Muscular Dystrophy - Pipeline Review, H2 2014

Published: Dec, 2014 | Pages: 113 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Muscular Dystrophy - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Muscular Dystrophy - Pipeline Review, H2 2014', provides an overview of the Muscular Dystrophy's therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Muscular Dystrophy, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Muscular Dystrophy and special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

- The report provides a snapshot of the global therapeutic landscape of Muscular Dystrophy
- The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities 
- The report reviews key players involved in the therapeutics development for Muscular Dystrophy and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects 
- A review of the Muscular Dystrophy products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
- Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- A detailed assessment of monotherapy and combination therapy pipeline projects
- Coverage of the Muscular Dystrophy pipeline on the basis of target, MoA, route of administration and molecule type
- Latest news and deals relating related to pipeline products

Reasons to buy

- Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Develop strategic initiatives by understanding the focus areas of leading companies
- Identify and understand important and diverse types of therapeutics under development for Muscular Dystrophy
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- 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

 Table of Contents
Table of Contents 2
List of Tables 8
List of Figures 9
Introduction 10
Global Markets Direct Report Coverage 10
Muscular Dystrophy Overview 11
Therapeutics Development 12
Pipeline Products for Muscular Dystrophy - Overview 12
Pipeline Products for Muscular Dystrophy - Comparative Analysis 13
Muscular Dystrophy - Therapeutics under Development by Companies 14
Muscular Dystrophy - Therapeutics under Investigation by Universities/Institutes 16
Muscular Dystrophy - Pipeline Products Glance 17
Clinical Stage Products 17
Early Stage Products 18
Muscular Dystrophy - Products under Development by Companies 19
Muscular Dystrophy - Products under Investigation by Universities/Institutes 21
Muscular Dystrophy - Companies Involved in Therapeutics Development 22
Acceleron Pharma, Inc. 22
aTyr Pharma, Inc. 23
Benitec Biopharma Limited 24
Bioblast Pharma Ltd. 25
Calzada Limited 26
Evotec AG 27
F. Hoffmann-La Roche Ltd. 28
Fate Therapeutics, Inc. 29
Genethon 30
Genzyme Corporation 31
Isis Pharmaceuticals, Inc. 32
Marina Biotech, Inc. 33
Novogen Limited 34
Prosensa Therapeutics B.V. 35
Prothelia, Inc. 36
SanBio, Inc. 37
Santhera Pharmaceuticals Holding AG 38
Sarepta Therapeutics, Inc. 39
Takeda Pharmaceutical Company Limited 40
Valentia Biopharma S.L. 41
Zambon Company S.p.A. 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
Product Description 52
Mechanism of Action 52
R&D Progress 52
Antisense Oligonucleotide for Myotonic Dystrophy - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
Antisense Oligonucleotide for Spinal Muscular Atrophy and Myotonic Dystrophy - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
AOD-9604 - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
ATYR-1940 - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Gene Therapy to Activate Dysferlin for Duchenne and Limb Girdle Muscular Dystrophies - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
Gene Therapy to Activate Dysferlin for Dysferlinopathies - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Gene Therapy to Activate Dystrophin for Muscular Dystrophy - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
ISIS-DMPKRx - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
omigapil - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
Pabparna - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
PRO-135 - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
PRT-01 - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
PRT-300 - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
RNAi Gene Therapy to Activate Myotilin for LGMD - Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
RNAi Oligonucleotide for Myotonic Dystrophy - Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
SB-308 - Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
Small Molecule for Muscular Dystrophy and Other Muscle Disorders - Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
Small Molecule to Target RNA for Myotonic Dystrophy - Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
Small Molecules for CNS and Muscular Dystrophy Diseases - Drug Profile 74
Product Description 74
Mechanism of Action 74
R&D Progress 74
Small Molecules for Dysferlinopathies - Drug Profile 75
Product Description 75
Mechanism of Action 75
R&D Progress 75
Small Molecules for Myotonic Dystrophy Type 1 - Drug Profile 76
Product Description 76
Mechanism of Action 76
R&D Progress 76
Small Molecules to Target CUG RNA for Myotonic Dystrophy 1 - Drug Profile 78
Product Description 78
Mechanism of Action 78
R&D Progress 78
Small Molecules to Target RNA for Myotonic Dystrophy - Drug Profile 79
Product Description 79
Mechanism of Action 79
R&D Progress 79
SRT-149 - Drug Profile 80
Product Description 80
Mechanism of Action 80
R&D Progress 80
SRT-152 - Drug Profile 81
Product Description 81
Mechanism of Action 81
R&D Progress 81
Stem Cell Therapy for Muscular Dystrophy - Drug Profile 82
Product Description 82
Mechanism of Action 82
R&D Progress 82
Stem Cell Therapy for Musculoskeletal and Hematological Disorders - Drug Profile 83
Product Description 83
Mechanism of Action 83
R&D Progress 83
trehalose - Drug Profile 84
Product Description 84
Mechanism of Action 84
R&D Progress 84
VAL-0411 - Drug Profile 85
Product Description 85
Mechanism of Action 85
R&D Progress 85
VLT-001 - Drug Profile 86
Product Description 86
Mechanism of Action 86
R&D Progress 86
VLT-002 - Drug Profile 87
Product Description 87
Mechanism of Action 87
R&D Progress 87
VLT-003 - Drug Profile 88
Product Description 88
Mechanism of Action 88
R&D Progress 88
VLT-004 - Drug Profile 89
Product Description 89
Mechanism of Action 89
R&D Progress 89
VLT-005 - Drug Profile 90
Product Description 90
Mechanism of Action 90
R&D Progress 90
VLT-014 - Drug Profile 91
Product Description 91
Mechanism of Action 91
R&D Progress 91
VLT-015 - Drug Profile 92
Product Description 92
Mechanism of Action 92
R&D Progress 92
VLT-016 - Drug Profile 93
Product Description 93
Mechanism of Action 93
R&D Progress 93
VLT-017 - Drug Profile 94
Product Description 94
Mechanism of Action 94
R&D Progress 94
VLT-025 - Drug Profile 95
Product Description 95
Mechanism of Action 95
R&D Progress 95
ZP-042 - Drug Profile 96
Product Description 96
Mechanism of Action 96
R&D Progress 96
Muscular Dystrophy - Recent Pipeline Updates 97
Muscular Dystrophy - Dormant Projects 102
Muscular Dystrophy - Discontinued Products 103
Muscular Dystrophy - Product Development Milestones 104
Featured News & Press Releases 104
Oct 14, 2014: Acceleron Initiates Phase 1 Clinical Trial with Innovative Muscle Drug ACE-083 104
Jul 17, 2014: Santhera Repositions Omigapil in Congenital Muscular Dystrophy and Initiates Clinical Development Program with Public-Private Partners 104
Feb 25, 2014: VLT Biopharma gets the Orphan Drug designation for VLT015 105
Sep 19, 2013: AmorChem invests in the development of a therapeutic to treat myotonic dystrophy 106
Dec 20, 2012: Scripps Florida Scientists Create New Approach to Destroy Disease-Associated RNAs in Cells 107
Dec 03, 2012: Benitec Biopharma's Muscular Dystrophy Program Demonstrates Significant Gene Silencing 107
Sep 12, 2012: An important breakthrough in the fight against muscular dystrophies 108
Jul 09, 2012: New gene transfer strategy shows promise for limb girdle and other muscular dystrophies 109
Apr 11, 2012: Santhera Pharma Joins EU-funded EndoStem Consortium In Preparation For Clinical Study With Omigapil In Congenital Muscular Dystrophies 110
Dec 14, 2011: Calzada Announces New Applications For AOD9604 111
Appendix 112
Methodology 112
Coverage 112
Secondary Research 112
Primary Research 112
Expert Panel Validation 112
Contact Us 113
Disclaimer 113

