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Muscular Dystrophy-Pipeline Review, H1 2015

Muscular Dystrophy-Pipeline Review, H1 2015


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Executive Summary

Muscular Dystrophy-Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Muscular Dystrophy-Pipeline Review, H1 2015', 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 4

List of Figures 5

Introduction 6

Global Markets Direct Report Coverage 6

Muscular Dystrophy Overview 7

Therapeutics Development 8

Pipeline Products for Muscular Dystrophy-Overview 8

Pipeline Products for Muscular Dystrophy-Comparative Analysis 9

Muscular Dystrophy-Therapeutics under Development by Companies 10

Muscular Dystrophy-Therapeutics under Investigation by Universities/Institutes 12

Muscular Dystrophy-Pipeline Products Glance 13

Late Stage Products 13

Clinical Stage Products 14

Early Stage Products 15

Muscular Dystrophy-Products under Development by Companies 16

Muscular Dystrophy-Products under Investigation by Universities/Institutes 19

Muscular Dystrophy-Companies Involved in Therapeutics Development 20

Acceleron Pharma, Inc. 20

aTyr Pharma, Inc. 21

Benitec Biopharma Limited 22

Bioblast Pharma Ltd. 23

BioMarin Pharmaceutical Inc. 24

Evotec AG 25

F. Hoffmann-La Roche Ltd. 26

Fate Therapeutics, Inc. 27

Genethon 28

Genzyme Corporation 29

Isis Pharmaceuticals, Inc. 30

Marina Biotech, Inc. 31

Novogen Limited 32

PolyNovo Limited 33

Prothelia, Inc. 34

SanBio, Inc. 35

Santhera Pharmaceuticals Holding AG 36

Sarepta Therapeutics, Inc. 37

Selecta Biosciences, Inc. 38

Takeda Pharmaceutical Company Limited 39

Valentia Biopharma S.L. 40

Muscular Dystrophy-Therapeutics Assessment 41

Assessment by Monotherapy Products 41

Assessment by Target 42

Assessment by Mechanism of Action 44

Assessment by Route of Administration 46

Assessment by Molecule Type 48

Drug Profiles 50

ACE-083-Drug Profile 50

Antisense Oligonucleotide for Myotonic Dystrophy-Drug Profile 51

Antisense Oligonucleotide for Spinal Muscular Atrophy and Myotonic Dystrophy-Drug Profile 52

AOD-9604-Drug Profile 53

ATYR-1940-Drug Profile 55

Gene Therapy for Muscular Dystrophy and Liver Diseases-Drug Profile 56

Gene Therapy to Activate Dysferlin for Duchenne and Limb Girdle Muscular Dystrophies-Drug Profile 57

Gene Therapy to Activate Dysferlin for Dysferlinopathies-Drug Profile 58

Gene Therapy to Activate Dystrophin for Muscular Dystrophy-Drug Profile 59

ISIS-DMPK-2.5-Drug Profile 60

ketoprofen-Drug Profile 61

omigapil-Drug Profile 62

Pabparna-Drug Profile 64

PRO-135-Drug Profile 65

PRT-01-Drug Profile 66

PRT-300-Drug Profile 67

Recombinant Protein to Activate Utrophin for Muscular Dystrophies-Drug Profile 68

RNAi Gene Therapy to Inhibit Myotilin for LGMD-Drug Profile 69

RNAi Oligonucleotides for Myotonic Dystrophy-Drug Profile 70

SB-308-Drug Profile 71

Small Molecule to Target CUG RNA for Myotonic Dystrophy 1-Drug Profile 72

Small Molecule to Target RNA for Myotonic Dystrophy-Drug Profile 73

Small Molecules for CNS and Muscular Dystrophy Diseases-Drug Profile 74

Small Molecules for Dysferlinopathies-Drug Profile 75

Small Molecules for Myotonic Dystrophy Type 1-Drug Profile 76

Small Molecules to Target RNA for Myotonic Dystrophy-Drug Profile 78

SRT-149-Drug Profile 79

SRT-152-Drug Profile 80

Stem Cell Therapy for Muscular Dystrophy-Drug Profile 81

Stem Cell Therapy for Musculoskeletal Disorders-Drug Profile 82

Stryka-232-Drug Profile 83

Stryka-234-Drug Profile 84

Stryka-425-Drug Profile 85

Stryka-533-Drug Profile 86

Stryka-978-Drug Profile 87

trehalose-Drug Profile 88

VAL-0411-Drug Profile 89

VLT-001-Drug Profile 90

VLT-002-Drug Profile 91

VLT-003-Drug Profile 92

VLT-004-Drug Profile 93

VLT-005-Drug Profile 94

VLT-014-Drug Profile 95

VLT-015-Drug Profile 96

VLT-016-Drug Profile 97

VLT-017-Drug Profile 98

VLT-025-Drug Profile 99

Muscular Dystrophy-Recent Pipeline Updates 100

Muscular Dystrophy-Dormant Projects 106

Muscular Dystrophy-Discontinued Products 107

Muscular Dystrophy-Product Development Milestones 108

Featured News & Press Releases 108

Appendix 116

Methodology 116

Coverage 116

Secondary Research 116

Primary Research 116

Expert Panel Validation 116

Contact Us 116

Disclaimer 117

List of Tables

Number of Products under Development for Muscular Dystrophy, H1 2015 12

Number of Products under Development for Muscular Dystrophy-Comparative Analysis, H1 2015 13

