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    Thalassemia-Pipeline Review H2 2017

    Thalassemia-Pipeline Review H2 2017

    • Report Code ID: RW00011022662
    • Category Life Sciences
    • No. of Pages 141
    • Publication Month Aug-17
    • Publisher Name Global Markets Direct
    Thalassemia-Pipeline Review, H2 2017

    Summary

    Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Thalassemia-Pipeline Review, H2 2017, provides an overview of the Thalassemia (Hematological Disorders) pipeline landscape.

    Thalassemia is a group of inherited blood disorders that affect the body's ability to produce hemoglobin and red blood cells. Symptoms include fussiness, paleness, frequent infections, failure to thrive, poor appetite and jaundice. Predisposing factors include family history. Treatment includes blood transfusions and bone marrow transplant.

    Report Highlights

    Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Thalassemia-Pipeline Review, H2 2017, provides comprehensive information on the therapeutics under development for Thalassemia (Hematological 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 Thalassemia (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Thalassemia and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 2, 5, 8, 15 and 2 respectively. Similarly, the Universities portfolio in Phase II, Preclinical and Discovery stages comprises 2, 2 and 1 molecules, respectively.

    Thalassemia (Hematological 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 Global Markets Direct'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 Thalassemia (Hematological Disorders).
    -The pipeline guide reviews pipeline therapeutics for Thalassemia (Hematological 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 Thalassemia (Hematological Disorders) therapeutics and enlists all their major and minor projects.
    -The pipeline guide evaluates Thalassemia (Hematological 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 Thalassemia (Hematological 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 Thalassemia (Hematological 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 Thalassemia (Hematological 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
    Table of Contents 2
    List of Tables 4
    List of Figures 4
    Introduction 5
    Global Markets Direct Report Coverage 5
    Thalassemia-Overview 6
    Thalassemia-Therapeutics Development 7
    Pipeline Overview 7
    Pipeline by Companies 8
    Pipeline by Universities/Institutes 11
    Products under Development by Companies 12
    Products under Development by Universities/Institutes 14
    Thalassemia-Therapeutics Assessment 15
    Assessment by Target 15
    Assessment by Mechanism of Action 17
    Assessment by Route of Administration 19
    Assessment by Molecule Type 21
    Thalassemia-Companies Involved in Therapeutics Development 23
    Acceleron Pharma Inc 23
    Agios Pharmaceuticals Inc 23
    Alnylam Pharmaceuticals Inc 24
    bluebird bio Inc 24
    Cadila Healthcare Ltd 25
    Calimmune Inc 25
    CRISPR Therapeutics 26
    Editas Medicine Inc 26
    Errant Gene Therapeutics LLC 27
    Gamida Cell Ltd 27
    Gilead Sciences Inc 28
    Incyte Corp 28
    Ionis Pharmaceuticals Inc 29
    IRBM Science Park SpA 29
    Kiadis Pharma NV 30
    La Jolla Pharmaceutical Company 30
    Merck & Co Inc 31
    Protagonist Therapeutics Inc 31
    Sangamo Therapeutics Inc 32
    Thalassemia-Drug Profiles 33
    (decitabine + tetrahydrouridine)-Drug Profile 33
    ACY-957-Drug Profile 34
    AG-348-Drug Profile 37
    ALN-TMP-Drug Profile 42
    ambrisentan-Drug Profile 44
    ATIR-201-Drug Profile 47
    BB-305-Drug Profile 48
    benserazide-Drug Profile 57
    CAL-H-Drug Profile 59
    CordIn-Drug Profile 60
    Drugs to Chelate Iron for Bone Degeneration, Postmenopausal Osteoporosis and Thalassemia-Drug Profile 61
    Gene Therapy for Sickle Cell Disease and Thalassemia-Drug Profile 62
    Gene Therapy to Activate IGF2BP1 for Sickle Cell Disease and Thalassemia-Drug Profile 63
    GSK-2696277-Drug Profile 64
    IMR-687-Drug Profile 65
    IONISTMPRSS-6LRx-Drug Profile 66
    LJPC-401-Drug Profile 67
    luspatercept-Drug Profile 69
    M-009-Drug Profile 78
    M-012-Drug Profile 79
    NiCord-Drug Profile 81
    PEG-EPO-Drug Profile 85
    PHBB-101-Drug Profile 86
    PTG-300-Drug Profile 87
    RCY-1497-Drug Profile 88
    RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia-Drug Profile 89
    ruxolitinib phosphate-Drug Profile 90
    SCD-101-Drug Profile 105
    sirolimus-Drug Profile 106
    Small Molecule to Inhibit Histone Deacetylase 1 for Beta Thalassemia-Drug Profile 107
    Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours-Drug Profile 108
    sotatercept-Drug Profile 109
    ST-400-Drug Profile 114
    Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease-Drug Profile 116
    Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease-Drug Profile 117
    Stem Cell Therapy to Activate Hemoglobin Beta for Beta-Thalassemia-Drug Profile 119
    Thalagen-Drug Profile 120
    Thalassemia-Dormant Projects 121
    Thalassemia-Product Development Milestones 122
    Featured News & Press Releases 122
    Appendix 137
    Methodology 137
    Coverage 137
    Secondary Research 137
    Primary Research 137
    Expert Panel Validation 137
    Contact Us 137
    Disclaimer 138

