JP2019509735A - デュシェンヌ型筋ジストロフィー治療における組換えフォリスタチン−Ｆｃ融合タンパク質及び使用 - Google Patents

デュシェンヌ型筋ジストロフィー治療における組換えフォリスタチン−Ｆｃ融合タンパク質及び使用 Download PDF

Info

Publication number
JP2019509735A
JP2019509735A JP2018546471A JP2018546471A JP2019509735A JP 2019509735 A JP2019509735 A JP 2019509735A JP 2018546471 A JP2018546471 A JP 2018546471A JP 2018546471 A JP2018546471 A JP 2018546471A JP 2019509735 A JP2019509735 A JP 2019509735A
Authority
JP
Japan
Prior art keywords
seq
recombinant follistatin
fusion protein
amino acid
recombinant
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2018546471A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019509735A5 (enExample
Inventor
アンドレア イスケンデリアン，
アンドレア イスケンデリアン，
アンジェラ ダブリュー． ノートン，
アンジェラ ダブリュー． ノートン，
チュアン シェン，
チュアン シェン，
クラーク パン，
クラーク パン，
Original Assignee
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
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 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 filed Critical 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
Publication of JP2019509735A publication Critical patent/JP2019509735A/ja
Publication of JP2019509735A5 publication Critical patent/JP2019509735A5/ja
Pending legal-status Critical Current

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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22—Immunoglobulins [IG], 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)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Endocrinology (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)
JP2018546471A 2016-03-04 2017-03-03 デュシェンヌ型筋ジストロフィー治療における組換えフォリスタチン−Ｆｃ融合タンパク質及び使用 Pending JP2019509735A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662303954P 2016-03-04 2016-03-04
US62/303,954 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 (2)

Publication Number Publication Date
JP2019509735A true JP2019509735A (ja) 2019-04-11
JP2019509735A5 JP2019509735A5 (enExample) 2020-04-09

Family

ID=58358939

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2018546471A Pending JP2019509735A (ja) 2016-03-04 2017-03-03 デュシェンヌ型筋ジストロフィー治療における組換えフォリスタチン−Ｆｃ融合タンパク質及び使用

Country Status (14)

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

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018266893A1 (en) * 2017-05-12 2019-12-05 Takeda Pharmaceutical Company Limited 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
KR20250024825A (ko) 2022-06-15 2025-02-19 유씨비 바이오파마 에스알엘 폴리스타틴-fc 융합 단백질
CA3259309A1 (en) 2022-06-15 2023-12-21 UCB Biopharma SRL Fusion protein for the prevention, treatment or improvement of kidney disease

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005534616A (ja) * 2002-02-21 2005-11-17 ワイエス Ｇａｓｐ１；フォリスタチンドメイン含有タンパク質
WO2008030367A2 (en) * 2006-09-01 2008-03-13 The General Hospital Corporation Selective myostatin inhibitors
WO2014116981A1 (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
JP2015531748A (ja) * 2012-06-21 2015-11-05 インディアナ ユニヴァーシティ リサーチ アンド テクノロジー コーポレイション 改変ｆｃエフェクター機能を有するインクレチン受容体リガンドポリペプチドｆｃ領域融合ポリペプチド及び複合物
WO2015187977A1 (en) * 2014-06-04 2015-12-10 Acceleron Pharma, Inc. Methods and compositions for treatment of disorders with follistatin polypeptides
JP2016501025A (ja) * 2012-12-18 2016-01-18 ノバルティス アーゲー 安定化されたインスリン様成長因子ポリペプチド

Family Cites Families (5)

* 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
EP1355919B1 (en) 2000-12-12 2010-11-24 MedImmune, LLC Molecules with extended half-lives, compositions and uses thereof
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
US8580922B2 (en) 2011-03-04 2013-11-12 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
LT2691417T (lt) * 2011-03-29 2018-10-25 Roche Glycart Ag Antikūno fc variantai

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005534616A (ja) * 2002-02-21 2005-11-17 ワイエス Ｇａｓｐ１；フォリスタチンドメイン含有タンパク質
WO2008030367A2 (en) * 2006-09-01 2008-03-13 The General Hospital Corporation Selective myostatin inhibitors
JP2015531748A (ja) * 2012-06-21 2015-11-05 インディアナ ユニヴァーシティ リサーチ アンド テクノロジー コーポレイション 改変ｆｃエフェクター機能を有するインクレチン受容体リガンドポリペプチドｆｃ領域融合ポリペプチド及び複合物
JP2016501025A (ja) * 2012-12-18 2016-01-18 ノバルティス アーゲー 安定化されたインスリン様成長因子ポリペプチド
WO2014116981A1 (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
WO2015187977A1 (en) * 2014-06-04 2015-12-10 Acceleron Pharma, Inc. Methods and compositions for treatment of disorders with follistatin polypeptides

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BIOCHEM. BIOPHYS. RES. COMMUN., 1995, VOL.208, NO.1, PP.1-9, JPN6021014154, ISSN: 0004635799 *
DRUG METAB. DISPOS., 2015, VOL.43, PP.1882-1890, JPN6021014156, ISSN: 0004491256 *
ENDOCRINOLOGY, 2005, VOL.146, PP.130-136, JPN7021001307, ISSN: 0004635798 *
J. PHARMACOL. EXP. THER., 2013, VOL.344, PP.616-623, JPN7021001308, ISSN: 0004635800 *

Also Published As

Publication number Publication date
EP3423479A2 (en) 2019-01-09
KR20180137487A (ko) 2018-12-27
IL261397A (en) 2018-10-31
EA201891990A1 (ru) 2019-04-30
WO2017152090A3 (en) 2017-10-19
US20200247856A1 (en) 2020-08-06
MX2018010470A (es) 2018-12-06
CA3016576A1 (en) 2017-09-08
CN109153708A (zh) 2019-01-04
AU2017228475A1 (en) 2018-09-13
SG11201807400TA (en) 2018-09-27
MA43588A (fr) 2021-06-02
BR112018067747A2 (pt) 2019-01-08
WO2017152090A2 (en) 2017-09-08

Similar Documents

Publication Publication Date Title
JP6475639B2 (ja) 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
JP6469022B2 (ja) デュシェンヌ型筋ジストロフィーの治療における抗−ｆｌｔ−１抗体
US20200247856A1 (en) Recombinant follistatin-fc fusion proteins and use in treating duchenne muscular dystrophy
JP2020519291A (ja) 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
JP2018518153A (ja) デュシェンヌ型筋ジストロフィーの治療における抗ｆｌｔ−１抗体
WO2023057404A1 (en) Novel combined administration
US10729746B2 (en) Recombinant placenta growth factor for treating Duchenne muscular dystrophy
HK1213804B (en) Follistatin in treating duchenne muscular dystrophy
HK1217637B (en) Follistatin in treating duchenne muscular dystrophy

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200302

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200302

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210421

A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20210624

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20210720

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20211110