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Dystrophin (DMD) - Pipeline Review, H2 2016

Published: Aug, 2016 | Pages: 81 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Dystrophin (DMD) - Pipeline Review, H2 2016

Summary

Global Markets Direct's, 'Dystrophin (DMD) - Pipeline Review, H2 2016', provides in depth analysis on Dystrophin (DMD) targeted pipeline therapeutics. 

The report provides comprehensive information on the Dystrophin (DMD), targeted therapeutics, complete with analysis by indications, stage of development, 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 Dystrophin (DMD) targeted therapeutics development 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.
*Updated report will be delivered in 48 hours of order confirmation.

Scope

- The report provides a snapshot of the global therapeutic landscape for Dystrophin (DMD)
- The report reviews Dystrophin (DMD) targeted therapeutics under development 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 in Dystrophin (DMD) targeted therapeutics and enlists all their major and minor projects 
- The report assesses Dystrophin (DMD) targeted therapeutics based on 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 and deals related to Dystrophin (DMD) targeted therapeutics

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 the targeted therapy areas and indications for Dystrophin (DMD)
- Identify the use of drugs for target identification and drug repurposing
- 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 Dystrophin (DMD) development landscape 
- 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
 Table of Contents
Table of Contents 2 List of Tables 6 List of Figures 6 Introduction 7 Global Markets Direct Report Coverage 7 Dystrophin (DMD) Overview 8 Therapeutics Development 9 Dystrophin (DMD) - Products under Development by Stage of Development 9 Dystrophin (DMD) - Products under Development by Therapy Area 10 Dystrophin (DMD) - Products under Development by Indication 11 Dystrophin (DMD) - Pipeline Products Glance 12 Late Stage Products 12 Early Stage Products 13 Dystrophin (DMD) - Products under Development by Companies 14 Dystrophin (DMD) - Products under Development by Universities/Institutes 16 Dystrophin (DMD) - Therapeutics Assessment 18 Assessment by Monotherapy/Combination Products 18 Assessment by Mechanism of Action 19 Assessment by Route of Administration 20 Assessment by Molecule Type 22 Dystrophin (DMD) - Companies Involved in Therapeutics Development 24 Asklepios BioPharmaceutical, Inc. 24 BioMarin Pharmaceutical Inc. 25 Daiichi Sankyo Company, Limited 26 Genethon 27 Nippon Shinyaku Co., Ltd. 28 PTC Therapeutics, Inc. 29 Sarepta Therapeutics, Inc. 30 WAVE Life Sciences Ltd. 31 Dystrophin (DMD) - Drug Profiles 32 Antisense Oligonucleotide 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 32 Product Description 32 Mechanism Of Action 32 R&D Progress 32 Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 33 Product Description 33 Mechanism Of Action 33 R&D Progress 33 Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 34 Product Description 34 Mechanism Of Action 34 R&D Progress 34 Biostrophin - Drug Profile 35 Product Description 35 Mechanism Of Action 35 R&D Progress 35 BMBD-001 - Drug Profile 36 Product Description 36 Mechanism Of Action 36 R&D Progress 36 DS-5141 - Drug Profile 37 Product Description 37 Mechanism Of Action 37 R&D Progress 37 eteplirsen - Drug Profile 38 Product Description 38 Mechanism Of Action 38 R&D Progress 38 Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 49 Product Description 49 Mechanism Of Action 49 R&D Progress 49 Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 50 Product Description 50 Mechanism Of Action 50 R&D Progress 50 Gene Therapy to Activate Dystrophin for Muscular Dystrophy - Drug Profile 51 Product Description 51 Mechanism Of Action 51 R&D Progress 51 NS-065 - Drug Profile 52 Product Description 52 Mechanism Of Action 52 R&D Progress 52 Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 53 Product Description 53 Mechanism Of Action 53 R&D Progress 53 Oligonucleotides 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 54 Product Description 54 Mechanism Of Action 54 R&D Progress 54 Oligonucleotides 2 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 55 Product Description 55 Mechanism Of Action 55 R&D Progress 55 Oligonucleotides 3 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 56 Product Description 56 Mechanism Of Action 56 R&D