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Genetic Disorder Cluster Muscular Dystrophy Drug Development Pipeline Review, 2017

Published By :

GBI Research

Published Date : May 2017

Category :

Pharmaceutical

No. of Pages : 79 Pages

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.

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

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

List of Table

List of Tables

Number of Products under Development for Becker Muscular Dystrophy 9
Number of Products under Development by Companies, Becker Muscular Dystrophy 10
Products under Development by Companies, Becker Muscular Dystrophy 11
Number of Products under Development for Duchenne Muscular Dystrophy 12
Number of Products under Development by Companies, Duchenne Muscular Dystrophy 14
Number of Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy 16
Products under Development by Companies, Duchenne Muscular Dystrophy 17
Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy 21
Number of Products by Stage and Target, Becker Muscular Dystrophy 23
Number of Products by Stage and Mechanism of Action, Becker Muscular Dystrophy 25
Number of Products by Stage and Route of Administration, Becker Muscular Dystrophy 27
Number of Products by Stage and Molecule Type, Becker Muscular Dystrophy 29
Number of Products by Stage and Target, Duchenne Muscular Dystrophy 31
Number of Products by Stage and Mechanism of Action, Duchenne Muscular Dystrophy 33
Number of Products by Stage and Route of Administration, Duchenne Muscular Dystrophy 35
Number of Products by Stage and Molecule Type, Duchenne Muscular Dystrophy 37
Becker Muscular Dystrophy – Pipeline by Cardero Therapeutics Inc 38
Becker Muscular Dystrophy – Pipeline by Milo Biotechnology LLC 38
Becker Muscular Dystrophy – Pipeline by PTC Therapeutics Inc 39
Becker Muscular Dystrophy – Pipeline by ReveraGen BioPharma Inc 39
Becker Muscular Dystrophy – Pipeline by Sarepta Therapeutics Inc 40
Duchenne Muscular Dystrophy – Pipeline by Acceleron Pharma Inc 40
Duchenne Muscular Dystrophy – Pipeline by Akashi Therapeutics Inc 41
Duchenne Muscular Dystrophy – Pipeline by Asklepios BioPharmaceutical Inc 41
Duchenne Muscular Dystrophy – Pipeline by BioMarin Pharmaceutical Inc 41
Duchenne Muscular Dystrophy – Pipeline by Bristol-Myers Squibb Company 42
Duchenne Muscular Dystrophy – Pipeline by Capricor Therapeutics Inc 42
Duchenne Muscular Dystrophy – Pipeline by Cardero Therapeutics Inc 43
Duchenne Muscular Dystrophy – Pipeline by Catabasis Pharmaceuticals Inc 43
Duchenne Muscular Dystrophy – Pipeline by CRISPR Therapeutics 44
Duchenne Muscular Dystrophy – Pipeline by Daiichi Sankyo Company Ltd 44
Duchenne Muscular Dystrophy – Pipeline by Debiopharm International SA 45
Duchenne Muscular Dystrophy – Pipeline by Editas Medicine Inc 45
Duchenne Muscular Dystrophy – Pipeline by Eloxx Pharmaceuticals Ltd 46
Duchenne Muscular Dystrophy – Pipeline by FibroGen Inc 46
Duchenne Muscular Dystrophy – Pipeline by Galapagos NV 47
Duchenne Muscular Dystrophy – Pipeline by Genethon SA 47
Duchenne Muscular Dystrophy – Pipeline by GTx Inc 48
Duchenne Muscular Dystrophy – Pipeline by GW Pharmaceuticals Plc 48
Duchenne Muscular Dystrophy – Pipeline by Idera Pharmaceuticals Inc 49
Duchenne Muscular Dystrophy – Pipeline by Italfarmaco SpA 49
Duchenne Muscular Dystrophy – Pipeline by La Jolla Pharmaceutical Company 50
Duchenne Muscular Dystrophy – Pipeline by Lexicon Pharmaceuticals Inc 50
Duchenne Muscular Dystrophy – Pipeline by Marina Biotech Inc 51
Duchenne Muscular Dystrophy – Pipeline by Merck KGaA 51
Duchenne Muscular Dystrophy – Pipeline by Milo Biotechnology LLC 52
Duchenne Muscular Dystrophy – Pipeline by Mitobridge Inc 52
Duchenne Muscular Dystrophy – Pipeline by Mitochon Pharmaceuticals Inc 53
Duchenne Muscular Dystrophy – Pipeline by MyoTherix Inc 53
Duchenne Muscular Dystrophy – Pipeline by NicOx SA 54
Duchenne Muscular Dystrophy – Pipeline by Nippon Shinyaku Co Ltd 54
Duchenne Muscular Dystrophy – Pipeline by Nobelpharma Co Ltd 55
Duchenne Muscular Dystrophy – Pipeline by Novartis AG 55
Duchenne Muscular Dystrophy – Pipeline by Pfizer Inc 56
Duchenne Muscular Dystrophy – Pipeline by Pluristem Therapeutics Inc 56
Duchenne Muscular Dystrophy – Pipeline by Prothelia Inc 57
Duchenne Muscular Dystrophy – Pipeline by PTC Therapeutics Inc 57
Duchenne Muscular Dystrophy – Pipeline by RASRx LLC 58
Duchenne Muscular Dystrophy – Pipeline by ReveraGen BioPharma Inc 58
Duchenne Muscular Dystrophy – Pipeline by Santhera Pharmaceuticals Holding AG 59
Duchenne Muscular Dystrophy – Pipeline by Sarepta Therapeutics Inc 60
Duchenne Muscular Dystrophy – Pipeline by Strykagen Corp 61
Duchenne Muscular Dystrophy – Pipeline by Summit Therapeutics Plc 61
Duchenne Muscular Dystrophy – Pipeline by Taiho Pharmaceutical Co Ltd 62
Duchenne Muscular Dystrophy – Pipeline by WAVE Life Sciences Ltd 62
Becker Muscular Dystrophy – Dormant Projects 63
Duchenne Muscular Dystrophy – Dormant Projects 64
Duchenne Muscular Dystrophy – Discontinued Products 66

