Duchenne Muscular Dystrophy – Pipeline Review, H1 2017

Global Markets Direct’s, ‘Duchenne Muscular Dystrophy – Pipeline Review, H1 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

Acceleron Pharma Inc

Akashi Therapeutics Inc

Asklepios BioPharmaceutical Inc

BioMarin Pharmaceutical Inc

Bristol-Myers Squibb Company

Capricor Therapeutics Inc

Cardero Therapeutics Inc

Catabasis Pharmaceuticals Inc

CRISPR Therapeutics

Daiichi Sankyo Company Ltd

Debiopharm International SA

Editas Medicine Inc

Eloxx Pharmaceuticals Ltd

FibroGen Inc

Galapagos NV

Genethon SA

GTx Inc

GW Pharmaceuticals Plc

Idera Pharmaceuticals Inc

Italfarmaco SpA

La Jolla Pharmaceutical Company

Lexicon Pharmaceuticals Inc

Marina Biotech Inc

Merck KGaA

Milo Biotechnology LLC

Mitobridge Inc

Mitochon Pharmaceuticals Inc

MyoTherix Inc

NicOx SA

Nippon Shinyaku Co Ltd

Nobelpharma Co Ltd

Novartis AG

Pfizer Inc

Pluristem Therapeutics Inc

Prothelia Inc

PTC Therapeutics Inc

RASRx LLC

ReveraGen BioPharma Inc

Santhera Pharmaceuticals Holding AG

Sarepta Therapeutics Inc

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 19

Duchenne Muscular Dystrophy - Companies Involved in Therapeutics Development 29

Duchenne Muscular Dystrophy - Drug Profiles 52

Duchenne Muscular Dystrophy - Dormant Projects 248

Duchenne Muscular Dystrophy - Discontinued Products 251

Duchenne Muscular Dystrophy - Product Development Milestones 252

Appendix 263

List of Tables

List of Tables

Number of Products under Development for Duchenne Muscular Dystrophy, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Companies, H1 2017 (Contd..1), H1 2017

Number of Products under Development by Companies, H1 2017 (Contd..2), H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Products under Development by Companies, H1 2017

Products under Development by Companies, H1 2017 (Contd..1), H1 2017

Products under Development by Companies, H1 2017 (Contd..2), H1 2017

Products under Development by Companies, H1 2017 (Contd..3), H1 2017

Products under Development by Companies, H1 2017 (Contd..4), H1 2017

Products under Development by Universities/Institutes, H1 2017

Number of Products by Stage and Target, H1 2017

Number of Products by Stage and Target, H1 2017 (Contd..1), H1 2017

Number of Products by Stage and Mechanism of Action, H1 2017

Number of Products by Stage and Mechanism of Action, H1 2017 (Contd..1), H1 2017

Number of Products by Stage and Route of Administration, H1 2017

Number of Products by Stage and Molecule Type, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Acceleron Pharma Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Akashi Therapeutics Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Asklepios BioPharmaceutical Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by BioMarin Pharmaceutical Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Bristol-Myers Squibb Company, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Capricor Therapeutics Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Cardero Therapeutics Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Catabasis Pharmaceuticals Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by CRISPR Therapeutics, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Daiichi Sankyo Company Ltd, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Debiopharm International SA, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Editas Medicine Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Eloxx Pharmaceuticals Ltd, H1 2017

Duchenne Muscular Dystrophy – Pipeline by FibroGen Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Galapagos NV, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Genethon SA, H1 2017

Duchenne Muscular Dystrophy – Pipeline by GTx Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by GW Pharmaceuticals Plc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Idera Pharmaceuticals Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Italfarmaco SpA, H1 2017

Duchenne Muscular Dystrophy – Pipeline by La Jolla Pharmaceutical Company, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Lexicon Pharmaceuticals Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Marina Biotech Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Merck KGaA, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Milo Biotechnology LLC, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Mitobridge Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Mitochon Pharmaceuticals Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by MyoTherix Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by NicOx SA, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Nippon Shinyaku Co Ltd, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Nobelpharma Co Ltd, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Novartis AG, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Pfizer Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Pluristem Therapeutics Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Prothelia Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by PTC Therapeutics Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by RASRx LLC, H1 2017

Duchenne Muscular Dystrophy – Pipeline by ReveraGen BioPharma Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Santhera Pharmaceuticals Holding AG, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Sarepta Therapeutics Inc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Strykagen Corp, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Summit Therapeutics Plc, H1 2017

Duchenne Muscular Dystrophy – Pipeline by Taiho Pharmaceutical Co Ltd, H1 2017

Duchenne Muscular Dystrophy – Pipeline by WAVE Life Sciences Ltd, H1 2017

Duchenne Muscular Dystrophy – Dormant Projects, H1 2017

Duchenne Muscular Dystrophy – Dormant Projects, H1 2017 (Contd..1), H1 2017

Duchenne Muscular Dystrophy – Dormant Projects, H1 2017 (Contd..2), H1 2017

Duchenne Muscular Dystrophy – Discontinued Products, H1 2017

List of Figures

List of Figures

Number of Products under Development for Duchenne Muscular Dystrophy, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Number of Products by Top 10 Targets, H1 2017

Number of Products by Stage and Top 10 Targets, H1 2017

Number of Products by Top 10 Mechanism of Actions, H1 2017

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2017

Number of Products by Routes of Administration, H1 2017

Number of Products by Stage and Routes of Administration, H1 2017

Number of Products by Molecule Types, H1 2017

Number of Products by Stage and Molecule Types, H1 2017

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