Market Research Logo

Genetic Disorder Cluster Muscular Dystrophy Drug Development Pipeline Review, 2017

Genetic Disorder Cluster Muscular Dystrophy Drug Development Pipeline Review, 2017

Summary


This report provides an overview of the genetic disorders pipeline landscape. 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. There are a total of six products in development for this indication, by six seperate companies; the companies operating in this pipeline space are all small pharmaceutical enterprises, and consist of Armgo Pharma, Cardero Therapeutics, Milo Biotechnology, PTC Therapeutics, ReveraGen BioPharma and Sarepta Therapeutics.

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.

There is a far larger pipeline for DMD than for BMD; there are a total of 108 products in development for DMD, by 57 companies and nine academic institutions. Key companies operating in this pipeline space include Sarepta Therapeutics, PTC Therapeutics, WAVE Life Sciences, Solid Biosciences, Summit Therapeutics and Catabasis Pharmaceuticals. Of particular importance are Sarepta, who are developing twelve distinct pipeline products for DMD. As with BMD, these are all smaller enterprises, although there is some involvement from larger pharmaceutical companies such as Pfizer and Merck in the DMD pipeline.

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


  • GBI Research Report Guidance
  • Executive Summary
  • Introduction
    • Genetic Disorder Cluster Muscular Dystrophy Report Coverage
    • Becker Muscular Dystrophy - Overview
    • Duchenne Muscular Dystrophy - Overview
  • Therapeutics Development
    • Becker Muscular Dystrophy
      • Table Figure 1: Number of Products under Development for Becker Muscular Dystrophy
      • Table Number of Products under Development for Becker Muscular Dystrophy
      • Table Figure 2: Number of Products under Development by Companies, Becker Muscular Dystrophy
      • Table Number of Products under Development by Companies, Becker Muscular Dystrophy
      • Table Products under Development by Companies, Becker Muscular Dystrophy
    • Duchenne Muscular Dystrophy
      • Table Figure 3: Number of Products under Development for Duchenne Muscular Dystrophy
      • Table Number of Products under Development for Duchenne Muscular Dystrophy
      • Table Figure 4: Number of Products under Development by Companies, Duchenne Muscular Dystrophy
      • Table Number of Products under Development by Companies, Duchenne Muscular Dystrophy
      • Table Figure 5: Number of Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy
      • Table Number of Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy
      • Table Products under Development by Companies, Duchenne Muscular Dystrophy
      • Table Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy
  • Therapeutics Assessment
    • Becker Muscular Dystrophy
      • Table Figure 6: Number of Products by Targets, Becker Muscular Dystrophy
      • Table Figure 7: Number of Products by Stage and Targets, Becker Muscular Dystrophy
      • Table Number of Products by Stage and Target, Becker Muscular Dystrophy
      • Table Figure 8: Number of Products by Mechanism of Actions, Becker Muscular Dystrophy
      • Table Figure 9: Number of Products by Stage and Mechanism of Actions, Becker Muscular Dystrophy
      • Table Number of Products by Stage and Mechanism of Action, Becker Muscular Dystrophy
      • Table Figure 10: Number of Products by Routes of Administration, Becker Muscular Dystrophy
      • Table Figure 11: Number of Products by Stage and Routes of Administration, Becker Muscular Dystrophy
      • Table Number of Products by Stage and Route of Administration, Becker Muscular Dystrophy
      • Table Figure 12: Number of Products by Molecule Types, Becker Muscular Dystrophy
      • Table Figure 13: Number of Products by Stage and Molecule Types, Becker Muscular Dystrophy
      • Table Number of Products by Stage and Molecule Type, Becker Muscular Dystrophy
    • Duchenne Muscular Dystrophy
      • Table Figure 14: Number of Products by Top 10 Targets, Duchenne Muscular Dystrophy
      • Table Figure 15: Number of Products by Stage and Top 10 Targets, Duchenne Muscular Dystrophy
      • Table Number of Products by Stage and Target, Duchenne Muscular Dystrophy
      • Table Figure 16: Number of Products by Top 10 Mechanism of Actions, Duchenne Muscular Dystrophy
      • Table Figure 17: Number of Products by Stage and Top 10 Mechanism of Actions, Duchenne Muscular Dystrophy
      • Table Number of Products by Stage and Mechanism of Action, Duchenne Muscular Dystrophy
      • Table Figure 18: Number of Products by Routes of Administration, Duchenne Muscular Dystrophy
      • Table Figure 19: Number of Products by Stage and Routes of Administration, Duchenne Muscular Dystrophy
