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Published: Oct, 2014 | Pages:
164 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)
Sickle Cell Disease - Pipeline Review, H2 2014 Summary Global Markets Direct's, 'Sickle Cell Disease - Pipeline Review, H2 2014', provides an overview of the Sickle Cell Disease's therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Sickle Cell Disease, 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 Sickle Cell Disease 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 Sickle Cell Disease - 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 Sickle Cell Disease and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the Sickle Cell Disease 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 Sickle Cell Disease 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 Sickle Cell Disease - Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline - Devise corrective measures for pipeline projects by understanding Sickle Cell Disease 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 4 List of Figures 5 Introduction 7 Global Markets Direct Report Coverage 7 Sickle Cell Disease Overview 8 Therapeutics Development 9 Pipeline Products for Sickle Cell Disease - Overview 9 Pipeline Products for Sickle Cell Disease - Comparative Analysis 10 Sickle Cell Disease - Therapeutics under Development by Companies 11 Sickle Cell Disease - Therapeutics under Investigation by Universities/Institutes 13 Sickle Cell Disease - Pipeline Products Glance 14 Late Stage Products 14 Clinical Stage Products 15 Early Stage Products 16 Sickle Cell Disease - Products under Development by Companies 17 Sickle Cell Disease - Products under Investigation by Universities/Institutes 20 Sickle Cell Disease - Companies Involved in Therapeutics Development 21 Acceleron Pharma, Inc. 21 Acetylon Pharmaceuticals, Inc. 22 Addex Therapeutics Ltd 23 Advinus Therapeutics Ltd. 24 AesRx, LLC. 25 Alnylam Pharmaceuticals, Inc. 26 Archemix Corp. 27 Bluebird bio, Inc. 28 Bristol-Myers Squibb Company 29 Children's Hospital Boston 30 Daiichi Sankyo Company, Limited 31 Dilaforette AB 32 Emmaus Medical, Inc. 33 Errant Gene Therapeutics, LLC 34 Erytech Pharma SA 35 Gamida Cell Ltd. 36 Gilead Sciences, Inc. 37 Global Blood Therapeutics, Inc. 38 Isis Pharmaceuticals, Inc. 39 Johnson & Johnson 40 Mast Therapeutics, Inc. 41 Morphogenesis, Inc. 42 NKT Therapeutics, Inc. 43 Novartis AG 44 OrphageniX, Inc. 45 Pfizer Inc. 46 Prolong Pharmaceuticals 47 ReveraGen BioPharma, Inc. 48 Sangamo BioSciences, Inc. 49 Sanofi 50 SynZyme Technologies, LLC 51 Sickle Cell Disease - Therapeutics Assessment 52 Assessment by Monotherapy Products 52 Assessment by Target 53 Assessment by Mechanism of Action 55 Assessment by Route of Administration 57 Assessment by Molecule Type 59 Drug Profiles 61 ACY-957 - Drug Profile 61 ADX-85142 - Drug Profile 62 ADX-94819 - Drug Profile 63 Aes-103 - Drug Profile 64 AIC-2011 - Drug Profile 66 AIC-6020 - Drug Profile 67 ALN-TMP - Drug Profile 68 Antisense Oligonucleotide to Inhibit BCL11A and KLF1 for Hematological Disorders - Drug Profile 69 apixaban - Drug Profile 70 ARC-5690 - Drug Profile 72 Cell Therapy for Sickle Cell Disease - Drug Profile 73 CNTO-530 - Drug Profile 74 DNA Oligonucleotides for Sickle Cell Anemia - Drug Profile 75 Drugs to Inhibit PRMT5 for Sickle Cell Anemia - Drug Profile 76 EdX-17 - Drug Profile 77 Enhoxy - Drug Profile 78 Gene Therapy for Hemoglobinopathies - Drug Profile 79 Gene Therapy for Sickle Cell Anemia - Drug Profile 80 Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile 81 Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile 82 Gene Therapy to Activate Gamma Globin for Sickle Cell Disease - Drug Profile 83 glutamine - Drug Profile 84 GTX-011 - Drug Profile 86 LentiGlobin - Drug Profile 87 luspatercept - Drug Profile 89 NiCord - Drug Profile 90 NKTT-120 - Drug Profile 91 panobinostat - Drug Profile 92 PB-04 - Drug Profile 98 PF-4447943 - Drug Profile 100 plerixafor - Drug Profile 101 PNQ-103 - Drug Profile 103 prasugrel hydrochloride - Drug Profile 104 regadenoson - Drug Profile 106 RN-1 - Drug Profile 108 Sanguinate - Drug Profile 109 SCPF - Drug Profile 111 sevuparin sodium - Drug Profile 112 Small Molecule to Agonize