Genetic Disorder Cluster Muscular Dystrophy Drug Development Pipeline Review, 2017

  • ID: 4258945
  • Report
  • Region: Global
  • 79 Pages
  • GBI Research
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FEATURED COMPANIES

  • Acceleron Pharma Inc
  • Editas Medicine Inc
  • GTx Inc
  • Merck KGaA
  • Nippon Shinyaku Co Ltd
  • PTC Therapeutics Inc
  • MORE

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
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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Acceleron Pharma Inc
  • Editas Medicine Inc
  • GTx Inc
  • Merck KGaA
  • Nippon Shinyaku Co Ltd
  • PTC Therapeutics Inc
  • MORE

1. Report Guidance

2. Executive Summary

3. Introduction

4. Genetic Disorder Cluster Muscular Dystrophy Report Coverage

  • Becker Muscular Dystrophy - Overview
  • Duchenne Muscular Dystrophy - Overview

5. Therapeutics Development

  • Becker Muscular Dystrophy
  • Duchenne Muscular Dystrophy

6. Therapeutics Assessment

  • Becker Muscular Dystrophy
  • Duchenne Muscular Dystrophy

7. Companies Involved in Therapeutics Development

  • Becker Muscular Dystrophy
  • Duchenne Muscular Dystrophy
  • Dormant Projects
  • Becker Muscular Dystrophy
  • Duchenne Muscular Dystrophy

8. Discontinued Products

  • Duchenne Muscular Dystrophy

9. Product Development Milestones

  • Duchenne Muscular Dystrophy

10. Appendix

  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation

List of Tables

  • Number of Products under Development for Becker Muscular Dystrophy
  • Number of Products under Development by Companies, Becker Muscular Dystrophy
  • Products under Development by Companies, Becker Muscular Dystrophy
  • Number of Products under Development for Duchenne Muscular Dystrophy
  • Number of Products under Development by Companies, Duchenne Muscular Dystrophy
  • Number of Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy
  • Products under Development by Companies, Duchenne Muscular Dystrophy
  • Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy
  • Number of Products by Stage and Target, Becker Muscular Dystrophy
  • Number of Products by Stage and Mechanism of Action, Becker Muscular Dystrophy
  • Number of Products by Stage and Route of Administration, Becker Muscular Dystrophy
  • Number of Products by Stage and Molecule Type, Becker Muscular Dystrophy
  • Number of Products by Stage and Target, Duchenne Muscular Dystrophy
  • Number of Products by Stage and Mechanism of Action, Duchenne Muscular Dystrophy
  • Number of Products by Stage and Route of Administration, Duchenne Muscular Dystrophy
  • Number of Products by Stage and Molecule Type, Duchenne Muscular Dystrophy
  • Becker Muscular Dystrophy - Pipeline by Cardero Therapeutics Inc
  • Becker Muscular Dystrophy - Pipeline by Milo Biotechnology LLC
  • Becker Muscular Dystrophy - Pipeline by PTC Therapeutics Inc
  • Becker Muscular Dystrophy - Pipeline by ReveraGen BioPharma Inc
  • Becker Muscular Dystrophy - Pipeline by Sarepta Therapeutics Inc
  • Duchenne Muscular Dystrophy - Pipeline by Acceleron Pharma Inc
  • Duchenne Muscular Dystrophy - Pipeline by Akashi Therapeutics Inc
  • Duchenne Muscular Dystrophy - Pipeline by Asklepios BioPharmaceutical Inc
  • Duchenne Muscular Dystrophy - Pipeline by BioMarin Pharmaceutical Inc
  • Duchenne Muscular Dystrophy - Pipeline by Bristol-Myers Squibb Company
  • Duchenne Muscular Dystrophy - Pipeline by Capricor Therapeutics Inc
  • Duchenne Muscular Dystrophy - Pipeline by Cardero Therapeutics Inc
  • Duchenne Muscular Dystrophy - Pipeline by Catabasis Pharmaceuticals Inc
  • Duchenne Muscular Dystrophy - Pipeline by CRISPR Therapeutics
  • Duchenne Muscular Dystrophy - Pipeline by Daiichi Sankyo Company Ltd
  • Duchenne Muscular Dystrophy - Pipeline by Debiopharm International SA
  • Duchenne Muscular Dystrophy - Pipeline by Editas Medicine Inc
  • Duchenne Muscular Dystrophy - Pipeline by Eloxx Pharmaceuticals Ltd
  • Duchenne Muscular Dystrophy - Pipeline by FibroGen Inc
  • Duchenne Muscular Dystrophy - Pipeline by Galapagos NV
  • Duchenne Muscular Dystrophy - Pipeline by Genethon SA
  • Duchenne Muscular Dystrophy - Pipeline by GTx Inc
  • Duchenne Muscular Dystrophy - Pipeline by GW Pharmaceuticals Plc
  • Duchenne Muscular Dystrophy - Pipeline by Idera Pharmaceuticals Inc
  • Duchenne Muscular Dystrophy - Pipeline by Italfarmaco SpA
  • Duchenne Muscular Dystrophy - Pipeline by La Jolla Pharmaceutical Company
  • Duchenne Muscular Dystrophy - Pipeline by Lexicon Pharmaceuticals Inc
  • Duchenne Muscular Dystrophy - Pipeline by Marina Biotech Inc
  • Duchenne Muscular Dystrophy - Pipeline by Merck KGaA
  • Duchenne Muscular Dystrophy - Pipeline by Milo Biotechnology LLC
  • Duchenne Muscular Dystrophy - Pipeline by Mitobridge Inc
  • Duchenne Muscular Dystrophy - Pipeline by Mitochon Pharmaceuticals Inc
  • Duchenne Muscular Dystrophy - Pipeline by MyoTherix Inc
  • Duchenne Muscular Dystrophy - Pipeline by NicOx SA
  • Duchenne Muscular Dystrophy - Pipeline by Nippon Shinyaku Co Ltd
  • Duchenne Muscular Dystrophy - Pipeline by Nobelpharma Co Ltd
  • Duchenne Muscular Dystrophy - Pipeline by Novartis AG
  • Duchenne Muscular Dystrophy - Pipeline by Pfizer Inc
  • Duchenne Muscular Dystrophy - Pipeline by Pluristem Therapeutics Inc
  • Duchenne Muscular Dystrophy - Pipeline by Prothelia Inc
  • Duchenne Muscular Dystrophy - Pipeline by PTC Therapeutics Inc
  • Duchenne Muscular Dystrophy - Pipeline by RASRx LLC
  • Duchenne Muscular Dystrophy - Pipeline by ReveraGen BioPharma Inc
  • Duchenne Muscular Dystrophy - Pipeline by Santhera Pharmaceuticals Holding AG
  • Duchenne Muscular Dystrophy - Pipeline by Sarepta Therapeutics Inc
  • Duchenne Muscular Dystrophy - Pipeline by Strykagen Corp
  • Duchenne Muscular Dystrophy - Pipeline by Summit Therapeutics Plc
  • Duchenne Muscular Dystrophy - Pipeline by Taiho Pharmaceutical Co Ltd
  • Duchenne Muscular Dystrophy - Pipeline by WAVE Life Sciences Ltd
  • Becker Muscular Dystrophy - Dormant Projects
  • Duchenne Muscular Dystrophy - Dormant Projects
  • Duchenne Muscular Dystrophy - Discontinued Products

