03-14 2020-01-01 Immusoft Corporation Methods for in vitro memory b cell differentiation and transduction with vsv-g pseudotyped viral vectors
WO2015187977A1 (en) 2014-06-04 2015-12-10 Acceleron Pharma, Inc. Methods and compositions for treatment of disorders with follistatin polypeptides
US10010498B2 (en) 2014-06-04 2018-07-03 Acceleron Pharma Inc. Methods for treatment of amyotrophic lateral sclerosis with follistatin fusion proteins
WO2016154601A1 (en) * 2015-03-26 2016-09-29 Acceleron Pharma Inc. Follistatin-related fusion proteins and uses thereof
EP4233887A1 (en) * 2016-01-06 2023-08-30 Stealth BioTherapeutics Inc. Methods for the treatment of duchenne muscular dystrophy
KR20180137487A (ko) * 2016-03-04 2018-12-27 샤이어 휴먼 지네틱 테라피즈 인크. 재조합 폴리스타틴-fc 융합 단백질 및 듀시엔형 근이영양증 치료에서의 용도
US20180362604A1 (en) * 2017-05-12 2018-12-20 Shire Human Genetic Therapies, Inc. Recombinant Follistatin-FC Fusion Proteins and Use in Treating Duchenne Muscular Dystrophy
KR101822633B1 (ko) * 2017-08-22 2018-01-26 (주)진셀팜 폴리스타틴 유래 생리활성 펩타이드, 및 이의 용도
WO2019046903A1 (en) * 2017-09-08 2019-03-14 Baker Heart and Diabetes Institute THERAPEUTIC METHOD FOR INCREASING MUSCLE MASS IN A SUBJECT
JP2021515576A (ja) * 2018-03-16 2021-06-24 イミュソフト コーポレーション フォリスタチンを分泌するように遺伝子操作されたｂ細胞ならびにフォリスタチン関連疾患、状態、障害を処置するために、ならびに筋肉の成長および強度を増強するためにこれを使用する方法
WO2019191204A1 (en) * 2018-03-28 2019-10-03 Acceleron Pharma Inc. Follistatin polypeptides for the treatment of muscle contracture
CA3094859A1 (en) * 2020-10-01 2022-04-01 Entos Pharmaceuticals Inc. Proteolipid vesicles formulated with fusion associated small transmembrane proteins
KR102361657B1 (ko) 2021-04-26 2022-02-14 주식회사 케이에스비튜젠 14-디옥시-11,12-디디히드로안드로그라폴라이드 석시네이트를 유효성분으로 함유하는 듀센형 근이영양증을 포함한 유전성 근육질환의 예방 또는 치료용 조성물

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JP6475639B2 (ja) * 2013-01-25 2019-02-27 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド デュシェンヌ筋ジストロフィーの治療におけるフォリスタチン
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Publication number Priority date Publication date Assignee Title
JP2019059730A (ja) * 2013-01-25 2019-04-18 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド デュシェンヌ筋ジストロフィーの治療におけるフォリスタチン

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HK1217637A1 (en) 2017-01-20
CN104902915A (zh) 2015-09-09
AU2014209178B2 (en) 2018-11-08
EP2948161B1 (en) 2018-12-12
EP2948161A1 (en) 2015-12-02
IL240024A0 (en) 2015-09-24
AU2019200797B2 (en) 2021-03-04
IL240024B (en) 2020-05-31
ES2715710T3 (es) 2019-06-05
US10723772B2 (en) 2020-07-28
MX362105B (es) 2019-01-04
KR102253597B1 (ko) 2021-05-17
AU2014209178A1 (en) 2015-05-21
HK1213804A1 (zh) 2016-07-15
EA201590719A1 (ru) 2015-12-30
JP2016511751A (ja) 2016-04-21
CN104902915B (zh) 2018-07-06
JP2019059730A (ja) 2019-04-18
US20180305421A1 (en) 2018-10-25
KR20150111347A (ko) 2015-10-05
US20160083440A1 (en) 2016-03-24
AU2019200797A1 (en) 2019-02-21
US9957309B2 (en) 2018-05-01
MX2015009631A (es) 2015-11-30
WO2014116981A1 (en) 2014-07-31
CA2898121A1 (en) 2014-07-31

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