Market Research Logo

Thalassemia - Pipeline Review, H2 2016

Thalassemia - Pipeline Review, H2 2016

Summary

Global Markets Direct’s, ‘Thalassemia - Pipeline Review, H2 2016’, provides an overview of the Thalassemia pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Thalassemia, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Thalassemia and features dormant and discontinued projects.

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note:

  • Certain sections in the report may be removed or altered based on the availability and relevance of data.


  • Scope
    • The report provides a snapshot of the global therapeutic landscape of Thalassemia
    • The report reviews pipeline therapeutics for Thalassemia 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 Thalassemia therapeutics and enlists all their major and minor projects
    • The report assesses Thalassemia therapeutics based on drug target, 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 related to pipeline therapeutics for Thalassemia
    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 important and diverse types of therapeutics under development for Thalassemia
    • 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 Thalassemia 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
    • Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline


    Introduction
    Global Markets Direct Report Coverage
    Thalassemia Overview
    Therapeutics Development
    Pipeline Products for Thalassemia - Overview
    Pipeline Products for Thalassemia - Comparative Analysis
    Thalassemia - Therapeutics under Development by Companies
    Thalassemia - Therapeutics under Investigation by Universities/Institutes
    Thalassemia - Pipeline Products Glance
    Late Stage Products
    Clinical Stage Products
    Early Stage Products
    Thalassemia - Products under Development by Companies
    Thalassemia - Products under Investigation by Universities/Institutes
    Thalassemia - Companies Involved in Therapeutics Development
    Acceleron Pharma, Inc.
    Acetylon Pharmaceuticals, Inc.
    Acino International AG
    Agios Pharmaceuticals, Inc.
    Alnylam Pharmaceuticals, Inc.
    Bellicum Pharmaceuticals, Inc.
    Bluebird bio, Inc.
    Editas Medicine, Inc.
    Emmaus Medical, Inc.
    Errant Gene Therapeutics, LLC
    Gamida Cell Ltd.
    Gilead Sciences, Inc.
    Incyte Corporation
    Ionis Pharmaceuticals, Inc.
    IRBM Science Park SpA
    Johnson & Johnson
    Kiadis Pharma N.V.
    La Jolla Pharmaceutical Company
    Merck & Co., Inc.
    Pfizer Inc.
    PharmaEssentia Corporation
    Sangamo BioSciences, Inc.
    Thalassemia - Therapeutics Assessment
    Assessment by Monotherapy Products
    Assessment by Target
    Assessment by Mechanism of Action
    Assessment by Route of Administration
    Assessment by Molecule Type
    Drug Profiles
    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
    Antisense Oligonucleotides to Inhibit BCL11A and KLF1 for Hematological Disorders - 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
    BPX-501 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    BtX-13 - 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
    Drug for Thalassemia and Hemochromatosis - 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
    glutamine - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    GSK-2696277 - 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
    PF-04447943 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Piggybac-HBB - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    PTG-300 - 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
    Thalagen - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Thalassemia - Dormant Projects
    Thalassemia - Product Development Milestones
    Featured News & Press Releases
    Jul 28, 2016: Protagonist Therapeutics Receives SBIR Funding for the Development of Injectable Hepcidin Mimetics for Treatment of Iron Overload Disorders
    Jun 10, 2016: Acceleron and Celgene Announce Updated Results from an Ongoing Phase 2 Study of Luspatercept in Beta-Thalassemia Presented at the 21st Congress of the European Hematology Association
    May 19, 2016: Agios to Present Clinical data from PKR Activator AG-348 at the 21st Congress of the European Hematology Association
    May 19, 2016: Acceleron Announces Data Presentations with Luspatercept at the 21st Congress of the European Hematology Association
    May 09, 2016: Merganser Biotech Announces Issuance of U.S. Patent 9,315,545 Covering Development Compound M012
    May 06, 2016: Drug-like Compound Shows Promise in Treating Two Blood Diseases
    May 04, 2016: Acetylon Pharmaceuticals Publishes New Mechanism for the Treatment of Sickle Cell Disease/Beta-Thalassemia with Selective HDAC1/2 Inhibition in PLoS One
