Summary
Global Markets Direct’s, ‘Sickle Cell Disease - Pipeline Review, H1 2016’, provides an overview of the Sickle Cell Disease pipeline landscape.
The report provides comprehensive information on the therapeutics under development for Sickle Cell Disease, 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 Sickle Cell Disease 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 Sickle Cell Disease
- The report reviews pipeline therapeutics for Sickle Cell Disease 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 Sickle Cell Disease therapeutics and enlists all their major and minor projects
- The report assesses Sickle Cell Disease 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 Sickle Cell Disease
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 Sickle Cell Disease
- 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 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
Companies Mentioned
Acceleron Pharma, Inc.
Acetylon Pharmaceuticals, Inc.
Addex Therapeutics Ltd
Advinus Therapeutics Ltd.
Alnylam Pharmaceuticals, Inc.
BioAegis Therapeutics, Inc.
Bluebird bio, Inc.
Bristol-Myers Squibb Company
Complexa, Inc.
CSL Limited
Dilaforette AB
Editas Medicine, Inc.
Emmaus Medical, Inc.
Errant Gene Therapeutics, LLC
Gamida Cell Ltd.
Gilead Sciences, Inc.
Global Blood Therapeutics, Inc.
Ionis Pharmaceuticals, Inc.
Johnson & Johnson
La Jolla Pharmaceutical Company
Merck & Co., Inc.
Morphogenesis, Inc.
NKT Therapeutics, Inc.
Novartis AG
Orphagen Pharmaceuticals, Inc.
Pfizer Inc.
PharmaEssentia Corporation
Prolong Pharmaceuticals, LLC
ReveraGen BioPharma, Inc.
Sancilio & Company, Inc.
Sangamo BioSciences, Inc.
Sanofi
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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 14
Sickle Cell Disease - Pipeline Products Glance 15
Late Stage Products 15
Clinical Stage Products 16
Early Stage Products 17
Unknown Stage Products 18
Sickle Cell Disease - Products under Development by Companies 19
Sickle Cell Disease - Products under Investigation by Universities/Institutes 22
Sickle Cell Disease - Companies Involved in Therapeutics Development 23
Acceleron Pharma, Inc. 23
Acetylon Pharmaceuticals, Inc. 24
Addex Therapeutics Ltd 25
Advinus Therapeutics Ltd. 26
Alnylam Pharmaceuticals, Inc. 27
BioAegis Therapeutics, Inc. 28
Bluebird bio, Inc. 29
Bristol-Myers Squibb Company 30
Complexa, Inc. 31
CSL Limited 32
Dilaforette AB 33
Editas Medicine, Inc. 34
Emmaus Medical, Inc. 35
Errant Gene Therapeutics, LLC 36
Gamida Cell Ltd. 37
Gilead Sciences, Inc. 38
Global Blood Therapeutics, Inc. 39
Ionis Pharmaceuticals, Inc. 40
Johnson & Johnson 41
La Jolla Pharmaceutical Company 42
Merck & Co., Inc. 43
Morphogenesis, Inc. 44
NKT Therapeutics, Inc. 45
Novartis AG 46
Orphagen Pharmaceuticals, Inc. 47
Pfizer Inc. 48
PharmaEssentia Corporation 49
Prolong Pharmaceuticals, LLC 50
ReveraGen BioPharma, Inc. 51
Sancilio & Company, Inc. 52
Sangamo BioSciences, Inc. 53
Sanofi 54
Sickle Cell Disease - Therapeutics Assessment 55
Assessment by Monotherapy Products 55
Assessment by Combination Products 56
Assessment by Target 57
Assessment by Mechanism of Action 59
Assessment by Route of Administration 61
Assessment by Molecule Type 63
Drug Profiles 65
ACY-957 - Drug Profile 65
AIC-6020 - Drug Profile 66
ALN-TMP - Drug Profile 67
ambrisentan - Drug Profile 68
Antisense Oligonucleotides to Inhibit BCL11A and KLF1 for Hematological Disorders - Drug Profile 70
apixaban - Drug Profile 71
BB-305 - Drug Profile 74
CNTO-530 - Drug Profile 76
CordIn - Drug Profile 77
CXA-10 - Drug Profile 78
didox - Drug Profile 79
