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    Lysosomal Alpha Glucosidase-Pipeline Review H1 2017

    Lysosomal Alpha Glucosidase-Pipeline Review H1 2017

    • Report Code ID: RW0001834344
    • Category Pharmaceuticals
    • No. of Pages 60
    • Publication Month May-17
    • Publisher Name Global Markets Direct
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Pipeline Review, H1 2017

    Summary

    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) pipeline Target constitutes close to 15 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes. The latest report Lysosomal Alpha Glucosidase - Pipeline Review, H1 2017, outlays comprehensive information on the Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA) , route of administration (RoA) and molecule type.

    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Lysosomal alpha-glucosidase is an enzyme encoded by the GAA gene. It is essential for the degradation of glygogen to glucose in lysosomes. Defects in this gene lead to glycogen storage disease II or Pompe disease. The molecules developed by companies in Phase III, Phase II, IND/CTA Filed, Preclinical and Discovery stages are 1, 1, 1, 7 and 2 respectively. Similarly, the universities portfolio in Phase II and Preclinical stages comprises 1 and 2 molecules, respectively. Report covers products from therapy areas Metabolic Disorders which include indications Pompe Disease.

    Furthermore, this report also reviews key players involved in Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics development with respective active and dormant or discontinued projects. Driven by data and information sourced from 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.

    Scope

    - The report provides a snapshot of the global therapeutic landscape for Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)
    - The report reviews Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources
    - The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
    - The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
    - The report reviews key players involved in Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics and enlists all their major and minor projects
    - The report assesses Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics based on mechanism of action (MoA) , route of administration (RoA) and molecule type
    - The report summarizes all the dormant and discontinued pipeline projects
    - The report reviews latest news and deals related to Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics

    Reasons to buy

    - Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
    - Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
    - Identify and understand the targeted therapy areas and indications for Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)
    - Identify the use of drugs for target identification and drug repurposing
    - Identify potential new clients or partners in the target demographic
    - Develop strategic initiatives by understanding the focus areas of leading companies
    - Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
    - Devise corrective measures for pipeline projects by understanding Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) development landscape
    - 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
    Table of Contents
    List of Tables
    List of Figures
    Introduction
    Publisher Report Coverage
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Overview
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Therapeutics Development
    Products under Development by Stage of Development
    Products under Development by Therapy Area
    Products under Development by Indication
    Products under Development by Companies
    Products under Development by Universities/Institutes
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Therapeutics Assessment
    Assessment by Mechanism of Action
    Assessment by Route of Administration
    Assessment by Molecule Type
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Companies Involved in Therapeutics Development
    Amicus Therapeutics Inc
    Audentes Therapeutics Inc
    Etubics Corp
    Genzyme Corp
    greenovation Biotech GmbH
    Huons Co Ltd
    JCR Pharmaceuticals Co Ltd
    Oxyrane Belgium NV
    Pharming Group NV
    Sarepta Therapeutics Inc
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drug Profiles
    Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    AT-982 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    ATB-200 + miglustat - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    GZ-402666 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    HU-023 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    JR-162 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    MOSS-GAA - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    OXY-2810 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    PGN-004 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Recombinant Alpha Glucosidase Replacement for Pompe Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    VAL-1221 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Dormant Products
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Discontinued Products
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Product Development Milestones
    Featured News & Press Releases
    May 15, 2017: Amicus Therapeutics Announces Positive Functional Data from Initial Patients in Pompe Phase 1/2 Study
    Mar 03, 2017: Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease
    Feb 15, 2017: Amicus Therapeutics Presents Important New Scientific Findings and Preclinical Data for Pompe Program at WORLDSymposium 2017
    Dec 08, 2016: Amicus Therapeutics Announces Positive Preliminary Data from Phase 1/2 Study of Novel Treatment Paradigm for Pompe Disease
    Nov 17, 2016: Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease to Begin in the UK
    Nov 04, 2016: Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease
    Mar 03, 2016: Sanofi Genzyme Presents Results from Phase 1/2 Study of Investigational Second-Generation Therapy for Pompe Disease
    Mar 01, 2016: Amicus Therapeutics Highlights Preclinical Pompe Data at WORLDSymposium 2016
    Feb 10, 2016: Amicus Therapeutics Announces Oral and Poster Presentations on Chaperone AT2221 at 12th Annual WORLDSymposium 2016
    Jan 11, 2016: Amicus Therapeutics Provides Update On Novel ERT for Pompe Disease (ATB200 + Chaperone)
    Dec 22, 2015: Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease
    Sep 03, 2015: Valerion Therapeutics Announces Positive Results Of Non-Clinical Research Study at SSIEM Annual Symposium
    May 11, 2015: Audentes Therapeutics To Present New Data On AT-002 at American Society of Gene and Cell Therapy Annual Meeting
    Feb 10, 2015: Amicus Therapeutics Presents Preclinical Data on Next-Generation Pompe ERT at WORLD Symposium 2015
    Appendix
    Methodology
    Coverage
    Secondary Research
    Primary Research
    Expert Panel Validation
    Contact Us
    Disclaimer

    List of Tables

    Number of Products under Development by Stage of Development, H1 2017
    Number of Products under Development by Therapy Areas, H1 2017
    Number of Products under Development by Indication, H1 2017
    Number of Products under Development by Companies, H1 2017
    Products under Development by Companies, H1 2017
    Number of Products under Investigation by Universities/Institutes, H1 2017
    Products under Investigation by Universities/Institutes, H1 2017
    Number of Products by Stage and Mechanism of Actions, H1 2017
    Number of Products by Stage and Route of Administration, H1 2017
    Number of Products by Stage and Molecule Type, H1 2017
    Pipeline by Amicus Therapeutics Inc, H1 2017
    Pipeline by Audentes Therapeutics Inc, H1 2017
    Pipeline by Etubics Corp, H1 2017
    Pipeline by Genzyme Corp, H1 2017
    Pipeline by greenovation Biotech GmbH, H1 2017
    Pipeline by Huons Co Ltd, H1 2017
    Pipeline by JCR Pharmaceuticals Co Ltd, H1 2017
    Pipeline by Oxyrane Belgium NV, H1 2017
    Pipeline by Pharming Group NV, H1 2017
    Pipeline by Sarepta Therapeutics Inc, H1 2017
    Dormant Projects, H1 2017
    Discontinued Products, H1 2017

    List of Figures

    Number of Products under Development by Stage of Development, H1 2017
    Number of Products by Mechanism of Actions, H1 2017
    Number of Products by Stage and 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
    Amicus Therapeutics Inc
    Audentes Therapeutics Inc
    Etubics Corp
    Genzyme Corp
    greenovation Biotech GmbH
    Huons Co Ltd
    JCR Pharmaceuticals Co Ltd
    Oxyrane Belgium NV
    Pharming Group NV
    Sarepta Therapeutics Inc

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