List of Tables
Number of Products under Development for Muscular Dystrophy, H2 2014 12
Number of Products under Development for Muscular Dystrophy - Comparative Analysis, H2 2014 13
Number of Products under Development by Companies, H2 2014 14
Number of Products under Investigation by Universities/Institutes, H2 2014 16
Comparative Analysis by Clinical Stage Development, H2 2014 17
Comparative Analysis by Early Stage Development, H2 2014 18
Products under Development by Companies, H2 2014 19
Products under Development by Companies, H2 2014 (Contd..1) 20
Products under Investigation by Universities/Institutes, H2 2014 21
Muscular Dystrophy - Pipeline by Acceleron Pharma, Inc., H2 2014 22
Muscular Dystrophy - Pipeline by aTyr Pharma, Inc., H2 2014 23
Muscular Dystrophy - Pipeline by Benitec Biopharma Limited, H2 2014 24
Muscular Dystrophy - Pipeline by Bioblast Pharma Ltd., H2 2014 25
Muscular Dystrophy - Pipeline by Calzada Limited, H2 2014 26
Muscular Dystrophy - Pipeline by Evotec AG, H2 2014 27
Muscular Dystrophy - Pipeline by F. Hoffmann-La Roche Ltd., H2 2014 28
Muscular Dystrophy - Pipeline by Fate Therapeutics, Inc., H2 2014 29
Muscular Dystrophy - Pipeline by Genethon, H2 2014 30
Muscular Dystrophy - Pipeline by Genzyme Corporation, H2 2014 31
Muscular Dystrophy - Pipeline by Isis Pharmaceuticals, Inc., H2 2014 32
Muscular Dystrophy - Pipeline by Marina Biotech, Inc., H2 2014 33
Muscular Dystrophy - Pipeline by Novogen Limited, H2 2014 34
Muscular Dystrophy - Pipeline by Prosensa Therapeutics B.V., H2 2014 35
Muscular Dystrophy - Pipeline by Prothelia, Inc., H2 2014 36
Muscular Dystrophy - Pipeline by SanBio, Inc., H2 2014 37
Muscular Dystrophy - Pipeline by Santhera Pharmaceuticals Holding AG, H2 2014 38
Muscular Dystrophy - Pipeline by Sarepta Therapeutics, Inc., H2 2014 39
Muscular Dystrophy - Pipeline by Takeda Pharmaceutical Company Limited, H2 2014 40
Muscular Dystrophy - Pipeline by Valentia Biopharma S.L., H2 2014 41
Muscular Dystrophy - Pipeline by Zambon Company S.p.A., H2 2014 42
Assessment by Monotherapy Products, H2 2014 43
Number of Products by Stage and Target, H2 2014 45
Number of Products by Stage and Mechanism of Action, H2 2014 47
Number of Products by Stage and Route of Administration, H2 2014 49
Number of Products by Stage and Molecule Type, H2 2014 51
Muscular Dystrophy Therapeutics - Recent Pipeline Updates, H2 2014 97
Muscular Dystrophy - Dormant Projects, H2 2014 102
Muscular Dystrophy - Discontinued Products, H2 2014 103