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    Thalassemia - Pipeline Review, H1 2017

    Thalassemia - Pipeline Review, H1 2017

    • Report Code ID: RW0001709482
    • Category Pharmaceuticals
    • No. of Pages 140
    • Publication Month Mar-17
    • Publisher Name Global Markets Direct
    Thalassemia - Pipeline Review, H1 2017

    Summary

    Publisher's latest Pharmaceutical and Healthcare disease pipeline guide Thalassemia - Pipeline Review, H1 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

    Publisher's Pharmaceutical and Healthcare latest pipeline guide Thalassemia - Pipeline Review, H1 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, 6, 17 and 4 respectively. Similarly, the Universities portfolio in Phase II, Preclinical and Discovery stages comprises 1, 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 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 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
    List of Tables
    List of Figures
    Introduction
    Publisher Report Coverage
    Thalassemia - Overview
    Thalassemia - Therapeutics Development
    Pipeline Overview
    Pipeline by Companies
    Pipeline by Universities/Institutes
    Products under Development by Companies
    Products under Development by Universities/Institutes
    Thalassemia - Therapeutics Assessment
    Assessment by Target
    Assessment by Mechanism of Action
    Assessment by Route of Administration
    Assessment by Molecule Type
    Thalassemia - Companies Involved in Therapeutics Development
    Acceleron Pharma Inc
    Agios Pharmaceuticals Inc
    Alnylam Pharmaceuticals Inc
    bluebird bio Inc
    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
    Johnson & Johnson
    Kiadis Pharma NV
    La Jolla Pharmaceutical Company
    Merck & Co Inc
    PharmaEssentia Corp
    Protagonist Therapeutics Inc
    Sangamo Therapeutics Inc
    Zydus Cadila Healthcare Ltd
    Thalassemia - Drug Profiles
    (decitabine + tetrahydrouridine) - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    ACY-957 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    AG-348 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    ALN-TMP - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    ambrisentan - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    ATIR-201 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    BB-305 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    benserazide - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    CAL-H - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    CNTO-530 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    CordIn - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Drugs to Chelate Iron for Bone Degeneration, Postmenopausal Osteoporosis and Thalassemia - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy to Activate IGF2BP1 for Sickle Cell Disease and Thalassemia - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    GSK-2696277 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    IMR-687 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    IONISTMPRSS-6LRx - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    LJPC-401 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    luspatercept - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    M-009 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    M-012 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    NiCord - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    PEG-EPO - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    PHBB-101 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    PTG-300 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    RCY-1497 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    ruxolitinib phosphate - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    SCD-101 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    sirolimus - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Small Molecule for Beta Thalassemia and Sickle Cell Anemia - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Small Molecule to Inhibit Histone Deacetylase 1 for Beta Thalassemia - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    sotatercept - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Stem Cell Therapy for Beta Thalassemia - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Thalagen - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Thalassemia - Dormant Projects
    Thalassemia - Product Development Milestones
    Featured News & Press Releases
    Feb 06, 2017: Kiadis Pharma Provides Update on ATIR201
    Dec 15, 2016: Kiadis Pharma announces initiation of Phase I/II clinical trial with ATIR201 for thalassemia
    Dec 14, 2016: bluebird bio Announces First Patient Treated with LentiGlobin Drug Product in Northstar-2 (HGB-207) Phase 3 Trial of Patients with Transfusion-Dependent ß-Thalassemia
    Dec 06, 2016: bluebird bio Provides Updates on HSC Gene Therapy Programs
    Dec 05, 2016: Acceleron and Celgene Announce Updated Results from Phase 2 Studies of Luspatercept in Beta-Thalassemia Presented at the 58th Annual Meeting of the American Society of Hematology
    Dec 04, 2016: Acetylon Presents Preclinical Data Demonstrating the Utility of Selective HDAC1,2 Inhibition by ACY-957 to Induce Gamma Globin (HBG) Protein Expression for the Treatment of Sickle Cell Disease and Beta-Thalassemia
    Dec 03, 2016: bluebird bio Presents New Data from HGB-205 Study of LentiGlobin Drug Product in Patients with Transfusion-Dependent ß-Thalassemia and Severe Sickle Cell Disease at American Society of Hematology (ASH) Annual Meeting
    Dec 02, 2016: Acetylon to present data on ACY-957 at the American Society of Hematology Annual Meeting
    Nov 21, 2016: CRISPR Therapeutics Announces Two Presentations Demonstrating the Potential for CRISPR Gene Editing To Treat Sickle Cell Disease and ß-Thalassemia
    Nov 03, 2016: bluebird bio to Present New Data from Three LentiGlobin Clinical Studies at American Society of Hematology (ASH) Annual Meeting
    Oct 13, 2016: bluebird bio Provides Update on LentiGlobin Programs and Research and Development Strategy at Gene Therapy Day
    Sep 21, 2016: LentiGlobin Investigational Gene Therapy for Transfusion-Dependent Beta-Thalassemia Accepted into European Medicines Agency's PRIME Program
    Sep 08, 2016: bluebird bio Opens Phase 3 Study of LentiGlobin Drug Product in Patients with Transfusion-Dependent Beta-Thalassemia
    Sep 07, 2016: La Jolla Pharmaceutical Company Reports Positive Results from Phase 1 Study of LJPC-401
    Appendix
    Methodology
    Coverage
    Secondary Research
    Primary Research
    Expert Panel Validation
    Contact Us
    Disclaimer

