Thalassemia - Pipeline Review, H2 2016

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Thalassemia - Pipeline Review, H2 2016 Summary Global Markets Directs, Thalassemia - Pipeline Review, H2 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 Directs 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 Directs 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 its 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 8 List of Figures 9 Introduction 10 Global Markets Direct Report Coverage 10 Thalassemia Overview 11 Therapeutics Development 12 Pipeline Products for Thalassemia - Overview 12 Pipeline Products for Thalassemia - Comparative Analysis 13 Thalassemia - Therapeutics under Development by Companies 14 Thalassemia - Therapeutics under Investigation by Universities/Institutes 16 Thalassemia - Pipeline Products Glance 17 Late Stage Products 17 Clinical Stage Products 18 Early Stage Products 19 Thalassemia - Products under Development by Companies 20 Thalassemia - Products under Investigation by Universities/Institutes 22 Thalassemia - Companies Involved in Therapeutics Development 23 Acceleron Pharma, Inc. 23 Acetylon Pharmaceuticals, Inc. 24 Acino International AG 25 Agios Pharmaceuticals, Inc. 26 Alnylam Pharmaceuticals, Inc. 27 Bellicum Pharmaceuticals, Inc. 28 Bluebird bio, Inc. 29 Editas Medicine, Inc. 30 Emmaus Medical, Inc. 31 Errant Gene Therapeutics, LLC 32 Gamida Cell Ltd. 33 Gilead Sciences, Inc. 34 Incyte Corporation 35 Ionis Pharmaceuticals, Inc. 36 IRBM Science Park SpA 37 Johnson & Johnson 38 Kiadis Pharma N.V. 39 La Jolla Pharmaceutical Company 40 Merck & Co., Inc. 41 Pfizer Inc. 42 PharmaEssentia Corporation 43 Sangamo BioSciences, Inc. 44 Thalassemia - Therapeutics Assessment 45 Assessment by Monotherapy Products 45 Assessment by Target 46 Assessment by Mechanism of Action 48 Assessment by Route of Administration 50 Assessment by Molecule Type 52 Drug Profiles 54 ACY-957 - Drug Profile 54 Product Description 54 Mechanism Of Action 54 R&D Progress 54 AG-348 - Drug Profile 56 Product Description 56 Mechanism Of Action 56 R&D Progress 56 ALN-TMP - Drug Profile 59 Product Description 59 Mechanism Of Action 59 R&D Progress 59 ambrisentan - Drug Profile 61 Product Description 61 Mechanism Of Action 61 R&D Progress 61 Antisense Oligonucleotides to Inhibit BCL11A and KLF1 for Hematological Disorders - Drug Profile 63 Product Description 63 Mechanism Of Action 63 R&D Progress 63 ATIR-201 - Drug Profile 64 Product Description 64 Mechanism Of Action 64 R&D Progress 64 BB-305 - Drug Profile 65 Product Description 65 Mechanism Of Action 65 R&D Progress 65 benserazide - Drug Profile 71 Product Description 71 Mechanism Of Action 71 R&D Progress 71 BPX-501 - Drug Profile 73 Product Description 73 Mechanism Of Action 73 R&D Progress 73 BtX-13 - Drug Profile 77 Product Description 77 Mechanism Of Action 77 R&D Progress 77 CNTO-530 - Drug Profile 78 Product Description 78 Mechanism Of Action 78 R&D Progress 78 CordIn - Drug Profile 79 Product Description 79 Mechanism Of Action 79 R&D Progress 79 Drug for Thalassemia and Hemochromatosis - Drug Profile 80 Product Description 80 Mechanism Of Action 80 R&D Progress 80 Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile 81 Product Description 81 Mechanism Of Action 81 R&D Progress 81 glutamine - Drug Profile 82 Product Description 82 Mechanism Of Action 82 R&D Progress 82 GSK-2696277 - Drug Profile 85 Product Description 85 Mechanism Of Action 85 R&D Progress 85 IONISTMPRSS-6LRx - Drug Profile 86 Product Description 86 Mechanism Of Action 86 R&D Progress 86 LJPC-401 - Drug Profile 87 Product Description 87 Mechanism Of Action 87 R&D Progress 87 luspatercept - Drug Profile 88 Product Description 88 Mechanism Of Action 88 R&D Progress 88 M-009 - Drug Profile 94 Product Description 94 Mechanism Of Action 94 R&D Progress 94 M-012 - Drug Profile 95 Product Description 95 Mechanism Of Action 95 R&D Progress 95 NiCord - Drug Profile 97 Product Description 97 Mechanism Of Action 97 R&D Progress 97 PF-04447943 - Drug Profile 100 Product Description 100 Mechanism Of Action 100 R&D Progress 100 Piggybac-HBB - Drug Profile 101 Product Description 101 Mechanism Of Action 101 R&D Progress 101 PTG-300 - Drug Profile 102 Product Description 102 Mechanism Of Action 102 R&D Progress 102 RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile 103 Product Description 103 Mechanism Of Action 103 R&D Progress 103 ruxolitinib phosphate - Drug Profile 104 Product Description 104 Mechanism Of Action 104 R&D Progress 104 SCD-101 - Drug Profile 117 Product Description 117 Mechanism Of Action 117 R&D Progress 117 sirolimus - Drug Profile 118 Product Description 118 Mechanism Of Action 118 R&D Progress 118 Small Molecule for Beta Thalassemia and Sickle Cell Anemia - Drug Profile 119 Product Description 119 Mechanism Of Action 119 R&D Progress 119 Small Molecule to Inhibit Histone Deacetylase 1 for Beta-Thalassemia - Drug Profile 120 Product Description 120 Mechanism Of Action 120 R&D Progress 120 Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours - Drug Profile 121 Product Description 121 Mechanism Of Action 121 R&D Progress 121 sotatercept - Drug Profile 122 Product Description 122 Mechanism Of Action 122 R&D Progress 122 Stem Cell Therapy for Beta Thalassemia - Drug Profile 127 Product Description 127 Mechanism Of Action 127 R&D Progress 127 Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease - Drug Profile 128 Product Description 128 Mechanism Of Action 128 R&D Progress 128 Thalagen - Drug Profile 129 Product Description 129 Mechanism Of Action 129 R&D Progress 129 Thalassemia - Dormant Projects 130 Thalassemia - Product Development Milestones 131 Featured News & Press Releases 131 Jul 28, 2016: Protagonist Therapeutics Receives SBIR Funding for the Development of Injectable Hepcidin Mimetics for Treatment of Iron Overload Disorders 131 Jun 10, 2016: Acceleron and Celgene Announce Updated Results from an Ongoing Phase 2 Study of Luspatercept in Beta-Thalassemia Presented at the 21st Congress of the European Hematology Association 131 May 19, 2016: Agios to Present Clinical data from PKR Activator AG-348 at the 21st Congress of the European Hematology Association 132 May 19, 2016: Acceleron Announces Data Presentations with Luspatercept at the 21st Congress of the European Hematology Association 133 May 09, 2016: Merganser Biotech Announces Issuance of U.S. Patent 9,315,545 Covering Development Compound M012 134 May 06, 2016: Drug-like Compound Shows Promise in Treating Two Blood Diseases 134 May 04, 2016: Acetylon Pharmaceuticals Publishes New Mechanism for the Treatment of Sickle Cell Disease/Beta-Thalassemia with Selective HDAC1/2 Inhibition in PLoS One 135 Feb 24, 2016: Merganser Biotech Initiates First Clinical Trial 136 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 136 Dec 02, 2015: Emmaus Life Sciences to Present Additional New Data from its Phase 3 Sickle Cell Disease Trial at 57th Annual ASH Meeting 137 Nov 05, 2015: Acceleron Announces New Data on Luspatercept at the 2015 American Society of Hematology Annual Meeting 138 Oct 23, 2015: Acceleron Highlights Phase 3 Studies, New Clinical Results and Research Strategies at Research and Development Day Event 139 Oct 19, 2015: La Jolla Pharmaceutical Doses First Patient in Phase 1 Clinical Trial of LJPC-401 in Patients at Risk of Iron Overload 139 Aug 11, 2015: La Jolla Pharmaceutical Company Announces FDA Acceptance of IND for LJPC-401 140 Jun 12, 2015: Acceleron Announces New Luspatercept Phase 2 Clinical Results at the 20th Congress of the European Hematology Association 141 Appendix 143 Methodology 143 Coverage 143 Secondary Research 143 Primary Research 143 Expert Panel Validation 143 Contact Us 143 Disclaimer 144

