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    Phenylketonuria (PKU) - Pipeline Review, H1 2017

    Phenylketonuria (PKU) - Pipeline Review, H1 2017

    • Report Code ID: RW0001689433
    • Category Healthcare
    • No. of Pages 47
    • Publication Month Feb-17
    • Publisher Name Global Markets Direct
    Report Summary

    Publisher's latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria (PKU) - Pipeline Review, H1 2017, provides an overview of the Phenylketonuria (PKU) (Metabolic Disorders) pipeline landscape.

    Phenylketonuria (also called PKU), is a rare inherited disorder that causes an amino acid called phenylalanine to build up in body. Symptoms include intellectual disability, psychiatric disorders, hyperactivity, skin rashes, poor bone strength and abnormally small head.

    Report Highlights

    Publisher's Pharmaceutical and Healthcare latest pipeline guide Phenylketonuria (PKU) - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Phenylketonuria (PKU) (Metabolic 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 Phenylketonuria (PKU) (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Phenylketonuria (PKU) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Preclinical and Discovery stages are 1, 9 and 1 respectively.

    Phenylketonuria (PKU) (Metabolic 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.

    Report Scope

    - The pipeline guide provides a snapshot of the global therapeutic landscape of Phenylketonuria (PKU) (Metabolic Disorders).
    - The pipeline guide reviews pipeline therapeutics for Phenylketonuria (PKU) (Metabolic 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 Phenylketonuria (PKU) (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
    - The pipeline guide evaluates Phenylketonuria (PKU) (Metabolic 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 Phenylketonuria (PKU) (Metabolic 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 Phenylketonuria (PKU) (Metabolic 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 Phenylketonuria (PKU) (Metabolic 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
    Phenylketonuria (PKU) - Overview
    Phenylketonuria (PKU) - Therapeutics Development
    Pipeline Overview
    Pipeline by Companies
    Products under Development by Companies
    Phenylketonuria (PKU) - Therapeutics Assessment
    Assessment by Target
    Assessment by Mechanism of Action
    Assessment by Route of Administration
    Assessment by Molecule Type
    Phenylketonuria (PKU) - Companies Involved in Therapeutics Development
    American Gene Technologies International Inc
    BioMarin Pharmaceutical Inc
    Codexis Inc
    Dimension Therapeutics Inc
    Erytech Pharma SA
    MipSalus ApS
    SOM Biotech SL
    Synlogic Inc
    Synthetic Biologics Inc
    Phenylketonuria (PKU) - Drug Profiles
    DTX-501 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy for Phenylketonuria - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    pegvaliase - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Phelimine - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Recombinant Enzyme for Phenylketonuria - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Recombinant Phenylalanine-4-Hydroxylase Replacement for Phenylketonuria - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Recombinant Phenylalanine-4-Hydroxylase Replacement for Phenylketonuria - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Small Molecules to Activate Phenylalanine Hydroxylase for Phenylketonuria - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    SOM-7400 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    SYN-200 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    SYNB-2010 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Phenylketonuria (PKU) - Dormant Projects
    Phenylketonuria (PKU) - Product Development Milestones
    Featured News & Press Releases
    Mar 31, 2016: Promising results with breakthrough treatment of orphan disease PKU
    Mar 21, 2016: BioMarin Phase 3 Study of Pegvaliase for Phenylketonuria Meets Primary Endpoint of Blood Phenylalanine Reduction (p0.0001)
    Jun 05, 2013: BioMarin Initiates Phase III Trial For PEG-PAL For Treatment Of Phenylketonuria
    Sep 26, 2012: BioMarin Pharma To Initiate Phase III Program For PEG-PAL In Second Quarter Of 2013
    Aug 02, 2010: BioMarin Reports Encouraging Preliminary Safety And Efficacy Trends In Phase II Clinical Study Of PEG-PAL In Phenylketonuria
    Sep 22, 2009: BioMarin Initiates Phase II Clinical Study Of PEG-PAL In PKU
    Jun 09, 2009: Results From Phase 1 Clinical Study Of PEG-PAL In PKU And Update On Phase 2 Clinical Study
    May 20, 2008: BioMarin Initiates Phase 1 Clinical Study Of PEG-PAL In PKU
    Nov 27, 2007: BioMarin Files Investigational New Drug Application For PEG-PAL For The Treatment Of PKU
    May 16, 2005: BioMarin And Serono Form Strategic Alliance For The Development And Commercialization Of Phenoptin And Phenylase
    Appendix
    Methodology
    Coverage
    Secondary Research
    Primary Research
    Expert Panel Validation
    Contact Us
    Disclaimer

    List of Tables

    Number of Products under Development for Phenylketonuria (PKU), H1 2017
    Number of Products under Development by Companies, H1 2017
    Products under Development by Companies, 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
    Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, H1 2017
    Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, H1 2017
    Phenylketonuria (PKU) - Pipeline by Codexis Inc, H1 2017
    Phenylketonuria (PKU) - Pipeline by Dimension Therapeutics Inc, H1 2017
    Phenylketonuria (PKU) - Pipeline by Erytech Pharma SA, H1 2017
    Phenylketonuria (PKU) - Pipeline by MipSalus ApS, H1 2017
    Phenylketonuria (PKU) - Pipeline by SOM Biotech SL, H1 2017
    Phenylketonuria (PKU) - Pipeline by Synlogic Inc, H1 2017
    Phenylketonuria (PKU) - Pipeline by Synthetic Biologics Inc, H1 2017
    Phenylketonuria (PKU) - Dormant Projects, H1 2017

    List of Figures

    Number of Products under Development for Phenylketonuria (PKU), H1 2017
    Number of Products under Development by Companies, H1 2017
    Number of Products by Targets, H1 2017
    Number of Products by Stage and Targets, 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
    American Gene Technologies International Inc
    BioMarin Pharmaceutical Inc
    Codexis Inc
    Dimension Therapeutics Inc
    Erytech Pharma SA
    MipSalus ApS
    SOM Biotech SL
    Synlogic Inc
    Synthetic Biologics Inc

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