Dystrophin (DMD) - Pipeline Review, H1 2018

June 2018 | 80 pages | ID: DE735048BBAEN
Global Markets Direct

Abstracts

Dystrophin (DMD) - Pipeline Review, H1 2018

SUMMARY

Dystrophin (DMD) pipeline Target constitutes close to 29 molecules. Out of which approximately 25 molecules are developed by companies and remaining by the universities/institutes. The latest report Dystrophin (DMD) - Pipeline Review, H1 2018, outlays 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.

Dystrophin (DMD) - Dystrophin is a cytoplasmic protein. It anchors the extracellular matrix to the cytoskeleton via F-actin. It acts as ligand for dystroglycan. It act as component of the dystrophin-associated glycoprotein complex which accumulates at the neuromuscular junction and at a variety of synapses in the peripheral and central nervous systems and has a structural function in stabilizing the sarcolemma.

The molecules developed by companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 2, 3, 2, 10 and 7 respectively. Similarly, the universities portfolio in Phase II and Preclinical stages comprises 1 and 3 molecules, respectively. Report covers products from therapy areas Genetic Disorders and Musculoskeletal Disorders which include indications Duchenne Muscular Dystrophy and Muscular Dystrophy.

Furthermore, this report also reviews key players involved in Dystrophin (DMD) targeted therapeutics development with respective active and dormant or discontinued projects. Driven by data and information sourced from 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.

Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

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

Contents

Introduction
Global Markets Direct Report Coverage
Dystrophin (DMD) - Overview
Dystrophin (DMD) - Therapeutics Development
Products under Development by Stage of Development
Products under Development by Therapy Area
Products under Development by Indication
Products under Development by Companies
Products under Development by Universities/Institutes
Dystrophin (DMD) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Dystrophin (DMD) - Companies Involved in Therapeutics Development
Daiichi Sankyo Co Ltd
Editas Medicine Inc
Genethon SA
NS Pharma Inc
Pfizer Inc
Sarepta Therapeutics Inc
WAVE Life Sciences Ltd
Dystrophin (DMD) - Drug Profiles
Antisense Oligonucleotide 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
casimersen - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
DS-5141 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
eteplirsen - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate DMD for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate DMD for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Dystrophin for Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
golodirsen - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NS-065 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides 2 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PF-06939926 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SGT-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-4008 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-4044 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-4050 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-4052 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-4055 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-5044 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-5045 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-5050 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-5051 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-5052 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-5053 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
WVE-210201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Dystrophin (DMD) - Dormant Products
Dystrophin (DMD) - Discontinued Products
Dystrophin (DMD) - Product Development Milestones
Featured News & Press Releases
May 18, 2018: Solid Biosciences Announces New Preclinical Data at the American Society of Gene and Cell Therapy Annual Meeting
May 15, 2018: Solid Biosciences Announces Upcoming Preclinical Data Presentations
May 10, 2018: Presentations on NS-065/ NCNP-01 at 2018 New Directions in Biology and Disease of Skeletal Muscle Conference
Apr 25, 2018: Daiichi Sankyo Announces Phase 1/2 Clinical Trial Results for DS-5141 (Therapeutic Agent for Duchenne Muscular Dystrophy) in Japan
Apr 25, 2018: Asklepios BioPharmaceutical Announces Former Portfolio Company’s Preclinical Asset for Duchenne Muscular Dystrophy Has Advanced into the Clinic
Apr 19, 2018: Results of Exploratory Investigator-Initiated Clinical Trial of NS-065/NCNP-01 for the Treatment of Duchenne Muscular Dystrophy Published in Science Translational Medicine
Apr 18, 2018: Solid Biosciences Provides Update On SGT-001 Clinical Development Program For Duchenne Muscular Dystrophy
Apr 12, 2018: Pfizer Doses First Patient Using Investigational Mini-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy
Apr 03, 2018: RM LAW Announces Class Action Lawsuit Against Solid Biosciences
Mar 14, 2018: Solid Biosciences Announces Clinical Hold On SGT-001 Phase I/II Clinical Trial For Duchenne Muscular Dystrophy
Mar 12, 2018: Sarepta Therapeutics Announces Plan to Submit a New Drug Application (NDA) for Accelerated Approval of Golodirsen (SRP-4053) in Patients with Duchenne Muscular Dystrophy (DMD) Amenable to Skipping Exon
Jan 17, 2018: First Duchenne Muscular Dystrophy Patient Dosed in Systemic Microdystrophin Gene Therapy
Jan 08, 2018: Sarepta Therapeutics Pre-Announces Fourth Quarter 2017 Revenue and Provides Full-Year 2018 Revenue Guidance for EXONDYS 51 (eteplirsen), Representing Approximately 100 Percent Year-over-Year Growth
Dec 27, 2017: Sarepta Therapeutics Announces Publication of Long-Term Pulmonary Function of Eteplirsen-Treated Patients Compared to Natural History of Duchenne Muscular Dystrophy in The Journal of Neuromuscular Diseases
Nov 30, 2017: Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate SGT-001 in Patients with Duchenne Muscular Dystrophy
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List Of Tables

LIST OF TABLES

Number of Products under Development by Stage of Development, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indication, H1 2018
Number of Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018 (Contd..1), H1 2018
Number of Products under Investigation by Universities/Institutes, H1 2018
Products under Investigation by Universities/Institutes, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Pipeline by Daiichi Sankyo Co Ltd, H1 2018
Pipeline by Editas Medicine Inc, H1 2018
Pipeline by Genethon SA, H1 2018
Pipeline by NS Pharma Inc, H1 2018
Pipeline by Pfizer Inc, H1 2018
Pipeline by Sarepta Therapeutics Inc, H1 2018
Pipeline by WAVE Life Sciences Ltd, H1 2018
Dormant Projects, H1 2018
Discontinued Products, H1 2018

List Of Figures

LIST OF FIGURES

Number of Products under Development by Stage of Development, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Top 10 Indications, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018

COMPANIES MENTIONED

Daiichi Sankyo Co Ltd
Editas Medicine Inc
Genethon SA
NS Pharma Inc
Pfizer Inc
Sarepta Therapeutics Inc
WAVE Life Sciences Ltd