Thalassemia - Pipeline Review, H2 2014

Published: Nov, 2014 | Pages: 97 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Thalassemia - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Thalassemia - Pipeline Review, H2 2014', provides an overview of the Thalassemia's therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Thalassemia, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Thalassemia and special features on late-stage 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, 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. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

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

Scope

- The report provides a snapshot of the global therapeutic landscape of Thalassemia
- The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities 
- The report reviews key players involved in the therapeutics development for Thalassemia and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects 
- A review of the Thalassemia products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
- Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- A detailed assessment of monotherapy and combination therapy pipeline projects
- Coverage of the Thalassemia pipeline on the basis of target, MoA, route of administration and molecule type
- Latest news and deals relating related to pipeline products

Reasons to buy

- Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Develop strategic initiatives by understanding the focus areas of leading companies
- Identify and understand important and diverse types of therapeutics under development for Thalassemia
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- 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

 Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Thalassemia Overview 8
Therapeutics Development 9
Pipeline Products for Thalassemia - Overview 9
Pipeline Products for Thalassemia - Comparative Analysis 10
Thalassemia - Therapeutics under Development by Companies 11
Thalassemia - Therapeutics under Investigation by Universities/Institutes 13
Thalassemia - Pipeline Products Glance 14
Late Stage Products 14
Clinical Stage Products 15
Early Stage Products 16
Thalassemia - Products under Development by Companies 17
Thalassemia - Products under Investigation by Universities/Institutes 18
Thalassemia - Companies Involved in Therapeutics Development 19
Acceleron Pharma, Inc. 19
Acetylon Pharmaceuticals, Inc. 20
Acino Pharma AG 21
Alnylam Pharmaceuticals, Inc. 22
Bluebird bio, Inc. 23
Children's Hospital Boston 24
Emmaus Medical, Inc. 25
Errant Gene Therapeutics, LLC 26
Gamida Cell Ltd. 27
Incyte Corporation 28
IRBM Science Park SpA 29
Isis Pharmaceuticals, Inc. 30
Johnson & Johnson 31
Protagonist Therapeutics Inc. 32
Sangamo BioSciences, Inc. 33
Thalassemia - Therapeutics Assessment 34
Assessment by Monotherapy Products 34
Assessment by Target 35
Assessment by Mechanism of Action 37
Assessment by Route of Administration 39
Assessment by Molecule Type 41
Drug Profiles 43
ACY-957 - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
ALN-TMP - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
Antisense Oligonucleotide to Inhibit BCL11A and KLF1 for Hematological Disorders - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
BtX-13 - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
CNTO-530 - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
Gene Therapy for Beta Thalassemia - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
glutamine - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
LentiGlobin - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
luspatercept - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
NiCord - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
PB-04 - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Peptide Agonists of Hepcidin for Beta-Thalassemia and Juvenile Hemochromatosis - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
Project x-1450 - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
ruxolitinib phosphate - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
Small Molecule to Inhibit Histone Deacetylase for Beta-Thalassemia - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
sotatercept - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
Synthetic Peptides Hematological and Genetic Disorders - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
Thalagen - Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
Thalassemia - Recent Pipeline Updates 70
Thalassemia - Dormant Projects 88
Thalassemia - Product Development Milestones 89
Featured News & Press Releases 89
Nov 06, 2014: Acceleron Announces New Clinical Data Presentations on luspatercept at the 2014 American Society of Hematology Annual Meeting 89
Nov 06, 2014: Celgene To Present Phase II trial Results Update On Luspatercept at American Society of Hematology Annual Meeting 89
Nov 06, 2014: Acceleron Announces New Clinical Data Presentations on sotatercept at the 2014 American Society of Hematology Annual Meeting 90
Oct 01, 2014: Acceleron to Host Educational Webcast on Luspatercept 90
Oct 01, 2014: Acceleron to Host Educational Webcast on Beta-thalassemia 91
Jun 14, 2014: Celgene and Acceleron announces interim data from ACE-536 at the 19th European Hematology Association annual congress 91
Jun 14, 2014: Studies Presented at 19th European Hematology Association Annual Congress Evaluate Activin Receptor Ligand Trap Programs in Beta-Thalassemia 92
May 22, 2014: Acceleron Announces New Interim Phase 2 Data on ACE-536 to be Reported at Three Oral Presentations at the 19th Annual Congress of the European Hematology Association 93
May 21, 2014: Acceleron Announces New Interim Phase 2 Data on Sotatercept to be Reported at Three Oral Presentations at the 19th Annual Congress of the European Hematology Association 94
Mar 24, 2014: Acceleron, Celgene and Collaborators Announce Publications in Nature Medicine Describing ACE-536 Therapeutic Potential in Beta-thalassemia and Myelodysplastic Syndromes 95
Appendix 96
Methodology 96
Coverage 96
Secondary Research 96
Primary Research 96
Expert Panel Validation 96
Contact Us 97
Disclaimer 97

List of Tables
Number of Products under Development for Thalassemia, H2 2014 9
Number of Products under Development for Thalassemia - Comparative Analysis, H2 2014 10
Number of Products under Development by Companies, H2 2014 12
Number of Products under Investigation by Universities/Institutes, H2 2014 13
Comparative Analysis by Late Stage Development, H2 2014 14
Comparative Analysis by Clinical Stage Development, H2 2014 15
Comparative Analysis by Early Stage Development, H2 2014 16
Products under Development by Companies, H2 2014 17
Products under Investigation by Universities/Institutes, H2 2014 18
Thalassemia - Pipeline by Acceleron Pharma, Inc., H2 2014 19
Thalassemia - Pipeline by Acetylon Pharmaceuticals, Inc., H2 2014 20
Thalassemia - Pipeline by Acino Pharma AG, H2 2014 21
Thalassemia - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2014 22
Thalassemia - Pipeline by Bluebird bio, Inc., H2 2014 23
Thalassemia - Pipeline by Children's Hospital Boston, H2 2014 24
Thalassemia - Pipeline by Emmaus Medical, Inc., H2 2014 25
Thalassemia - Pipeline by Errant Gene Therapeutics, LLC, H2 2014 26
Thalassemia - Pipeline by Gamida Cell Ltd., H2 2014 27
Thalassemia - Pipeline by Incyte Corporation, H2 2014 28
Thalassemia - Pipeline by IRBM Science Park SpA, H2 2014 29
Thalassemia - Pipeline by Isis Pharmaceuticals, Inc., H2 2014 30
Thalassemia - Pipeline by Johnson & Johnson, H2 2014 31
Thalassemia - Pipeline by Protagonist Therapeutics Inc., H2 2014 32
Thalassemia - Pipeline by Sangamo BioSciences, Inc., H2 2014 33
Assessment by Monotherapy Products, H2 2014 34
Number of Products by Stage and Target, H2 2014 36
Number of Products by Stage and Mechanism of Action, H2 2014 38
Number of Products by Stage and Route of Administration, H2 2014 40
Number of Products by Stage and Molecule Type, H2 2014 42
Thalassemia Therapeutics - Recent Pipeline Updates, H2 2014 70
Thalassemia - Dormant Projects, H2 2014 88