EA201590719A1 - Фоллистатин в лечении мышечной дистрофии дюшенна - Google Patents

Фоллистатин в лечении мышечной дистрофии дюшенна

Info

Publication number
EA201590719A1
EA201590719A1 EA201590719A EA201590719A EA201590719A1 EA 201590719 A1 EA201590719 A1 EA 201590719A1 EA 201590719 A EA201590719 A EA 201590719A EA 201590719 A EA201590719 A EA 201590719A EA 201590719 A1 EA201590719 A1 EA 201590719A1
Authority
EA
Eurasian Patent Office
Prior art keywords
treatment
follystatin
dushenna
distrophia
muscular
Prior art date
Application number
EA201590719A
Other languages
English (en)
Russian (ru)
Inventor
Рошель Мино
Original Assignee
Шир Хьюман Дженетик Терапис, Инк.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Шир Хьюман Дженетик Терапис, Инк. filed Critical Шир Хьюман Дженетик Терапис, Инк.
Publication of EA201590719A1 publication Critical patent/EA201590719A1/ru

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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)
EA201590719A 2013-01-25 2014-01-24 Фоллистатин в лечении мышечной дистрофии дюшенна EA201590719A1 (ru)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
EA201590719A1 true EA201590719A1 (ru) 2015-12-30

Family

ID=50159515

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201590719A EA201590719A1 (ru) 2013-01-25 2014-01-24 Фоллистатин в лечении мышечной дистрофии дюшенна

Country Status (12)

Country Link
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)
WO (1) WO2014116981A1 (enExample)

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US8128933B2 (en) 2005-11-23 2012-03-06 Acceleron Pharma, Inc. Method of promoting bone growth by an anti-activin B antibody
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
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
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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US6277375B1 (en) 1997-03-03 2001-08-21 Board Of Regents, The University Of Texas System Immunoglobulin-like domains with increased half-lives
PT1355919E (pt) 2000-12-12 2011-03-02 Medimmune Llc Moléculas com semivida longa, composições que as contêm e suas utilizações
MXPA04008149A (es) 2002-02-21 2005-06-17 Wyeth Corp Proteinas que contienen dominios de folistatina.
WO2005033134A2 (en) * 2003-09-30 2005-04-14 Regeneron Pharmaceuticals, Inc. Secreted protein therapeutics and uses thereof
JP4688483B2 (ja) * 2004-04-15 2011-05-25 株式会社テクノネットワーク四国 フォリスタチン変異体ポリペプチド
EP1896503B1 (en) 2005-05-31 2014-10-29 Board of Regents, The University of Texas System IgG1 ANTIBODIES WITH MUTATED Fc PORTION FOR INCREASED BINDING TO FcRn RECEPTOR AND USES TEHEREOF
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NZ590326A (en) * 2008-06-26 2013-05-31 Acceleron Pharma Inc Antagonists of activin-actriia and uses for increasing red blood cell levels
US20100008918A1 (en) 2008-06-26 2010-01-14 Acceleron Pharma Inc. Methods for dosing an actriib antagonist and monitoring of treated patients
CA2773494A1 (en) * 2009-09-09 2011-03-17 Acceleron Pharma Inc. Actriib antagonists and dosing and uses thereof
US9023890B2 (en) * 2009-12-02 2015-05-05 The Charlotte-Mecklenburg Hospital Authority Nitrate esters and their use for the treatment of muscle and muscle related diseases
US8580922B2 (en) 2011-03-04 2013-11-12 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
WO2013186719A1 (en) 2012-06-15 2013-12-19 Pfizer Inc. Improved antagonist antibodies against gdf-8 and uses therefor
LT3835310T (lt) 2012-09-13 2024-04-25 Bristol-Myers Squibb Company Fibronektino pagrindo karkaso domeno baltymai, kurie rišasi prie miostatino
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
WO2014187807A1 (en) 2013-05-21 2014-11-27 Arcarios B.V. Follistatin derivatives
WO2015187977A1 (en) 2014-06-04 2015-12-10 Acceleron Pharma, Inc. Methods and compositions for treatment of disorders with follistatin polypeptides
WO2016057975A2 (en) 2014-10-10 2016-04-14 Research Institute At Nationwide Children's Hospital Guided injections for aav gene transfer to muscle

Also Published As

Publication number Publication date
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
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
JP6475639B2 (ja) 2019-02-27
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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