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    Gaucher Disease-Pipeline Review H1 2017

    Gaucher Disease-Pipeline Review H1 2017

    • Report Code ID: RW0001834393
    • Category Pharmaceuticals
    • No. of Pages 74
    • Publication Month Jun-17
    • Publisher Name Global Markets Direct
    Gaucher Disease - Pipeline Review, H1 2017

    Summary

    Publisher'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

    Publisher'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 Publisher'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
    List of Tables
    List of Figures
    Introduction
    Publisher 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
    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

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