Hemophilia A – Pipeline Review, H2 2018

Global Markets Direct’s, ‘Hemophilia A – Pipeline Review, H2 2018’, provides an overview of the Hemophilia A pipeline landscape.

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

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

– The report assesses Hemophilia A 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 Hemophilia A

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 Hemophilia A

- 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 Hemophilia A 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

Amarna Therapeutics BV

Apitope International NV

Bayer AG

Beijing Tiantan Biological Products Co Ltd

BioMarin Pharmaceutical Inc

Bioverativ Inc

Casebia Therapeutics

Catalyst Biosciences Inc

Chugai Pharmaceutical Co Ltd

CSL Ltd

Daiichi Sankyo Co Ltd

DBV Technologies SA

EpiVax Inc

Expression Therapeutics LLC

GC Pharma

ID Pharma Co Ltd

Idogen AB

Immusoft Corp

Innovare R & D SA De CV

LFB SA

Novo Nordisk AS

Octapharma AG

Okklo Life Sciences BV

OPKO Biologics Ltd

Pangen Biotech Inc.

Pfizer Inc

Pharming Group NV

Promethera Biosciences SA

Recursion Pharmaceuticals Inc

Sangamo Therapeutics Inc

Sanofi

Shire Plc

Sino Biopharmaceutical Ltd

SK Chemicals Co Ltd

Spark Therapeutics Inc

Ultragenyx Pharmaceutical Inc

UniQure NV

XL-protein GmbH

Table of Contents

Table of Contents

Table of Contents 2

Introduction 5

Hemophilia A - Overview 6

Hemophilia A - Therapeutics Development 7

Hemophilia A - Therapeutics Assessment 18

Hemophilia A - Companies Involved in Therapeutics Development 26

Hemophilia A - Drug Profiles 45

Hemophilia A - Dormant Projects 164

Hemophilia A - Discontinued Products 166

Hemophilia A - Product Development Milestones 167

Appendix 178

List of Tables

List of Tables

Number of Products under Development for Hemophilia A, H2 2018

Number of Products under Development by Companies, H2 2018

Number of Products under Development by Universities/Institutes, H2 2018

Products under Development by Companies, H2 2018

Products under Development by Universities/Institutes, H2 2018

Number of Products by Stage and Target, H2 2018

Number of Products by Stage and Mechanism of Action, H2 2018

Number of Products by Stage and Route of Administration, H2 2018

Number of Products by Stage and Molecule Type, H2 2018

Hemophilia A – Pipeline by Amarna Therapeutics BV, H2 2018

Hemophilia A – Pipeline by Apitope International NV, H2 2018

Hemophilia A – Pipeline by Bayer AG, H2 2018

Hemophilia A – Pipeline by Beijing Tiantan Biological Products Co Ltd, H2 2018

Hemophilia A – Pipeline by BioMarin Pharmaceutical Inc, H2 2018

Hemophilia A – Pipeline by Bioverativ Inc, H2 2018

Hemophilia A – Pipeline by Casebia Therapeutics, H2 2018

Hemophilia A – Pipeline by Catalyst Biosciences Inc, H2 2018

Hemophilia A – Pipeline by Chugai Pharmaceutical Co Ltd, H2 2018

Hemophilia A – Pipeline by CSL Ltd, H2 2018

Hemophilia A – Pipeline by Daiichi Sankyo Co Ltd, H2 2018

Hemophilia A – Pipeline by DBV Technologies SA, H2 2018

Hemophilia A – Pipeline by EpiVax Inc, H2 2018

Hemophilia A – Pipeline by Expression Therapeutics LLC, H2 2018

Hemophilia A – Pipeline by GC Pharma, H2 2018

Hemophilia A – Pipeline by ID Pharma Co Ltd, H2 2018

Hemophilia A – Pipeline by Idogen AB, H2 2018

Hemophilia A – Pipeline by Immusoft Corp, H2 2018

Hemophilia A – Pipeline by Innovare R & D SA De CV, H2 2018

Hemophilia A – Pipeline by LFB SA, H2 2018

Hemophilia A – Pipeline by Novo Nordisk AS, H2 2018

Hemophilia A – Pipeline by Octapharma AG, H2 2018

Hemophilia A – Pipeline by Okklo Life Sciences BV, H2 2018

Hemophilia A – Pipeline by OPKO Biologics Ltd, H2 2018

Hemophilia A – Pipeline by Pangen Biotech Inc., H2 2018

Hemophilia A – Pipeline by Pfizer Inc, H2 2018

Hemophilia A – Pipeline by Pharming Group NV, H2 2018

Hemophilia A – Pipeline by Promethera Biosciences SA, H2 2018

Hemophilia A – Pipeline by Recursion Pharmaceuticals Inc, H2 2018

Hemophilia A – Pipeline by Sangamo Therapeutics Inc, H2 2018

Hemophilia A – Pipeline by Sanofi, H2 2018

Hemophilia A – Pipeline by Shire Plc, H2 2018

Hemophilia A – Pipeline by Sino Biopharmaceutical Ltd, H2 2018

Hemophilia A – Pipeline by SK Chemicals Co Ltd, H2 2018

Hemophilia A – Pipeline by Spark Therapeutics Inc, H2 2018

Hemophilia A – Pipeline by Ultragenyx Pharmaceutical Inc, H2 2018

Hemophilia A – Pipeline by UniQure NV, H2 2018

Hemophilia A – Pipeline by XL-protein GmbH, H2 2018

Hemophilia A – Dormant Projects, H2 2018

Hemophilia A – Discontinued Products, H2 2018

List of Figures

List of Figures

Number of Products under Development for Hemophilia A, H2 2018

Number of Products under Development by Companies, H2 2018

Number of Products under Development by Universities/Institutes, H2 2018

Number of Products by Targets, H2 2018

Number of Products by Stage and Targets, H2 2018

Number of Products by Top 10 Mechanism of Actions, H2 2018

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2018

Number of Products by Routes of Administration, H2 2018

Number of Products by Stage and Routes of Administration, H2 2018

Number of Products by Top 10 Molecule Types, H2 2018

Number of Products by Stage and Top 10 Molecule Types, H2 2018

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