Sickle Cell Disease - Pipeline Review, H2 2015 Now

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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 Diseases 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 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, 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 preregistration 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.
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Table of Contents

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
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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
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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

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