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)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Neurosurgery (AREA)
  • Rheumatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (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
TW201627320A (zh) 2007-02-02 2016-08-01 艾瑟勒朗法瑪公司 衍生自ＡｃｔＲＩＩＢ的變體與其用途
EP2315602A4 (en) 2008-06-26 2011-11-02 Acceleron Pharma Inc METHOD OF DOSING AN ACTRIIB ANTAGONIST AND MONITORING 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
CA2898121A1 (en) * 2013-01-25 2014-07-31 Shire Human Genetic Therapies, Inc. Follistatin in treating duchenne muscular dystrophy
ES2786253T3 (es) 2013-03-14 2020-10-09 Immusoft Corp Métodos para la diferenciación y transducción in vitro de células b de memoria con vectores virales pseudotipados vsv-g
US10010498B2 (en) * 2014-06-04 2018-07-03 Acceleron Pharma Inc. Methods for treatment of amyotrophic lateral sclerosis with follistatin fusion proteins
JP6568110B2 (ja) 2014-06-04 2019-08-28 アクセルロン ファーマ， インコーポレイテッド フォリスタチンポリペプチドによる障害の処置のための方法および組成物
CA2980757A1 (en) * 2015-03-26 2016-09-29 Acceleron Pharma Inc. Follistatin-related fusion proteins and uses thereof
CN109152810A (zh) * 2016-01-06 2019-01-04 康德生物医疗技术公司 用于预防和治疗杜氏肌肉萎缩症的方法和组合物
BR112018067747A2 (pt) * 2016-03-04 2019-01-08 Shire Human Genetic Therapies proteínas de fusão recombinantes de folistatina-fc e uso no tratamento da distrofia muscular de duchenne
AU2018266893A1 (en) * 2017-05-12 2019-12-05 Takeda Pharmaceutical Company Limited 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
CA3093716A1 (en) * 2018-03-16 2019-09-19 Immusoft Corporation B cells genetically engineered to secrete follistatin and methods of using the same to treat follistatin-related diseases, conditions, disorders and to enhance muscle growth and strength
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
EP1355919B1 (en) 2000-12-12 2010-11-24 MedImmune, LLC Molecules with extended half-lives, compositions and uses thereof
PL374966A1 (en) 2002-02-21 2005-11-14 Wyeth Follistatin domain containing proteins
US7355018B2 (en) * 2003-09-30 2008-04-08 Regeneron Pharmaceuticals, Inc. Modified IGF1 polypeptides with increased stability and potency
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
WO2006130834A2 (en) 2005-05-31 2006-12-07 Board Of Regents, The University Of Texas System IGGl ANTIBODIES WITH MUTATED FC PORTION FOR INCREASED BINDING TO FCRN RECEPTOR AND USES THEREOF
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KR20110031951A (ko) * 2008-06-26 2011-03-29 악셀레론 파마 인코포레이티드 액티빈-ａｃｔｒｉｉａ의 길항물질 및 적혈구 수준을 증가시키기 위한 이들의 용도
EP3202459B1 (en) 2009-09-09 2021-04-14 Acceleron Pharma Inc. Actriib antagonists and dosing and uses thereof for treating obesity or type 2 diabetes by regulating body fat content
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
MY177331A (en) 2012-06-15 2020-09-12 Pfizer Improved antagonist antibodies against gdf-8 and uses therefor
RS58801B1 (sr) 2012-09-13 2019-07-31 Bristol Myers Squibb Co Skafold domenski proteini na bazi fibronektina koji se vezuju za miostatin
CA2898121A1 (en) * 2013-01-25 2014-07-31 Shire Human Genetic Therapies, Inc. Follistatin in treating duchenne muscular dystrophy
WO2014187807A1 (en) 2013-05-21 2014-11-27 Arcarios B.V. Follistatin derivatives
JP6568110B2 (ja) 2014-06-04 2019-08-28 アクセルロン ファーマ， インコーポレイテッド フォリスタチンポリペプチドによる障害の処置のための方法および組成物
US10842886B2 (en) 2014-10-10 2020-11-24 Research Institute At Nationwide Children's Hospital Guided injections for AAV gene transfer to muscle

Also Published As

Publication number Publication date
JP2019059730A (ja) 2019-04-18
HK1213804A1 (zh) 2016-07-15
CN104902915A (zh) 2015-09-09
ES2715710T3 (es) 2019-06-05
US10723772B2 (en) 2020-07-28
JP6475639B2 (ja) 2019-02-27
KR20150111347A (ko) 2015-10-05
MX2015009631A (es) 2015-11-30
JP2016511751A (ja) 2016-04-21
IL240024A0 (en) 2015-09-24
CN104902915B (zh) 2018-07-06
HK1217637A1 (en) 2017-01-20
IL240024B (en) 2020-05-31
WO2014116981A1 (en) 2014-07-31
MX362105B (es) 2019-01-04
US20180305421A1 (en) 2018-10-25
AU2019200797B2 (en) 2021-03-04
AU2014209178A1 (en) 2015-05-21
KR102253597B1 (ko) 2021-05-17
EP2948161A1 (en) 2015-12-02
US20160083440A1 (en) 2016-03-24
EP2948161B1 (en) 2018-12-12
AU2019200797A1 (en) 2019-02-21
US9957309B2 (en) 2018-05-01
CA2898121A1 (en) 2014-07-31
AU2014209178B2 (en) 2018-11-08

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