Muscular Dystrophy – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Muscular Dystrophy – Pipeline Review, H2 2017’, 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

Achelios Therapeutics Inc

AMO Pharma Ltd

aTyr Pharma Inc

Benitec Biopharma Ltd

Bio Blast Pharma Ltd

Biophytis SAS

Corcept Therapeutics Inc

Evotec AG

Fulcrum Therapeutics Inc

Genethon SA

Ionis Pharmaceuticals Inc

Marina Biotech Inc

Medestea Research & Production SpA

Novogen Ltd

Pfizer Inc

Prothelia Inc

Recursion Pharmaceuticals Inc

SanBio Inc

Santhera Pharmaceuticals Holding AG

Sarepta Therapeutics Inc

Strykagen Corp

Takeda Pharmaceutical Co Ltd

Ultragenyx Pharmaceutical Inc

WAVE Life Sciences Ltd

Table of Contents

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

Muscular Dystrophy - Therapeutics Development 9

Pipeline Overview 9

Pipeline by Companies 10

Pipeline by Universities/Institutes 13

Products under Development by Companies 14

Products under Development by Universities/Institutes 17

Muscular Dystrophy - Therapeutics Assessment 18

Assessment by Target 18

Assessment by Mechanism of Action 20

Assessment by Route of Administration 22

Assessment by Molecule Type 24

Muscular Dystrophy - Companies Involved in Therapeutics Development 26

Acceleron Pharma Inc 26

Achelios Therapeutics Inc 26

AMO Pharma Ltd 26

aTyr Pharma Inc 27

Benitec Biopharma Ltd 27

Bio Blast Pharma Ltd 28

Biophytis SAS 28

Corcept Therapeutics Inc 28

Evotec AG 29

Fulcrum Therapeutics Inc 30

Genethon SA 30

Ionis Pharmaceuticals Inc 30

Marina Biotech Inc 31

Medestea Research & Production SpA 31

Novogen Ltd 32

Pfizer Inc 32

Prothelia Inc 33

Recursion Pharmaceuticals Inc 33

SanBio Inc 33

Santhera Pharmaceuticals Holding AG 34

Sarepta Therapeutics Inc 34

Strykagen Corp 35

Takeda Pharmaceutical Co Ltd 35

Ultragenyx Pharmaceutical Inc 36

WAVE Life Sciences Ltd 36

Muscular Dystrophy - Drug Profiles 38

ACE-083 - Drug Profile 38

Antisense Oligonucleotide to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile 41

Antisense RNAi Oligonucleotide to Inhibit DUX4 for Facioscapulohumeral Muscular Dystrophy - Drug Profile 42

Antisense RNAi Oligonucleotides for Myotonic Dystrophy - Drug Profile 43

ATYR-1940 - Drug Profile 44

AVGN-7 - Drug Profile 48

baliforsen - Drug Profile 49

BB-301 - Drug Profile 50

BIO-103 - Drug Profile 52

domagrozumab - Drug Profile 53

Fusion Protein to Activate Utrophin for Muscular Dystrophies - Drug Profile 55

Gene Therapy to Activate Dysferlin for Dysferlinopathies - Drug Profile 56

Gene Therapy to Activate Dystrophin for Muscular Dystrophy - Drug Profile 57

IUCT-169 - Drug Profile 58

IUCT-290 - Drug Profile 59

IUCT-309 - Drug Profile 60

ketoprofen - Drug Profile 61

LR-08 - Drug Profile 62

mexiletine hydrochloride - Drug Profile 63

MYO-101 - Drug Profile 64

MYO-102 - Drug Profile 65

MYO-103 - Drug Profile 66

MYO-201 - Drug Profile 67

MYO-301 - Drug Profile 68

Oligonucleotide 1 to Target Dystrophia Myotonica Protein Kinase for Myotonic Dystrophy Type 1 - Drug Profile 69

Oligonucleotides to Inhibit DM1 Protein Kinase for Myotonic Dystrophy Type 1 - Drug Profile 70

omigapil - Drug Profile 71

poloxamer - Drug Profile 73

PRT-01 - Drug Profile 76

RNAi Gene Therapy to Inhibit Myotilin for LGMD - Drug Profile 78

RP-33 - Drug Profile 79

SB-308 - Drug Profile 80

SIWA-318 - Drug Profile 81

Small Molecule to Inhibit DUX4 for Muscular Dystrophy - Drug Profile 82

Small Molecule to Target CUG RNA for Myotonic Dystrophy 1 - Drug Profile 83

Small Molecule to Target RNA for Myotonic Dystrophy Type 1 - Drug Profile 84

Small Molecules for Dysferlinopathies - Drug Profile 85

Small Molecules for Emery-Dreifuss Muscular Dystrophy - Drug Profile 86

Small Molecules for Facioscapulohumeral Muscular Dystrophy - Drug Profile 87

Small Molecules for Facioscapulohumeral Muscular Dystrophy - Drug Profile 88

Small Molecules for Merosin-Deficient Congenital Muscular Dystrophy Type 1A - Drug Profile 89

Small Molecules for Myotonic Dystrophy - Drug Profile 90

Small Molecules for Pain - Drug Profile 91

Small Molecules for Walker-Warburg syndrome - Drug Profile 92

Small Molecules to Activate SMCHD1 for Facioscapulohumeral Dystrophy - Drug Profile 93

