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    Lysosomal Alpha Glucosidase-Pipeline Review H2 2017

    Lysosomal Alpha Glucosidase-Pipeline Review H2 2017

    • Report Code ID: RW00011220235
    • Category Life Sciences
    • No. of Pages 62
    • Publication Month Oct-17
    • Publisher Name Global Markets Direct
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)-Pipeline Review, H2 2017

    Summary

    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.

    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 molecules developed by companies in Phase III, Phase II, IND/CTA Filed, Preclinical and Discovery stages are 1, 2, 1, 7 and 1 respectively. Similarly, the universities portfolio in Phase II, Phase I and Preclinical stages comprises 1, 1 and 1 molecules, respectively. Report covers products from therapy areas Metabolic Disorders which include indications Pompe Disease.

    The latest report Lysosomal Alpha Glucosidase-Pipeline Review, H2 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. It 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.

    The report is built using 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.

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

    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
    Table of Contents 2
    List of Tables 5
    List of Figures 5
    Introduction 6
    Publisher Report Coverage 6
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)-Overview 7
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)-Therapeutics Development 8
    Products under Development by Stage of Development 8
    Products under Development by Therapy Area 9
    Products under Development by Indication 10
    Products under Development by Companies 11
    Products under Development by Universities/Institutes 13
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)-Therapeutics Assessment 15
    Assessment by Mechanism of Action 15
    Assessment by Route of Administration 17
    Assessment by Molecule Type 19
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)-Companies Involved in Therapeutics Development 21
    Amicus Therapeutics Inc 21
    Audentes Therapeutics Inc 21
    Etubics Corp 22
    Genzyme Corp 22
    greenovation Biotech GmbH 22
    JCR Pharmaceuticals Co Ltd 23
    NanoMedSyn SAS 23
    Oxyrane Belgium NV 23
    Pharming Group NV 24
    Sarepta Therapeutics Inc 24
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)-Drug Profiles 26
    Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease-Drug Profile 26
    Product Description 26
    Mechanism Of Action 26
    R&D Progress 26
    AT-982-Drug Profile 27
    Product Description 27
    Mechanism Of Action 27
    R&D Progress 27
    ATB-200 + miglustat-Drug Profile 29
    Product Description 29
    Mechanism Of Action 29
    R&D Progress 29
    AVRRD-03-Drug Profile 32
    Product Description 32
    Mechanism Of Action 32
    R&D Progress 32
    Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease-Drug Profile 33
    Product Description 33
    Mechanism Of Action 33
    R&D Progress 33
    Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease-Drug Profile 34
    Product Description 34
    Mechanism Of Action 34
    R&D Progress 34
    Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease-Drug Profile 35
    Product Description 35
    Mechanism Of Action 35
    R&D Progress 35
    Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease-Drug Profile 36
    Product Description 36
    Mechanism Of Action 36
    R&D Progress 36
    GZ-402666-Drug Profile 37
    Product Description 37
    Mechanism Of Action 37
    R&D Progress 37
    JR-162-Drug Profile 39
    Product Description 39
    Mechanism Of Action 39
    R&D Progress 39
    MOSS-GAA-Drug Profile 40
    Product Description 40
    Mechanism Of Action 40
    R&D Progress 40
    OXY-2810-Drug Profile 41
    Product Description 41
    Mechanism Of Action 41
    R&D Progress 41
    PGN-004-Drug Profile 42
    Product Description 42
    Mechanism Of Action 42
    R&D Progress 42
    Recombinant Alpha Glucosidase Replacement for Pompe Disease-Drug Profile 43
    Product Description 43
    Mechanism Of Action 43
    R&D Progress 43
    VAL-1221-Drug Profile 44
    Product Description 44
    Mechanism Of Action 44
    R&D Progress 44
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)-Dormant Products 46
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)-Discontinued Products 47
    Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)-Product Development Milestones 48
    Featured News & Press Releases 48
    Oct 04, 2017: Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study at World Muscle Society 48
    Sep 21, 2017: U.S. FDA Grants Orphan Drug Designation for ATB200/AT2221 for Pompe Disease 49
    Jul 11, 2017: Valerion Initiates VAL-1221 Dosing in Patients with Pompe Disease 50
    May 15, 2017: Amicus Therapeutics Announces Positive Functional Data from Initial Patients in Pompe Phase 1/2 Study 51
    Mar 03, 2017: Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease 52
    Feb 23, 2017: JCR to Initiate Development of Jr-162, A New Drug Candidate For Pompe Disease Using J-brain Cargo 53
    Feb 15, 2017: Amicus Therapeutics Presents Important New Scientific Findings and Preclinical Data for Pompe Program at WORLDSymposium 2017 53
    Dec 08, 2016: Amicus Therapeutics Announces Positive Preliminary Data from Phase 1/2 Study of Novel Treatment Paradigm for Pompe Disease 54
    Nov 17, 2016: Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease to Begin in the UK 55
    Nov 04, 2016: Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease 56
    Mar 03, 2016: Sanofi Genzyme Presents Results from Phase 1/2 Study of Investigational Second-Generation Therapy for Pompe Disease 57
    Mar 01, 2016: Amicus Therapeutics Highlights Preclinical Pompe Data at WORLDSymposium 2016 58
    Feb 10, 2016: Amicus Therapeutics Announces Oral and Poster Presentations on Chaperone AT2221 at 12th Annual WORLDSymposium 2016 58
    Jan 11, 2016: Amicus Therapeutics Provides Update On Novel ERT for Pompe Disease (ATB200 + Chaperone) 59
    Dec 22, 2015: Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease 60
    Appendix 61
    Methodology 61
    Coverage 61
    Secondary Research 61
    Primary Research 61
    Expert Panel Validation 61
    Contact Us 61
    Disclaimer 6