Progress 56 PRO-055 - Drug Profile 57 Product Description 57 Mechanism Of Action 57 R&D Progress 57 SGT-001 - Drug Profile 59 Product Description 59 Mechanism Of Action 59 R&D Progress 59 Small Molecule to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 60 Product Description 60 Mechanism Of Action 60 R&D Progress 60 SRP-4008 - Drug Profile 61 Product Description 61 Mechanism Of Action 61 R&D Progress 61 SRP-4044 - Drug Profile 62 Product Description 62 Mechanism Of Action 62 R&D Progress 62 SRP-4045 - Drug Profile 63 Product Description 63 Mechanism Of Action 63 R&D Progress 63 SRP-4050 - Drug Profile 64 Product Description 64 Mechanism Of Action 64 R&D Progress 64 SRP-4052 - Drug Profile 65 Product Description 65 Mechanism Of Action 65 R&D Progress 65 SRP-4053 - Drug Profile 66 Product Description 66 Mechanism Of Action 66 R&D Progress 66 SRP-4055 - Drug Profile 67 Product Description 67 Mechanism Of Action 67 R&D Progress 67 Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 68 Product Description 68 Mechanism Of Action 68 R&D Progress 68 Dystrophin (DMD) - Dormant Projects 69 Dystrophin (DMD) - Discontinued Products 70 Dystrophin (DMD) - Featured News & Press Releases 71 Jun 06, 2016: Sarepta Therapeutics Announces FDA Request For Dystrophin Data Prior To Making A Decision on Eteplirsen NDA 71 May 25, 2016: Sarepta Therapeutics Announces FDA Will Not Complete the Review of the Eteplirsen New Drug Application By The PDUFA Date 71 Apr 25, 2016: MDA Chief Medical and Scientific Officer Testifies at Landmark FDA Hearing for Duchenne Drug 71 Apr 25, 2016: Sarepta Issues Statement on Advisory Committee Outcome for Use of Eteplirsen in the Treatment of Duchenne Muscular Dystrophy 72 Mar 14, 2016: Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen 73 Mar 14, 2016: Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy 73 Feb 25, 2016: Daiichi Sankyo Announces Commencement of Clinical Trials in Japan for Duchenne Muscular Dystrophy Treatment 73 Feb 08, 2016: Sarepta Therapeutics Receives Notification of PDUFA Extension for Eteplirsen 74 Jan 20, 2016: FDA Postpones Advisory Committee Meeting to Review Eteplirsen Due to Severe Weather Storm in the Washington D.C. Area 74 Dec 18, 2015: Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping 75 Nov 17, 2015: Sarepta Therapeutics Announces Publication of Positive Long-Term Safety and Efficacy Data for Eteplirsen in the Annals of Neurology 75 Oct 22, 2015: Gene Therapy Treats All Muscles in the Body in Muscular Dystrophy Dogs 76 Oct 14, 2015: Sarepta Therapeutics Announces Tentative FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy 77 Oct 01, 2015: Sarepta Therapeutics Announces Additional Long-Term Efficacy and Safety Data from Pivotal Phase IIb Program of Eteplirsen for Treatment of Duchenne Muscular Dystrophy 77 Aug 25, 2015: Sarepta Therapeutics Announces FDA Has Filed Eteplirsen NDA for the Potential Treatment of Duchenne Muscular Dystrophy for Patients Amenable to Exon 51 Skipping 79 Appendix 80 Methodology 80 Coverage 80 Secondary Research 80 Primary Research 80 Expert Panel Validation 80 Contact Us 80 Disclaimer 81
List of Tables
Number of Products under Development for, H2 2016 9 Number of Products under Development by Therapy Area, H2 2016 10 Number of Products under Development by Indication, H2 2016 11 Comparative Analysis by Late Stage Development, H2 2016 12 Comparative Analysis by Early Stage Products, H2 2016 13 Number of Products under Development by Companies, H2 2016 14 Products under Development by Companies, H2 2016 15 Number of Products under Investigation by Universities/Institutes, H2 2016 16 Products under Investigation by Universities/Institutes, H2 2016 17 Assessment by Monotherapy/Combination Products, H2 2016 18 Number of Products by Stage and Mechanism of Action, H2 2016 19 Number of Products by Stage and Route of Administration, H2 2016 21 Number of Products by Stage and Molecule Type, H2 2016 23 Pipeline by Asklepios BioPharmaceutical, Inc., H2 2016 24 Pipeline by BioMarin Pharmaceutical Inc., H2 2016 25 Pipeline by Daiichi Sankyo Company, Limited, H2 2016 26 Pipeline by Genethon, H2 2016 27 Pipeline by Nippon Shinyaku Co., Ltd., H2 2016 28 Pipeline by PTC Therapeutics, Inc., H2 2016 29 Pipeline by Sarepta Therapeutics, Inc., H2 2016 30 Pipeline by WAVE Life Sciences Ltd., H2 2016 31 Dormant Projects, H2 2016 69 Discontinued Products, H2 2016 70



                                

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