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Published: Dec, 2015 | Pages:
178 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Sickle Cell Disease - Pipeline Review, H2 2015 Summary Global Markets Direct's, 'Sickle Cell Disease - Pipeline Review, H2 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 5 List of Figures 6 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 15 Sickle Cell Disease - Pipeline Products Glance 16 Late Stage Products 16 Clinical Stage Products 17 Early Stage Products 18 Unknown Stage Products 19 Sickle Cell Disease - Products under Development by Companies 20 Sickle Cell Disease - Products under Investigation by Universities/Institutes 23 Sickle Cell Disease - Companies Involved in Therapeutics Development 24 Acceleron Pharma, Inc. 24 Acetylon Pharmaceuticals, Inc. 25 Addex Therapeutics Ltd 26 Advinus Therapeutics Ltd. 27 Alnylam Pharmaceuticals, Inc. 28 Bluebird bio, Inc. 29 Bristol-Myers Squibb Company 30 Complexa, Inc. 31 Daiichi Sankyo Company, Limited 32 Dilaforette AB 33 Emmaus Medical, Inc. 34 Errant Gene Therapeutics, LLC 35 Gamida Cell Ltd. 36 Gilead Sciences, Inc. 37 Global Blood Therapeutics, Inc. 38 Isis Pharmaceuticals, Inc. 39 Johnson & Johnson 40 La Jolla Pharmaceutical Company 41 Morphogenesis, Inc. 42 NKT Therapeutics, Inc. 43 Novartis AG 44 Orphagen Pharmaceuticals, Inc. 45 Pfizer Inc. 46 PharmaEssentia Corporation 47 Prolong Pharmaceuticals 48 ReveraGen BioPharma, Inc. 49 Sancilio & Company, Inc. 50 Sangamo BioSciences, Inc. 51 Sanofi 52 Sickle Cell Disease - Therapeutics Assessment 53 Assessment by Monotherapy Products 53 Assessment by Target 54 Assessment by Mechanism of Action 56 Assessment by Route of Administration 58 Assessment by Molecule Type 60 Drug Profiles 62 ACY-957 - Drug Profile 62 AIC-2011 - Drug Profile 63 AIC-6020 - Drug Profile 64 ALN-TMP - Drug Profile 65 Antisense Oligonucleotides to Inhibit BCL11A and KLF1 for Hematological Disorders - Drug Profile 66 apixaban - Drug Profile 67 BB-305 - Drug Profile 69 CNTO-530 - Drug Profile 71 CordIn - Drug Profile 72 CXA-10 - Drug Profile 73 Didox - Drug Profile 74 Drugs to Inhibit PRMT5 for Sickle Cell Anemia - Drug Profile 78 DRX-194 - Drug Profile 79 EdX-17 - Drug Profile 80 GBT-440 - Drug Profile 81 Gene Therapy for Sickle Cell Anemia - Drug Profile 82 Gene Therapy for Sickle Cell Disease - Drug Profile 83 Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile 84 Gene Therapy to Activate Gamma Globin for Sickle Cell Disease - Drug Profile 85 glutamine - Drug Profile 86 HBI-002 - Drug Profile 88 HBI-137 - Drug Profile 89 LJPC-401 - Drug Profile 90 luspatercept - Drug Profile 91 NiCord - Drug Profile 92 NKTT-120 - Drug Profile 93 NM-96 - Drug Profile 94 panobinostat - Drug Profile 95 PB-04 - Drug Profile 101 pentosan polysulfate sodium - Drug Profile 103 PF-4447943 - Drug Profile 104 plerixafor - Drug Profile 105 PNQ-103 - Drug Profile 107 prasugrel hydrochloride - Drug Profile 108 Protein for Sickle Cell Disease - Drug Profile 111 regadenoson - Drug Profile 112 RN-1 - Drug Profile 114 RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile 115 Sanguinate - Drug Profile 116 SC-411 - Drug Profile 118 SCD-101 - Drug Profile 119 SCPF - Drug Profile 120 sevuparin sodium - Drug Profile 121 Small Molecule for Beta Thalassemia and Sickle Cell Anemia - Drug Profile 123 Small Molecule to Agonize A2A for Immunology, Hematological and Neurological Disorders - Drug Profile 124 Small Molecules for Sickle Cell Anemia - Drug Profile 125 Small Molecules to Antagonize TNFR1 for Immunology, Gastrointestinal, Neurology and Hematological Disorders - Drug Profile 126 Small Molecules to Inhibit HDAC for Oncology and Sickle Cell Disease - Drug Profile 127 Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours - Drug Profile 128 Stem Cell Therapy to Activate Beta Globin for Sickle Cell Disease - Drug Profile 129 Trichosic - Drug Profile 130 Verolone - Drug Profile 131 Sickle Cell Disease - Recent Pipeline Updates 133 Sickle Cell Disease - Dormant Projects 164 Sickle Cell Disease - Discontinued Products 166 Sickle Cell Disease - Product Development Milestones 167 Featured News & Press Releases 167 Appendix 173 Methodology 173 Coverage 173 Secondary Research 173 Primary Research 173 Expert Panel Validation 173 Contact Us 173 Disclaimer 174