Drugs to Inhibit PRMT5 for Sickle Cell Anemia - Drug Profile 83
DRX-194 - Drug Profile 84
EdX-17 - Drug Profile 85
GBT-440 - Drug Profile 86
Gene Therapy for Sickle Cell Anemia - Drug Profile 87
Gene Therapy for Sickle Cell Disease - Drug Profile 88
Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile 89
Gene Therapy to Activate Gamma Globin for Sickle Cell Disease - Drug Profile 90
glutamine - Drug Profile 91
haptoglobin + hemopexin - Drug Profile 93
HBI-002 - Drug Profile 94
HBI-137 - Drug Profile 95
IMR-687 - Drug Profile 96
LJPC-401 - Drug Profile 97
luspatercept - Drug Profile 98
NiCord - Drug Profile 100
NKTT-120 - Drug Profile 101
NM-96 - Drug Profile 102
panobinostat - Drug Profile 103
PB-04 - Drug Profile 110
pentosan polysulfate sodium - Drug Profile 112
PF-04447943 - Drug Profile 113
plerixafor - Drug Profile 114
PNQ-103 - Drug Profile 117
Protein for Sickle Cell Disease - Drug Profile 118
Recombinant Plasma Gelsolin Replacement for Sickle Cell Disease - Drug Profile 119
RN-1 - Drug Profile 120
RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile 121
Sanguinate - Drug Profile 122
SC-411 - Drug Profile 124
SCD-101 - Drug Profile 125
SCPF - Drug Profile 126
sevuparin sodium - Drug Profile 127
Small Molecule for Beta Thalassemia and Sickle Cell Anemia - Drug Profile 129
Small Molecule for Sickle Cell Disease - Drug Profile 130
Small Molecule to Agonize A2A for Immunology, Hematological and Neurological Disorders - Drug Profile 131
Small Molecules for Sickle Cell Anemia - Drug Profile 132
Small Molecules to Antagonize TNFR1 for Immunology, Gastrointestinal, Neurology and Hematological Disorders - Drug Profile 133
Small Molecules to Inhibit HDAC for Oncology and Sickle Cell Disease - Drug Profile 134
Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours - Drug Profile 135
sotatercept - Drug Profile 136
Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease - Drug Profile 138
Stem Cell Therapy to Activate Beta Globin for Sickle Cell Disease - Drug Profile 139
Trichosic - Drug Profile 140
vamorolone - Drug Profile 141
Sickle Cell Disease - Recent Pipeline Updates 143
Sickle Cell Disease - Dormant Projects 179
Sickle Cell Disease - Discontinued Products 181
Sickle Cell Disease - Product Development Milestones 182
Featured News & Press Releases 182
Appendix 189
Methodology 189
Coverage 189
Secondary Research 189
Primary Research 189
Expert Panel Validation 189
Contact Us 189
Disclaimer 190
List of Tables
Number of Products under Development for Sickle Cell Disease, H1 2016 13
Number of Products under Development for Sickle Cell Disease - Comparative Analysis, H1 2016 14
Number of Products under Development by Companies, H1 2016 15
Number of Products under Development by Companies, H1 2016 (Contd..1) 16
Number of Products under Development by Companies, H1 2016 (Contd..2) 17
Number of Products under Investigation by Universities/Institutes, H1 2016 18
Comparative Analysis by Late Stage Development, H1 2016 19
Comparative Analysis by Clinical Stage Development, H1 2016 20
Comparative Analysis by Early Stage Development, H1 2016 21
Comparative Analysis by Unknown Stage Development, H1 2016 22
Products under Development by Companies, H1 2016 23
Products under Development by Companies, H1 2016 (Contd..1) 24
Products under Development by Companies, H1 2016 (Contd..2) 25
Products under Investigation by Universities/Institutes, H1 2016 26
Sickle Cell Disease - Pipeline by Acceleron Pharma, Inc., H1 2016 27
Sickle Cell Disease - Pipeline by Acetylon Pharmaceuticals, Inc., H1 2016 28
