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Myotonic Dystrophy - Pipeline Review, H1 2016

  • February 2016
  • -
  • Global Markets Direct
  • -
  • 63 pages

Myotonic Dystrophy - Pipeline Review, H1 2016

Summary

Global Markets Direct’s, ‘Myotonic Dystrophy - Pipeline Review, H1 2016’, provides an overview of the Myotonic Dystrophy pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Myotonic 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 Myotonic 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 Myotonic Dystrophy
- The report reviews pipeline therapeutics for Myotonic 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 Myotonic Dystrophy therapeutics and enlists all their major and minor projects
- The report assesses Myotonic 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 Myotonic 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 Myotonic 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 Myotonic 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

Myotonic Dystrophy - Pipeline Review, H1 2016
Table of Contents
Table of Contents 2
List of Tables 6
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Myotonic Dystrophy Overview 8
Therapeutics Development 9
Pipeline Products for Myotonic Dystrophy - Overview 9
Pipeline Products for Myotonic Dystrophy - Comparative Analysis 10
Myotonic Dystrophy - Therapeutics under Development by Companies 11
Myotonic Dystrophy - Therapeutics under Investigation by Universities/Institutes 12
Myotonic Dystrophy - Pipeline Products Glance 13
Clinical Stage Products 13
Early Stage Products 14
Myotonic Dystrophy - Products under Development by Companies 15
Myotonic Dystrophy - Products under Investigation by Universities/Institutes 16
Myotonic Dystrophy - Companies Involved in Therapeutics Development 17
AMO Pharma Limited 17
BioMarin Pharmaceutical Inc. 18
F. Hoffmann-La Roche Ltd. 19
Ionis Pharmaceuticals, Inc. 20
Marina Biotech, Inc. 21
Valentia Biopharma S.L. 22
WAVE Life Sciences Ltd. 23
Myotonic Dystrophy - Therapeutics Assessment 24
Assessment by Monotherapy Products 24
Assessment by Target 25
Assessment by Mechanism of Action 27
Assessment by Route of Administration 29
Assessment by Molecule Type 31
Drug Profiles 33
AMO-02 - Drug Profile 33
Product Description 33
Mechanism of Action 33
RandD Progress 33
Antisense RNAi Oligonucleotides for Myotonic Dystrophy - Drug Profile 34
Product Description 34
Mechanism of Action 34
RandD Progress 34
IONIS-DMPK2.5Rx - Drug Profile 35
Product Description 35
Mechanism of Action 35
RandD Progress 35
Oligonucleotide 1 to Target Dystrophia Myotonica Protein Kinase for Myotonic Dystrophy - Drug Profile 36
Product Description 36
Mechanism of Action 36
RandD Progress 36
Oligonucleotides to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile 37
Product Description 37
Mechanism of Action 37
RandD Progress 37
PRO-135 - Drug Profile 38
Product Description 38
Mechanism of Action 38
RandD Progress 38
Small Molecule to Target CUG RNA for Myotonic Dystrophy 1 - Drug Profile 39
Product Description 39
Mechanism of Action 39
RandD Progress 39
Small Molecule to Target RNA for Myotonic Dystrophy - Drug Profile 40
Product Description 40
Mechanism of Action 40
RandD Progress 40
Small Molecules for Myotonic Dystrophy Type 1 - Drug Profile 41
Product Description 41
Mechanism of Action 41
RandD Progress 41
Small Molecules to Target RNA for Myotonic Dystrophy - Drug Profile 43
Product Description 43
Mechanism of Action 43
RandD Progress 43
SRT-149 - Drug Profile 44
Product Description 44
Mechanism of Action 44
RandD Progress 44
SRT-152 - Drug Profile 45
Product Description 45
Mechanism of Action 45
RandD Progress 45
VAL-0411 - Drug Profile 46
Product Description 46
Mechanism of Action 46
RandD Progress 46
VLT-001 - Drug Profile 47
Product Description 47
Mechanism of Action 47
RandD Progress 47
VLT-002 - Drug Profile 48
Product Description 48
Mechanism of Action 48
RandD Progress 48
VLT-003 - Drug Profile 49
Product Description 49
Mechanism of Action 49
RandD Progress 49
VLT-004 - Drug Profile 50
Product Description 50
Mechanism of Action 50
RandD Progress 50
VLT-005 - Drug Profile 51
Product Description 51
Mechanism of Action 51
RandD Progress 51
VLT-014 - Drug Profile 52
Product Description 52
Mechanism of Action 52
RandD Progress 52
VLT-015 - Drug Profile 53
Product Description 53
Mechanism of Action 53
RandD Progress 53
VLT-016 - Drug Profile 54
Product Description 54
Mechanism of Action 54
RandD Progress 54
VLT-017 - Drug Profile 55
Product Description 55
Mechanism of Action 55
RandD Progress 55
VLT-025 - Drug Profile 56
Product Description 56
Mechanism of Action 56
RandD Progress 56
Myotonic Dystrophy - Recent Pipeline Updates 57
Myotonic Dystrophy - Dormant Projects 58
Myotonic Dystrophy - Product Development Milestones 59
Featured News and Press Releases 59
Feb 25, 2014: VLT Biopharma gets the Orphan Drug designation for VLT015 59
Sep 19, 2013: AmorChem invests in the development of a therapeutic to treat myotonic dystrophy 59
Dec 20, 2012: Scripps Florida Scientists Create New Approach to Destroy Disease-Associated RNAs in Cells 60
Appendix 62
Methodology 62
Coverage 62
Secondary Research 62
Primary Research 62
Expert Panel Validation 62
Contact Us 62
Disclaimer 63

