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Hemoglobinopathies - Pipeline Review, H2 2013

  • December 2013
  • -
  • Global Markets Direct
  • -
  • 36 pages

Hemoglobinopathies - Pipeline Review, H2 2013

Summary

Global Markets Direct’s, 'Hemoglobinopathies - Pipeline Review, H2 2013', provides an overview of the indication’s therapeutic pipeline. This report provides information on the therapeutic development for Hemoglobinopathies, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Hemoglobinopathies. Hemoglobinopathies - Pipeline Review, Half Year is built using data and information sourced from Global Markets Direct’s proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct’s team.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

- A snapshot of the global therapeutic scenario for Hemoglobinopathies.
- A review of the Hemoglobinopathies products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.
- Coverage of products based on various stages of development ranging from discovery till registration stages.
- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
- Coverage of the Hemoglobinopathies pipeline on the basis of route of administration and molecule type.
- Key discontinued pipeline projects.
- Latest news and deals relating to the products.

Reasons to buy

- Identify and understand important and diverse types of therapeutics under development for Hemoglobinopathies.
- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
- Plan mergers and acquisitions effectively by identifying players of the most promising pipeline.
- Devise corrective measures for pipeline projects by understanding Hemoglobinopathies 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.

Table Of Contents

Hemoglobinopathies - Pipeline Review, H2 2013
Table of Contents

Table of Contents 2
List of Tables 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Hemoglobinopathies Overview 6
Therapeutics Development 7
Pipeline Products for Hemoglobinopathies - Overview 7
Pipeline Products for Hemoglobinopathies - Comparative Analysis 8
Hemoglobinopathies - Therapeutics under Development by Companies 9
Hemoglobinopathies - Pipeline Products Glance 10
Clinical Stage Products 10
Early Stage Products 11
Hemoglobinopathies - Products under Development by Companies 12
Hemoglobinopathies - Companies Involved in Therapeutics Development 13
Sangamo BioSciences, Inc. 13
Gamida Cell Ltd. 14
Alnylam Pharmaceuticals, Inc. 15
Cancer Therapeutics CRC Pty Ltd 16
Hemoglobinopathies - Therapeutics Assessment 17
Assessment by Monotherapy Products 17
Assessment by Target 18
Assessment by Mechanism of Action 19
Assessment by Route of Administration 20
Assessment by Molecule Type 21
Assessment by Therapeutic Class 23
Drug Profiles 25
NiCord - Drug Profile 25
Product Description 25
Mechanism of Action 25
RandD Progress 25
ZFP Transcription Factors For Hemoglobinopathies - Drug Profile 26
Product Description 26
Mechanism of Action 26
RandD Progress 26
ALN-TMP - Drug Profile 27
Product Description 27
Mechanism of Action 27
RandD Progress 27
Drug Targeting PRMT5 - Drug Profile 28
Product Description 28
Mechanism of Action 28
RandD Progress 28
Drug Targeting Protein Arginine Methyltransferase 5 - Drug Profile 29
Product Description 29
Mechanism of Action 29
RandD Progress 29
Hemoglobinopathies - Recent Pipeline Updates 30
Hemoglobinopathies - Product Development Milestones 33
Featured News and Press Releases 33
Dec 08, 2012: Alnylam Pharma Presents New Preclinical Data On RNAi Therapeutics For Treatment Of Hemoglobinopathies At ASH Annual Meeting 2012 33
Appendix 35
Methodology 35
Coverage 35
Secondary Research 35
Primary Research 35
Expert Panel Validation 35
Contact Us 36
Disclaimer 36



List of Tables

Number of Products under Development for Hemoglobinopathies, H2 2013 7
Number of Products under Development for Hemoglobinopathies - Comparative Analysis, H2 2013 8
Number of Products under Development by Companies, H2 2013 9
Comparative Analysis by Clinical Stage Development, H2 2013 10
Comparative Analysis by Early Stage Development, H2 2013 11
Products under Development by Companies, H2 2013 12
Hemoglobinopathies - Pipeline by Sangamo BioSciences, Inc., H2 2013 13
Hemoglobinopathies - Pipeline by Gamida Cell Ltd., H2 2013 14
Hemoglobinopathies - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2013 15
Hemoglobinopathies - Pipeline by Cancer Therapeutics CRC Pty Ltd, H2 2013 16
Assessment by Monotherapy Products, H2 2013 17
Number of Products by Stage and Target, H2 2013 18
Number of Products by Stage and Mechanism of Action, H2 2013 19
Number of Products by Stage and Route of Administration, H2 2013 20
Number of Products by Stage and Molecule Type, H2 2013 22
Number of Products by Stage and Therapeutic Class, H2 2013 24
Hemoglobinopathies Therapeutics - Recent Pipeline Updates, H2 2013 30



List of Figures

Number of Products under Development for Hemoglobinopathies, H2 2013 7
Number of Products under Development for Hemoglobinopathies - Comparative Analysis, H2 2013 8
Number of Products under Development by Companies, H2 2013 9
Comparative Analysis by Early Stage Products, H2 2013 11
Assessment by Monotherapy Products, H2 2013 17
Number of Products by Top 10 Molecule Type, H2 2013 21
Number of Products by Stage and Top 10 Molecule Type, H2 2013 22
Number of Products by Top 10 Therapeutic Class, H2 2013 23
Number of Products by Stage and Top 10 Therapeutic Class, H2 2013 24



Companies Mentioned

Sangamo BioSciences, Inc.
Gamida Cell Ltd.
Alnylam Pharmaceuticals, Inc.
Cancer Therapeutics CRC Pty Ltd

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