Thalassemia - Pipeline Review, H1 2016 Summary 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
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