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Duchenne Muscular Dystrophy - Pipeline Review, H1 2016

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

Duchenne Muscular Dystrophy - Pipeline Review, H1 2016

Summary

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

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

Duchenne Muscular Dystrophy - Pipeline Review, H1 2016
Table of Contents
Table of Contents 2
List of Tables 6
List of Figures 8
Introduction 9
Global Markets Direct Report Coverage 9
Duchenne Muscular Dystrophy Overview 10
Therapeutics Development 11
Pipeline Products for Duchenne Muscular Dystrophy - Overview 11
Pipeline Products for Duchenne Muscular Dystrophy - Comparative Analysis 12
Duchenne Muscular Dystrophy - Therapeutics under Development by Companies 13
Duchenne Muscular Dystrophy - Therapeutics under Investigation by Universities/Institutes 18
Duchenne Muscular Dystrophy - Pipeline Products Glance 19
Late Stage Products 19
Clinical Stage Products 20
Early Stage Products 21
Unknown Stage Products 22
Duchenne Muscular Dystrophy - Products under Development by Companies 23
Duchenne Muscular Dystrophy - Products under Investigation by Universities/Institutes 29
Duchenne Muscular Dystrophy - Companies Involved in Therapeutics Development 30
Acceleron Pharma, Inc. 30
Anagenesis Biotechnology 31
Asklepios BioPharmaceutical, Inc. 32
Bamboo Therapeutics, Inc. 33
BioMarin Pharmaceutical Inc. 34
Bristol-Myers Squibb Company 35
Capricor Therapeutics, Inc. 36
Catabasis Pharmaceuticals, Inc. 37
Daiichi Sankyo Company, Limited 38
Debiopharm International S.A. 39
Editas Medicine, Inc. 40
Eli Lilly and Company 41
EryDel SPA 42
FibroGen, Inc. 43
Galapagos NV 44
Genethon 45
GTx, Inc. 46
Idera Pharmaceuticals, Inc. 47
Italfarmaco S.p.A. 48
La Jolla Pharmaceutical Company 49
Marathon Pharmaceuticals, LLC 50
Marina Biotech, Inc. 51
Merck KGaA 52
Milo Biotechnology LLC 53
MyoTherix Inc. 54
NicOx S.A. 55
Nippon Shinyaku Co., Ltd. 56
nLife Therapeutics, S.L. 57
Nobelpharma Co., Ltd. 58
Pfizer Inc. 59
Prothelia, Inc. 60
PTC Therapeutics, Inc. 61
RASRx, LLC 62
ReveraGen BioPharma, Inc. 63
Sanofi 64
Santhera Pharmaceuticals Holding AG 65
Sarepta Therapeutics, Inc. 66
Summit Therapeutics Plc 67
Taiho Pharmaceutical Co., Ltd. 68
WAVE Life Sciences Ltd. 69
Zambon Company S.p.A. 70
Duchenne Muscular Dystrophy - Therapeutics Assessment 71
Assessment by Monotherapy Products 71
Assessment by Target 72
Assessment by Mechanism of Action 75
Assessment by Route of Administration 78
Assessment by Molecule Type 80
Drug Profiles 82
4-P001 - Drug Profile 82
AAV1-Foliistatin - Drug Profile 83
ACE-083 - Drug Profile 84
alisporivir - Drug Profile 85
Antisense Oligonucleotide 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 87
Antisense Oligonucleotide to Activate Dystrophin for Duchene Muscular Dystrophy - Drug Profile 88
Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 89
Antisense Oligonucleotide to Inhibit Myostatin for DMD and Becker MD - Drug Profile 90
Antisense Oligonucleotides to Inhibit ACVR2B for Duchenne Muscular Dystrophy - Drug Profile 91
Antisense RNAi Oligonucleotides for Duchenne Muscular Dystrophy - Drug Profile 92
Aptamer to Activate Calcineurin A for Genetic Disorders and Musculoskeletal Disorders - Drug Profile 93
arbekacin - Drug Profile 94
ARM-210 - Drug Profile 95
AT-300 - Drug Profile 96
ataluren - Drug Profile 98
Biostrophin - Drug Profile 102
BMBD-001 - Drug Profile 103
BMN-044 - Drug Profile 104
BMN-045 - Drug Profile 106
BMN-053 - Drug Profile 108
BMS-986089 - Drug Profile 110
CAP-1002 - Drug Profile 111
