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    Mucopolysaccharidosis I-Pipeline Review H1 2017

    Mucopolysaccharidosis I-Pipeline Review H1 2017

    • Report Code ID: RW0001834383
    • Category Pharmaceuticals
    • No. of Pages 73
    • Publication Month Jun-17
    • Publisher Name Global Markets Direct
    Mucopolysaccharidosis I - Pipeline Review, H1 2017

    Summary

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

    MPS I (Mucopolysaccharidosis I) is an inherited lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs) . Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy.

    Report Highlights

    Publisher's Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis I - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I (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 Mucopolysaccharidosis I (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) 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 5, 1, 6 and 3 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 2 and 1 molecules, respectively.

    Mucopolysaccharidosis I (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.

    Scope

    - The pipeline guide provides a snapshot of the global therapeutic landscape of Mucopolysaccharidosis I (Metabolic Disorders) .
    - The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis I (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 Mucopolysaccharidosis I (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
    - The pipeline guide evaluates Mucopolysaccharidosis I (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 Mucopolysaccharidosis I (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 Mucopolysaccharidosis I (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 Mucopolysaccharidosis I (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
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Overview
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Development
    Pipeline Overview
    Pipeline by Companies
    Pipeline by Universities/Institutes
    Products under Development by Companies
    Products under Development by Universities/Institutes
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Assessment
    Assessment by Target
    Assessment by Mechanism of Action
    Assessment by Route of Administration
    Assessment by Molecule Type
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Companies Involved in Therapeutics Development
    AngioChem Inc
    ArmaGen Inc
    Bioasis Technologies Inc
    CRISPR Therapeutics
    Eloxx Pharmaceuticals Ltd
    Immusoft Corp
    Inventiva
    OPKO Health Inc
    PTC Therapeutics Inc
    RegenxBio Inc
    Sangamo Therapeutics Inc
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Drug Profiles
    AGT-181 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    ataluren - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Cell Therapy for Mucopolysaccharidosis I - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Cell Therapy to Activate Alpha L-Iduronidase for Hurler Syndrome - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    ELX-02 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    JOT-102 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    MGTA-456 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    odiparcil - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Oligonucleotide for Mucopolysaccharidosis I - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    pentosan polysulfate sodium - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Recombinant Alpha-L-Iduronidase Replacement for Hurler Syndrome - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    RGX-111 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    SB-318 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Stem Cell Therapy for Hurler Sydrome - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    X-372 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Product Development Milestones
    Featured News & Press Releases
    Feb 16, 2017: ArmaGen Reports Preliminary Evidence of Cognitive Improvement in Children with Hurler Syndrome (MPS I) Treated with AGT-181
    Feb 07, 2017: ArmaGen Announces Oral Presentation of Preliminary Results from its Phase 2 Clinical Trial of AGT-181 in Patients with MPS 1 to be Presented at WORLDSymposium 2017
    Nov 07, 2016: Eloxx Pharmaceuticals Announces Orphan Drug Designation in the U.S. and Europe for ELX-02 in Mucopolysacchardisis Type 1 (MPS 1)
    Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-111
    Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-111
    Mar 31, 2016: ArmaGen Announces Initiation of Phase 2 Proof-of-Concept Clinical Trial in Brazil to Study AGT-181 for the Treatment of Hurler Syndrome
    Dec 30, 2015: FDA Grants Rare Pediatric Disease Designation to REGENXBIO RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I (MPS I)
    Nov 05, 2015: ArmaGen Receives Rare Pediatric Disease Designation from FDA for AGT-181 for the Potential Treatment of Hurler Syndrome
    Oct 01, 2015: FDA Grants Orphan Drug Designation to REGENXBIO's RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I
    Sep 02, 2015: ArmaGen Announces First Patient Dosed in Phase 1/2a Clinical Trial of AGT-181 for the Treatment of Hurler Syndrome
    Apr 08, 2015: ArmaGen Announces FDA Acceptance of IND Application for AGT-181 for the Treatment of Hurler Syndrome
    Dec 19, 2014: Translarna Granted Orphan Drug Designation in the U.S. and Europe for the Treatment of Mucopolysaccharidosis I
    Feb 05, 2014: Mouse Study Shows Gene Therapy May Be Possible Cure for Hurler Syndrome
    Appendix
    Methodology
    Coverage
    Secondary Research
    Primary Research
    Expert Panel Validation
    Contact Us
    Disclaimer

    List of Tables

    Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) , 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
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by AngioChem Inc, H1 2017
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Inc, H1 2017
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Bioasis Technologies Inc, H1 2017
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by CRISPR Therapeutics, H1 2017
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Eloxx Pharmaceuticals Ltd, H1 2017
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Immusoft Corp, H1 2017
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Inventiva, H1 2017
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by OPKO Health Inc, H1 2017
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by PTC Therapeutics Inc, H1 2017
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by RegenxBio Inc, H1 2017
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sangamo Therapeutics Inc, H1 2017
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, H1 2017
    Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products, H1 2017

    List of Figures

    Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) , 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 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
    AngioChem Inc
    ArmaGen Inc
    Bioasis Technologies Inc
    CRISPR Therapeutics
    Eloxx Pharmaceuticals Ltd
    Immusoft Corp
    Inventiva
    OPKO Health Inc
    PTC Therapeutics Inc
    RegenxBio Inc
    Sangamo Therapeutics Inc

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