Thalassemia – Pipeline Review, H1 2016

$2,000

The report provides comprehensive information on the therapeutics under development for Thalassemia, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history……

Total Pages: 135

Global Markets Direct’s, ‘Thalassemia - Pipeline Review, H1 2016’, provides an overview of the Thalassemia pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Thalassemia, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Thalassemia and features dormant 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, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

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

Scope

- The report provides a snapshot of the global therapeutic landscape of Thalassemia
- The report reviews pipeline therapeutics for Thalassemia by companies and universities/research institutes based on information derived from company and industry-specific sources
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
- The report reviews key players involved Thalassemia therapeutics and enlists all their major and minor projects
- The report assesses Thalassemia therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type
- The report summarizes all the dormant and discontinued pipeline projects
- The report reviews latest news related to pipeline therapeutics for Thalassemia

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Identify and understand important and diverse types of therapeutics under development for Thalassemia
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies
- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
- 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 7
List of Figures 8
Introduction 9
Global Markets Direct Report Coverage 9
Thalassemia Overview 10
Therapeutics Development 11
Pipeline Products for Thalassemia - Overview 11
Pipeline Products for Thalassemia - Comparative Analysis 12
Thalassemia - Therapeutics under Development by Companies 13
Thalassemia - Therapeutics under Investigation by Universities/Institutes 15
Thalassemia - Pipeline Products Glance 16
Late Stage Products 16
Clinical Stage Products 17
Early Stage Products 18
Thalassemia - Products under Development by Companies 19
Thalassemia - Products under Investigation by Universities/Institutes 21
Thalassemia - Companies Involved in Therapeutics Development 22
Acceleron Pharma, Inc. 22
Acetylon Pharmaceuticals, Inc. 23
Acino Pharma AG 24
Agios Pharmaceuticals, Inc. 25
Alnylam Pharmaceuticals, Inc. 26
Bellicum Pharmaceuticals, Inc. 27
Bluebird bio, Inc. 28
Editas Medicine, Inc. 29
Emmaus Medical, Inc. 30
Errant Gene Therapeutics, LLC 31
Gamida Cell Ltd. 32
Incyte Corporation 33
Ionis Pharmaceuticals, Inc. 34
IRBM Science Park SpA 35
Johnson & Johnson 36
Kiadis Pharma B.V. 37
La Jolla Pharmaceutical Company 38
Merck & Co., Inc. 39
PharmaEssentia Corporation 40
Protagonist Therapeutics Inc. 41
Sangamo BioSciences, Inc. 42
Thalassemia - Therapeutics Assessment 43
Assessment by Monotherapy Products 43
Assessment by Target 44
Assessment by Mechanism of Action 46
Assessment by Route of Administration 48
Assessment by Molecule Type 50
Drug Profiles 52
ACY-957 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
AG-348 - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
ALN-TMP - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
Antisense Oligonucleotides to Inhibit BCL11A and KLF1 for Hematological Disorders - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
ATIR-201 - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
BB-305 - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
BPX-501 - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
BtX-13 - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
CNTO-530 - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
CordIn - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
Drug for Thalassemia and Hemochromatosis - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
glutamine - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
GSK-2696277 - Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
ISIS-TMPRSS6LRx - Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
LJPC-401 - Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
luspatercept - Drug Profile 72
Product Description 72
Mechanism of Action 72
R&D Progress 72
M-012 - Drug Profile 74
Product Description 74
Mechanism of Action 74
R&D Progress 74
NiCord - Drug Profile 76
Product Description 76
Mechanism of Action 76
R&D Progress 76
PB-04 - Drug Profile 77
Product Description 77
Mechanism of Action 77
R&D Progress 77
Peptide Agonists of Hepcidin for Beta-Thalassemia and Juvenile Hemochromatosis - Drug Profile 79
Product Description 79
Mechanism of Action 79
R&D Progress 79
RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile 80
Product Description 80
Mechanism of Action 80
R&D Progress 80
ruxolitinib phosphate - Drug Profile 81
Product Description 81
Mechanism of Action 81
R&D Progress 81
SCD-101 - Drug Profile 86
Product Description 86
Mechanism of Action 86
R&D Progress 86
Small Molecule for Beta Thalassemia and Sickle Cell Anemia - Drug Profile 87
Product Description 87
Mechanism of Action 87
R&D Progress 87
Small Molecule to Inhibit Histone Deacetylase 1 for Beta-Thalassemia - Drug Profile 88
Product Description 88
Mechanism of Action 88
R&D Progress 88
Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours - Drug Profile 89
Product Description 89
Mechanism of Action 89
R&D Progress 89
sotatercept - Drug Profile 90
Product Description 90
Mechanism of Action 90
R&D Progress 90
Stem Cell Therapy for Beta Thalassemia - Drug Profile 92
Product Description 92
Mechanism of Action 92
R&D Progress 92
Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease - Drug Profile 93
Product Description 93
Mechanism of Action 93
R&D Progress 93
Thalagen - Drug Profile 94
Product Description 94
Mechanism of Action 94
R&D Progress 94
Thalassemia - Recent Pipeline Updates 95
Thalassemia - Dormant Projects 126
Thalassemia - Product Development Milestones 127
Featured News & Press Releases 127
Feb 24, 2016: Merganser Biotech Initiates First Clinical Trial 127
Dec 07, 2015: Acetylon Presents Data on the Pharmacokinetics and HbE/HbG Hemoglobin Induction Following Once Daily Dosing of an Oral HDAC1,2 Inhibitor in Animal Models 127
Dec 02, 2015: Emmaus Life Sciences to Present Additional New Data from its Phase 3 Sickle Cell Disease Trial at 57th Annual ASH Meeting 128
Nov 05, 2015: Acceleron Announces New Data on Luspatercept at the 2015 American Society of Hematology Annual Meeting 128
Oct 23, 2015: Acceleron Highlights Phase 3 Studies, New Clinical Results and Research Strategies at Research and Development Day Event 129
Oct 19, 2015: La Jolla Pharmaceutical Doses First Patient in Phase 1 Clinical Trial of LJPC-401 in Patients at Risk of Iron Overload 130
Aug 11, 2015: La Jolla Pharmaceutical Company Announces FDA Acceptance of IND for LJPC-401 131
Jun 12, 2015: Acceleron Announces New Luspatercept Phase 2 Clinical Results at the 20th Congress of the European Hematology Association 131
Jun 09, 2015: Acceleron Pharma to Host Conference Call and Webcast to Review New Luspatercept Data Presented at the 20th Congress of the European Hematology Association 132
May 21, 2015: Acceleron to Present Luspatercept Clinical Trials at the 20th Congress of the European Hematology Association 133
Appendix 134
Methodology 134
Coverage 134
Secondary Research 134
Primary Research 134
Expert Panel Validation 134
Contact Us 134
Disclaimer 135

