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市場調查報告書

高雪氏症:開發中產品分析

Gaucher Disease - Pipeline Review, H1 2017

出版商 Global Markets Direct 商品編碼 363112
出版日期 內容資訊 英文 74 Pages
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高雪氏症:開發中產品分析 Gaucher Disease - Pipeline Review, H1 2017
出版日期: 2017年06月13日 內容資訊: 英文 74 Pages
簡介

本報告提供高雪氏症治療藥的開發情形相關資訊,提供您各開發階段,藥物標的,作用機制,給藥途徑及分子類型別分析,開發治療藥的企業概要,最新消息和新聞稿等資訊。

簡介

  • 調查範圍

高雪氏症概要

治療藥的開發

  • 開發中產品的概要
  • 開發中產品比較分析

高雪氏症:企業開發中的治療藥

高雪氏症:大學/機關研究中的治療藥

高雪氏症:開發中產品概況

  • 臨床階段的產品
  • 初期階段的產品

高雪氏症:企業開發中的產品

高雪氏症:大學/機關研究中的產品

高雪氏症治療藥的開發企業

  • Actelion Ltd
  • Bioorganic Research and Services S.A.
  • Genzyme Corporation
  • greenovation Biotech GmbH
  • JCR Pharmaceuticals Co., Ltd.
  • Lixte Biotechnology Holdings, Inc.
  • Neuraltus Pharmaceuticals, Inc.
  • Okklo Life Sciences BV
  • Pharming Group N.V.
  • Protalix BioTherapeutics, Inc.
  • Sangamo BioSciences, Inc.
  • Shire Plc
  • The International Biotechnology Center (IBC) Generium

高雪氏症:治療藥的評估

  • 單劑產品
  • 標的別
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

藥物簡介

高雪氏症:暫停中的計劃

高雪氏症:中止開發的產品

高雪氏症:產品開發的里程碑

  • 主要消息和新聞稿

附錄

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目錄
Product Code: GMDHC9429IDB

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Gaucher Disease - Pipeline Review, H1 2017, provides an overview of the Gaucher Disease (Genetic Disorders) pipeline landscape.

Gaucher disease is a rare genetic disorder occurs due to lacks an enzyme called glucocerebrosidase. Symptoms include cognitive impairment, enlarged spleen, severe swelling, lung disease, fatigue and seizures. Treatment includes enzyme replacement therapy.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Gaucher Disease - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Gaucher Disease (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Gaucher Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Gaucher Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, Phase I, Preclinical and Discovery stages are 4, 2, 11 and 2 respectively. Similarly, the Universities portfolio in Phase I, Preclinical and Discovery stages comprises 1, 1 and 5 molecules, respectively.

Gaucher Disease (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Gaucher Disease (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Gaucher Disease (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Gaucher Disease (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Gaucher Disease (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews latest news related to pipeline therapeutics for Gaucher Disease (Genetic Disorders)

Reasons to buy

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Gaucher Disease (Genetic Disorders).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Gaucher Disease (Genetic Disorders) 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.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Table of Contents

  • Table of Contents
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Gaucher Disease - Overview
    • Gaucher Disease - Therapeutics Development
    • Pipeline Overview
    • Pipeline by Companies
    • Pipeline by Universities/Institutes
    • Products under Development by Companies
    • Products under Development by Universities/Institutes
  • Gaucher Disease - Therapeutics Assessment
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Gaucher Disease - Companies Involved in Therapeutics Development
    • Actelion Ltd
    • Genzyme Corp
    • greenovation Biotech GmbH
    • JCR Pharmaceuticals Co Ltd
    • Lixte Biotechnology Holdings Inc
    • Neuraltus Pharmaceuticals Inc
    • Orphazyme ApS
    • Pharming Group NV
    • Protalix BioTherapeutics Inc
    • Shire Plc
  • Gaucher Disease - Drug Profiles
    • ADN-LYS - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ambroxol - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • arimoclomol - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • AVRRD-02 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy for Gaucher Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy for Gaucher Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ibiglustat - 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
    • LTI-291 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • miglustat - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • MOSS-GBA - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • NCGC-607 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • NP-003 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Pcgin - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • PRX-112 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Beta Glucosidase 2 Replacement for Gaucher's Disease and Male Infertility - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Beta-Cerebrosidase Replacement for Gaucher's Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Protein for Type I and Type III Gaucher's Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RND-001 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecule to Activate Beta Galactosidase for Gaucher Disease and GM1 Gangliosidosis - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Inhibit Glucocerebrosidase for Gaucher Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Inhibit 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
    • velaglucerase alfa - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Gaucher Disease - Dormant Projects
  • Gaucher Disease - Discontinued Products
  • Gaucher Disease - Product Development Milestones
    • Featured News & Press Releases
      • Mar 22, 2017: AVROBIO Expands Rare Disease Pipeline with Gene Therapy to Treat Gaucher Disease
      • Oct 25, 2016: Asthma research unexpectedly yields new treatment approach for inherited enzyme disease
      • 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
      • Jun 28, 2012: Shire's Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease
      • Feb 22, 2012: Shire Announces European Approval Of Manufacturing Facility For VPRIV
      • Dec 19, 2011: Lixte's LB-205 Shows Therapeutic Potential For Gaucher Disease
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
  • Disclaimer

List of Tables

  • Number of Products under Development for Gaucher Disease, H1 2017
  • Number of Products under Development by Companies, H1 2017
  • Number of Products under Development by Universities/Institutes, H1 2017
  • Products under Development by Companies, H1 2017
  • Products under Development by Universities/Institutes, H1 2017
  • Number of Products by Stage and Target, H1 2017
  • Number of Products by Stage and Mechanism of Action, H1 2017
  • Number of Products by Stage and Route of Administration, H1 2017
  • Number of Products by Stage and Molecule Type, H1 2017
  • Gaucher Disease - Pipeline by Actelion Ltd, H1 2017
  • Gaucher Disease - Pipeline by Genzyme Corp, H1 2017
  • Gaucher Disease - Pipeline by greenovation Biotech GmbH, H1 2017
  • Gaucher Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H1 2017
  • Gaucher Disease - Pipeline by Lixte Biotechnology Holdings Inc, H1 2017
  • Gaucher Disease - Pipeline by Neuraltus Pharmaceuticals Inc, H1 2017
  • Gaucher Disease - Pipeline by Orphazyme ApS, H1 2017
  • Gaucher Disease - Pipeline by Pharming Group NV, H1 2017
  • Gaucher Disease - Pipeline by Protalix BioTherapeutics Inc, H1 2017
  • Gaucher Disease - Pipeline by Shire Plc, H1 2017
  • Gaucher Disease - Dormant Projects, H1 2017
  • Gaucher Disease - Discontinued Products, H1 2017

List of Figures

  • Number of Products under Development for Gaucher Disease, H1 2017
  • Number of Products under Development by Companies, H1 2017
  • Number of Products under Development by Universities/Institutes, H1 2017
  • Number of Products by Targets, H1 2017
  • Number of Products by Stage and Targets, H1 2017
  • Number of Products by Top 10 Mechanism of Actions, H1 2017
  • Number of Products by Stage and Top 10 Mechanism of Actions, H1 2017
  • Number of Products by Routes of Administration, H1 2017
  • Number of Products by Stage and Routes of Administration, H1 2017
  • Number of Products by Molecule Types, H1 2017
  • Number of Products by Stage and Molecule Types, H1 2017
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