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Thalassemia - Pipeline Review, H2 2017
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Published by Global Markets Direct: 22 Aug 2017 | 120506 | In Stock
Related Topics: Healthcare

Introduction

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
for Thalassemia - Pipeline Review, H2 2017

  • 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

Additional Details

Publisher

Global Markets Direct

Publisher Information

Reference

120506 | GMDHC9638IDB

Number of Pages

141

Report Format

PDF

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