List of Tables Number of Products under Development for Thalassemia, H2 2016 12 Number of Products under Development for Thalassemia - Comparative Analysis, H2 2016 13 Number of Products under Development by Companies, H2 2016 14 Number of Products under Development by Companies, H2 2016 (Contd..1) 15 Number of Products under Investigation by Universities/Institutes, H2 2016 16 Comparative Analysis by Late Stage Development, H2 2016 17 Comparative Analysis by Clinical Stage Development, H2 2016 18 Comparative Analysis by Early Stage Development, H2 2016 19 Products under Development by Companies, H2 2016 20 Products under Development by Companies, H2 2016 (Contd..1) 21 Products under Investigation by Universities/Institutes, H2 2016 22 Thalassemia - Pipeline by Acceleron Pharma, Inc., H2 2016 23 Thalassemia - Pipeline by Acetylon Pharmaceuticals, Inc., H2 2016 24 Thalassemia - Pipeline by Acino International AG, H2 2016 25 Thalassemia - Pipeline by Agios Pharmaceuticals, Inc., H2 2016 26 Thalassemia - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2016 27 Thalassemia - Pipeline by Bellicum Pharmaceuticals, Inc., H2 2016 28 Thalassemia - Pipeline by bluebird bio, Inc., H2 2016 29 Thalassemia - Pipeline by Editas Medicine, Inc., H2 2016 30 Thalassemia - Pipeline by Emmaus Medical, Inc., H2 2016 31 Thalassemia - Pipeline by Errant Gene Therapeutics, LLC, H2 2016 32 Thalassemia - Pipeline by Gamida Cell Ltd., H2 2016 33 Thalassemia - Pipeline by Gilead Sciences, Inc., H2 2016 34 Thalassemia - Pipeline by Incyte Corporation, H2 2016 35 Thalassemia - Pipeline by Ionis Pharmaceuticals, Inc., H2 2016 36 Thalassemia - Pipeline by IRBM Science Park SpA, H2 2016 37 Thalassemia - Pipeline by Johnson & Johnson, H2 2016 38 Thalassemia - Pipeline by Kiadis Pharma N.V., H2 2016 39 Thalassemia - Pipeline by La Jolla Pharmaceutical Company, H2 2016 40 Thalassemia - Pipeline by Merck & Co., Inc., H2 2016 41 Thalassemia - Pipeline by Pfizer Inc., H2 2016 42 Thalassemia - Pipeline by PharmaEssentia Corporation, H2 2016 43 Thalassemia - Pipeline by Sangamo BioSciences, Inc., H2 2016 44 Assessment by Monotherapy Products, H2 2016 45 Number of Products by Stage and Target, H2 2016 47 Number of Products by Stage and Mechanism of Action, H2 2016 49 Number of Products by Stage and Route of Administration, H2 2016 51 Number of Products by Stage and Molecule Type, H2 2016 53 Thalassemia - Dormant Projects, H2 2016 130


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