      • Table Number of Products by Stage and Route of Administration, Duchenne Muscular Dystrophy
      • Table Figure 20: Number of Products by Molecule Types, Duchenne Muscular Dystrophy
      • Table Figure 21: Number of Products by Stage and Molecule Types, Duchenne Muscular Dystrophy
      • Table Number of Products by Stage and Molecule Type, Duchenne Muscular Dystrophy
  • Companies Involved in Therapeutics Development
    • Becker Muscular Dystrophy
      • Table Becker Muscular Dystrophy - Pipeline by Cardero Therapeutics Inc
      • Table Becker Muscular Dystrophy - Pipeline by Milo Biotechnology LLC
      • Table Becker Muscular Dystrophy - Pipeline by PTC Therapeutics Inc
      • Table Becker Muscular Dystrophy - Pipeline by ReveraGen BioPharma Inc
      • Table Becker Muscular Dystrophy - Pipeline by Sarepta Therapeutics Inc
    • Duchenne Muscular Dystrophy
      • Table Duchenne Muscular Dystrophy - Pipeline by Acceleron Pharma Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Akashi Therapeutics Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Asklepios BioPharmaceutical Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by BioMarin Pharmaceutical Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Bristol-Myers Squibb Company
      • Table Duchenne Muscular Dystrophy - Pipeline by Capricor Therapeutics Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Cardero Therapeutics Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Catabasis Pharmaceuticals Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by CRISPR Therapeutics
      • Table Duchenne Muscular Dystrophy - Pipeline by Daiichi Sankyo Company Ltd
      • Table Duchenne Muscular Dystrophy - Pipeline by Debiopharm International SA
      • Table Duchenne Muscular Dystrophy - Pipeline by Editas Medicine Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Eloxx Pharmaceuticals Ltd
      • Table Duchenne Muscular Dystrophy - Pipeline by FibroGen Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Galapagos NV
      • Table Duchenne Muscular Dystrophy - Pipeline by Genethon SA
      • Table Duchenne Muscular Dystrophy - Pipeline by GTx Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by GW Pharmaceuticals Plc
      • Table Duchenne Muscular Dystrophy - Pipeline by Idera Pharmaceuticals Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Italfarmaco SpA
      • Table Duchenne Muscular Dystrophy - Pipeline by La Jolla Pharmaceutical Company
      • Table Duchenne Muscular Dystrophy - Pipeline by Lexicon Pharmaceuticals Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Marina Biotech Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Merck KGaA
      • Table Duchenne Muscular Dystrophy - Pipeline by Milo Biotechnology LLC
      • Table Duchenne Muscular Dystrophy - Pipeline by Mitobridge Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Mitochon Pharmaceuticals Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by MyoTherix Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by NicOx SA
      • Table Duchenne Muscular Dystrophy - Pipeline by Nippon Shinyaku Co Ltd
      • Table Duchenne Muscular Dystrophy - Pipeline by Nobelpharma Co Ltd
      • Table Duchenne Muscular Dystrophy - Pipeline by Novartis AG
      • Table Duchenne Muscular Dystrophy - Pipeline by Pfizer Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Pluristem Therapeutics Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Prothelia Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by PTC Therapeutics Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by RASRx LLC
      • Table Duchenne Muscular Dystrophy - Pipeline by ReveraGen BioPharma Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Santhera Pharmaceuticals Holding AG
      • Table Duchenne Muscular Dystrophy - Pipeline by Sarepta Therapeutics Inc
      • Table Duchenne Muscular Dystrophy - Pipeline by Strykagen Corp
      • Table Duchenne Muscular Dystrophy - Pipeline by Summit Therapeutics Plc
      • Table Duchenne Muscular Dystrophy - Pipeline by Taiho Pharmaceutical Co Ltd
      • Table Duchenne Muscular Dystrophy - Pipeline by WAVE Life Sciences Ltd
  • Dormant Projects
    • Becker Muscular Dystrophy
      • Table Becker Muscular Dystrophy - Dormant Projects
    • Duchenne Muscular Dystrophy
      • Table Duchenne Muscular Dystrophy - Dormant Projects
  • Discontinued Products
    • Duchenne Muscular Dystrophy
      • Table Duchenne Muscular Dystrophy - Discontinued Products
  • Product Development Milestones
    • Duchenne Muscular Dystrophy
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

Download our eBook: How to Succeed Using Market Research

Learn how to effectively navigate the market research process to help guide your organization on the journey to success.

Download eBook

Share this report