    List of Tables

    Number of Products under Development for Thalassemia, H1 2017
    Number of Products under Development by Companies, H1 2017
    Number of Products under Development by Companies, H1 2017 (Contd..1) , H1 2017
    Number of Products under Development by Universities/Institutes, H1 2017
    Products under Development by Companies, H1 2017
    Products under Development by Companies, H1 2017 (Contd..1) , 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
    Thalassemia - Pipeline by Acceleron Pharma Inc, H1 2017
    Thalassemia - Pipeline by Agios Pharmaceuticals Inc, H1 2017
    Thalassemia - Pipeline by Alnylam Pharmaceuticals Inc, H1 2017
    Thalassemia - Pipeline by bluebird bio Inc, H1 2017
    Thalassemia - Pipeline by Calimmune Inc, H1 2017
    Thalassemia - Pipeline by CRISPR Therapeutics, H1 2017
    Thalassemia - Pipeline by Editas Medicine Inc, H1 2017
    Thalassemia - Pipeline by Errant Gene Therapeutics LLC, H1 2017
    Thalassemia - Pipeline by Gamida Cell Ltd, H1 2017
    Thalassemia - Pipeline by Gilead Sciences Inc, H1 2017
    Thalassemia - Pipeline by Incyte Corp, H1 2017
    Thalassemia - Pipeline by Ionis Pharmaceuticals Inc, H1 2017
    Thalassemia - Pipeline by IRBM Science Park SpA, H1 2017
    Thalassemia - Pipeline by Johnson & Johnson, H1 2017
    Thalassemia - Pipeline by Kiadis Pharma NV, H1 2017
    Thalassemia - Pipeline by La Jolla Pharmaceutical Company, H1 2017
    Thalassemia - Pipeline by Merck & Co Inc, H1 2017
    Thalassemia - Pipeline by PharmaEssentia Corp, H1 2017
    Thalassemia - Pipeline by Protagonist Therapeutics Inc, H1 2017
    Thalassemia - Pipeline by Sangamo Therapeutics Inc, H1 2017
    Thalassemia - Pipeline by Zydus Cadila Healthcare Ltd, H1 2017
    Thalassemia - Dormant Projects, H1 2017

    List of Figures

    Number of Products under Development for Thalassemia, 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 Top 10 Targets, H1 2017
    Number of Products by Stage and Top 10 Targets, H1 2017
    Number of Products by Top 10 Mechanism of Actions, H1 2017
    Number of Products by Stage and Top 10 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 Top 10 Molecule Types, H1 2017
    Number of Products by Stage and Top 10 Molecule Types, H1 2017
    Acceleron Pharma Inc
    Agios Pharmaceuticals Inc
    Alnylam Pharmaceuticals Inc
    bluebird bio Inc
    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
    Johnson & Johnson
    Kiadis Pharma NV
    La Jolla Pharmaceutical Company
    Merck & Co Inc
    PharmaEssentia Corp
    Protagonist Therapeutics Inc
    Sangamo Therapeutics Inc
    Zydus Cadila Healthcare Ltd

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