Sickle Cell Disease-Pipeline Review, H1 2017

Sickle Cell Disease-Pipeline Review, H1 2017


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Executive Summary

Sickle Cell Disease-Pipeline Review, H1 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Sickle Cell Disease-Pipeline Review, H1 2017, provides an overview of the Sickle Cell Disease (Hematological Disorders) pipeline landscape.

Sickle cell anemia is a genetic (inherited) blood disorder in which red blood cells, which carry oxygen around the body, develop abnormally. Signs and symptoms include anemia, delayed growth, vision problems, pain and frequent infections. Treatment includes antibiotics, pain relievers, blood transfusion and stem cell transplant.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Sickle Cell Disease-Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Sickle Cell Disease (Hematological Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Sickle Cell Disease (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Sickle Cell Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 1, 5, 5, 9, 27, 6 and 1 respectively. Similarly, the Universities portfolio in Phase II, Phase I, Preclinical and Discovery stages comprises 1, 1, 5 and 2 molecules, respectively.

Sickle Cell Disease (Hematological Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

The pipeline guide provides a snapshot of the global therapeutic landscape of Sickle Cell Disease (Hematological Disorders).

The pipeline guide reviews pipeline therapeutics for Sickle Cell Disease (Hematological Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.

The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.

The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.

The pipeline guide reviews key companies involved in Sickle Cell Disease (Hematological Disorders) therapeutics and enlists all their major and minor projects.

The pipeline guide evaluates Sickle Cell Disease (Hematological Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.

The pipeline guide encapsulates all the dormant and discontinued pipeline projects.

The pipeline guide reviews latest news related to pipeline therapeutics for Sickle Cell Disease (Hematological Disorders)

Reasons to buy

Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.

Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.

Find and recognize significant and varied types of therapeutics under development for Sickle Cell Disease (Hematological Disorders).

Classify potential new clients or partners in the target demographic.

Develop tactical initiatives by understanding the focus areas of leading companies.

Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.

Formulate corrective measures for pipeline projects by understanding Sickle Cell Disease (Hematological Disorders) 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.

Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.



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Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 6

