Summary
Global Markets Direct’s, ‘Muscular Dystrophy - Pipeline Review, H1 2016’, provides an overview of the Muscular Dystrophy pipeline landscape.
The report provides comprehensive information on the therapeutics under development for Muscular Dystrophy, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Muscular Dystrophy and features dormant and discontinued projects.
Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
The report 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.
Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.
Scope
- The report provides a snapshot of the global therapeutic landscape of Muscular Dystrophy
- The report reviews pipeline therapeutics for Muscular Dystrophy by companies and universities/research institutes based on information derived from company and industry-specific sources
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
- The report reviews key players involved Muscular Dystrophy therapeutics and enlists all their major and minor projects
- The report assesses Muscular Dystrophy therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type
- The report summarizes all the dormant and discontinued pipeline projects
- The report reviews latest news related to pipeline therapeutics for Muscular Dystrophy
Reasons to buy
- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Identify and understand important and diverse types of therapeutics under development for Muscular Dystrophy
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies
- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding Muscular Dystrophy 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
- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline
Companies Mentioned
Acceleron Pharma, Inc.
AMO Pharma Limited
Asahi Kasei Pharma Corp.
aTyr Pharma, Inc.
Benitec Biopharma Limited
Bioblast Pharma Ltd.
BioMarin Pharmaceutical Inc.
Biophytis SAS
Evotec AG
F. Hoffmann-La Roche Ltd.
Fate Therapeutics, Inc.
Genethon
Ionis Pharmaceuticals, Inc.
Marina Biotech, Inc.
Novogen Limited
Prothelia, Inc.
SanBio, Inc.
Santhera Pharmaceuticals Holding AG
Sarepta Therapeutics, Inc.
Selecta Biosciences, Inc.
Takeda Pharmaceutical Company Limited
WAVE Life Sciences Ltd.
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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
Bioblast Pharma Ltd. 26
BioMarin Pharmaceutical Inc. 27
Biophytis SAS 28
Evotec AG 29
F. Hoffmann-La Roche Ltd. 30
Fate Therapeutics, Inc. 31
Genethon 32
Ionis Pharmaceuticals, Inc. 33
Marina Biotech, Inc. 34
Novogen Limited 35
Prothelia, Inc. 36
SanBio, Inc. 37
Santhera Pharmaceuticals Holding AG 38
Sarepta Therapeutics, Inc. 39
Selecta Biosciences, Inc. 40
Takeda Pharmaceutical Company Limited 41
WAVE Life Sciences Ltd. 42
Muscular Dystrophy - Therapeutics Assessment 43
Assessment by Monotherapy Products 43
Assessment by Target 44
Assessment by Mechanism of Action 46
Assessment by Route of Administration 48
Assessment by Molecule Type 50
Drug Profiles 52
ACE-083 - Drug Profile 52
AMO-02 - Drug Profile 54
Antisense Oligonucleotide to Activate Dystrophin for Muscular Dystrophy - Drug Profile 55
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
BIO-101 - Drug Profile 60
BIO-103 - Drug Profile 61
Drugs for Merosin-Deficient Congenital Muscular Dystrophy Type 1A - Drug Profile 62
elcatonin - Drug Profile 63
Gene Therapy for Muscular Dystrophy and Liver Diseases - Drug Profile 64
Gene Therapy to Activate Dysferlin for Duchenne and Limb Girdle Muscular Dystrophies - Drug Profile 65
Gene Therapy to Activate Dysferlin for Dysferlinopathies - Drug Profile 66
Gene Therapy to Activate Dystrophin for Muscular Dystrophy - Drug Profile 67
IONISDMPK-2.5Rx - Drug Profile 68
IUCT-169 - Drug Profile 70
IUCT-290 - Drug Profile 71
IUCT-309 - Drug Profile 72
ketoprofen - Drug Profile 73
LR-08 - Drug Profile 74
Oligonucleotide 1 to Target Dystrophia Myotonica Protein Kinase for Myotonic Dystrophy - Drug Profile 75
Oligonucleotides to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile 76
omigapil - Drug Profile 77
Pabparna - Drug Profile 79
PRO-135 - Drug Profile 80
PRT-01 - Drug Profile 81
Recombinant Protein to Activate Utrophin for Muscular Dystrophies - Drug Profile 83
RNAi Gene Therapy to Inhibit Myotilin for LGMD - Drug Profile 84
RP-33 - Drug Profile 85
SB-308 - Drug Profile 86
Small Molecule to Target CUG RNA for Myotonic Dystrophy 1 - Drug Profile 87
Small Molecule to Target RNA for Myotonic Dystrophy - Drug Profile 88
Small Molecules for Dysferlinopathies - Drug Profile 89
Small Molecules for Facioscapulohumeral Muscular Dystrophy - Drug Profile 90
Small Molecules for Myotonic Dystrophy - Drug Profile 91
Small Molecules for Myotonic Dystrophy Type 1 - Drug Profile 92
