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Muscular Dystrophy - Pipeline Review, H2 2016

  • November 2016
  • -
  • Global Markets Direct
  • -
  • 144 pages

Muscular Dystrophy - Pipeline Review, H2 2016

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Muscular Dystrophy – Pipeline Review, H2 2016, provides an overview of the Muscular Dystrophy (Musculoskeletal Disorders) pipeline landscape.

Muscular dystrophy is a group of diseases in which muscle fibers are unusually susceptible to damage. These damaged muscles become progressively weaker. Symptoms usually appear before age 6 and may appear as early as infancy. They may include fatigue, learning difficulties, intellectual disability, muscle weakness and progressive difficulty walking.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Muscular Dystrophy – Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Muscular Dystrophy (Musculoskeletal Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Muscular Dystrophy (Musculoskeletal Disorders) pipeline guide also reviews of key players involved in therapeutic development for Muscular Dystrophy and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 5, 2, 24 and 14 respectively. Similarly, the Universities portfolio in Phase II, Phase I, Preclinical and Discovery stages comprises 1, 1, 3 and 4 molecules, respectively.Muscular Dystrophy.

Muscular Dystrophy (Musculoskeletal Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note:Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Muscular Dystrophy (Musculoskeletal Disorders).
- The pipeline guide reviews pipeline therapeutics for Muscular Dystrophy (Musculoskeletal Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Muscular Dystrophy (Musculoskeletal Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Muscular Dystrophy (Musculoskeletal Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Muscular Dystrophy (Musculoskeletal Disorders)

Reasons to buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Muscular Dystrophy (Musculoskeletal Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Muscular Dystrophy (Musculoskeletal Disorders) pipeline depth and focus of Indication therapeutics.
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Table Of Contents

