Genetic Disorder Cluster Muscular Dystrophy Drug Development Pipeline Review 2017 - 108 Products in Development for DMD, by 57 Companies and Nine Academic Institutions - Research and Markets - Seite 2

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?

Key Topics Covered:

1. Report Guidance

2. Executive Summary

3. Introduction

4. Genetic Disorder Cluster Muscular Dystrophy Report Coverage

5. Therapeutics Development

6. Therapeutics Assessment

7. Companies Involved in Therapeutics Development

8. Discontinued Products

9. Product Development Milestones

10. Appendix

Companies Mentioned

• Acceleron Pharma Inc
• Akashi Therapeutics Inc
• Asklepios BioPharmaceutical Inc
• BioMarin Pharmaceutical Inc
• Bristol-Myers Squibb Company
• CRISPR Therapeutics
• Catabasis Pharmaceuticals Inc
• Daiichi Sankyo Company Ltd
• Debiopharm International SA
• Editas Medicine Inc
• GW Pharmaceuticals Plc
• Galapagos NV
• Genethon SA
• Idera Pharmaceuticals Inc
• Italfarmaco SpA
• La Jolla Pharmaceutical Company
• Lexicon Pharmaceuticals Inc
• Merck KGaA
• Mitobridge Inc
• Mitochon Pharmaceuticals Inc
• MyoTherix Inc
• NicOx SA
• Nippon Shinyaku Co Ltd
• Nobelpharma Co Ltd
• Novartis AG
• Pfizer Inc
• Pluristem Therapeutics Inc
• Prothelia Inc
• RASRx LLC
• ReveraGen BioPharma Inc
• Santhera Pharmaceuticals Holding AG
• Sarepta Therapeutics Inc
• Strykagen Corp
• Summit Therapeutics Plc
• WAVE Life Sciences Ltd

For more information about this report visit http://www.researchandmarkets.com/research/xnlrl3/genetic_disorder