List of Figures

  • Number of Products under Development for Becker Muscular Dystrophy
  • Number of Products under Development by Companies, Becker Muscular Dystrophy
  • Number of Products under Development for Duchenne Muscular Dystrophy
  • Number of Products under Development by Companies, Duchenne Muscular Dystrophy
  • Number of Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy
  • Number of Products by Targets, Becker Muscular Dystrophy
  • Number of Products by Stage and Targets, Becker Muscular Dystrophy
  • Number of Products by Mechanism of Actions, Becker Muscular Dystrophy
  • Number of Products by Stage and Mechanism of Actions, Becker Muscular Dystrophy
  • Number of Products by Routes of Administration, Becker Muscular Dystrophy
  • Number of Products by Stage and Routes of Administration, Becker Muscular Dystrophy
  • Number of Products by Molecule Types, Becker Muscular Dystrophy
  • Number of Products by Stage and Molecule Types, Becker Muscular Dystrophy
  • Number of Products by Top 10 Targets, Duchenne Muscular Dystrophy
  • Number of Products by Stage and Top 10 Targets, Duchenne Muscular Dystrophy
  • Number of Products by Top 10 Mechanism of Actions, Duchenne Muscular Dystrophy
  • Number of Products by Stage and Top 10 Mechanism of Actions, Duchenne Muscular Dystrophy
  • Number of Products by Routes of Administration, Duchenne Muscular Dystrophy
  • Number of Products by Stage and Routes of Administration, Duchenne Muscular Dystrophy
  • Number of Products by Molecule Types, Duchenne Muscular Dystrophy
  • Number of Products by Stage and Molecule Types, Duchenne Muscular Dystrophy
Note: Product cover images may vary from those shown
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  • Acceleron Pharma Inc
  • Akashi Therapeutics Inc
  • Asklepios BioPharmaceutical Inc
  • BioMarin Pharmaceutical Inc
  • Bristol-Myers Squibb Company
  • CRISPR Therapeutics
  • Capricor Therapeutics Inc
  • Cardero Therapeutics Inc
  • Catabasis Pharmaceuticals Inc
  • Daiichi Sankyo Company Ltd
  • Debiopharm International SA
  • Editas Medicine Inc
  • Eloxx Pharmaceuticals Ltd
  • FibroGen Inc
  • GTx Inc
  • GW Pharmaceuticals Plc
  • Galapagos NV
  • Genethon SA
  • 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
  • PTC Therapeutics Inc
  • Pfizer Inc
  • Pluristem Therapeutics Inc
  • Prothelia 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
Note: Product cover images may vary from those shown
5 of 5
Note: Product cover images may vary from those shown
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