Muscular Dystrophy - Pipeline Review, H2 2016
Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Muscular Dystrophy Overview 8
Therapeutics Development 9
Pipeline Products for Muscular Dystrophy - Overview 9
Pipeline Products for Muscular Dystrophy - Comparative Analysis 10
Muscular Dystrophy - Therapeutics under Development by Companies 11
Muscular Dystrophy - Therapeutics under Investigation by Universities/Institutes 13
Muscular Dystrophy - Pipeline Products Glance 14
Late Stage Products 14
Clinical Stage Products 15
Early Stage Products 16
Muscular Dystrophy - Products under Development by Companies 17
Muscular Dystrophy - Products under Investigation by Universities/Institutes 20
Muscular Dystrophy - Companies Involved in Therapeutics Development 21
Acceleron Pharma, Inc. 21
AMO Pharma Limited 22
Asahi Kasei Pharma Corp. 23
aTyr Pharma, Inc. 24
Benitec Biopharma Limited 25
Bio Blast Pharma Ltd. 26
Biophytis SAS 27
Corcept Therapeutics Incorporated 28
Evotec AG 29
F. Hoffmann-La Roche Ltd. 30
Genethon 31
Ionis Pharmaceuticals, Inc. 32
Marina Biotech, Inc. 33
Medestea Research and Production S.p.A. 34
Novogen Limited 35
Pfizer Inc. 36
Prothelia, Inc. 37
SanBio, Inc. 38
Santhera Pharmaceuticals Holding AG 39
Sarepta Therapeutics, Inc. 40
Selecta Biosciences, Inc. 41
Strykagen Corporation 42
Takeda Pharmaceutical Company Limited 43
WAVE Life Sciences Ltd. 44
Muscular Dystrophy - Therapeutics Assessment 45
Assessment by Monotherapy Products 45
Assessment by Target 46
Assessment by Mechanism of Action 48
Assessment by Route of Administration 50
Assessment by Molecule Type 52
Drug Profiles 54
ACE-083 - Drug Profile 54
Antisense Oligonucleotide to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile 56
Antisense RNAi Oligonucleotides for Myotonic Dystrophy - Drug Profile 57
ATYR-1940 - Drug Profile 58
baliforsen - Drug Profile 61
BIO-103 - Drug Profile 62
domagrozumab - Drug Profile 63
Drugs for Merosin-Deficient Congenital Muscular Dystrophy Type 1A - Drug Profile 65
elcatonin - Drug Profile 66
Gene Therapy for Muscular Dystrophy and Liver Diseases - Drug Profile 67
Gene Therapy to Activate Dysferlin for Duchenne and Limb Girdle Muscular Dystrophies - Drug Profile 68
Gene Therapy to Activate Dysferlin for Dysferlinopathies - Drug Profile 69
Gene Therapy to Activate Dystrophin for Muscular Dystrophy - Drug Profile 70
IUCT-169 - Drug Profile 71
IUCT-290 - Drug Profile 72
IUCT-309 - Drug Profile 73
ketoprofen - Drug Profile 74
LR-08 - Drug Profile 75
MED-1101 - Drug Profile 76
mexiletine hydrochloride - Drug Profile 77
Oligonucleotide 1 to Target Dystrophia Myotonica Protein Kinase for Myotonic Dystrophy - Drug Profile 78
Oligonucleotides to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile 79
omigapil - Drug Profile 80
Pabparna - Drug Profile 82
poloxamer - Drug Profile 83
PRT-01 - Drug Profile 85
Recombinant Protein to Activate Utrophin for Muscular Dystrophies - Drug Profile 87
RNAi Gene Therapy to Inhibit Myotilin for LGMD - Drug Profile 88
RP-33 - Drug Profile 89
SB-308 - Drug Profile 90
SIWA-318 - Drug Profile 91
Small Molecule to Inhibit DUX4 for Muscular Dystrophy - Drug Profile 92
Small Molecule to Target CUG RNA for Myotonic Dystrophy 1 - Drug Profile 93
Small Molecule to Target RNA for Myotonic Dystrophy - Drug Profile 94
Small Molecules for Dysferlinopathies - Drug Profile 95
Small Molecules for Facioscapulohumeral Muscular Dystrophy - Drug Profile 96
Small Molecules for Myotonic Dystrophy - Drug Profile 97
Small Molecules for Myotonic Dystrophy Type 1 - Drug Profile 98
Small Molecules to Activate SMCHD1 for Facioscapulohumeral Dystrophy - Drug Profile 99
Small Molecules to Antagonize Glucocorticoid Receptor II for Muscular Dystrophy - Drug Profile 100
Small Molecules to Inhibit MBNL1 for Myotonic Dystrophy Type I - Drug Profile 101
Small Molecules to Target RNA for Myotonic Dystrophy - Drug Profile 102
SRT-149 - Drug Profile 103
SRT-152 - Drug Profile 104
Stem Cell Therapy for Muscular Dystrophy - Drug Profile 105
Stryka-232 - Drug Profile 106
Stryka-234 - Drug Profile 107
Stryka-425 - Drug Profile 108
Stryka-533 - Drug Profile 109
Stryka-978 - Drug Profile 110
tideglusib - Drug Profile 111
trehalose - Drug Profile 112
TXA-127 - Drug Profile 116
VAL-0411 - Drug Profile 119
VAL-1205 - Drug Profile 120
Muscular Dystrophy - Dormant Projects 121
Muscular Dystrophy - Discontinued Products 123
Muscular Dystrophy - Product Development Milestones 124
Featured News and Press Releases 124
Appendix 139
Methodology 139
Coverage 139
Secondary Research 139
Primary Research 139
Expert Panel Validation 139
Contact Us 139
Disclaimer 140


