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ゴーシェ病 : パイプライン分析

Gaucher's Disease - Pipeline Review, H2 2015

発行 Global Markets Direct 商品コード 200948
出版日 ページ情報 英文 86 Pages
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ゴーシェ病 : パイプライン分析 Gaucher's Disease - Pipeline Review, H2 2015
出版日: 2015年08月31日 ページ情報: 英文 86 Pages
概要

ゴーシェ病は脂質貯蔵に関する疾患で、マクロファージ単球系内部でのグルコセレブロシドの堆積という特徴があります。この疾患は、リソソーム加水分解酵素の一種であるグルコセレブロシダーゼ(酸性βグルコシダーゼやグルコシルセラミダーゼとも呼ばれる)の欠乏によって生じます。また、数多くの表現型が存在するという特徴もあります。その重症度は非常に幅広く、幼児期の内に合併症のほぼ全てを発症する患者がいる一方で、無症候状態のまま80歳代を迎える患者もいます。

当レポートでは、世界各国でのゴーシェ病治療用のパイプライン製品の開発状況について分析し、パイプライン製品の最新動向や、治験の各段階にある製品の一覧、主要企業のプロファイル、主な薬剤の概要(製品概要、機能メカニズム、研究開発(R&D)の進展状況)、最新の業界動向などを調査して、その結果を概略以下の構成でお届けします。

イントロダクション

  • 分析範囲

ゴーシェ病の概要

治療薬の開発

  • ゴーシェ病向けパイプライン製品:概要
  • ゴーシェ病向けパイプライン製品:比較分析

各企業で開発中のゴーシェ病治療薬

大学/研究機関で研究中のゴーシェ病治療薬

パイプライン製品の概略

  • 後期段階の製品
  • 治験段階の製品
  • 初期段階の製品

ゴーシェ病治療薬:開発中の製品の一覧(企業別)

ゴーシェ病治療薬:研究中の製品の一覧(大学/研究機関別)

ゴーシェ病治療薬の開発に従事している企業

  • Actelion Ltd
  • Amicus Therapeutics, Inc.
  • Bioorganic Research and Services S.A.
  • Biosidus S.A.
  • Dong-A Socio Group
  • Genzyme Corporation
  • greenovation Biotech GmbH
  • ISU ABXIS Co.,Ltd.
  • JCR Pharmaceuticals Co., Ltd.
  • Lixte Biotechnology Holdings, Inc.
  • Neuraltus Pharmaceuticals, Inc.
  • Okklo Life Sciences BV
  • Pharming Group N.V.
  • Protalix BioTherapeutics, Inc.
  • The International Biotechnology Center (IBC) Generium
  • UAB Profarma

ゴーシェ病:治療薬の評価

  • 単剤製品
  • 併用製品
  • 標的別
  • 機能メカニズム別
  • 投与方法別
  • 分子の種類別
  • 薬効分類別

薬剤のプロファイル

  • afegostat tartrate + ERT
  • ambroxol
  • AT-3375
  • BNT-001
  • グルコセレブロシダーゼ
  • GZ-402671
  • imigluceraseのバイオシミラー
  • LB-201
  • LB-205
  • miglustat
  • NP-003
  • OKL-1014
  • PRX-112
  • Recombinant Enzyme to Replace Beta Glucosidase 2 for Gaucher's Disease and Male Infertility
  • Recombinant Enzyme to Replace Beta-Cerebrosidase for Gaucher's Disease
  • Small Molecule to Activate Beta-Galactosidase for GM1 Gangliosidosis and Gaucher's Disease
  • Small Molecule to Activate Glucocerebrosidase for Parkinson's, Gaucher's and Neurodegenerative Diseases
  • Small Molecules to Activate Glucocerebrosidase for Gaucher and Parkinson Disease
  • Small Molecules to Inhibit Alpha-glucosidase for Diabetes and Gaucher's Disease
  • taliglucerase alfa

ゴーシェ病治療薬:最新の薬剤プロファイル

ゴーシェ病治療薬:開発が休止状態の製品

ゴーシェ病治療薬:開発が中止された製品

ゴーシェ病関連製品の開発のマイルストーン

  • 注目すべき最新動向・プレスリリース

付録

図表一覧

このページに掲載されている内容は最新版と異なる場合があります。詳細はお問い合わせください。

目次
Product Code: GMDHC6989IDB

Summary

Global Markets Direct's, 'Gaucher's Disease - Pipeline Review, H2 2015', provides an overview of the Gaucher's Disease's therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Gaucher's Disease, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Gaucher's Disease and special features on late-stage and discontinued projects.

Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct's proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

  • The report provides a snapshot of the global therapeutic landscape of Gaucher's Disease
  • The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities
  • The report reviews key players involved in the therapeutics development for Gaucher's Disease and enlists all their major and minor projects
  • The report summarizes all the dormant and discontinued pipeline projects
  • A review of the Gaucher's Disease products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
  • Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • A detailed assessment of monotherapy and combination therapy pipeline projects
  • Coverage of the Gaucher's Disease pipeline on the basis of target, MoA, route of administration and molecule type
  • Latest news and deals relating related to pipeline products

Reasons to buy

  • Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
  • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Identify and understand important and diverse types of therapeutics under development for Gaucher's Disease
  • Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
  • Devise corrective measures for pipeline projects by understanding Gaucher's Disease pipeline depth and focus of Indication therapeutics
  • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope
  • Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Table of Contents

  • Table of Contents
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Gaucher's Disease Overview
  • Therapeutics Development
    • Pipeline Products for Gaucher's Disease - Overview
    • Pipeline Products for Gaucher's Disease - Comparative Analysis
  • Gaucher's Disease - Therapeutics under Development by Companies
  • Gaucher's Disease - Therapeutics under Investigation by Universities/Institutes
  • Gaucher's Disease - Pipeline Products Glance
    • Late Stage Products
    • Clinical Stage Products
    • Early Stage Products
  • Gaucher's Disease - Products under Development by Companies
  • Gaucher's Disease - Products under Investigation by Universities/Institutes
  • Gaucher's Disease - Companies Involved in Therapeutics Development
    • Actelion Ltd
    • Amicus Therapeutics, Inc.
    • Bioorganic Research and Services S.A.
    • Biosidus S.A.
    • Dong-A Socio Group
    • Genzyme Corporation
    • greenovation Biotech GmbH
    • ISU ABXIS Co.,Ltd.
    • JCR Pharmaceuticals Co., Ltd.
    • Lixte Biotechnology Holdings, Inc.
    • Neuraltus Pharmaceuticals, Inc.
    • Okklo Life Sciences BV
    • Pharming Group N.V.
    • Protalix BioTherapeutics, Inc.
    • The International Biotechnology Center (IBC) Generium
    • UAB Profarma
  • Gaucher's Disease - Therapeutics Assessment
    • Assessment by Monotherapy Products
    • Assessment by Combination Products
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Drug Profiles
    • afegostat tartrate + ERT - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • ambroxol - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • AT-3375 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • BNT-001 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Gluco-Cerebosidase - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • GZ-402671 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • imiglucerase biosimilar - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • imiglucerase biosimilar - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • imiglucerase biosimilar - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • imiglucerase biosimilar - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • imiglucerase biosimilar - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • LB-201 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • LB-205 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • miglustat - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • NP-003 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • OKL-1014 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • PRX-112 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Recombinant Enzyme to Replace Beta Glucosidase 2 for Gaucher's Disease and Male Infertility - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Recombinant Enzyme to Replace Beta-Cerebrosidase for Gaucher's Disease - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Small Molecule to Activate Beta-Galactosidase for GM1 Gangliosidosis and Gaucher's disease - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Small Molecule to Activate Glucocerebrosidase for Parkinson's, Gaucher's and Neurodegenerative Diseases - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Small Molecules to Activate Glucocerebrosidase for Gaucher and Parkinson Disease - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Small Molecules to Inhibit Alpha-glucosidase for Diabetes and Gaucher's Disease - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • taliglucerase alfa - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
