BR112018067747A2 - proteínas de fusão recombinantes de folistatina-fc e uso no tratamento da distrofia muscular de duchenne - Google Patents

proteínas de fusão recombinantes de folistatina-fc e uso no tratamento da distrofia muscular de duchenne

Info

Publication number
BR112018067747A2
BR112018067747A2 BR112018067747A BR112018067747A BR112018067747A2 BR 112018067747 A2 BR112018067747 A2 BR 112018067747A2 BR 112018067747 A BR112018067747 A BR 112018067747A BR 112018067747 A BR112018067747 A BR 112018067747A BR 112018067747 A2 BR112018067747 A2 BR 112018067747A2
Authority
BR
Brazil
Prior art keywords
muscular dystrophy
folistatin
duchenne muscular
treatment
fusion proteins
Prior art date
Application number
BR112018067747A
Other languages
English (en)
Inventor
Iskenderian Andrea
W Norton Angela
Shen Chuan
Pan Clark
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 BR112018067747A2 publication Critical patent/BR112018067747A2/pt

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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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/473—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used alpha-Glycoproteins
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • 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
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22—Immunoglobulins [IGs], 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
    • 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
    • A61K2039/545—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
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
    • C07K2317/92—Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
    • 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)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Toxicology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Endocrinology (AREA)
  • Microbiology (AREA)
  • Mycology (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Prostheses (AREA)

Abstract

a presente invenção fornece, dentre outras coisas, métodos e composições para o tratamento da distrofia muscular, em particular, a distrofia muscular de duchenne (dmd). em algumas modalidades, um método de acordo com a presente invenção inclui a administração, a um indivíduo que sofre ou que esteja suscetível à dmd, de uma quantidade eficaz de uma proteína de fusão de folistatina recombinante de modo que pelo menos um sintoma ou característica da dmd tenha sua intensidade, gravidade, ou frequência reduzida, ou seu início retardado.
BR112018067747A 2016-03-04 2017-03-03 proteínas de fusão recombinantes de folistatina-fc e uso no tratamento da distrofia muscular de duchenne BR112018067747A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662303954P 2016-03-04 2016-03-04
PCT/US2017/020723 WO2017152090A2 (en) 2016-03-04 2017-03-03 Recombinant follistatin-fc fusion proteins and use in treating duchenne muscular dystrophy

Publications (1)

Publication Number Publication Date
BR112018067747A2 true BR112018067747A2 (pt) 2019-01-08

Family

ID=58358939

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018067747A BR112018067747A2 (pt) 2016-03-04 2017-03-03 proteínas de fusão recombinantes de folistatina-fc e uso no tratamento da distrofia muscular de duchenne

Country Status (14)

Country Link
US (1) US20200247856A1 (pt)
EP (1) EP3423479A2 (pt)
JP (1) JP2019509735A (pt)
KR (1) KR20180137487A (pt)
CN (1) CN109153708A (pt)
AU (1) AU2017228475A1 (pt)
BR (1) BR112018067747A2 (pt)
CA (1) CA3016576A1 (pt)
EA (1) EA201891990A1 (pt)
IL (1) IL261397A (pt)
MA (1) MA43588A (pt)
MX (1) MX2018010470A (pt)
SG (1) SG11201807400TA (pt)
WO (1) WO2017152090A2 (pt)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3621986A1 (en) * 2017-05-12 2020-03-18 Shire Human Genetic Therapies, Inc. Recombinant follistatin-fc fusion proteins and use in treating duchenne muscular dystrophy
TW201936201A (zh) 2017-12-14 2019-09-16 美商堅固生物科技公司 基因之非病毒生產及遞送
CN108303860B (zh) * 2018-03-26 2019-11-08 西安电子科技大学 一种用于无掩模光刻扫描的分布式曝光方法
WO2019191204A1 (en) * 2018-03-28 2019-10-03 Acceleron Pharma Inc. Follistatin polypeptides for the treatment of muscle contracture
KR20210121137A (ko) 2019-01-29 2021-10-07 샤이어-엔피에스 파마슈티칼즈, 인크. 부갑상선 호르몬 변이체
WO2023242271A1 (en) 2022-06-15 2023-12-21 UCB Biopharma SRL Fusion protein for the prevention, treatment or amelioration of kidney diseases
TW202409093A (zh) 2022-06-15 2024-03-01 比利時商Ｕｃｂ生物製藥公司 融合蛋白

Family Cites Families (12)

* Cited by examiner, † Cited by third party
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
DE60143544D1 (de) 2000-12-12 2011-01-05 Medimmune Llc Moleküle mit längeren halbwertszeiten, zusammensetzungen und deren verwendung
MXPA04008150A (es) * 2002-02-21 2005-06-17 Wyeth Corp Gasp1: una proteina que contiene dominio de folistatina.
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
US8580922B2 (en) 2011-03-04 2013-11-12 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
ES2692268T3 (es) * 2011-03-29 2018-12-03 Roche Glycart Ag Variantes de Fc de anticuerpo
CN104582736A (zh) * 2012-06-21 2015-04-29 印第安纳大学研究及科技有限公司 Fc效应子功能改变的肠降血糖素受体配体多肽Fc区融合多肽和缀合物
PE20151612A1 (es) * 2012-12-18 2015-11-19 Novartis Ag Polipeptidos estabilizados del factor de crecimiento tipo insulina
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
KR20210119546A (ko) * 2014-06-04 2021-10-05 악셀레론 파마 인코포레이티드 폴리스타틴 폴리펩티드를 이용한 장애의 치료방법 및 치료를 위한 조성물

Also Published As

Publication number Publication date
SG11201807400TA (en) 2018-09-27
AU2017228475A1 (en) 2018-09-13
WO2017152090A2 (en) 2017-09-08
MA43588A (fr) 2021-06-02
EP3423479A2 (en) 2019-01-09
IL261397A (en) 2018-10-31
CN109153708A (zh) 2019-01-04
US20200247856A1 (en) 2020-08-06
CA3016576A1 (en) 2017-09-08
KR20180137487A (ko) 2018-12-27
JP2019509735A (ja) 2019-04-11
WO2017152090A3 (en) 2017-10-19
EA201891990A1 (ru) 2019-04-30
MX2018010470A (es) 2018-12-06

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Legal Events

Date Code Title Description
B11A Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing
B11Y Definitive dismissal - extension of time limit for request of examination expired [chapter 11.1.1 patent gazette]
B350 Update of information on the portal [chapter 15.35 patent gazette]