IL240024B - Polystatin in the treatment of Duchenne muscular dystrophy - Google Patents

Polystatin in the treatment of Duchenne muscular dystrophy

Info

Publication number
IL240024B
IL240024B IL240024A IL24002415A IL240024B IL 240024 B IL240024 B IL 240024B IL 240024 A IL240024 A IL 240024A IL 24002415 A IL24002415 A IL 24002415A IL 240024 B IL240024 B IL 240024B
Authority
IL
Israel
Prior art keywords
ducenne
follistatin
treating
muscular dystrophy
dystrophy
Prior art date
Application number
IL240024A
Other languages
English (en)
Hebrew (he)
Other versions
IL240024A0 (en
Original Assignee
Shire Human Genetic Therapies
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 Shire Human Genetic Therapies filed Critical Shire Human Genetic Therapies
Publication of IL240024A0 publication Critical patent/IL240024A0/en
Publication of IL240024B publication Critical patent/IL240024B/en

Links

Classifications

    • 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

Landscapes

  • 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)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
  • Rheumatology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
IL240024A 2013-01-25 2015-07-20 Polystatin in the treatment of Duchenne muscular dystrophy IL240024B (en)

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 (2)

Publication Number Publication Date
IL240024A0 IL240024A0 (en) 2015-09-24
IL240024B true IL240024B (en) 2020-05-31

Family

ID=50159515

Family Applications (1)

Application Number Title Priority Date Filing Date
IL240024A IL240024B (en) 2013-01-25 2015-07-20 Polystatin in the treatment of Duchenne muscular dystrophy

Country Status (12)

Country Link
US (2) US9957309B2 (OSRAM)
EP (1) EP2948161B1 (OSRAM)
JP (2) JP6475639B2 (OSRAM)
KR (1) KR102253597B1 (OSRAM)
CN (1) CN104902915B (OSRAM)
AU (2) AU2014209178B2 (OSRAM)
CA (1) CA2898121A1 (OSRAM)
EA (1) EA201590719A1 (OSRAM)
ES (1) ES2715710T3 (OSRAM)
IL (1) IL240024B (OSRAM)
MX (1) MX362105B (OSRAM)
WO (1) WO2014116981A1 (OSRAM)

Families Citing this family (18)

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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
TWI432449B (zh) 2007-02-02 2014-04-01 Acceleron Pharma Inc 衍生自ＡｃｔＲＩＩＢ的變體與其用途
KR20220002710A (ko) 2008-06-26 2022-01-06 악셀레론 파마 인코포레이티드 액티빈-ａｃｔｒｉｉａ 길항물질을 투약하는 방법 및 치료된 환자의 모니터링
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
AU2014209178B2 (en) * 2013-01-25 2018-11-08 Takeda Pharmaceutical Company Limited Follistatin in treating Duchenne muscular dystrophy
WO2014152832A1 (en) 2013-03-14 2014-09-25 Immusoft Corporation Methods for in vitro memory b cell differentiation and transduction with vsv-g pseudotyped viral vectors
US10010498B2 (en) 2014-06-04 2018-07-03 Acceleron Pharma Inc. Methods for treatment of amyotrophic lateral sclerosis with follistatin fusion proteins
KR102305109B1 (ko) * 2014-06-04 2021-09-27 악셀레론 파마 인코포레이티드 폴리스타틴 폴리펩티드를 이용한 장애의 치료방법 및 치료를 위한 조성물
CN107636154A (zh) * 2015-03-26 2018-01-26 阿塞勒隆制药公司 促滤泡素抑制素相关的融合蛋白及其用途
CN116196389A (zh) * 2016-01-06 2023-06-02 康德生物医疗有限公司 用于预防和治疗杜氏肌肉萎缩症的方法和组合物
EP3423479A2 (en) * 2016-03-04 2019-01-09 Shire Human Genetic Therapies, Inc. Recombinant follistatin-fc fusion proteins and use in treating duchenne muscular dystrophy
JP2020519291A (ja) * 2017-05-12 2020-07-02 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 組み換えフォリスタチン−ｆｃ融合タンパク質およびデュシェンヌ型筋ジストロフィーの治療における使用
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
WO2019178613A1 (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

Family Cites Families (18)

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Publication number Priority date Publication date Assignee Title
US6277375B1 (en) 1997-03-03 2001-08-21 Board Of Regents, The University Of Texas System Immunoglobulin-like domains with increased half-lives
DK1355919T3 (da) 2000-12-12 2011-03-14 Medimmune Llc Molekyler med længere halveringstider, sammensætninger og anvendelser deraf
CA2476654A1 (en) 2002-02-21 2003-09-04 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
US8163881B2 (en) 2005-05-31 2012-04-24 The Board Of Regents Of The University Of Texas System Immunoglobulin molecules with improved characteristics
WO2008030367A2 (en) * 2006-09-01 2008-03-13 The General Hospital Corporation Selective myostatin inhibitors
EP2318028B1 (en) * 2008-06-26 2020-02-19 Acceleron Pharma Inc. Antagonists of soluble activin-actriia and uses for increasing red blood cell levels
KR20220002710A (ko) * 2008-06-26 2022-01-06 악셀레론 파마 인코포레이티드 액티빈-ａｃｔｒｉｉａ 길항물질을 투약하는 방법 및 치료된 환자의 모니터링
BR112012005225B8 (pt) * 2009-09-09 2023-01-10 Acceleron Pharma Inc Uso de uma proteína de fusão actriib-fc para o tratamento de um transtorno relacionado ao osso ou associado à perda de músculo por crescimento muscular insuficiente
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
SA113340642B1 (ar) 2012-06-15 2015-09-15 فايزر إنك أجسام مضادة معارضة محسنة ضد gdf-8 واستخداماتها
RS65556B1 (sr) 2012-09-13 2024-06-28 Bristol Myers Squibb Co Proteini domena skele na bazi fibronektina koji vezuju miostatin
AU2014209178B2 (en) * 2013-01-25 2018-11-08 Takeda Pharmaceutical Company Limited Follistatin in treating Duchenne muscular dystrophy
WO2014187807A1 (en) 2013-05-21 2014-11-27 Arcarios B.V. Follistatin derivatives
KR102305109B1 (ko) 2014-06-04 2021-09-27 악셀레론 파마 인코포레이티드 폴리스타틴 폴리펩티드를 이용한 장애의 치료방법 및 치료를 위한 조성물
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
AU2014209178A1 (en) 2015-05-21
CA2898121A1 (en) 2014-07-31
CN104902915B (zh) 2018-07-06
JP6475639B2 (ja) 2019-02-27
EP2948161A1 (en) 2015-12-02
WO2014116981A1 (en) 2014-07-31
HK1217637A1 (en) 2017-01-20
AU2019200797A1 (en) 2019-02-21
JP2019059730A (ja) 2019-04-18
JP2016511751A (ja) 2016-04-21
AU2014209178B2 (en) 2018-11-08
MX362105B (es) 2019-01-04
MX2015009631A (es) 2015-11-30
ES2715710T3 (es) 2019-06-05
CN104902915A (zh) 2015-09-09
US20180305421A1 (en) 2018-10-25
US9957309B2 (en) 2018-05-01
US10723772B2 (en) 2020-07-28
KR20150111347A (ko) 2015-10-05
IL240024A0 (en) 2015-09-24
KR102253597B1 (ko) 2021-05-17
AU2019200797B2 (en) 2021-03-04
HK1213804A1 (zh) 2016-07-15
EA201590719A1 (ru) 2015-12-30
US20160083440A1 (en) 2016-03-24
EP2948161B1 (en) 2018-12-12

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