Sickle Cell Disease – Pipeline Review, H2 2016

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

Angiocrine Bioscience, Inc.

Bio Products Laboratory Ltd

bluebird bio, Inc.

Bristol-Myers Squibb Company

Complexa, Inc.

CRISPR Therapeutics

CSL Limited

Editas Medicine, Inc.

Emmaus Medical, Inc.

Errant Gene Therapeutics, LLC

Gamida Cell Ltd.

Gilead Sciences, Inc.

Global Blood Therapeutics, Inc.

Johnson & Johnson

La Jolla Pharmaceutical Company

Merck & Co., Inc.

Modus Therapeutics Holding AB

Morphogenesis, Inc.

NKT Therapeutics, Inc.

Novartis AG

Orphagen Pharmaceuticals, Inc.

Pfizer Inc.

PharmaEssentia Corporation

Prolong Pharmaceuticals, LLC

Protagonist Therapeutics Inc.

ReveraGen BioPharma, Inc.

Sancilio & Company, Inc.

Sangamo BioSciences, Inc.

Selexys Pharmaceuticals Corporation

Table of Contents

Table of Contents

Table of Contents 2

Introduction 5

Sickle Cell Disease Overview 6

Therapeutics Development 7

Sickle Cell Disease - Therapeutics under Development by Companies 9

Sickle Cell Disease - Therapeutics under Investigation by Universities/Institutes 13

Sickle Cell Disease - Pipeline Products Glance 14

Sickle Cell Disease - Products under Development by Companies 18

Sickle Cell Disease - Products under Investigation by Universities/Institutes 21

Sickle Cell Disease - Companies Involved in Therapeutics Development 22

Sickle Cell Disease - Therapeutics Assessment 56

Drug Profiles 68

Sickle Cell Disease - Dormant Projects 176

Sickle Cell Disease - Discontinued Products 179

Sickle Cell Disease - Product Development Milestones 180

Appendix 191

List of Tables

List of Tables

Number of Products under Development for Sickle Cell Disease, H2 2016 14

Number of Products under Development for Sickle Cell Disease – Comparative Analysis, H2 2016 15