Small Molecules to Activate SMCHD1 for Facioscapulohumeral Dystrophy - Drug Profile 94
Small Molecules to Inhibit MBNL1 for Myotonic Dystrophy Type I - Drug Profile 95
Small Molecules to Target RNA for Myotonic Dystrophy - Drug Profile 96
SRT-149 - Drug Profile 97
SRT-152 - Drug Profile 98
Stem Cell Therapy for Muscular Dystrophy - Drug Profile 99
Stem Cell Therapy for Musculoskeletal Disorders - Drug Profile 100
Stryka-232 - Drug Profile 101
Stryka-234 - Drug Profile 102
Stryka-425 - Drug Profile 103
Stryka-533 - Drug Profile 104
Stryka-978 - Drug Profile 105
trehalose - Drug Profile 106
VAL-0411 - Drug Profile 107
VAL-1205 - Drug Profile 108
Muscular Dystrophy - Recent Pipeline Updates 109
Muscular Dystrophy - Dormant Projects 117
Muscular Dystrophy - Discontinued Products 119
Muscular Dystrophy - Product Development Milestones 120
Featured News & Press Releases 120
Appendix 130
Methodology 130
Coverage 130
Secondary Research 130
Primary Research 130
Expert Panel Validation 130
Contact Us 130
Disclaimer 131
List of Tables
Number of Products under Development for Muscular Dystrophy, H1 2016 13
Number of Products under Development for Muscular Dystrophy - Comparative Analysis, H1 2016 14
Number of Products under Development by Companies, H1 2016 15
Number of Products under Development by Companies, H1 2016 (Contd..1) 16
Number of Products under Investigation by Universities/Institutes, H1 2016 17
Comparative Analysis by Late Stage Development, H1 2016 18
Comparative Analysis by Clinical Stage Development, H1 2016 19
Comparative Analysis by Early Stage Development, H1 2016 20
Products under Development by Companies, H1 2016 21
Products under Development by Companies, H1 2016 (Contd..1) 22
Products under Development by Companies, H1 2016 (Contd..2) 23
Products under Investigation by Universities/Institutes, H1 2016 24
Muscular Dystrophy - Pipeline by Acceleron Pharma, Inc., H1 2016 25
Muscular Dystrophy - Pipeline by AMO Pharma Limited, H1 2016 26
Muscular Dystrophy - Pipeline by Asahi Kasei Pharma Corp., H1 2016 27
Muscular Dystrophy - Pipeline by aTyr Pharma, Inc., H1 2016 28
Muscular Dystrophy - Pipeline by Benitec Biopharma Limited, H1 2016 29
Muscular Dystrophy - Pipeline by Bioblast Pharma Ltd., H1 2016 30
Muscular Dystrophy - Pipeline by BioMarin Pharmaceutical Inc., H1 2016 31
Muscular Dystrophy - Pipeline by Biophytis SAS, H1 2016 32
Muscular Dystrophy - Pipeline by Evotec AG, H1 2016 33
Muscular Dystrophy - Pipeline by F. Hoffmann-La Roche Ltd., H1 2016 34
Muscular Dystrophy - Pipeline by Fate Therapeutics, Inc., H1 2016 35
Muscular Dystrophy - Pipeline by Genethon, H1 2016 36
Muscular Dystrophy - Pipeline by Ionis Pharmaceuticals, Inc., H1 2016 37
Muscular Dystrophy - Pipeline by Marina Biotech, Inc., H1 2016 38
Muscular Dystrophy - Pipeline by Novogen Limited, H1 2016 39
Muscular Dystrophy - Pipeline by Prothelia, Inc., H1 2016 40
Muscular Dystrophy - Pipeline by SanBio, Inc., H1 2016 41
Muscular Dystrophy - Pipeline by Santhera Pharmaceuticals Holding AG, H1 2016 42
Muscular Dystrophy - Pipeline by Sarepta Therapeutics, Inc., H1 2016 43
Muscular Dystrophy - Pipeline by Selecta Biosciences, Inc., H1 2016 44
Muscular Dystrophy - Pipeline by Takeda Pharmaceutical Company Limited, H1 2016 45
Muscular Dystrophy - Pipeline by WAVE Life Sciences Ltd., H1 2016 46
Assessment by Monotherapy Products, H1 2016 47
Number of Products by Stage and Target, H1 2016 49
Number of Products by Stage and Mechanism of Action, H1 2016 51
Number of Products by Stage and Route of Administration, H1 2016 53
Number of Products by Stage and Molecule Type, H1 2016 55
Muscular Dystrophy Therapeutics - Recent Pipeline Updates, H1 2016 113
Muscular Dystrophy - Dormant Projects, H1 2016 121
Muscular Dystrophy - Dormant Projects (Contd..1), H1 2016 122
Muscular Dystrophy - Discontinued Products, H1 2016 123
List of Figures
Number of Products under Development for Muscular Dystrophy, H1 2016 13
Number of Products under Development for Muscular Dystrophy - Comparative Analysis, H1 2016 14
Number of Products under Development by Companies, H1 2016 15
Number of Products under Investigation by Universities/Institutes, H1 2016 17
Comparative Analysis by Clinical Stage Development, H1 2016 19
Comparative Analysis by Early Stage Products, H1 2016 20
Assessment by Monotherapy Products, H1 2016 47
Number of Products by Top 10 Targets, H1 2016 48
Number of Products by Stage and Top 10 Targets, H1 2016 48
Number of Products by Top 10 Mechanism of Actions, H1 2016 50
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2016 50
Number of Products by Routes of Administration, H1 2016 52
Number of Products by Stage and Routes of Administration, H1 2016 52
Number of Products by Molecule Types, H1 2016 54
Number of Products by Stage and Molecule Types, H1 2016 54