    Feb 24, 2016: Merganser Biotech Initiates First Clinical Trial
    Dec 07, 2015: Acetylon Presents Data on the Pharmacokinetics and HbE/HbG Hemoglobin Induction Following Once Daily Dosing of an Oral HDAC1,2 Inhibitor in Animal Models
    Dec 02, 2015: Emmaus Life Sciences to Present Additional New Data from its Phase 3 Sickle Cell Disease Trial at 57th Annual ASH Meeting
    Nov 05, 2015: Acceleron Announces New Data on Luspatercept at the 2015 American Society of Hematology Annual Meeting
    Oct 23, 2015: Acceleron Highlights Phase 3 Studies, New Clinical Results and Research Strategies at Research and Development Day Event
    Oct 19, 2015: La Jolla Pharmaceutical Doses First Patient in Phase 1 Clinical Trial of LJPC-401 in Patients at Risk of Iron Overload
    Aug 11, 2015: La Jolla Pharmaceutical Company Announces FDA Acceptance of IND for LJPC-401
    Jun 12, 2015: Acceleron Announces New Luspatercept Phase 2 Clinical Results at the 20th Congress of the European Hematology Association
    Appendix
    Methodology
    Coverage
    Secondary Research
    Primary Research
    Expert Panel Validation
    Contact Us
    Disclaimer
    List of Tables
    Number of Products under Development for Thalassemia, H2 2016
    Number of Products under Development for Thalassemia - Comparative Analysis, H2 2016
    Number of Products under Development by Companies, H2 2016
    Number of Products under Development by Companies, H2 2016 (Contd..1)
    Number of Products under Investigation by Universities/Institutes, H2 2016
    Comparative Analysis by Late Stage Development, H2 2016
    Comparative Analysis by Clinical Stage Development, H2 2016
    Comparative Analysis by Early Stage Development, H2 2016
    Products under Development by Companies, H2 2016
    Products under Development by Companies, H2 2016 (Contd..1)
    Products under Investigation by Universities/Institutes, H2 2016
    Thalassemia - Pipeline by Acceleron Pharma, Inc., H2 2016
    Thalassemia - Pipeline by Acetylon Pharmaceuticals, Inc., H2 2016
    Thalassemia - Pipeline by Acino International AG, H2 2016
    Thalassemia - Pipeline by Agios Pharmaceuticals, Inc., H2 2016
    Thalassemia - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2016
    Thalassemia - Pipeline by Bellicum Pharmaceuticals, Inc., H2 2016
    Thalassemia - Pipeline by bluebird bio, Inc., H2 2016
    Thalassemia - Pipeline by Editas Medicine, Inc., H2 2016
    Thalassemia - Pipeline by Emmaus Medical, Inc., H2 2016
    Thalassemia - Pipeline by Errant Gene Therapeutics, LLC, H2 2016
    Thalassemia - Pipeline by Gamida Cell Ltd., H2 2016
    Thalassemia - Pipeline by Gilead Sciences, Inc., H2 2016
    Thalassemia - Pipeline by Incyte Corporation, H2 2016
    Thalassemia - Pipeline by Ionis Pharmaceuticals, Inc., H2 2016
    Thalassemia - Pipeline by IRBM Science Park SpA, H2 2016
    Thalassemia - Pipeline by Johnson & Johnson, H2 2016
    Thalassemia - Pipeline by Kiadis Pharma N.V., H2 2016
    Thalassemia - Pipeline by La Jolla Pharmaceutical Company, H2 2016
    Thalassemia - Pipeline by Merck & Co., Inc., H2 2016
    Thalassemia - Pipeline by Pfizer Inc., H2 2016
    Thalassemia - Pipeline by PharmaEssentia Corporation, H2 2016
    Thalassemia - Pipeline by Sangamo BioSciences, Inc., H2 2016
    Assessment by Monotherapy Products, H2 2016
    Number of Products by Stage and Target, H2 2016
    Number of Products by Stage and Mechanism of Action, H2 2016
    Number of Products by Stage and Route of Administration, H2 2016
    Number of Products by Stage and Molecule Type, H2 2016
    Thalassemia - Dormant Projects, H2 2016
    List of Figures
    Number of Products under Development for Thalassemia, H2 2016
    Number of Products under Development for Thalassemia - Comparative Analysis, H2 2016
    Number of Products under Development by Companies, H2 2016
    Number of Products under Investigation by Universities/Institutes, H2 2016
    Comparative Analysis by Clinical Stage Development, H2 2016
    Comparative Analysis by Early Stage Products, H2 2016
    Assessment by Monotherapy Products, H2 2016
    Number of Products by Top 10 Targets, H2 2016
    Number of Products by Stage and Top 10 Targets, H2 2016
    Number of Products by Top 10 Mechanism of Actions, H2 2016
    Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016
    Number of Products by Routes of Administration, H2 2016
    Number of Products by Stage and Routes of Administration, H2 2016
    Number of Products by Top 10 Molecule Types, H2 2016
    Number of Products by Stage and Molecule Types, H2 2016

    Download our eBook: How to Succeed Using Market Research

    Learn how to effectively navigate the market research process to help guide your organization on the journey to success.

    Download eBook

    Share this report