Genetic Disorder Cluster Muscular Dystrophy Drug Development Pipeline Review, 2017

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This report provides an overview of the genetic disorders pipeline landscape, specifically focusing on muscular dystrophies. The report provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for Becker and Duchenne Muscular Dystrophy (BMD and DMD) and features dormant and discontinued projects. Both indications covered are inherited muscular dystrophies, a group of genetic, degenerative diseases primarily affecting voluntary muscles.

BMD, the first type of muscular dystrophy, leads to symptoms include pain and sensation, difficulty with running, hopping, and jumping, toe walking, breathing problems, cognitive problems and frequent falls. DMD is a condition which causes muscle weakness, and is an X-linked disorder. The gene for DMD is present on the X chromosome. It codes for a protein named dystrophin. Dystrophin is essential for providing structural support inside muscle cells. Symptoms usually appear before age 6 and may appear as early as infancy. They include fatigue, learning difficulties, intellectual disability, muscle weakness and progressive difficulty walking.

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The size of these two pipelines ranges from just six in BMD to 108 in DMD. The majority of pipeline products for DMD target dystrophin which, as mentioned, is the causative gene for the disease. However, a number of products act on other molecular targets such as growth differentiation factor 8, nuclear factor kappa B and utrophin. The first two of these make up the pipeline for BMD, along with forkhead box protein 1B.

Scope

• Which companies are the most active within the pipeline for genetic disorder therapeutics?
• Which pharmaceutical approaches are the most prominent at each stage of the pipeline and within each indication?
• To what extent do universities and institutions play a role within this pipeline, compared to pharmaceutical companies?
• What are the most important R&D milestones and data publications to have happened in the field of genetic disorder therapeutics?

Reasons to buy

• Understand the overall pipeline, with an at-a-glance overview of all products in therapeutic development for each indication
• Assess the products in development in granular detail, with an up-to-date overview of each individual pipeline program in each indication, and a comprehensive picture of recent updates and milestones for each
• Analyze the companies, institutions and universities currently operating in the pipeline, and the products being fielded by each of these
• Understand the composition of the pipeline in terms of molecule type, molecular target, mechanism of action and route of administration

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Table of Contents

GBI Research Report Guidance 2
Executive Summary 3
Table of Contents 4
List of Tables 5
List of Figures 7

Introduction 8
Genetic Disorder Cluster Muscular Dystrophy Report Coverage 8
Becker Muscular Dystrophy – Overview 8
Duchenne Muscular Dystrophy – Overview 8
Therapeutics Development 9
Becker Muscular Dystrophy 9
Duchenne Muscular Dystrophy 12
Therapeutics Assessment 22
Becker Muscular Dystrophy 22
Duchenne Muscular Dystrophy 30
Companies Involved in Therapeutics Development 38
Becker Muscular Dystrophy 38
Duchenne Muscular Dystrophy 40
Dormant Projects 63
Becker Muscular Dystrophy 63
Duchenne Muscular Dystrophy 64
Discontinued Products 66
Duchenne Muscular Dystrophy 66
Product Development Milestones 67
Duchenne Muscular Dystrophy 67

Appendix 78
Methodology 78
Coverage 78
Secondary Research 78
Primary Research 78
Expert Panel Validation 78
Contact Us 79
Disclaimer 79