List of Tables
Number of Products under Development for Myotonic Dystrophy, H1 2016 9
Number of Products under Development for Myotonic Dystrophy - Comparative Analysis, H1 2016 10
Number of Products under Development by Companies, H1 2016 11
Number of Products under Investigation by Universities/Institutes, H1 2016 12
Comparative Analysis by Clinical Stage Development, H1 2016 13
Comparative Analysis by Early Stage Development, H1 2016 14
Products under Development by Companies, H1 2016 15
Products under Investigation by Universities/Institutes, H1 2016 16
Myotonic Dystrophy - Pipeline by AMO Pharma Limited, H1 2016 17
Myotonic Dystrophy - Pipeline by BioMarin Pharmaceutical Inc., H1 2016 18
Myotonic Dystrophy - Pipeline by F. Hoffmann-La Roche Ltd., H1 2016 19
Myotonic Dystrophy - Pipeline by Ionis Pharmaceuticals, Inc., H1 2016 20
Myotonic Dystrophy - Pipeline by Marina Biotech, Inc., H1 2016 21
Myotonic Dystrophy - Pipeline by Valentia Biopharma S.L., H1 2016 22
Myotonic Dystrophy - Pipeline by WAVE Life Sciences Ltd., H1 2016 23
Assessment by Monotherapy Products, H1 2016 24
Number of Products by Stage and Target, H1 2016 26
Number of Products by Stage and Mechanism of Action, H1 2016 28
Number of Products by Stage and Route of Administration, H1 2016 30
Number of Products by Stage and Molecule Type, H1 2016 32
Myotonic Dystrophy Therapeutics - Recent Pipeline Updates, H1 2016 57
Myotonic Dystrophy - Dormant Projects, H1 2016 58

List of Figures
Number of Products under Development for Myotonic Dystrophy, H1 2016 9
Number of Products under Development for Myotonic Dystrophy - Comparative Analysis, H1 2016 10
Number of Products under Development by Companies, H1 2016 11
Comparative Analysis by Early Stage Products, H1 2016 14
Assessment by Monotherapy Products, H1 2016 24
Number of Products by Targets, H1 2016 25
Number of Products by Stage and Targets, H1 2016 25
Number of Products by Mechanism of Actions, H1 2016 27
Number of Products by Stage and Mechanism of Actions, H1 2016 27
Number of Products by Routes of Administration, H1 2016 29
Number of Products by Stage and Routes of Administration, H1 2016 29
Number of Products by Molecule Types, H1 2016 31
Number of Products by Stage and Molecule Types, H1 2016 31

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