Thalassemia – Pipeline Review, H2 2019

“Global Markets Direct’s, ‘Thalassemia – Pipeline Review, H2 2019’, provides an overview of the Thalassemia pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Thalassemia, 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 Thalassemia 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 Thalassemia

– The report reviews pipeline therapeutics for Thalassemia 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 Thalassemia therapeutics and enlists all their major and minor projects

– The report assesses Thalassemia 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 Thalassemia”

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 Thalassemia

- 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 Thalassemia 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

Agios Pharmaceuticals Inc

Allife Medical Science and Technology Co Ltd

Aruvant Sciences Inc

ASC Therapeutics Inc

Beam Therapeutics Inc

bluebird bio Inc

Cell Source Inc

CRISPR Therapeutics AG

CSL Ltd

EdiGene Inc

Editas Medicine Inc

EmeraMed Ltd

Epidestiny Inc

Errant Gene Therapeutics LLC

ExCellThera Inc

Fulcrum Therapeutics Inc

Gamida Cell Ltd

Gilead Sciences Inc

Imara Inc

Incyte Corp

Invenux LLC

Ionis Pharmaceuticals Inc

IRBM Science Park SpA

La Jolla Pharmaceutical Company

Merck & Co Inc

Merganser Biotech Inc

Nanomedic Inc

Orchard Therapeutics Plc

Phoenicia Biosciences Inc

Protagonist Therapeutics Inc

Rare Partners Srl

Regenacy Pharmaceuticals LLC

Sangamo Therapeutics Inc

Sanquin Plasma Products BV

Silence Therapeutics Plc

Trucode Gene Repair Inc

Vifor Pharma AG”

Table of Contents

Table of Contents

Table of Contents 2

Introduction 5

Thalassemia Overview 6

Thalassemia Therapeutics Development 7

Thalassemia Therapeutics Assessment 15

Thalassemia Companies Involved in Therapeutics Development 25

Thalassemia Drug Profiles 35

Thalassemia Dormant Projects 143

Thalassemia Discontinued Products 144

Thalassemia Product Development Milestones 145

Appendix 158

List of Tables

“List of Tables

Number of Products under Development for Thalassemia, 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 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 Universities/Institutes, H2 2019

Number of Products by Stage and Target, H2 2019

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

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

Number of Products by Stage and Molecule Type, H2 2019

Thalassemia – Pipeline by Acceleron Pharma Inc, H2 2019

Thalassemia – Pipeline by Agios Pharmaceuticals Inc, H2 2019

Thalassemia – Pipeline by Allife Medical Science and Technology Co Ltd, H2 2019

Thalassemia – Pipeline by Aruvant Sciences Inc, H2 2019

Thalassemia – Pipeline by ASC Therapeutics Inc, H2 2019

Thalassemia – Pipeline by Beam Therapeutics Inc, H2 2019

Thalassemia – Pipeline by bluebird bio Inc, H2 2019

Thalassemia – Pipeline by Cell Source Inc, H2 2019

Thalassemia – Pipeline by CRISPR Therapeutics AG, H2 2019

Thalassemia – Pipeline by CSL Ltd, H2 2019

Thalassemia – Pipeline by EdiGene Inc, H2 2019

Thalassemia – Pipeline by Editas Medicine Inc, H2 2019

Thalassemia – Pipeline by EmeraMed Ltd, H2 2019

Thalassemia – Pipeline by Epidestiny Inc, H2 2019

Thalassemia – Pipeline by Errant Gene Therapeutics LLC, H2 2019

Thalassemia – Pipeline by ExCellThera Inc, H2 2019

Thalassemia – Pipeline by Fulcrum Therapeutics Inc, H2 2019

Thalassemia – Pipeline by Gamida Cell Ltd, H2 2019

Thalassemia – Pipeline by Gilead Sciences Inc, H2 2019

Thalassemia – Pipeline by Imara Inc, H2 2019

Thalassemia – Pipeline by Incyte Corp, H2 2019

Thalassemia – Pipeline by Invenux LLC, H2 2019

Thalassemia – Pipeline by Ionis Pharmaceuticals Inc, H2 2019

Thalassemia – Pipeline by IRBM Science Park SpA, H2 2019

Thalassemia – Pipeline by La Jolla Pharmaceutical Company, H2 2019

Thalassemia – Pipeline by Merck & Co Inc, H2 2019

Thalassemia – Pipeline by Merganser Biotech Inc, H2 2019

Thalassemia – Pipeline by Nanomedic Inc, H2 2019

Thalassemia – Pipeline by Orchard Therapeutics Plc, H2 2019

Thalassemia – Pipeline by Phoenicia Biosciences Inc, H2 2019

Thalassemia – Pipeline by Protagonist Therapeutics Inc, H2 2019

Thalassemia – Pipeline by Rare Partners Srl, H2 2019

Thalassemia – Pipeline by Regenacy Pharmaceuticals LLC, H2 2019

Thalassemia – Pipeline by Sangamo Therapeutics Inc, H2 2019

Thalassemia – Pipeline by Sanquin Plasma Products BV, H2 2019

Thalassemia – Pipeline by Silence Therapeutics Plc, H2 2019

Thalassemia – Pipeline by Trucode Gene Repair Inc, H2 2019

Thalassemia – Pipeline by Vifor Pharma AG, H2 2019

Thalassemia – Dormant Projects, H2 2019

Thalassemia – Dormant Projects, H2 2019 (Contd..1), H2 2019

Thalassemia – Discontinued Products, H2 2019”

List of Figures

“List of Figures

Number of Products under Development for Thalassemia, 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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