List of Tables
Number of Products under Development for Thalassemia, H1 2016 11
Number of Products under Development for Thalassemia - Comparative Analysis, H1 2016 12
Number of Products under Development by Companies, H1 2016 14
Number of Products under Investigation by Universities/Institutes, H1 2016 15
Comparative Analysis by Late Stage Development, H1 2016 16
Comparative Analysis by Clinical Stage Development, H1 2016 17
Comparative Analysis by Early Stage Development, H1 2016 18
Products under Development by Companies, H1 2016 19
Products under Development by Companies, H1 2016 (Contd..1) 20
Products under Investigation by Universities/Institutes, H1 2016 21
Thalassemia - Pipeline by Acceleron Pharma, Inc., H1 2016 22
Thalassemia - Pipeline by Acetylon Pharmaceuticals, Inc., H1 2016 23
Thalassemia - Pipeline by Acino Pharma AG, H1 2016 24
Thalassemia - Pipeline by Agios Pharmaceuticals, Inc., H1 2016 25
Thalassemia - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2016 26
Thalassemia - Pipeline by Bellicum Pharmaceuticals, Inc., H1 2016 27
Thalassemia - Pipeline by Bluebird bio, Inc., H1 2016 28
Thalassemia - Pipeline by Editas Medicine, Inc., H1 2016 29
Thalassemia - Pipeline by Emmaus Medical, Inc., H1 2016 30
Thalassemia - Pipeline by Errant Gene Therapeutics, LLC, H1 2016 31
Thalassemia - Pipeline by Gamida Cell Ltd., H1 2016 32
Thalassemia - Pipeline by Incyte Corporation, H1 2016 33
Thalassemia - Pipeline by Ionis Pharmaceuticals, Inc., H1 2016 34
Thalassemia - Pipeline by IRBM Science Park SpA, H1 2016 35
Thalassemia - Pipeline by Johnson & Johnson, H1 2016 36
Thalassemia - Pipeline by Kiadis Pharma B.V., H1 2016 37
Thalassemia - Pipeline by La Jolla Pharmaceutical Company, H1 2016 38
Thalassemia - Pipeline by Merck & Co., Inc., H1 2016 39
Thalassemia - Pipeline by PharmaEssentia Corporation, H1 2016 40
Thalassemia - Pipeline by Protagonist Therapeutics Inc., H1 2016 41
Thalassemia - Pipeline by Sangamo BioSciences, Inc., H1 2016 42
Assessment by Monotherapy Products, H1 2016 43
Number of Products by Stage and Target, H1 2016 45
Number of Products by Stage and Mechanism of Action, H1 2016 47
Number of Products by Stage and Route of Administration, H1 2016 49
Number of Products by Stage and Molecule Type, H1 2016 51
Thalassemia Therapeutics - Recent Pipeline Updates, H1 2016 95
Thalassemia - Dormant Projects, H1 2016 126

List of Figures
Number of Products under Development for Thalassemia, H1 2016 11
Number of Products under Development for Thalassemia - Comparative Analysis, H1 2016 12
Number of Products under Development by Companies, H1 2016 13
Number of Products under Investigation by Universities/Institutes, H1 2016 15
Comparative Analysis by Clinical Stage Development, H1 2016 17
Comparative Analysis by Early Stage Products, H1 2016 18
Assessment by Monotherapy Products, H1 2016 43
Number of Products by Top 10 Targets, H1 2016 44
Number of Products by Stage and Top 10 Targets, H1 2016 44
Number of Products by Top 10 Mechanism of Actions, H1 2016 46
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2016 46
Number of Products by Routes of Administration, H1 2016 48
Number of Products by Stage and Routes of Administration, H1 2016 48
Number of Products by Top 10 Molecule Types, H1 2016 50
Number of Products by Stage and Top 10 Molecule Types, H1 2016 50

Companies Mentioned

Acceleron Pharma, Inc.
Acetylon Pharmaceuticals, Inc.
Acino Pharma AG
Agios Pharmaceuticals, Inc.
Alnylam Pharmaceuticals, Inc.
Bellicum Pharmaceuticals, Inc.
Bluebird bio, Inc.
Editas Medicine, Inc.
Emmaus Medical, Inc.
Errant Gene Therapeutics, LLC
Gamida Cell Ltd.
Incyte Corporation
Ionis Pharmaceuticals, Inc.
IRBM Science Park SpA
Johnson & Johnson
Kiadis Pharma B.V.
La Jolla Pharmaceutical Company
Merck & Co., Inc.
PharmaEssentia Corporation
Protagonist Therapeutics Inc.
Sangamo BioSciences, Inc.


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