Sickle Cell Disease – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Sickle Cell Disease – Pipeline Review, H2 2017’, 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

Addmedica SAS

Angiocrine Bioscience Inc

Annexin Pharmaceuticals AB

ArQule Inc

Bio Products Laboratory Ltd

Bioverativ Inc

bluebird bio Inc

Bristol-Myers Squibb Co

Calimmune Inc

CRISPR Therapeutics

CSL Ltd

Daiichi Sankyo Co Ltd

Editas Medicine Inc

Errant Gene Therapeutics LLC

Gamida Cell Ltd

Genethon SA

Gilead Sciences Inc

Global Blood Therapeutics Inc

Incyte Corp

Ironwood Pharmaceuticals Inc

La Jolla Pharmaceutical Company

MaxCyte Inc

Merck & Co Inc

Morphogenesis Inc

NKT Therapeutics Inc

Novartis AG

Orphagen Pharmaceuticals Inc

Prolong Pharmaceuticals LLC

Protagonist Therapeutics Inc

ReveraGen BioPharma Inc

Sancilio & Company Inc

Sangamo Therapeutics Inc

Table of Contents

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

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

Addmedica SAS 29

Angiocrine Bioscience Inc 29

Annexin Pharmaceuticals AB 30

ArQule Inc 30

Bio Products Laboratory Ltd 31

Bioverativ Inc 31

bluebird bio Inc 31

Bristol-Myers Squibb Co 32

Calimmune Inc 32

CRISPR Therapeutics 33

CSL Ltd 33

Daiichi Sankyo Co Ltd 34

Editas Medicine Inc 34

Errant Gene Therapeutics LLC 35

Gamida Cell Ltd 35

Genethon SA 36

Gilead Sciences Inc 36

Global Blood Therapeutics Inc 36

Incyte Corp 37

Ironwood Pharmaceuticals Inc 37

La Jolla Pharmaceutical Company 38

MaxCyte Inc 38

Merck & Co Inc 39

Morphogenesis Inc 39

NKT Therapeutics Inc 40

Novartis AG 40

Orphagen Pharmaceuticals Inc 41

Prolong Pharmaceuticals LLC 41

Protagonist Therapeutics Inc 41

ReveraGen BioPharma Inc 42

Sancilio & Company Inc 42

Sangamo Therapeutics Inc 42

Sickle Cell Disease - Drug Profiles 44

(decitabine + tetrahydrouridine) - Drug Profile 44

AB-110 - Drug Profile 45

ACY-957 - Drug Profile 46

ambrisentan - Drug Profile 49

AP-01 - Drug Profile 52

apixaban - Drug Profile 54

ARQ-092 - Drug Profile 62

BB-305 - Drug Profile 66

benserazide - Drug Profile 75

BIVV-003 - Drug Profile 77

CAL-H - Drug Profile 79

CordIn - Drug Profile 80

crizanlizumab - Drug Profile 81

CTX-001 - Drug Profile 84

didox - Drug Profile 86

Drugs to Inhibit PRMT5 for Sickle Cell Anemia - Drug Profile 89

DRX-194 - Drug Profile 90

GBT-440 - Drug Profile 91

Gene Therapy for Sickle Cell Anemia - Drug Profile 97

Gene Therapy for Sickle Cell Disease - Drug Profile 98

Gene Therapy for Sickle Cell Disease - Drug Profile 99

Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile 100

Gene Therapy to Activate Gamma Globin for Sickle Cell Disease - Drug Profile 101

Gene Therapy to Activate IGF2BP1 for Sickle Cell Disease and Thalassemia - Drug Profile 102

haptoglobin (human) - Drug Profile 103

haptoglobin + hemopexin - Drug Profile 104

HBI-002 - Drug Profile 105

HBI-137 - Drug Profile 106

hydroxyurea - Drug Profile 107

hydroxyurea - Drug Profile 108

IMR-687 - Drug Profile 109

INCB-59872 - Drug Profile 111

IW-1701 - Drug Profile 112

LJPC-401 - Drug Profile 113

luspatercept - Drug Profile 115

M-012 - Drug Profile 125

NiCord - Drug Profile 127

NKTT-120 - Drug Profile 131

panobinostat - Drug Profile 133

pentosan polysulfate sodium - Drug Profile 140

Polysaccharide to Inhibit Coagulation Factor X for Deep Vein Thrombosis and Sickle Cell Disease - Drug Profile 141

prasugrel hydrochloride - Drug Profile 142

Protein for Sickle Cell Disease - Drug Profile 147

PTG-300 - Drug Profile 148

RCY-1497 - Drug Profile 150

RN-1 - Drug Profile 151

RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile 152

Sanguinate - Drug Profile 153

SC-411 - Drug Profile 156

SCD-101 - Drug Profile 157

SCPF - Drug Profile 158

Small Molecules for Sickle Cell Disease - Drug Profile 159

Small Molecules for Sickle Cell Disease - Drug Profile 160

Small Molecules to Inhibit HDAC for Oncology and Sickle Cell Disease - Drug Profile 161

Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours - Drug Profile 162

sotatercept - Drug Profile 163

Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease - Drug Profile 168

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 174

Sickle Cell Disease - Product Development Milestones 176

Featured News & Press Releases 176

Appendix 185

Methodology 185

Coverage 185

Secondary Research 185

Primary Research 185

Expert Panel Validation 185

Contact Us 185

Disclaimer 186

List of Tables

List of Tables

Number of Products under Development for Sickle Cell Disease, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Companies, H2 2017 (Contd..1), H2 2017

Number of Products under Development by Companies, H2 2017 (Contd..2), H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Companies, H2 2017 (Contd..1), H2 2017

Products under Development by Companies, H2 2017 (Contd..2), H2 2017

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Target, H2 2017 (Contd..1), H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017 (Contd..1), H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Sickle Cell Disease – Pipeline by Acceleron Pharma Inc, H2 2017

Sickle Cell Disease – Pipeline by Addmedica SAS, H2 2017

Sickle Cell Disease – Pipeline by Angiocrine Bioscience Inc, H2 2017

Sickle Cell Disease – Pipeline by Annexin Pharmaceuticals AB, H2 2017

Sickle Cell Disease – Pipeline by ArQule Inc, H2 2017

Sickle Cell Disease – Pipeline by Bio Products Laboratory Ltd, H2 2017

Sickle Cell Disease – Pipeline by Bioverativ Inc, H2 2017

Sickle Cell Disease – Pipeline by bluebird bio Inc, H2 2017

Sickle Cell Disease – Pipeline by Bristol-Myers Squibb Co, H2 2017

Sickle Cell Disease – Pipeline by Calimmune Inc, H2 2017

Sickle Cell Disease – Pipeline by CRISPR Therapeutics, H2 2017

Sickle Cell Disease – Pipeline by CSL Ltd, H2 2017

Sickle Cell Disease – Pipeline by Daiichi Sankyo Co Ltd, H2 2017

Sickle Cell Disease – Pipeline by Editas Medicine Inc, H2 2017

Sickle Cell Disease – Pipeline by Errant Gene Therapeutics LLC, H2 2017

Sickle Cell Disease – Pipeline by Gamida Cell Ltd, H2 2017

Sickle Cell Disease – Pipeline by Genethon SA, H2 2017

Sickle Cell Disease – Pipeline by Gilead Sciences Inc, H2 2017

Sickle Cell Disease – Pipeline by Global Blood Therapeutics Inc, H2 2017

Sickle Cell Disease – Pipeline by Incyte Corp, H2 2017

Sickle Cell Disease – Pipeline by Ironwood Pharmaceuticals Inc, H2 2017

Sickle Cell Disease – Pipeline by La Jolla Pharmaceutical Company, H2 2017

Sickle Cell Disease – Pipeline by MaxCyte Inc, H2 2017

Sickle Cell Disease – Pipeline by Merck & Co Inc, H2 2017

Sickle Cell Disease – Pipeline by Morphogenesis Inc, H2 2017

Sickle Cell Disease – Pipeline by NKT Therapeutics Inc, H2 2017

Sickle Cell Disease – Pipeline by Novartis AG, H2 2017

Sickle Cell Disease – Pipeline by Orphagen Pharmaceuticals Inc, H2 2017

Sickle Cell Disease – Pipeline by Prolong Pharmaceuticals LLC, H2 2017

Sickle Cell Disease – Pipeline by Protagonist Therapeutics Inc, H2 2017

Sickle Cell Disease – Pipeline by ReveraGen BioPharma Inc, H2 2017

Sickle Cell Disease – Pipeline by Sancilio & Company Inc, H2 2017

Sickle Cell Disease – Pipeline by Sangamo Therapeutics Inc, H2 2017

Sickle Cell Disease – Dormant Projects, H2 2017

Sickle Cell Disease – Dormant Projects, H2 2017 (Contd..1), H2 2017

List of Figures

List of Figures

Number of Products under Development for Sickle Cell Disease, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Number of Products by Top 10 Targets, H2 2017

Number of Products by Stage and Top 10 Targets, H2 2017

Number of Products by Top 10 Mechanism of Actions, H2 2017

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2017

Number of Products by Routes of Administration, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Top 10 Molecule Types, H2 2017

Number of Products by Stage and Top 10 Molecule Types, H2 2017

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