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Thalassemia - Pipeline Review, H1 2015

Published: Feb-2015 | Format: PDF | Global Markets Direct | Number of pages: 100 | Code: MRS - 7023

 

Global Markets Direct’s, ‘Thalassemia Pipeline Review, H1 2015’, 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.

 

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 12
Thalassemia - Pipeline Products Glance 13
Late Stage Products 13
Clinical Stage Products 14
Early Stage Products 15
Thalassemia - Products under Development by Companies 16
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
Emmaus Medical, Inc. 24
Errant Gene Therapeutics, LLC 25
Gamida Cell Ltd. 26
Incyte Corporation 27
IRBM Science Park SpA 28
Isis Pharmaceuticals, Inc. 29
Johnson & Johnson 30
Protagonist Therapeutics Inc. 31
Sangamo BioSciences, Inc. 32
Thalassemia - Therapeutics Assessment 33
Assessment by Monotherapy Products 33
Assessment by Target 34
Assessment by Mechanism of Action 36
Assessment by Route of Administration 38
Assessment by Molecule Type 40
Drug Profiles 42
ACY-957 - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
ALN-TMP - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
Antisense Oligonucleotides to Inhibit BCL11A and KLF1 for Hematological Disorders - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
BB-305 - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
BtX-13 - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
CNTO-530 - 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
luspatercept - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
NiCord - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
PB-04 - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
Peptide Agonists of Hepcidin for Beta-Thalassemia and Juvenile Hemochromatosis - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Project x-1450 - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
ruxolitinib phosphate - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
Small Molecule to Inhibit Histone Deacetylase for Beta-Thalassemia - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
sotatercept - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
Stem Cell Therapy for Beta Thalassemia - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
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 90
Thalassemia - Product Development Milestones 91
Featured News & Press Releases 91
Feb 04, 2015: Sangamo BioSciences Announces FDA Acceptance of IND to Initiate Clinical Trial of its Novel ZFP Therapeutic for Beta-Thalassemia 91
Dec 08, 2014: Acetylon Pharmaceuticals Presents Positive Preclinical Data on Selective HDAC1/2 Inhibition for the Treatment of Sickle Cell Disease/ß-thalassemia 92
Dec 07, 2014: New Data from Luspatercept Phase 2 Clinical Trial in Beta-Thalassemia Presented at the 56th American Society of Hematology (ASH) Annual Meeting and Exposition 92
Nov 06, 2014: Acceleron Announces New Clinical Data Presentations on luspatercept at the 2014 American Society of Hematology Annual Meeting 94
Nov 06, 2014: Celgene To Present Phase II trial Results Update On Luspatercept at American Society of Hematology Annual Meeting 95
Nov 06, 2014: Acceleron Announces New Clinical Data Presentations on sotatercept at the 2014 American Society of Hematology Annual Meeting 95
Oct 01, 2014: Acceleron to Host Educational Webcast on Luspatercept 96
Oct 01, 2014: Acceleron to Host Educational Webcast on Beta-thalassemia 96
Jun 14, 2014: Celgene and Acceleron announces interim data from ACE-536 at the 19th European Hematology Association annual congress 96
Jun 14, 2014: Studies Presented at 19th European Hematology Association Annual Congress Evaluate Activin Receptor Ligand Trap Programs in Beta-Thalassemia 97

 

Appendix 99
Methodology 99
Coverage 99
Secondary Research 99
Primary Research 99
Expert Panel Validation 99
Contact Us 99
Disclaimer 100


List of Tables
 

Number of Products under Development for Thalassemia, H1 2015 9
Number of Products under Development for Thalassemia - Comparative Analysis, H1 2015 10
Number of Products under Development by Companies, H1 2015 11
Number of Products under Investigation by Universities/Institutes, H1 2015 12
Comparative Analysis by Late Stage Development, H1 2015 13
Comparative Analysis by Clinical Stage Development, H1 2015 14
Comparative Analysis by Early Stage Development, H1 2015 15
Products under Development by Companies, H1 2015 16
Products under Development by Companies, H1 2015 (Contd..1) 17
Products under Investigation by Universities/Institutes, H1 2015 18
Thalassemia - Pipeline by Acceleron Pharma, Inc., H1 2015 19
Thalassemia - Pipeline by Acetylon Pharmaceuticals, Inc., H1 2015 20
Thalassemia - Pipeline by Acino Pharma AG, H1 2015 21
Thalassemia - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2015 22
Thalassemia - Pipeline by Bluebird bio, Inc., H1 2015 23
Thalassemia - Pipeline by Emmaus Medical, Inc., H1 2015 24
Thalassemia - Pipeline by Errant Gene Therapeutics, LLC, H1 2015 25
Thalassemia - Pipeline by Gamida Cell Ltd., H1 2015 26
Thalassemia - Pipeline by Incyte Corporation, H1 2015 27
Thalassemia - Pipeline by IRBM Science Park SpA, H1 2015 28
Thalassemia - Pipeline by Isis Pharmaceuticals, Inc., H1 2015 29
Thalassemia - Pipeline by Johnson & Johnson, H1 2015 30
Thalassemia - Pipeline by Protagonist Therapeutics Inc., H1 2015 31
Thalassemia - Pipeline by Sangamo BioSciences, Inc., H1 2015 32
Assessment by Monotherapy Products, H1 2015 33
Number of Products by Stage and Target, H1 2015 35
Number of Products by Stage and Mechanism of Action, H1 2015 37
Number of Products by Stage and Route of Administration, H1 2015 39
Number of Products by Stage and Molecule Type, H1 2015 41
Thalassemia Therapeutics - Recent Pipeline Updates, H1 2015 70
Thalassemia - Dormant Projects, H1 2015 90"

 

List of Figures
 

Number of Products under Development for Thalassemia, H1 2015 9
Number of Products under Development for Thalassemia - Comparative Analysis, H1 2015 10
Number of Products under Development by Companies, H1 2015 11
Comparative Analysis by Clinical Stage Development, H1 2015 14
Comparative Analysis by Early Stage Products, H1 2015 15
Assessment by Monotherapy Products, H1 2015 33
Number of Products by Top 10 Targets, H1 2015 34
Number of Products by Stage and Top 10 Targets, H1 2015 34
Number of Products by Top 10 Mechanism of Actions, H1 2015 36
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 37
Number of Products by Top 10 Routes of Administration, H1 2015 38
Number of Products by Stage and Top 10 Routes of Administration, H1 2015 39
Number of Products by Top 10 Molecule Types, H1 2015 40
Number of Products by Stage and Top 10 Molecule Types, H1 2015 41

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