Sickle Cell Disease – Pipeline Review, H2 2019

Global Markets Direct’s, ‘Sickle Cell Disease – Pipeline Review, H2 2019’, 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

Agios Pharmaceuticals Inc

Alkermes Plc

Angiocrine Bioscience Inc

Annexin Pharmaceuticals AB

ArQule Inc

Aruvant Sciences Inc

Axcella Health Inc

Beam Therapeutics Inc

bluebird bio Inc

Bristol-Myers Squibb Co

C4X Discovery Holdings Plc

Cannabis Science Inc

Cell Source Inc

CRISPR Therapeutics AG

CSL Ltd

Cyclerion Therapeutics Inc

Dystrogen Therapeutics SA

Ebelle Pharmaceuticals Inc

Editas Medicine Inc

Emmaus Life Sciences Inc

Epidestiny Inc

Epizyme Inc

Errant Gene Therapeutics LLC

ExCellThera Inc

Forma Therapeutics Inc

Fulcrum Therapeutics Inc

Functional Fluidics LLC

Gamida Cell Ltd

Gilead Sciences Inc

Global Blood Therapeutics Inc

Glycan Therapeutics Inc

Hillhurst Biopharmaceuticals Inc

Homology Medicines Inc

Imara Inc

Intellia Therapeutics Inc

Invenux LLC

La Jolla Pharmaceutical Company

MaxCyte Inc

Merck & Co Inc

Micelle BioPharma Inc

MimeTech Srl

Modus Therapeutics AB

Molecules For Health Inc

Morphogenesis Inc

Novartis AG

Oryzon Genomics SA

Pfizer Inc

PHD Biosciences

Phoenicia Biosciences Inc

Protagonist Therapeutics Inc

Rare Partners Srl

Regenacy Pharmaceuticals LLC

ReNeuroGen LLC

Sangamo Therapeutics Inc

Secura Bio Inc

Syros Pharmaceuticals Inc

Trucode Gene Repair Inc

Vanguard Therapeutics Inc

Table of Contents

Table of Contents

Table of Contents 2

Introduction 5

Sickle Cell Disease - Overview 6

Sickle Cell Disease - Therapeutics Development 7

Sickle Cell Disease - Therapeutics Assessment 17

Sickle Cell Disease - Companies Involved in Therapeutics Development 25

Sickle Cell Disease - Drug Profiles 43

Sickle Cell Disease - Dormant Projects 196

Sickle Cell Disease - Product Development Milestones 199

Appendix 211

List of Tables

List of Tables

Number of Products under Development for Sickle Cell Disease, H2 2019

Number of Products under Development by Companies, H2 2019

Number of Products under Development by Companies, H2 2019 (Contd..1), H2 2019

Number of Products under Development by Companies, H2 2019 (Contd..2), H2 2019

Number of Products under Development by Universities/Institutes, H2 2019

Products under Development by Companies, H2 2019

Products under Development by Companies, H2 2019 (Contd..1), H2 2019

Products under Development by Companies, H2 2019 (Contd..2), H2 2019

Products under Development by Companies, H2 2019 (Contd..3), H2 2019

Products under Development by Universities/Institutes, H2 2019

Number of Products by Stage and Target, H2 2019

Number of Products by Stage and Target, H2 2019 (Contd..1), H2 2019

Number of Products by Stage and Mechanism of Action, H2 2019

Number of Products by Stage and Mechanism of Action, H2 2019 (Contd..1), H2 2019

Number of Products by Stage and Route of Administration, H2 2019

Number of Products by Stage and Molecule Type, H2 2019

Sickle Cell Disease – Pipeline by Agios Pharmaceuticals Inc, H2 2019

Sickle Cell Disease – Pipeline by Alkermes Plc, H2 2019

Sickle Cell Disease – Pipeline by Angiocrine Bioscience Inc, H2 2019

Sickle Cell Disease – Dormant Projects, H2 2019

Sickle Cell Disease – Dormant Projects, H2 2019 (Contd..1), H2 2019

Sickle Cell Disease – Dormant Projects, H2 2019 (Contd..2), H2 2019

Sickle Cell Disease – Discontinued Products, H2 2019

List of Figures

List of Figures

Number of Products under Development for Sickle Cell Disease, H2 2019

Number of Products under Development by Companies, H2 2019

Number of Products under Development by Universities/Institutes, H2 2019

Number of Products by Top 10 Targets, H2 2019

Number of Products by Stage and Top 10 Targets, H2 2019

Number of Products by Top 10 Mechanism of Actions, H2 2019

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2019

Number of Products by Routes of Administration, H2 2019

Number of Products by Stage and Routes of Administration, H2 2019

Number of Products by Top 10 Molecule Types, H2 2019

Number of Products by Stage and Top 10 Molecule Types, H2 2019

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