CAT-1004 - Drug Profile 113
CAT-1040 - Drug Profile 115
CAT-1041 - Drug Profile 116
DA-4210 - Drug Profile 117
deflazacort - Drug Profile 118
dexamethasone sodium phosphate - Drug Profile 119
domagrozumab - Drug Profile 121
drisapersen - Drug Profile 122
Drug for Duchenne Muscular Dystrophy - Drug Profile 124
Drugs for Duchenne Muscular Dystrophy - Drug Profile 125
DS-5141b - Drug Profile 126
DT-200 - Drug Profile 127
enobosarm - Drug Profile 129
eteplirsen - Drug Profile 131
FG-3019 - Drug Profile 134
Gene Therapy for Duchenne Muscular Dystrophy - Drug Profile 136
Gene Therapy to Activate Dysferlin for Duchenne and Limb Girdle Muscular Dystrophies - Drug Profile 137
Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 138
Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 139
Gene Therapy to Activate Dystrophine for Duchenne Muscular Dystrophy - Drug Profile 140
givinostat - Drug Profile 141
Gtx-027 - Drug Profile 143
IB-DMD - Drug Profile 144
idebenone - Drug Profile 145
IMO-8400 - Drug Profile 148
LJPC-30Sa - Drug Profile 150
LJPC-30Sb - Drug Profile 151
MG-53 - Drug Profile 152
MP-101 - Drug Profile 154
Myomir - Drug Profile 155
naproxcinod - Drug Profile 156
NBD Peptide - Drug Profile 158
NS-065 - Drug Profile 160
Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 161
Oligonucleotides 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 162
Oligonucleotides 2 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 163
Oligonucleotides 3 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 164
Oligonucleotides for Ischemic Heart Disease and Duchenne Muscular Dystrophy - Drug Profile 165
OSX-200 - Drug Profile 166
poloxamer - Drug Profile 167
PRO-052 - Drug Profile 169
PRO-055 - Drug Profile 171
PRT-01 - Drug Profile 173
Recombinant Protein for Muscular Dystrophy - Drug Profile 175
Recombinant Protein to Activate Utrophin for Muscular Dystrophies - Drug Profile 176
Recombinant Proteins for Duchenne Muscular Dystrophy - Drug Profile 177
rimeporide - Drug Profile 178
RTC-13 - Drug Profile 179
Small Molecule for Duchenne Muscular Dystrophy - Drug Profile 181
Small Molecule for Duchenne Muscular Dystrophy - Drug Profile 182
Small Molecule to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 183
Small Molecules for Duchenne Muscular Dystrophy - Drug Profile 184
Small Molecules to Target Utrophin for Duchenne Muscular Dystrophy - Drug Profile 185
SMT-022357 - Drug Profile 186
SMTC-1100 - Drug Profile 187
SRP-4008 - Drug Profile 189
SRP-4044 - Drug Profile 190
SRP-4045 - Drug Profile 191
SRP-4050 - Drug Profile 192
SRP-4052 - Drug Profile 193
SRP-4053 - Drug Profile 194
SRP-4055 - Drug Profile 195
Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 196
Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 198
Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 199
Stryka-516 - Drug Profile 200
Stryka-969 - Drug Profile 201
StrykaPro-1 - Drug Profile 202
Synthetic Peptides for Duchene Muscle Dystrophy - Drug Profile 203
tadalafil - Drug Profile 204
TAS-205 - Drug Profile 206
TVN-102 - Drug Profile 207
TXA-127 - Drug Profile 208
Verolone - Drug Profile 211
ZP-049 - Drug Profile 213
Duchenne Muscular Dystrophy - Recent Pipeline Updates 214
Duchenne Muscular Dystrophy - Dormant Projects 279
Duchenne Muscular Dystrophy - Dormant Projects 279
Duchenne Muscular Dystrophy - Discontinued Products 282
Duchenne Muscular Dystrophy - Product Development Milestones 283
Featured News and Press Releases 283
Appendix 289
Methodology 289
Coverage 289
Secondary Research 289
Primary Research 289
Expert Panel Validation 289
Contact Us 289
Disclaimer 290