A2A for Immunology, Hematological and Neurological Disorders - Drug Profile 113 Small Molecules to Inhibit TNFR1 for Immunology, Gastrointestinal, Neurology and Hematological Disorders - Drug Profile 114 Stem Cell Therapy to Activate Beta Globin for Sickle Cell Disease - Drug Profile 115 Synthetic Peptides Hematological and Genetic Disorders - Drug Profile 116 Trichostatin A - Drug Profile 118 VBP-15 - Drug Profile 119 vepoloxamer - Drug Profile 121 VitalHeme - Drug Profile 122 Sickle Cell Disease - Recent Pipeline Updates 124 Sickle Cell Disease - Dormant Projects 150 Sickle Cell Disease - Discontinued Products 151 Sickle Cell Disease - Product Development Milestones 152 Featured News & Press Releases 152 Appendix 160 Methodology 160 Coverage 160 Secondary Research 160 Primary Research 160 Expert Panel Validation 160 Contact Us 161 Disclaimer 161
List of Tables Number of Products under Development for Sickle Cell Disease, H2 2014 12 Number of Products under Development for Sickle Cell Disease - Comparative Analysis, H2 2014 13 Number of Products under Development by Companies, H2 2014 14 Number of Products under Investigation by Universities/Institutes, H2 2014 16 Comparative Analysis by Late Stage Development, H2 2014 17 Comparative Analysis by Clinical Stage Development, H2 2014 18 Comparative Analysis by Early Stage Development, H2 2014 19 Products under Development by Companies, H2 2014 20 Products under Development by Companies, H2 2014 (Contd..1) 21 Products under Development by Companies, H2 2014 (Contd..2) 22 Products under Investigation by Universities/Institutes, H2 2014 23 Sickle Cell Disease - Pipeline by Acceleron Pharma, Inc., H2 2014 24 Sickle Cell Disease - Pipeline by Acetylon Pharmaceuticals, Inc., H2 2014 25 Sickle Cell Disease - Pipeline by Addex Therapeutics Ltd, H2 2014 26 Sickle Cell Disease - Pipeline by Advinus Therapeutics Ltd., H2 2014 27 Sickle Cell Disease - Pipeline by AesRx, LLC., H2 2014 28 Sickle Cell Disease - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2014 29 Sickle Cell Disease - Pipeline by Archemix Corp., H2 2014 30 Sickle Cell Disease - Pipeline by Bluebird bio, Inc., H2 2014 31 Sickle Cell Disease - Pipeline by Bristol-Myers Squibb Company, H2 2014 32 Sickle Cell Disease - Pipeline by Children's Hospital Boston, H2 2014 33 Sickle Cell Disease - Pipeline by Daiichi Sankyo Company, Limited, H2 2014 34 Sickle Cell Disease - Pipeline by Dilaforette AB, H2 2014 35 Sickle Cell Disease - Pipeline by Emmaus Medical, Inc., H2 2014 36 Sickle Cell Disease - Pipeline by Errant Gene Therapeutics, LLC, H2 2014 37 Sickle Cell Disease - Pipeline by Erytech Pharma SA, H2 2014 38 Sickle Cell Disease - Pipeline by Gamida Cell Ltd., H2 2014 39 Sickle Cell Disease - Pipeline by Gilead Sciences, Inc., H2 2014 40 Sickle Cell Disease - Pipeline by Global Blood Therapeutics, Inc., H2 2014 41 Sickle Cell Disease - Pipeline by Isis Pharmaceuticals, Inc., H2 2014 42 Sickle Cell Disease - Pipeline by Johnson & Johnson, H2 2014 43 Sickle Cell Disease - Pipeline by Mast Therapeutics, Inc., H2 2014 44 Sickle Cell Disease - Pipeline by Morphogenesis, Inc., H2 2014 45 Sickle Cell Disease - Pipeline by NKT Therapeutics, Inc., H2 2014 46 Sickle Cell Disease - Pipeline by Novartis AG, H2 2014 47 Sickle Cell Disease - Pipeline by OrphageniX, Inc., H2 2014 48 Sickle Cell Disease - Pipeline by Pfizer Inc., H2 2014 49 Sickle Cell Disease - Pipeline by Prolong Pharmaceuticals, H2 2014 50 Sickle Cell Disease - Pipeline by ReveraGen BioPharma, Inc., H2 2014 51 Sickle Cell Disease - Pipeline by Sangamo BioSciences, Inc., H2 2014 52 Sickle Cell Disease - Pipeline by Sanofi, H2 2014 53 Sickle Cell Disease - Pipeline by SynZyme Technologies, LLC, H2 2014 54 Assessment by Monotherapy Products, H2 2014 55 Number of Products by Stage and Target, H2 2014 57 Number of Products by Stage and Mechanism of Action, H2 2014 59 Number of Products by Stage and Route of Administration, H2 2014 61 Number of Products by Stage and Molecule Type, H2 2014 63 Sickle Cell Disease Therapeutics - Recent Pipeline Updates, H2 2014 127 Sickle Cell Disease - Dormant Projects, H2 2014 153 Sickle Cell Disease - Discontinued Products, H2 2014 154
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