    List of Tables
    Number of Products under Development for Thalassemia, H2 2017
    Number of Products under Development by Companies, H2 2017
    Number of Products under Development by Companies, H2 2017 (Contd..1), H2 2017
    Number of Products under Development by Universities/Institutes, H2 2017
    Products under Development by Companies, H2 2017
    Products under Development by Companies, H2 2017 (Contd..1), H2 2017
    Products under Development by Universities/Institutes, H2 2017
    Number of Products by Stage and Target, H2 2017
    Number of Products by Stage and Mechanism of Action, H2 2017
    Number of Products by Stage and Route of Administration, H2 2017
    Number of Products by Stage and Molecule Type, H2 2017
    Thalassemia-Pipeline by Acceleron Pharma Inc, H2 2017
    Thalassemia-Pipeline by Agios Pharmaceuticals Inc, H2 2017
    Thalassemia-Pipeline by Alnylam Pharmaceuticals Inc, H2 2017
    Thalassemia-Pipeline by bluebird bio Inc, H2 2017
    Thalassemia-Pipeline by Cadila Healthcare Ltd, H2 2017
    Thalassemia-Pipeline by Calimmune Inc, H2 2017
    Thalassemia-Pipeline by CRISPR Therapeutics, H2 2017
    Thalassemia-Pipeline by Editas Medicine Inc, H2 2017
    Thalassemia-Pipeline by Errant Gene Therapeutics LLC, H2 2017
    Thalassemia-Pipeline by Gamida Cell Ltd, H2 2017
    Thalassemia-Pipeline by Gilead Sciences Inc, H2 2017
    Thalassemia-Pipeline by Incyte Corp, H2 2017
    Thalassemia-Pipeline by Ionis Pharmaceuticals Inc, H2 2017
    Thalassemia-Pipeline by IRBM Science Park SpA, H2 2017
    Thalassemia-Pipeline by Kiadis Pharma NV, H2 2017
    Thalassemia-Pipeline by La Jolla Pharmaceutical Company, H2 2017
    Thalassemia-Pipeline by Merck & Co Inc, H2 2017
    Thalassemia-Pipeline by Protagonist Therapeutics Inc, H2 2017
    Thalassemia-Pipeline by Sangamo Therapeutics Inc, H2 2017
    Thalassemia-Dormant Projects, H2 2017

    List of Figures
    Number of Products under Development for Thalassemia, H2 2017
    Number of Products under Development by Companies, H2 2017
    Number of Products under Development by Universities/Institutes, H2 2017
    Number of Products by Top 10 Targets, H2 2017
    Number of Products by Stage and Top 10 Targets, H2 2017
    Number of Products by Top 10 Mechanism of Actions, H2 2017
    Number of Products by Stage and Top 10 Mechanism of Actions, H2 2017
    Number of Products by Routes of Administration, H2 2017
    Number of Products by Stage and Routes of Administration, H2 2017
    Number of Products by Top 10 Molecule Types, H2 2017
    Number of Products by Stage and Top 10 Molecule Types, H2 2017
    Acceleron Pharma Inc
    Agios Pharmaceuticals Inc
    Alnylam Pharmaceuticals Inc
    bluebird bio Inc
    Cadila Healthcare Ltd
    Calimmune Inc
    CRISPR Therapeutics
    Editas Medicine Inc
    Errant Gene Therapeutics LLC
    Gamida Cell Ltd
    Gilead Sciences Inc
    Incyte Corp
    Ionis Pharmaceuticals Inc
    IRBM Science Park SpA
    Kiadis Pharma NV
    La Jolla Pharmaceutical Company
    Merck & Co Inc
    Protagonist Therapeutics Inc
    Sangamo Therapeutics Inc

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