List of Tables
Number of Products under Development for Duchenne Muscular Dystrophy, H1 2016 18
Number of Products under Development for Duchenne Muscular Dystrophy - Comparative Analysis, H1 2016 19
Number of Products under Development by Companies, H1 2016 21
Number of Products under Development by Companies, H1 2016 (Contd..1) 22
Number of Products under Development by Companies, H1 2016 (Contd..2) 23
Number of Products under Development by Companies, H1 2016 (Contd..3) 24
Number of Products under Investigation by Universities/Institutes, H1 2016 25
Comparative Analysis by Late Stage Development, H1 2016 26
Comparative Analysis by Clinical Stage Development, H1 2016 27
Comparative Analysis by Early Stage Development, H1 2016 28
Comparative Analysis by Unknown Stage Development, H1 2016 29
Products under Development by Companies, H1 2016 30
Products under Development by Companies, H1 2016 (Contd..1) 31
Products under Development by Companies, H1 2016 (Contd..2) 32
Products under Development by Companies, H1 2016 (Contd..3) 33
Products under Development by Companies, H1 2016 (Contd..4) 34
Products under Development by Companies, H1 2016 (Contd..5) 35
Products under Investigation by Universities/Institutes, H1 2016 36
Duchenne Muscular Dystrophy - Pipeline by Acceleron Pharma, Inc., H1 2016 37
Duchenne Muscular Dystrophy - Pipeline by Anagenesis Biotechnology, H1 2016 38
Duchenne Muscular Dystrophy - Pipeline by Asklepios BioPharmaceutical, Inc., H1 2016 39
Duchenne Muscular Dystrophy - Pipeline by Bamboo Therapeutics, Inc., H1 2016 40
Duchenne Muscular Dystrophy - Pipeline by BioMarin Pharmaceutical Inc., H1 2016 41
Duchenne Muscular Dystrophy - Pipeline by Bristol-Myers Squibb Company, H1 2016 42
Duchenne Muscular Dystrophy - Pipeline by Capricor Therapeutics, Inc., H1 2016 43
Duchenne Muscular Dystrophy - Pipeline by Catabasis Pharmaceuticals, Inc., H1 2016 44
Duchenne Muscular Dystrophy - Pipeline by Daiichi Sankyo Company, Limited, H1 2016 45
Duchenne Muscular Dystrophy - Pipeline by Debiopharm International S.A., H1 2016 46
Duchenne Muscular Dystrophy - Pipeline by Editas Medicine, Inc., H1 2016 47
Duchenne Muscular Dystrophy - Pipeline by Eli Lilly and Company, H1 2016 48
Duchenne Muscular Dystrophy - Pipeline by EryDel SPA, H1 2016 49
Duchenne Muscular Dystrophy - Pipeline by FibroGen, Inc., H1 2016 50
Duchenne Muscular Dystrophy - Pipeline by Galapagos NV, H1 2016 51
Duchenne Muscular Dystrophy - Pipeline by Genethon, H1 2016 52
Duchenne Muscular Dystrophy - Pipeline by GTx, Inc., H1 2016 53
Duchenne Muscular Dystrophy - Pipeline by Idera Pharmaceuticals, Inc., H1 2016 54
Duchenne Muscular Dystrophy - Pipeline by Italfarmaco S.p.A., H1 2016 55
Duchenne Muscular Dystrophy - Pipeline by La Jolla Pharmaceutical Company, H1 2016 56
Duchenne Muscular Dystrophy - Pipeline by Marathon Pharmaceuticals, LLC, H1 2016 57
Duchenne Muscular Dystrophy - Pipeline by Marina Biotech, Inc., H1 2016 58
Duchenne Muscular Dystrophy - Pipeline by Merck KGaA, H1 2016 59