  • Gaucher's Disease - Recent Pipeline Updates
  • Gaucher's Disease - Dormant Projects
  • Gaucher's Disease - Discontinued Products
  • Gaucher's Disease - Product Development Milestones
    • Featured News & Press Releases
      • Sep 02, 2014: Shire's VPRIV400 units/vial launched in Japan for the improvement of symptoms of Gaucher disease
      • Jul 04, 2014: Shire's VPRIV approved in Japan for the improvement of symptoms of Gaucher disease
      • Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study With PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease
      • Mar 14, 2014: Shire Pharmaceuticals Initiates Voluntary Nationwide Recall Of One Batch, Packaged into Three Lots, of VPRIV (velaglucerase alfa for injection)
      • Feb 14, 2014: Shire Announces FDA Approval of Manufacturing Facility for VPRIV (Velaglucerase Alfa for Injection) Drug Substance
      • Feb 12, 2014: Protalix BioTherapeutics Announces Oral GCD Data to be Presented at WORLD Symposium 2014
      • Oct 14, 2013: Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for Oral GCD in Gaucher Disease Patients
      • Apr 02, 2013: Protalix BioTherapeutics Treats First Gaucher Patient In Phase I Study With PRX-112
      • Mar 07, 2013: Protalix BioTherapeutics Receives Approval From Israeli Ministry Of Health To Initiate Phase I Clinical Trial Of PRX-112 For Treatment Of Gaucher Disease
      • Jun 28, 2012: Shire's Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Gaucher's Disease, H2 2015
  • Number of Products under Development for Gaucher's Disease - Comparative Analysis, H2 2015
  • Number of Products under Development by Companies, H2 2015
  • Number of Products under Investigation by Universities/Institutes, H2 2015
  • Comparative Analysis by Late Stage Development, H2 2015
  • Comparative Analysis by Clinical Stage Development, H2 2015
  • Comparative Analysis by Early Stage Development, H2 2015
  • Products under Development by Companies, H2 2015
  • Products under Development by Companies, H2 2015 (Contd..1)
  • Products under Investigation by Universities/Institutes, H2 2015
  • Gaucher's Disease - Pipeline by Actelion Ltd, H2 2015
  • Gaucher's Disease - Pipeline by Amicus Therapeutics, Inc., H2 2015
  • Gaucher's Disease - Pipeline by Bioorganic Research and Services S.A., H2 2015
  • Gaucher's Disease - Pipeline by Biosidus S.A., H2 2015
  • Gaucher's Disease - Pipeline by Dong-A Socio Group, H2 2015
  • Gaucher's Disease - Pipeline by Genzyme Corporation, H2 2015
  • Gaucher's Disease - Pipeline by greenovation Biotech GmbH, H2 2015
  • Gaucher's Disease - Pipeline by ISU ABXIS Co.,Ltd., H2 2015
  • Gaucher's Disease - Pipeline by JCR Pharmaceuticals Co., Ltd., H2 2015
  • Gaucher's Disease - Pipeline by Lixte Biotechnology Holdings, Inc., H2 2015
  • Gaucher's Disease - Pipeline by Neuraltus Pharmaceuticals, Inc., H2 2015
  • Gaucher's Disease - Pipeline by Okklo Life Sciences BV, H2 2015
  • Gaucher's Disease - Pipeline by Pharming Group N.V., H2 2015
  • Gaucher's Disease - Pipeline by Protalix BioTherapeutics, Inc., H2 2015
  • Gaucher's Disease - Pipeline by The International Biotechnology Center (IBC) Generium, H2 2015
  • Gaucher's Disease - Pipeline by UAB Profarma, H2 2015
  • Assessment by Monotherapy Products, H2 2015
  • Assessment by Combination Products, H2 2015
  • Number of Products by Stage and Target, H2 2015
  • Number of Products by Stage and Mechanism of Action, H2 2015
  • Number of Products by Stage and Route of Administration, H2 2015
  • Number of Products by Stage and Molecule Type, H2 2015
  • Gaucher's Disease Therapeutics - Recent Pipeline Updates, H2 2015
  • Gaucher's Disease - Dormant Projects, H2 2015
  • Gaucher's Disease - Discontinued Products, H2 2015

List of Figures

  • Number of Products under Development for Gaucher's Disease, H2 2015
  • Number of Products under Development for Gaucher's Disease - Comparative Analysis, H2 2015
  • Number of Products under Development by Companies, H2 2015
  • Number of Products under Investigation by Universities/Institutes, H2 2015
  • Comparative Analysis by Clinical Stage Development, H2 2015
  • Comparative Analysis by Early Stage Products, H2 2015
  • Assessment by Monotherapy Products, H2 2015
  • Number of Products by Top 10 Targets, H2 2015
  • Number of Products by Stage and Top 10 Targets, H2 2015
  • Number of Products by Top 10 Mechanism of Actions, H2 2015
  • Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015
  • Number of Products by Top 10 Routes of Administration, H2 2015
  • Number of Products by Stage and Top 10 Routes of Administration, H2 2015
  • Number of Products by Top 10 Molecule Types, H2 2015
  • Number of Products by Stage and Top 10 Molecule Types, H2 2015
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