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Published: May, 2015 | Pages:
168 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Sickle Cell Disease - Pipeline Review, H1 2015 Summary Global Markets Direct's, 'Sickle Cell Disease - Pipeline Review, H1 2015', 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 14 Sickle Cell Disease - Pipeline Products Glance 15 Late Stage Products 15 Clinical Stage Products 16 Early Stage Products 17 Sickle Cell Disease - Products under Development by Companies 18 Sickle Cell Disease - Products under Investigation by Universities/Institutes 21 Sickle Cell Disease - Companies Involved in Therapeutics Development 22 Acceleron Pharma, Inc. 22 Acetylon Pharmaceuticals, Inc. 23 Addex Therapeutics Ltd 24 Advinus Therapeutics Ltd. 25 Alnylam Pharmaceuticals, Inc. 26 Bluebird bio, Inc. 27 Bristol-Myers Squibb Company 28 Daiichi Sankyo Company, Limited 29 Dilaforette AB 30 Emmaus Medical, Inc. 31 Errant Gene Therapeutics, LLC 32 Erytech Pharma SA 33 Gamida Cell Ltd. 34 Gilead Sciences, Inc. 35 Global Blood Therapeutics, Inc. 36 Isis Pharmaceuticals, Inc. 37 Johnson & Johnson 38 Morphogenesis, Inc. 39 NKT Therapeutics, Inc. 40 Novartis AG 41 Pfizer Inc. 42 PharmaEssentia Corporation 43 Prolong Pharmaceuticals 44 ReveraGen BioPharma, Inc. 45 Sangamo BioSciences, Inc. 46 Sanofi 47 SynZyme Technologies, LLC 48 Sickle Cell Disease - Therapeutics Assessment 49 Assessment by Monotherapy Products 49 Assessment by Target 50 Assessment by Mechanism of Action 52 Assessment by Route of Administration 54 Assessment by Molecule Type 56 Drug Profiles 58 ACY-957 - Drug Profile 58 ADX-85142 - Drug Profile 59 ADX-94819 - Drug Profile 60 AIC-2011 - Drug Profile 61 AIC-6020 - Drug Profile 62 ALN-TMP - Drug Profile 63 Antisense Oligonucleotides to Inhibit BCL11A and KLF1 for Hematological Disorders - Drug Profile 64 apixaban - Drug Profile 65 BAX-555 - Drug Profile 67 BB-305 - Drug Profile 69 CNTO-530 - Drug Profile 71 Didox - Drug Profile 72 Drugs to Inhibit PRMT5 for Sickle Cell Anemia - Drug Profile 76 EdX-17 - Drug Profile 77 Enhoxy - Drug Profile 78 GBT-440 - Drug Profile 79 Gene Therapy for Sickle Cell Anemia - Drug Profile 80 Gene Therapy for Sickle Cell Disease - Drug Profile 81 Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile 82 Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile 83 Gene Therapy to Activate Gamma Globin for Sickle Cell Disease - Drug Profile 84 glutamine - Drug Profile 85 luspatercept - Drug Profile 87 NiCord - Drug Profile 88 NKTT-120 - Drug Profile 89 panobinostat - Drug Profile 90 PB-04 - Drug Profile 96 PF-4447943 - Drug Profile 97 plerixafor - Drug Profile 98 PNQ-103 - Drug Profile 100 prasugrel hydrochloride - Drug Profile 101 Protein for Sickle Cell Disease - Drug Profile 104 regadenoson - Drug Profile 105 RN-1 - Drug Profile 107 Sanguinate - Drug Profile 108 SCD-101 - Drug Profile 110 SCPF - Drug Profile 111 sevuparin sodium - Drug Profile 112 Small Molecule for Beta Thalassemia and Sickle Cell Anemia - Drug Profile 114 Small Molecule to Agonize A2A for Immunology, Hematological and Neurological Disorders - Drug Profile 115 Small Molecule to Inhibit KDM1A for AML, MDS and Sickle Cell Disease - Drug Profile 116 Small Molecules to Antagonize TNFR1 for Immunology, Gastrointestinal, Neurology and Hematological Disorders - Drug Profile 117 Small Molecules to Inhibit HDAC for Oncology and Sickle Cell Disease - Drug Profile 118 Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours - Drug Profile 119 Stem Cell Therapy to Activate Beta Globin for Sickle Cell Disease - Drug Profile 120 Trichosic - Drug Profile 121 VBP-15 - Drug Profile 122 VitalHeme - Drug Profile 124 Sickle Cell Disease - Recent Pipeline Updates 126 Sickle Cell Disease - Dormant Projects 155 Sickle Cell Disease - Discontinued Products 157 Sickle Cell Disease - Product Development Milestones 158 Featured News & Press Releases 158 Appendix 164 Methodology 164 Coverage 164 Secondary Research 164 Primary Research 164 Expert Panel Validation 164 Contact Us 164 Disclaimer 165
