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Sickle Cell Disease - Pipeline Review, H2 2015 Now Available at iData Insights

Press Release   •   Jan 23, 2016 14:41 IST

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 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.

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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

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

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