    List of Tables
    Number of Products under Development by Stage of Development, H2 2017 8
    Number of Products under Development by Therapy Areas, H2 2017 9
    Number of Products under Development by Indication, H2 2017 10
    Number of Products under Development by Companies, H2 2017 11
    Products under Development by Companies, H2 2017 12
    Number of Products under Investigation by Universities/Institutes, H2 2017 13
    Products under Investigation by Universities/Institutes, H2 2017 14
    Number of Products by Stage and Mechanism of Actions, H2 2017 16
    Number of Products by Stage and Route of Administration, H2 2017 18
    Number of Products by Stage and Molecule Type, H2 2017 20
    Pipeline by Amicus Therapeutics Inc, H2 2017 21
    Pipeline by Audentes Therapeutics Inc, H2 2017 21
    Pipeline by Etubics Corp, H2 2017 22
    Pipeline by Genzyme Corp, H2 2017 22
    Pipeline by greenovation Biotech GmbH, H2 2017 23
    Pipeline by JCR Pharmaceuticals Co Ltd, H2 2017 23
    Pipeline by NanoMedSyn SAS, H2 2017 23
    Pipeline by Oxyrane Belgium NV, H2 2017 24
    Pipeline by Pharming Group NV, H2 2017 24
    Pipeline by Sarepta Therapeutics Inc, H2 2017 25
    Dormant Projects, H2 2017 46
    Discontinued Products, H2 2017 47

    List of Figures
    Number of Products under Development by Stage of Development, H2 2017 8
    Number of Products by Mechanism of Actions, H2 2017 15
    Number of Products by Stage and Mechanism of Actions, H2 2017 15
    Number of Products by Routes of Administration, H2 2017 17
    Number of Products by Stage and Routes of Administration, H2 2017 17
    Number of Products by Molecule Types, H2 2017 19
    Number of Products by Stage and Molecule Types, H2 2017 19
    Amicus Therapeutics Inc
    Audentes Therapeutics Inc
    Etubics Corp
    Genzyme Corp
    greenovation Biotech GmbH
    JCR Pharmaceuticals Co Ltd
    NanoMedSyn SAS
    Oxyrane Belgium NV
    Pharming Group NV
    Sarepta Therapeutics Inc

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