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Thalassemia - Pipeline Review, H2 2017

Published: Aug, 2017 | Pages: 141 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

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



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