Sickle Cell Disease - Pipeline by Addex Therapeutics Ltd, H1 2016 29
Sickle Cell Disease - Pipeline by Advinus Therapeutics Ltd., H1 2016 30
Sickle Cell Disease - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2016 31
Sickle Cell Disease - Pipeline by BioAegis Therapeutics, Inc., H1 2016 32
Sickle Cell Disease - Pipeline by Bluebird bio, Inc., H1 2016 33
Sickle Cell Disease - Pipeline by Bristol-Myers Squibb Company, H1 2016 34
Sickle Cell Disease - Pipeline by Complexa, Inc., H1 2016 35
Sickle Cell Disease - Pipeline by CSL Limited, H1 2016 36
Sickle Cell Disease - Pipeline by Dilaforette AB, H1 2016 37
Sickle Cell Disease - Pipeline by Editas Medicine, Inc., H1 2016 38
Sickle Cell Disease - Pipeline by Emmaus Medical, Inc., H1 2016 39
Sickle Cell Disease - Pipeline by Errant Gene Therapeutics, LLC, H1 2016 40
Sickle Cell Disease - Pipeline by Gamida Cell Ltd., H1 2016 41
Sickle Cell Disease - Pipeline by Gilead Sciences, Inc., H1 2016 42
Sickle Cell Disease - Pipeline by Global Blood Therapeutics, Inc., H1 2016 43
Sickle Cell Disease - Pipeline by Ionis Pharmaceuticals, Inc., H1 2016 44
Sickle Cell Disease - Pipeline by Johnson & Johnson, H1 2016 45
Sickle Cell Disease - Pipeline by La Jolla Pharmaceutical Company, H1 2016 46
Sickle Cell Disease - Pipeline by Merck & Co., Inc., H1 2016 47
Sickle Cell Disease - Pipeline by Morphogenesis, Inc., H1 2016 48
Sickle Cell Disease - Pipeline by NKT Therapeutics, Inc., H1 2016 49
Sickle Cell Disease - Pipeline by Novartis AG, H1 2016 50
Sickle Cell Disease - Pipeline by Orphagen Pharmaceuticals, Inc., H1 2016 51
Sickle Cell Disease - Pipeline by Pfizer Inc., H1 2016 52
Sickle Cell Disease - Pipeline by PharmaEssentia Corporation, H1 2016 53
Sickle Cell Disease - Pipeline by Prolong Pharmaceuticals, LLC, H1 2016 54
Sickle Cell Disease - Pipeline by ReveraGen BioPharma, Inc., H1 2016 55
Sickle Cell Disease - Pipeline by Sancilio & Company, Inc., H1 2016 56
Sickle Cell Disease - Pipeline by Sangamo BioSciences, Inc., H1 2016 57
Sickle Cell Disease - Pipeline by Sanofi, H1 2016 58
Assessment by Monotherapy Products, H1 2016 59
Assessment by Combination Products, H1 2016 60
Number of Products by Stage and Target, H1 2016 62
Number of Products by Stage and Mechanism of Action, H1 2016 64
Number of Products by Stage and Route of Administration, H1 2016 66
Number of Products by Stage and Molecule Type, H1 2016 68
Sickle Cell Disease Therapeutics - Recent Pipeline Updates, H1 2016 147
Sickle Cell Disease - Dormant Projects, H1 2016 183
Sickle Cell Disease - Dormant Projects (Contd..1), H1 2016 184
Sickle Cell Disease - Discontinued Products, H1 2016 185
List of Figures
Number of Products under Development for Sickle Cell Disease, H1 2016 13
Number of Products under Development for Sickle Cell Disease - Comparative Analysis, H1 2016 14
Number of Products under Development by Companies, H1 2016 15
Number of Products under Investigation by Universities/Institutes, H1 2016 18
Comparative Analysis by Clinical Stage Development, H1 2016 20
Comparative Analysis by Early Stage Products, H1 2016 21
Assessment by Monotherapy Products, H1 2016 59
Number of Products by Top 10 Targets, H1 2016 61
Number of Products by Stage and Top 10 Targets, H1 2016 61
Number of Products by Top 10 Mechanism of Actions, H1 2016 63
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2016 63
Number of Products by Routes of Administration, H1 2016 65
Number of Products by Stage and Routes of Administration, H1 2016 65
Number of Products by Top 10 Molecule Types, H1 2016 67
Number of Products by Stage and Top 10 Molecule Types, H1 2016 67
