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An analysis of the latest treatments for thalassemia just published

Medical Market Research

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.

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

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Report Highlights

The 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 The 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.


- 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)

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This report helps to

- 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.

Companies Mentioned in the Report

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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Table of Contents

List of Tables 4
List of Figures 4
Introduction 5
The 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


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