List of Tables
Number of Products under Development for Sickle Cell Disease, H2 2015 13 Number of Products under Development for Sickle Cell Disease - Comparative Analysis, H2 2015 14 Number of Products under Development by Companies, H2 2015 16 Number of Products under Development by Companies, H2 2015 (Contd..1) 17 Number of Products under Development by Companies, H2 2015 (Contd..2) 18 Number of Products under Investigation by Universities/Institutes, H2 2015 19 Comparative Analysis by Late Stage Development, H2 2015 20 Comparative Analysis by Clinical Stage Development, H2 2015 21 Comparative Analysis by Early Stage Development, H2 2015 22 Comparative Analysis by Unknown Stage Development, H2 2015 23 Products under Development by Companies, H2 2015 24 Products under Development by Companies, H2 2015 (Contd..1) 25 Products under Development by Companies, H2 2015 (Contd..2) 26 Products under Investigation by Universities/Institutes, H2 2015 27 Sickle Cell Disease - Pipeline by Acceleron Pharma, Inc., H2 2015 28 Sickle Cell Disease - Pipeline by Acetylon Pharmaceuticals, Inc., H2 2015 29 Sickle Cell Disease - Pipeline by Addex Therapeutics Ltd, H2 2015 30 Sickle Cell Disease - Pipeline by Advinus Therapeutics Ltd., H2 2015 31 Sickle Cell Disease - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2015 32 Sickle Cell Disease - Pipeline by Bluebird bio, Inc., H2 2015 33 Sickle Cell Disease - Pipeline by Bristol-Myers Squibb Company, H2 2015 34 Sickle Cell Disease - Pipeline by Complexa, Inc., H2 2015 35 Sickle Cell Disease - Pipeline by Daiichi Sankyo Company, Limited, H2 2015 36 Sickle Cell Disease - Pipeline by Dilaforette AB, H2 2015 37 Sickle Cell Disease - Pipeline by Emmaus Medical, Inc., H2 2015 38 Sickle Cell Disease - Pipeline by Errant Gene Therapeutics, LLC, H2 2015 39 Sickle Cell Disease - Pipeline by Gamida Cell Ltd., H2 2015 40 Sickle Cell Disease - Pipeline by Gilead Sciences, Inc., H2 2015 41 Sickle Cell Disease - Pipeline by Global Blood Therapeutics, Inc., H2 2015 42 Sickle Cell Disease - Pipeline by Isis Pharmaceuticals, Inc., H2 2015 43 Sickle Cell Disease - Pipeline by Johnson & Johnson, H2 2015 44 Sickle Cell Disease - Pipeline by La Jolla Pharmaceutical Company, H2 2015 45 Sickle Cell Disease - Pipeline by Morphogenesis, Inc., H2 2015 46 Sickle Cell Disease - Pipeline by NKT Therapeutics, Inc., H2 2015 47 Sickle Cell Disease - Pipeline by Novartis AG, H2 2015 48 Sickle Cell Disease - Pipeline by Orphagen Pharmaceuticals, Inc., H2 2015 49 Sickle Cell Disease - Pipeline by Pfizer Inc., H2 2015 50 Sickle Cell Disease - Pipeline by PharmaEssentia Corporation, H2 2015 51 Sickle Cell Disease - Pipeline by Prolong Pharmaceuticals, H2 2015 52 Sickle Cell Disease - Pipeline by ReveraGen BioPharma, Inc., H2 2015 53 Sickle Cell Disease - Pipeline by Sancilio & Company, Inc., H2 2015 54 Sickle Cell Disease - Pipeline by Sangamo BioSciences, Inc., H2 2015 55 Sickle Cell Disease - Pipeline by Sanofi, H2 2015 56 Assessment by Monotherapy Products, H2 2015 57 Number of Products by Stage and Target, H2 2015 59 Number of Products by Stage and Mechanism of Action, H2 2015 61 Number of Products by Stage and Route of Administration, H2 2015 63 Number of Products by Stage and Molecule Type, H2 2015 65 Sickle Cell Disease Therapeutics - Recent Pipeline Updates, H2 2015 137 Sickle Cell Disease - Dormant Projects, H2 2015 168 Sickle Cell Disease - Dormant Projects (Contd..1), H2 2015 169 Sickle Cell Disease - Discontinued Products, H2 2015 170
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