List of Tables
Number of Products under Development for Sickle Cell Disease, H1 2015 12 Number of Products under Development for Sickle Cell Disease - Comparative Analysis, H1 2015 13 Number of Products under Development by Companies, H1 2015 15 Number of Products under Development by Companies, H1 2015 (Contd..1) 16 Number of Products under Investigation by Universities/Institutes, H1 2015 17 Comparative Analysis by Late Stage Development, H1 2015 18 Comparative Analysis by Clinical Stage Development, H1 2015 19 Comparative Analysis by Early Stage Development, H1 2015 20 Products under Development by Companies, H1 2015 21 Products under Development by Companies, H1 2015 (Contd..1) 22 Products under Development by Companies, H1 2015 (Contd..2) 23 Products under Investigation by Universities/Institutes, H1 2015 24 Sickle Cell Disease - Pipeline by Acceleron Pharma, Inc., H1 2015 25 Sickle Cell Disease - Pipeline by Acetylon Pharmaceuticals, Inc., H1 2015 26 Sickle Cell Disease - Pipeline by Addex Therapeutics Ltd, H1 2015 27 Sickle Cell Disease - Pipeline by Advinus Therapeutics Ltd., H1 2015 28 Sickle Cell Disease - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2015 29 Sickle Cell Disease - Pipeline by Bluebird bio, Inc., H1 2015 30 Sickle Cell Disease - Pipeline by Bristol-Myers Squibb Company, H1 2015 31 Sickle Cell Disease - Pipeline by Daiichi Sankyo Company, Limited, H1 2015 32 Sickle Cell Disease - Pipeline by Dilaforette AB, H1 2015 33 Sickle Cell Disease - Pipeline by Emmaus Medical, Inc., H1 2015 34 Sickle Cell Disease - Pipeline by Errant Gene Therapeutics, LLC, H1 2015 35 Sickle Cell Disease - Pipeline by Erytech Pharma SA, H1 2015 36 Sickle Cell Disease - Pipeline by Gamida Cell Ltd., H1 2015 37 Sickle Cell Disease - Pipeline by Gilead Sciences, Inc., H1 2015 38 Sickle Cell Disease - Pipeline by Global Blood Therapeutics, Inc., H1 2015 39 Sickle Cell Disease - Pipeline by Isis Pharmaceuticals, Inc., H1 2015 40 Sickle Cell Disease - Pipeline by Johnson & Johnson, H1 2015 41 Sickle Cell Disease - Pipeline by Morphogenesis, Inc., H1 2015 42 Sickle Cell Disease - Pipeline by NKT Therapeutics, Inc., H1 2015 43 Sickle Cell Disease - Pipeline by Novartis AG, H1 2015 44 Sickle Cell Disease - Pipeline by Pfizer Inc., H1 2015 45 Sickle Cell Disease - Pipeline by PharmaEssentia Corporation, H1 2015 46 Sickle Cell Disease - Pipeline by Prolong Pharmaceuticals, H1 2015 47 Sickle Cell Disease - Pipeline by ReveraGen BioPharma, Inc., H1 2015 48 Sickle Cell Disease - Pipeline by Sangamo BioSciences, Inc., H1 2015 49 Sickle Cell Disease - Pipeline by Sanofi, H1 2015 50 Sickle Cell Disease - Pipeline by SynZyme Technologies, LLC, H1 2015 51 Assessment by Monotherapy Products, H1 2015 52 Number of Products by Stage and Target, H1 2015 54 Number of Products by Stage and Mechanism of Action, H1 2015 56 Number of Products by Stage and Route of Administration, H1 2015 58 Number of Products by Stage and Molecule Type, H1 2015 60 Sickle Cell Disease Therapeutics - Recent Pipeline Updates, H1 2015 129 Sickle Cell Disease - Dormant Projects, H1 2015 158 Sickle Cell Disease - Dormant Projects (Contd..1), H1 2015 159 Sickle Cell Disease - Discontinued Products, H1 2015 160
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