Small Molecules to Antagonize Glucocorticoid Receptor II for Muscular 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

Stem Cell Therapy for Muscular Dystrophy - Drug Profile 97

Stem Cell Therapy to Activate ZSCAN4 for Muscular Dystrophy - Drug Profile 98

Stryka-232 - Drug Profile 99

Stryka-234 - Drug Profile 100

Stryka-425 - Drug Profile 101

Stryka-533 - Drug Profile 102

Stryka-978 - Drug Profile 103

tideglusib - Drug Profile 104

trehalose - Drug Profile 107

TXA-127 - Drug Profile 112

VAL-0411 - Drug Profile 116

WITH-1101 - Drug Profile 117

XN-001 - Drug Profile 118

Muscular Dystrophy - Dormant Projects 119

Muscular Dystrophy - Discontinued Products 121

Muscular Dystrophy - Product Development Milestones 122

Featured News & Press Releases 122

Appendix 134

Methodology 134

Coverage 134

Secondary Research 134

Primary Research 134

Expert Panel Validation 134

Contact Us 134

Disclaimer 135

List of Tables

List of Tables

Number of Products under Development for Muscular Dystrophy, H2 2017 13

Number of Products under Development by Companies, H2 2017 15

Number of Products under Development by Companies, H2 2017 (Contd..1), H2 2017 16

Number of Products under Development by Universities/Institutes, H2 2017 17

Products under Development by Companies, H2 2017 18

Products under Development by Companies, H2 2017 (Contd..1), H2 2017 19

Products under Development by Companies, H2 2017 (Contd..2), H2 2017 20

Products under Development by Universities/Institutes, H2 2017 21

Number of Products by Stage and Target, H2 2017 23

Number of Products by Stage and Mechanism of Action, H2 2017 25

Number of Products by Stage and Route of Administration, H2 2017 27

Number of Products by Stage and Molecule Type, H2 2017 29

Muscular Dystrophy – Pipeline by Acceleron Pharma Inc, H2 2017 30

Muscular Dystrophy – Pipeline by Achelios Therapeutics Inc, H2 2017 30

Muscular Dystrophy – Pipeline by AMO Pharma Ltd, H2 2017 31

Muscular Dystrophy – Pipeline by aTyr Pharma Inc, H2 2017 31

Muscular Dystrophy – Pipeline by Benitec Biopharma Ltd, H2 2017 31

Muscular Dystrophy – Pipeline by Bio Blast Pharma Ltd, H2 2017 32

Muscular Dystrophy – Pipeline by Biophytis SAS, H2 2017 32

Muscular Dystrophy – Pipeline by Corcept Therapeutics Inc, H2 2017 33

Muscular Dystrophy – Pipeline by Evotec AG, H2 2017 33

Muscular Dystrophy – Pipeline by Fulcrum Therapeutics Inc, H2 2017 34

Muscular Dystrophy – Pipeline by Genethon SA, H2 2017 34

Muscular Dystrophy – Pipeline by Ionis Pharmaceuticals Inc, H2 2017 35

Muscular Dystrophy – Pipeline by Marina Biotech Inc, H2 2017 35

Muscular Dystrophy – Pipeline by Medestea Research & Production SpA, H2 2017 36

Muscular Dystrophy – Pipeline by Novogen Ltd, H2 2017 36

Muscular Dystrophy – Pipeline by Pfizer Inc, H2 2017 37

Muscular Dystrophy – Pipeline by Prothelia Inc, H2 2017 37

Muscular Dystrophy – Pipeline by Recursion Pharmaceuticals Inc, H2 2017 37

Muscular Dystrophy – Pipeline by SanBio Inc, H2 2017 38

Muscular Dystrophy – Pipeline by Santhera Pharmaceuticals Holding AG, H2 2017 38

Muscular Dystrophy – Pipeline by Sarepta Therapeutics Inc, H2 2017 39

Muscular Dystrophy – Pipeline by Strykagen Corp, H2 2017 39

Muscular Dystrophy – Pipeline by Takeda Pharmaceutical Co Ltd, H2 2017 40

Muscular Dystrophy – Pipeline by Ultragenyx Pharmaceutical Inc, H2 2017 40

Muscular Dystrophy – Pipeline by WAVE Life Sciences Ltd, H2 2017 41

Muscular Dystrophy – Dormant Projects, H2 2017 123

Muscular Dystrophy – Dormant Projects, H2 2017 (Contd..1), H2 2017 124

Muscular Dystrophy – Discontinued Products, H2 2017 125

List of Figures

List of Figures

Number of Products under Development for Muscular Dystrophy, H2 2017 13

Number of Products under Development by Companies, H2 2017 14

Number of Products under Development by Universities/Institutes, H2 2017 17

Number of Products by Top 10 Targets, H2 2017 22

Number of Products by Stage and Top 10 Targets, H2 2017 22

Number of Products by Top 10 Mechanism of Actions, H2 2017 24

Number of Products by Stage and Mechanism of Actions, H2 2017 24

Number of Products by Routes of Administration, H2 2017 26

Number of Products by Stage and Routes of Administration, H2 2017 26

Number of Products by Top 10 Molecule Types, H2 2017 28

Number of Products by Stage and Top 10 Molecule Types, H2 2017 28

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