Duchenne Muscular Dystrophy - Pipeline by Milo Biotechnology LLC, H1 2016 60
Duchenne Muscular Dystrophy - Pipeline by MyoTherix Inc., H1 2016 61
Duchenne Muscular Dystrophy - Pipeline by NicOx S.A., H1 2016 62
Duchenne Muscular Dystrophy - Pipeline by Nippon Shinyaku Co., Ltd., H1 2016 63
Duchenne Muscular Dystrophy - Pipeline by nLife Therapeutics, S.L., H1 2016 64
Duchenne Muscular Dystrophy - Pipeline by Nobelpharma Co., Ltd., H1 2016 65
Duchenne Muscular Dystrophy - Pipeline by Pfizer Inc., H1 2016 66
Duchenne Muscular Dystrophy - Pipeline by Prothelia, Inc., H1 2016 67
Duchenne Muscular Dystrophy - Pipeline by PTC Therapeutics, Inc., H1 2016 68
Duchenne Muscular Dystrophy - Pipeline by RASRx, LLC, H1 2016 69
Duchenne Muscular Dystrophy - Pipeline by ReveraGen BioPharma, Inc., H1 2016 70
Duchenne Muscular Dystrophy - Pipeline by Sanofi, H1 2016 71
Duchenne Muscular Dystrophy - Pipeline by Santhera Pharmaceuticals Holding AG, H1 2016 72
Duchenne Muscular Dystrophy - Pipeline by Sarepta Therapeutics, Inc., H1 2016 73
Duchenne Muscular Dystrophy - Pipeline by Summit Therapeutics Plc, H1 2016 74
Duchenne Muscular Dystrophy - Pipeline by Taiho Pharmaceutical Co., Ltd., H1 2016 75
Duchenne Muscular Dystrophy - Pipeline by WAVE Life Sciences Ltd., H1 2016 76
Duchenne Muscular Dystrophy - Pipeline by Zambon Company S.p.A., H1 2016 77
Assessment by Monotherapy Products, H1 2016 78
Number of Products by Stage and Target, H1 2016 80
Number of Products by Stage and Mechanism of Action, H1 2016 83
Number of Products by Stage and Route of Administration, H1 2016 86
Number of Products by Stage and Molecule Type, H1 2016 88
Duchenne Muscular Dystrophy Therapeutics - Recent Pipeline Updates, H1 2016 221
Duchenne Muscular Dystrophy - Dormant Projects, H1 2016 286
Duchenne Muscular Dystrophy - Dormant Projects (Contd..1), H1 2016 287
Duchenne Muscular Dystrophy - Dormant Projects (Contd..2), H1 2016 288
Duchenne Muscular Dystrophy - Discontinued Products, H1 2016 289

List of Figures
Number of Products under Development for Duchenne Muscular Dystrophy, H1 2016 18
Number of Products under Development for Duchenne Muscular Dystrophy - Comparative Analysis, H1 2016 19
Number of Products under Development by Companies, H1 2016 20
Number of Products under Investigation by Universities/Institutes, H1 2016 25
Comparative Analysis by Late Stage Development, H1 2016 26
Comparative Analysis by Clinical Stage Development, H1 2016 27
Comparative Analysis by Early Stage Products, H1 2016 28
Assessment by Monotherapy Products, H1 2016 78
Number of Products by Top 10 Targets, H1 2016 79
Number of Products by Stage and Top 10 Targets, H1 2016 79
Number of Products by Top 10 Mechanism of Actions, H1 2016 82
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2016 82
Number of Products by Routes of Administration, H1 2016 85
Number of Products by Stage and Routes of Administration, H1 2016 85
Number of Products by Top 10 Molecule Types, H1 2016 87
Number of Products by Stage and Molecule Types, H1 2016 87

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