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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


Executive Summary
Introduction
Genetic Disorder Cluster Muscular Dystrophy Report Coverage
Becker Muscular Dystrophy – Overview
Duchenne Muscular Dystrophy – Overview
Therapeutics Development
Becker Muscular Dystrophy
Duchenne Muscular Dystrophy
Therapeutics Assessment
Becker Muscular Dystrophy
Duchenne Muscular Dystrophy
Companies Involved in Therapeutics Development
Becker Muscular Dystrophy
Duchenne Muscular Dystrophy
Dormant Projects
Becker Muscular Dystrophy
Duchenne Muscular Dystrophy
Discontinued Products
Duchenne Muscular Dystrophy
Product Development Milestones
Duchenne Muscular Dystrophy
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
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

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