List of Chart

List of Figures

Number of Products under Development for Becker Muscular Dystrophy 9
Number of Products under Development by Companies, Becker Muscular Dystrophy 10
Number of Products under Development for Duchenne Muscular Dystrophy 12
Number of Products under Development by Companies, Duchenne Muscular Dystrophy 13
Number of Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy 16
Number of Products by Targets, Becker Muscular Dystrophy 22
Number of Products by Stage and Targets, Becker Muscular Dystrophy 22
Number of Products by Mechanism of Actions, Becker Muscular Dystrophy 24
Number of Products by Stage and Mechanism of Actions, Becker Muscular Dystrophy 24
Number of Products by Routes of Administration, Becker Muscular Dystrophy 26
Number of Products by Stage and Routes of Administration, Becker Muscular Dystrophy 26
Number of Products by Molecule Types, Becker Muscular Dystrophy 28
Number of Products by Stage and Molecule Types, Becker Muscular Dystrophy 28
Number of Products by Top 10 Targets, Duchenne Muscular Dystrophy 30
Number of Products by Stage and Top 10 Targets, Duchenne Muscular Dystrophy 30
Number of Products by Top 10 Mechanism of Actions, Duchenne Muscular Dystrophy 32
Number of Products by Stage and Top 10 Mechanism of Actions, Duchenne Muscular Dystrophy 32
Number of Products by Routes of Administration, Duchenne Muscular Dystrophy 34
Number of Products by Stage and Routes of Administration, Duchenne Muscular Dystrophy 34
Number of Products by Molecule Types, Duchenne Muscular Dystrophy 36
Number of Products by Stage and Molecule Types, Duchenne Muscular Dystrophy 36

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