Introduction 7

Global Markets Direct Report Coverage 7

Sickle Cell Disease-Overview 8

Sickle Cell Disease-Therapeutics Development 9

Pipeline Overview 9

Pipeline by Companies 10

Pipeline by Universities/Institutes 14

Products under Development by Companies 15

Products under Development by Universities/Institutes 18

Sickle Cell Disease-Therapeutics Assessment 19

Assessment by Target 19

Assessment by Mechanism of Action 22

Assessment by Route of Administration 25

Assessment by Molecule Type 27

Sickle Cell Disease-Companies Involved in Therapeutics Development 29

Acceleron Pharma Inc 29

Addex Therapeutics Ltd 29

Addmedica SAS 30

Advinus Therapeutics Ltd 30

Alnylam Pharmaceuticals Inc 31

Angiocrine Bioscience Inc 31

ArQule Inc 32

Bio Products Laboratory Ltd 32

Bioverativ Inc 33

bluebird bio Inc 33

Bristol-Myers Squibb Company 34

Calimmune Inc 34

CRISPR Therapeutics 35

CSL Ltd 35

Editas Medicine Inc 36

Errant Gene Therapeutics LLC 36

Gamida Cell Ltd 37

Genethon SA 37

Gilead Sciences Inc 38

Global Blood Therapeutics Inc 38

Incyte Corp 39

Johnson & Johnson 39

La Jolla Pharmaceutical Company 40

MaxCyte Inc 40

Merck & Co Inc 41

Modus Therapeutics Holding AB 41

Morphogenesis Inc 42

NKT Therapeutics Inc 42

Novartis AG 43

Orphagen Pharmaceuticals Inc 43

Pfizer Inc 44

Prolong Pharmaceuticals LLC 44

Protagonist Therapeutics Inc 45

ReveraGen BioPharma Inc 45

Sancilio & Company Inc 46

Sangamo Therapeutics Inc 46

Sickle Cell Disease-Drug Profiles 47

(decitabine + tetrahydrouridine)-Drug Profile 47

AB-110-Drug Profile 48

ACY-957-Drug Profile 49

ALN-TMP-Drug Profile 52

ambrisentan-Drug Profile 54

apixaban-Drug Profile 57

ARQ-092-Drug Profile 65

BB-305-Drug Profile 68

benserazide-Drug Profile 76

CAL-H-Drug Profile 78

CNTO-530-Drug Profile 79

CordIn-Drug Profile 80

crizanlizumab-Drug Profile 81

didox-Drug Profile 83

Drugs to Inhibit PRMT5 for Sickle Cell Anemia-Drug Profile 86

DRX-194-Drug Profile 87

GBT-440-Drug Profile 88

Gene Therapy for Sickle Cell Anemia-Drug Profile 94

Gene Therapy for Sickle Cell Disease-Drug Profile 95

Gene therapy for Sickle Cell Disease-Drug Profile 96

Gene Therapy for Sickle Cell Disease and Thalassemia-Drug Profile 97

Gene Therapy to Activate Gamma Globin for Sickle Cell Disease-Drug Profile 98

Gene Therapy to Activate IGF2BP1 for Sickle Cell Disease and Thalassemia-Drug Profile 99

glutamine-Drug Profile 100

haptoglobin (human)-Drug Profile 104

haptoglobin + hemopexin-Drug Profile 105

HBI-002-Drug Profile 106

HBI-137-Drug Profile 107

hydroxyurea-Drug Profile 108

hydroxyurea-Drug Profile 109

IMR-687-Drug Profile 110

INCB-59872-Drug Profile 111

LJPC-401-Drug Profile 112

luspatercept-Drug Profile 114

M-012-Drug Profile 122

NiCord-Drug Profile 124

NKTT-120-Drug Profile 128

panobinostat-Drug Profile 130

pentosan polysulfate sodium-Drug Profile 138

PF-04447943-Drug Profile 139

PNQ-103-Drug Profile 140

Protein for Sickle Cell Disease-Drug Profile 141

PTG-300-Drug Profile 142

RCY-1497-Drug Profile 143

RN-1-Drug Profile 144

RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia-Drug Profile 145

Sanguinate-Drug Profile 146

SC-411-Drug Profile 149

SCD-101-Drug Profile 150

SCPF-Drug Profile 151

sevuparin sodium-Drug Profile 152

Small Molecule to Agonize A2A for Hematological, Immunology and Neurological Disorders-Drug Profile 155

Small Molecules for Sickle Cell Disease-Drug Profile 156

Small Molecules for Sickle Cell Disease-Drug Profile 157

Small Molecules to Inhibit HDAC for Oncology and Sickle Cell Disease-Drug Profile 158

Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours-Drug Profile 159

sotatercept-Drug Profile 160

Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease-Drug Profile 165

Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease-Drug Profile 166

Stem Cell Therapy for Sickle Cell Disease-Drug Profile 167

Stem Cell Therapy to Activate Beta Globin for Sickle Cell Disease-Drug Profile 169

Trichosic-Drug Profile 170

vamorolone-Drug Profile 171

Sickle Cell Disease-Dormant Projects 173

Sickle Cell Disease-Discontinued Products 175

Sickle Cell Disease-Product Development Milestones 176

Featured News & Press Releases 176

Appendix 186

Methodology 186

Coverage 186

Secondary Research 186

Primary Research 186

Expert Panel Validation 186

Contact Us 186

Disclaimer 187

List of Figures

Number of Products under Development for Sickle Cell Disease, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Number of Products by Top 10 Targets, H1 2017

Number of Products by Stage and Top 10 Targets, H1 2017

Number of Products by Top 10 Mechanism of Actions, H1 2017

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2017

Number of Products by Routes of Administration, H1 2017

Number of Products by Stage and Top 10 Routes of Administration, H1 2017

Number of Products by Top 10 Molecule Types, H1 2017

Number of Products by Stage and Top 10 Molecule Types, H1 2017

List of Tables

Number of Products under Development for Sickle Cell Disease, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Companies, H1 2017 (Contd..1), H1 2017

Number of Products under Development by Companies, H1 2017 (Contd..2), H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Products under Development by Companies, H1 2017