Number of Products under Development by Companies, H2 2016 17

Number of Products under Development by Companies, H2 2016 (Contd..1) 18

Number of Products under Development by Companies, H2 2016 (Contd..2) 19

Number of Products under Investigation by Universities/Institutes, H2 2016 20

Comparative Analysis by Late Stage Development, H2 2016 21

Comparative Analysis by Clinical Stage Development, H2 2016 22

Comparative Analysis by Early Stage Development, H2 2016 23

Comparative Analysis by Unknown Stage Development, H2 2016 24

Products under Development by Companies, H2 2016 25

Products under Development by Companies, H2 2016 (Contd..1) 26

Products under Development by Companies, H2 2016 (Contd..2) 27

Products under Investigation by Universities/Institutes, H2 2016 28

Sickle Cell Disease – Pipeline by Acceleron Pharma, Inc., H2 2016 29

Sickle Cell Disease – Pipeline by Acetylon Pharmaceuticals, Inc., H2 2016 30

Sickle Cell Disease – Pipeline by Addex Therapeutics Ltd, H2 2016 31

Sickle Cell Disease – Pipeline by Advinus Therapeutics Ltd, H2 2016 32

Sickle Cell Disease – Pipeline by Alnylam Pharmaceuticals, Inc., H2 2016 33

Sickle Cell Disease – Pipeline by Angiocrine Bioscience, Inc., H2 2016 34

Sickle Cell Disease – Pipeline by Bio Products Laboratory Ltd, H2 2016 35

Sickle Cell Disease – Pipeline by bluebird bio, Inc., H2 2016 36

Sickle Cell Disease – Pipeline by Bristol-Myers Squibb Company, H2 2016 37

Sickle Cell Disease – Pipeline by Complexa, Inc., H2 2016 38

Sickle Cell Disease – Pipeline by CRISPR Therapeutics, H2 2016 39

Sickle Cell Disease – Pipeline by CSL Limited, H2 2016 40

Sickle Cell Disease – Pipeline by Editas Medicine, Inc., H2 2016 41

Sickle Cell Disease – Pipeline by Emmaus Medical, Inc., H2 2016 42

Sickle Cell Disease – Pipeline by Errant Gene Therapeutics, LLC, H2 2016 43

Sickle Cell Disease – Pipeline by Gamida Cell Ltd., H2 2016 44

Sickle Cell Disease – Pipeline by Gilead Sciences, Inc., H2 2016 45

Sickle Cell Disease – Pipeline by Global Blood Therapeutics, Inc., H2 2016 46

Sickle Cell Disease – Pipeline by Johnson & Johnson, H2 2016 47

Sickle Cell Disease – Pipeline by La Jolla Pharmaceutical Company, H2 2016 48

Sickle Cell Disease – Pipeline by Merck & Co., Inc., H2 2016 49

Sickle Cell Disease – Pipeline by Modus Therapeutics Holding AB, H2 2016 50

Sickle Cell Disease – Pipeline by Morphogenesis, Inc., H2 2016 51

Sickle Cell Disease – Pipeline by NKT Therapeutics, Inc., H2 2016 52

Sickle Cell Disease – Pipeline by Novartis AG, H2 2016 53

Sickle Cell Disease – Pipeline by Orphagen Pharmaceuticals, Inc., H2 2016 54

Sickle Cell Disease – Pipeline by Pfizer Inc., H2 2016 55

Sickle Cell Disease – Pipeline by PharmaEssentia Corporation, H2 2016 56

Sickle Cell Disease – Pipeline by Prolong Pharmaceuticals, LLC, H2 2016 57

Sickle Cell Disease – Pipeline by Protagonist Therapeutics Inc., H2 2016 58

Sickle Cell Disease – Pipeline by ReveraGen BioPharma, Inc., H2 2016 59

Cell Disease – Pipeline by Sancilio & Company, Inc., H2 2016 60

Sickle Cell Disease – Pipeline by Sangamo BioSciences, Inc., H2 2016 61

Sickle Cell Disease – Pipeline by Selexys Pharmaceuticals Corporation, H2 2016 62

Assessment by Monotherapy Products, H2 2016 63

Assessment by Combination Products, H2 2016 64

Number of Products by Stage and Target, H2 2016 66

Number of Products by Stage and Mechanism of Action, H2 2016 69

Number of Products by Stage and Route of Administration, H2 2016 72

Number of Products by Stage and Molecule Type, H2 2016 74

Sickle Cell Disease – Dormant Projects, H2 2016 183

Sickle Cell Disease – Dormant Projects (Contd..1), H2 2016 184

Sickle Cell Disease – Dormant Projects (Contd..2), H2 2016 185

Sickle Cell Disease – Discontinued Products, H2 2016 186

List of Figures

List of Figures

Number of Products under Development for Sickle Cell Disease, H2 2016 14

Number of Products under Development for Sickle Cell Disease – Comparative Analysis, H2 2016 15

Number of Products under Development by Companies, H2 2016 16

Number of Products under Investigation by Universities/Institutes, H2 2016 20

Comparative Analysis by Late Stage Development, H2 2016 21

Comparative Analysis by Clinical Stage Development, H2 2016 22

Comparative Analysis by Early Stage Products, H2 2016 23

Assessment by Monotherapy Products, H2 2016 63

Assessment by Combination Products, H2 2016 64

Number of Products by Top 10 Targets, H2 2016 65

Number of Products by Stage and Top 10 Targets, H2 2016 65

Number of Products by Top 10 Mechanism of Actions, H2 2016 68

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016 68

Number of Products by Routes of Administration, H2 2016 71

Number of Products by Stage and Routes of Administration, H2 2016 71

Number of Products by Top 10 Molecule Types, H2 2016 73

Number of Products by Stage and Top 10 Molecule Types, H2 2016 73

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