Duchenne Muscular Dystrophy – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Duchenne Muscular Dystrophy – Pipeline Review, H2 2017’, provides an overview of the Duchenne Muscular Dystrophy pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Duchenne Muscular Dystrophy, 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 Duchenne Muscular Dystrophy 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 Duchenne Muscular Dystrophy

The report reviews pipeline therapeutics for Duchenne Muscular Dystrophy 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 Duchenne Muscular Dystrophy therapeutics and enlists all their major and minor projects

The report assesses Duchenne Muscular Dystrophy 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 Duchenne Muscular Dystrophy

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 Duchenne Muscular Dystrophy

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 Duchenne Muscular Dystrophy 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

4P-Pharma SAS

Acceleron Pharma Inc

Akashi Therapeutics Inc

Antisense Therapeutics Ltd

Asklepios BioPharmaceutical Inc

Beech Tree Labs Inc

Bioleaders Corp

BioMarin Pharmaceutical Inc

Capricor Therapeutics Inc

Catabasis Pharmaceuticals Inc

CRISPR Therapeutics

Daiichi Sankyo Company Ltd

Debiopharm International SA

Editas Medicine Inc

Eloxx Pharmaceuticals Ltd

F. Hoffmann-La Roche Ltd

FibroGen Inc

Galapagos NV

Genethon SA

GTx Inc

GW Pharmaceuticals Plc

Italfarmaco SpA

Karyopharm Therapeutics Inc

La Jolla Pharmaceutical Company

Lexicon Pharmaceuticals Inc

Marathon Pharmaceuticals LLC

Marina Biotech Inc

Merck KGaA

Milo Biotechnology LLC

Mitobridge Inc

Mitochon Pharmaceuticals Inc

N-Gene Research Laboratories Inc

NicOx SA

Nobelpharma Co Ltd

Novartis AG

NS Pharma Inc

Pfizer Inc

Pluristem Therapeutics Inc

Prothelia Inc

PTC Therapeutics Inc

ReveraGen BioPharma Inc

Santhera Pharmaceuticals Holding AG

Sarepta Therapeutics Inc

SOM Biotech SL

Strykagen Corp

Summit Therapeutics Plc

Taiho Pharmaceutical Co Ltd

WAVE Life Sciences Ltd

Table of Contents

Table of Contents

Table of Contents 2

Introduction 5

Duchenne Muscular Dystrophy - Overview 6

Duchenne Muscular Dystrophy - Therapeutics Development 7

Duchenne Muscular Dystrophy - Therapeutics Assessment 21

Duchenne Muscular Dystrophy - Companies Involved in Therapeutics Development 31

Duchenne Muscular Dystrophy - Drug Profiles 56

Duchenne Muscular Dystrophy - Dormant Projects 270

Duchenne Muscular Dystrophy - Discontinued Products 273

Duchenne Muscular Dystrophy - Product Development Milestones 274

Appendix 284

List of Tables

List of Tables

Number of Products under Development for Duchenne Muscular Dystrophy, 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 Companies, H2 2017 (Contd..3), 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 Companies, H2 2017 (Contd..3), H2 2017

Products under Development by Companies, H2 2017 (Contd..4), H2 2017

Products under Development by Companies, H2 2017 (Contd..5), 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

Duchenne Muscular Dystrophy – Pipeline by 4P-Pharma SAS, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Acceleron Pharma Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Akashi Therapeutics Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Antisense Therapeutics Ltd, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Asklepios BioPharmaceutical Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Beech Tree Labs Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Bioleaders Corp, H2 2017

Duchenne Muscular Dystrophy – Pipeline by BioMarin Pharmaceutical Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Capricor Therapeutics Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Catabasis Pharmaceuticals Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by CRISPR Therapeutics, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Daiichi Sankyo Company Ltd, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Debiopharm International SA, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Editas Medicine Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Eloxx Pharmaceuticals Ltd, H2 2017

Duchenne Muscular Dystrophy – Pipeline by F. Hoffmann-La Roche Ltd, H2 2017

Duchenne Muscular Dystrophy – Pipeline by FibroGen Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Galapagos NV, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Genethon SA, H2 2017

Duchenne Muscular Dystrophy – Pipeline by GTx Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by GW Pharmaceuticals Plc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Italfarmaco SpA, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Karyopharm Therapeutics Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by La Jolla Pharmaceutical Company, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Lexicon Pharmaceuticals Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Marathon Pharmaceuticals LLC, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Marina Biotech Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Merck KGaA, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Milo Biotechnology LLC, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Mitobridge Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Mitochon Pharmaceuticals Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by N-Gene Research Laboratories Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by NicOx SA, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Nobelpharma Co Ltd, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Novartis AG, H2 2017

Duchenne Muscular Dystrophy – Pipeline by NS Pharma Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Pfizer Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Pluristem Therapeutics Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Prothelia Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by PTC Therapeutics Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by ReveraGen BioPharma Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Santhera Pharmaceuticals Holding AG, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Sarepta Therapeutics Inc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by SOM Biotech SL, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Strykagen Corp, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Summit Therapeutics Plc, H2 2017

Duchenne Muscular Dystrophy – Pipeline by Taiho Pharmaceutical Co Ltd, H2 2017

Duchenne Muscular Dystrophy – Pipeline by WAVE Life Sciences Ltd, H2 2017

Duchenne Muscular Dystrophy – Dormant Projects, H2 2017

Duchenne Muscular Dystrophy – Dormant Projects, H2 2017 (Contd..1), H2 2017

Duchenne Muscular Dystrophy – Dormant Projects, H2 2017 (Contd..2), H2 2017

Duchenne Muscular Dystrophy – Discontinued Products, H2 2017

List of Figures

List of Figures

Number of Products under Development for Duchenne Muscular Dystrophy, 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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