Products under Development by Companies, H1 2017 (Contd..1), H1 2017

Products under Development by Companies, H1 2017 (Contd..2), H1 2017

Products under Development by Universities/Institutes, H1 2017

Number of Products by Stage and Target, H1 2017

Number of Products by Stage and Target, H1 2017 (Contd..1), H1 2017

Number of Products by Stage and Mechanism of Action, H1 2017

Number of Products by Stage and Mechanism of Action, H1 2017 (Contd..1), H1 2017

Number of Products by Stage and Route of Administration, H1 2017

Number of Products by Stage and Molecule Type, H1 2017

Sickle Cell Disease-Pipeline by Acceleron Pharma Inc, H1 2017

Sickle Cell Disease-Pipeline by Addex Therapeutics Ltd, H1 2017

Sickle Cell Disease-Pipeline by Addmedica SAS, H1 2017

Sickle Cell Disease-Pipeline by Advinus Therapeutics Ltd, H1 2017

Sickle Cell Disease-Pipeline by Alnylam Pharmaceuticals Inc, H1 2017

Sickle Cell Disease-Pipeline by Angiocrine Bioscience Inc, H1 2017

Sickle Cell Disease-Pipeline by ArQule Inc, H1 2017

Sickle Cell Disease-Pipeline by Bio Products Laboratory Ltd, H1 2017

Sickle Cell Disease-Pipeline by Bioverativ Inc, H1 2017

Sickle Cell Disease-Pipeline by bluebird bio Inc, H1 2017

Sickle Cell Disease-Pipeline by Bristol-Myers Squibb Company, H1 2017

Sickle Cell Disease-Pipeline by Calimmune Inc, H1 2017

Sickle Cell Disease-Pipeline by CRISPR Therapeutics, H1 2017

Sickle Cell Disease-Pipeline by CSL Ltd, H1 2017

Sickle Cell Disease-Pipeline by Editas Medicine Inc, H1 2017

Sickle Cell Disease-Pipeline by Errant Gene Therapeutics LLC, H1 2017

Sickle Cell Disease-Pipeline by Gamida Cell Ltd, H1 2017

Sickle Cell Disease-Pipeline by Genethon SA, H1 2017

Sickle Cell Disease-Pipeline by Gilead Sciences Inc, H1 2017

Sickle Cell Disease-Pipeline by Global Blood Therapeutics Inc, H1 2017

Sickle Cell Disease-Pipeline by Incyte Corp, H1 2017

Sickle Cell Disease-Pipeline by Johnson & Johnson, H1 2017

Sickle Cell Disease-Pipeline by La Jolla Pharmaceutical Company, H1 2017

Sickle Cell Disease-Pipeline by MaxCyte Inc, H1 2017

Sickle Cell Disease-Pipeline by Merck & Co Inc, H1 2017

Sickle Cell Disease-Pipeline by Modus Therapeutics Holding AB, H1 2017

Sickle Cell Disease-Pipeline by Morphogenesis Inc, H1 2017

Sickle Cell Disease-Pipeline by NKT Therapeutics Inc, H1 2017

Sickle Cell Disease-Pipeline by Novartis AG, H1 2017

Sickle Cell Disease-Pipeline by Orphagen Pharmaceuticals Inc, H1 2017

Sickle Cell Disease-Pipeline by Pfizer Inc, H1 2017

Sickle Cell Disease-Pipeline by Prolong Pharmaceuticals LLC, H1 2017

Sickle Cell Disease-Pipeline by Protagonist Therapeutics Inc, H1 2017

Sickle Cell Disease-Pipeline by ReveraGen BioPharma Inc, H1 2017

Sickle Cell Disease-Pipeline by Sancilio & Company Inc, H1 2017

Sickle Cell Disease-Pipeline by Sangamo Therapeutics Inc, H1 2017

Sickle Cell Disease-Dormant Projects, H1 2017

Sickle Cell Disease-Dormant Projects, H1 2017 (Contd..1), H1 2017

Sickle Cell Disease-Discontinued Products, H1 2017

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Acceleron Pharma Inc, Addex Therapeutics Ltd, Addmedica SAS, Advinus Therapeutics Ltd, Alnylam Pharmaceuticals Inc, Angiocrine Bioscience Inc, ArQule Inc, Bio Products Laboratory Ltd, Bioverativ Inc, bluebird bio Inc, Bristol-Myers Squibb Company, Calimmune Inc, CRISPR Therapeutics, CSL Ltd, Editas Medicine Inc, Errant Gene Therapeutics LLC, Gamida Cell Ltd, Genethon SA, Gilead Sciences Inc, Global Blood Therapeutics Inc, Incyte Corp, Johnson & Johnson, La Jolla Pharmaceutical Company, MaxCyte Inc, Merck & Co Inc, Modus Therapeutics Holding AB, Morphogenesis Inc, NKT Therapeutics Inc, Novartis AG, Orphagen Pharmaceuticals Inc, Pfizer Inc, Prolong Pharmaceuticals LLC, Protagonist Therapeutics Inc, ReveraGen BioPharma Inc, Sancilio & Company Inc, Sangamo Therapeutics Inc

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