Number of Products under Development by Companies, H1 2015 14

Number of Products under Development by Companies, H1 2015 (Contd..1) 15

Number of Products under Investigation by Universities/Institutes, H1 2015 16

Comparative Analysis by Late Stage Development, H1 2015 17

Comparative Analysis by Clinical Stage Development, H1 2015 18

Comparative Analysis by Early Stage Development, H1 2015 19

Products under Development by Companies, H1 2015 20

Products under Development by Companies, H1 2015 (Contd..1) 21

Products under Development by Companies, H1 2015 (Contd..2) 22

Products under Investigation by Universities/Institutes, H1 2015 23

Muscular Dystrophy-Pipeline by Acceleron Pharma, Inc., H1 2015 24

Muscular Dystrophy-Pipeline by aTyr Pharma, Inc., H1 2015 25

Muscular Dystrophy-Pipeline by Benitec Biopharma Limited, H1 2015 26

Muscular Dystrophy-Pipeline by Bioblast Pharma Ltd., H1 2015 27

Muscular Dystrophy-Pipeline by BioMarin Pharmaceutical Inc., H1 2015 28

Muscular Dystrophy-Pipeline by Evotec AG, H1 2015 29

Muscular Dystrophy-Pipeline by F. Hoffmann-La Roche Ltd., H1 2015 30

Muscular Dystrophy-Pipeline by Fate Therapeutics, Inc., H1 2015 31

Muscular Dystrophy-Pipeline by Genethon, H1 2015 32

Muscular Dystrophy-Pipeline by Genzyme Corporation, H1 2015 33

Muscular Dystrophy-Pipeline by Isis Pharmaceuticals, Inc., H1 2015 34

Muscular Dystrophy-Pipeline by Marina Biotech, Inc., H1 2015 35

Muscular Dystrophy-Pipeline by Novogen Limited, H1 2015 36

Muscular Dystrophy-Pipeline by PolyNovo Limited, H1 2015 37

Muscular Dystrophy-Pipeline by Prothelia, Inc., H1 2015 38

Muscular Dystrophy-Pipeline by SanBio, Inc., H1 2015 39

Muscular Dystrophy-Pipeline by Santhera Pharmaceuticals Holding AG, H1 2015 40

Muscular Dystrophy-Pipeline by Sarepta Therapeutics, Inc., H1 2015 41

Muscular Dystrophy-Pipeline by Selecta Biosciences, Inc., H1 2015 42

Muscular Dystrophy-Pipeline by Takeda Pharmaceutical Company Limited, H1 2015 43

Muscular Dystrophy-Pipeline by Valentia Biopharma S.L., H1 2015 44

Assessment by Monotherapy Products, H1 2015 45

Number of Products by Stage and Target, H1 2015 47

Number of Products by Stage and Mechanism of Action, H1 2015 49

Number of Products by Stage and Route of Administration, H1 2015 51

Number of Products by Stage and Molecule Type, H1 2015 53

Muscular Dystrophy Therapeutics-Recent Pipeline Updates, H1 2015 104

Muscular Dystrophy-Dormant Projects, H1 2015 110

Muscular Dystrophy-Discontinued Products, H1 2015 111

List of Figures

Number of Products under Development for Muscular Dystrophy, H1 2015 12

Number of Products under Development for Muscular Dystrophy-Comparative Analysis, H1 2015 13

Number of Products under Development by Companies, H1 2015 14

Number of Products under Investigation by Universities/Institutes, H1 2015 16

Comparative Analysis by Early Stage Products, H1 2015 19

Assessment by Monotherapy Products, H1 2015 45

Number of Products by Top 10 Targets, H1 2015 46

Number of Products by Stage and Top 10 Targets, H1 2015 47

Number of Products by Top 10 Mechanism of Actions, H1 2015 48

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 49

Number of Products by Top 10 Routes of Administration, H1 2015 50

Number of Products by Stage and Top 10 Routes of Administration, H1 2015 51

Number of Products by Top 10 Molecule Types, H1 2015 52

Number of Products by Stage and Top 10 Molecule Types, H1 2015 53

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Acceleron Pharma, Inc.

aTyr Pharma, Inc.

Benitec Biopharma Limited

Bioblast Pharma Ltd.

BioMarin Pharmaceutical Inc.

Evotec AG

F. Hoffmann-La Roche Ltd.

Fate Therapeutics, Inc.

Genethon

Genzyme Corporation

Isis Pharmaceuticals, Inc.

Marina Biotech, Inc.

Novogen Limited

PolyNovo Limited

Prothelia, Inc.

SanBio, Inc.

Santhera Pharmaceuticals Holding AG

Sarepta Therapeutics, Inc.

Selecta Biosciences, Inc.

Takeda Pharmaceutical Company Limited

Valentia Biopharma S.L.

Muscular Dystrophy Therapeutic Products under Development, Key Players in Muscular Dystrophy Therapeutics, Muscular Dystrophy Pipeline Overview, Muscular Dystrophy Pipeline, Muscular Dystrophy Pipeline Assessment


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