List of Tables
Number of Products under Development for Muscular Dystrophy, H2 2016 13
Number of Products under Development for Muscular Dystrophy - Comparative Analysis, H2 2016 14
Number of Products under Development by Companies, H2 2016 15
Number of Products under Development by Companies, H2 2016 (Contd..1) 16
Number of Products under Investigation by Universities/Institutes, H2 2016 17
Comparative Analysis by Late Stage Development, H2 2016 18
Comparative Analysis by Clinical Stage Development, H2 2016 19
Comparative Analysis by Early Stage Development, H2 2016 20
Products under Development by Companies, H2 2016 21
Products under Development by Companies, H2 2016 (Contd..1) 22
Products under Development by Companies, H2 2016 (Contd..2) 23
Products under Investigation by Universities/Institutes, H2 2016 24
Muscular Dystrophy - Pipeline by Acceleron Pharma, Inc., H2 2016 25
Muscular Dystrophy - Pipeline by AMO Pharma Limited, H2 2016 26
Muscular Dystrophy - Pipeline by Asahi Kasei Pharma Corp., H2 2016 27
Muscular Dystrophy - Pipeline by aTyr Pharma, Inc., H2 2016 28
Muscular Dystrophy - Pipeline by Benitec Biopharma Limited, H2 2016 29
Muscular Dystrophy - Pipeline by Bio Blast Pharma Ltd., H2 2016 30
Muscular Dystrophy - Pipeline by Biophytis SAS, H2 2016 31
Muscular Dystrophy - Pipeline by Corcept Therapeutics Incorporated, H2 2016 32
Muscular Dystrophy - Pipeline by Evotec AG, H2 2016 33
Muscular Dystrophy - Pipeline by F. Hoffmann-La Roche Ltd., H2 2016 34
Muscular Dystrophy - Pipeline by Genethon, H2 2016 35
Muscular Dystrophy - Pipeline by Ionis Pharmaceuticals, Inc., H2 2016 36
Muscular Dystrophy - Pipeline by Marina Biotech, Inc., H2 2016 37
Muscular Dystrophy - Pipeline by Medestea Research and Production S.p.A., H2 2016 38
Muscular Dystrophy - Pipeline by Novogen Limited, H2 2016 39
Muscular Dystrophy - Pipeline by Pfizer Inc., H2 2016 40
Muscular Dystrophy - Pipeline by Prothelia, Inc., H2 2016 41
Muscular Dystrophy - Pipeline by SanBio, Inc., H2 2016 42
Muscular Dystrophy - Pipeline by Santhera Pharmaceuticals Holding AG, H2 2016 43
Muscular Dystrophy - Pipeline by Sarepta Therapeutics, Inc., H2 2016 44
Muscular Dystrophy - Pipeline by Selecta Biosciences, Inc., H2 2016 45
Muscular Dystrophy - Pipeline by Strykagen Corporation, H2 2016 46
Muscular Dystrophy - Pipeline by Takeda Pharmaceutical Company Limited, H2 2016 47
Muscular Dystrophy - Pipeline by WAVE Life Sciences Ltd., H2 2016 48
Assessment by Monotherapy Products, H2 2016 49
Number of Products by Stage and Target, H2 2016 51
Number of Products by Stage and Mechanism of Action, H2 2016 53
Number of Products by Stage and Route of Administration, H2 2016 55
Number of Products by Stage and Molecule Type, H2 2016 57
Muscular Dystrophy - Dormant Projects, H2 2016 125
Muscular Dystrophy - Dormant Projects (Contd..1), H2 2016 126
Muscular Dystrophy - Discontinued Products, H2 2016 127


List of Figures
Number of Products under Development for Muscular Dystrophy, H2 2016 13
Number of Products under Development for Muscular Dystrophy - Comparative Analysis, H2 2016 14
Number of Products under Development by Companies, H2 2016 15
Number of Products under Investigation by Universities/Institutes, H2 2016 17
Comparative Analysis by Clinical Stage Development, H2 2016 19
Comparative Analysis by Early Stage Products, H2 2016 20
Assessment by Monotherapy Products, H2 2016 49
Number of Products by Top 10 Targets, H2 2016 50
Number of Products by Stage and Top 10 Targets, H2 2016 50
Number of Products by Top 10 Mechanism of Actions, H2 2016 52
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016 52
Number of Products by Routes of Administration, H2 2016 54
Number of Products by Stage and Routes of Administration, H2 2016 54
Number of Products by Top 10 Molecule Types, H2 2016 56
Number of Products by Stage and Top 10 Molecule Types, H2 2016 56

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