Dystrophin (DMD) - Pipeline Review, H2 2019

Dystrophin (DMD) - Pipeline Review, H2 2019

Summary

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.

Dystrophin (DMD) pipeline Target constitutes close to 48 molecules. Out of which approximately 44 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Pre-Registration, Filing rejected/Withdrawn, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 2, 4, 1, 23 and 12 respectively. Similarly, the universities portfolio in Preclinical stages comprises 4 molecules, respectively. Report covers products from therapy areas Genetic Disorders and Musculoskeletal Disorders which include indications Duchenne Muscular Dystrophy and Muscular Dystrophy.

The latest report Dystrophin (DMD) - Pipeline Review, H2 2019, 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. It also reviews key players involved in Dystrophin (DMD) targeted therapeutics development with respective active and dormant or discontinued projects.

The report is built using 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.

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


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
Alpha Anomeric
Audentes Therapeutics Inc
Daiichi Sankyo Co Ltd
Dystrogen Therapeutics SA
Editas Medicine Inc
Evox Therapeutics Ltd
MyoGene Bio LLC
Nippon Shinyaku Co Ltd
NS Pharma Inc
OliPass Corporation
Pepgen Ltd
Pfizer Inc
Sarepta Therapeutics Inc
Spinalcyte Llc
Sutura Therapeutics Ltd
Tolerion Inc
Vertex Pharmaceuticals 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
Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Antisense Oligonucleotides to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AT-702 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AT-751 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AT-753 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
casimersen - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CYMD-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
DMD-1742 - 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 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 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 Duchenne Muscular Dystrophy 1 - 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-089 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides 3 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides 5 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides 6 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides 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
Protein to Target Dystrophin for Duchenne Muscular Dystrophy - 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
SRP-9001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy 2 to Activate Dystrophin for Duchenne Muscular Dystrophy - 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
Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SUT-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
viltolarsen - 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
Dec 13, 2019: Sarepta’s DMD therapy Vyondys 53 secures FDA accelerated approval
Nov 05, 2019: Dystrogen Therapeutics announces that treatment with Dystrophin expressing Chimeric (DEC) Cells improves cardiac function in Preclinical Duchenne's Study
Aug 21, 2019: Sarepta fails to receive FDA approval for DMD drug golodirsen
Aug 08, 2019: Sarepta Therapeutics comments on erroneous submission to US FDA Adverse Event Reporting System (FAERS)
Jul 09, 2019: New analysis shows drug slows down respiratory decline
Jul 01, 2019: AskBio's Perspective on Pfizer's 1b clinical trial results on Duchenne MD Gene
Jun 28, 2019: Pfizer presents initial clinical data on phase 1b gene therapy study for Duchenne Muscular Dystrophy (DMD)
Apr 18, 2019: Parent Project Muscular Dystrophy awards $100,000 grant to Nationwide Children's Hospital to further explore GALGT2 gene therapy in duchenne muscular dystrophy
Mar 28, 2019: Sarepta Therapeutics announces positive expression results from the Casimersen (SRP-4045) arm of the ESSENCE study
Mar 25, 2019: Sarepta Therapeutics to provide update on Duchenne Muscular Dystrophy gene therapy program
Feb 15, 2019: FDA accepts Sarepta’s NDA for precision medicine Golodirsen
Feb 06, 2019: NS Pharma begins rolling NDA submission to FDA for Viltolarsen (NS-065/NCNP-01)
Dec 20, 2018: Sarepta Therapeutics completes Submission of New Drug Application Seeking approval of Golodirsen (SRP-4053) in patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53
Nov 13, 2018: Evox Therapeutics wins funding from Duchenne UK to explore exosome-mediated delivery of dystrophin
Oct 03, 2018: NS-065/NCNP-01 (Viltolarsen) of Nippon Shinyaku’ in-house product presentation on results of Phase I/II study in Japan
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Number of Products under Development by Stage of Development, H2 2019
Number of Products under Development by Therapy Areas, H2 2019
Number of Products under Development by Indication, H2 2019
Number of Products under Development by Companies, H2 2019
Products under Development by Companies, H2 2019
Products under Development by Companies, H2 2019 (Contd..1), H2 2019
Products under Development by Companies, H2 2019 (Contd..2), H2 2019
Number of Products under Investigation by Universities/Institutes, H2 2019
Products under Investigation by Universities/Institutes, H2 2019
Number of Products by Stage and Mechanism of Actions, H2 2019
Number of Products by Stage and Route of Administration, H2 2019
Number of Products by Stage and Molecule Type, H2 2019
Pipeline by Alpha Anomeric, H2 2019
Pipeline by Audentes Therapeutics Inc, H2 2019
Pipeline by Daiichi Sankyo Co Ltd, H2 2019
Pipeline by Dystrogen Therapeutics SA, H2 2019
Pipeline by Editas Medicine Inc, H2 2019
Pipeline by Evox Therapeutics Ltd, H2 2019
Pipeline by MyoGene Bio LLC, H2 2019
Pipeline by Nippon Shinyaku Co Ltd, H2 2019
Pipeline by NS Pharma Inc, H2 2019
Pipeline by OliPass Corporation, H2 2019
Pipeline by Pepgen Ltd, H2 2019
Pipeline by Pfizer Inc, H2 2019
Pipeline by Sarepta Therapeutics Inc, H2 2019
Pipeline by Spinalcyte Llc, H2 2019
Pipeline by Sutura Therapeutics Ltd, H2 2019
Pipeline by Tolerion Inc, H2 2019
Pipeline by Vertex Pharmaceuticals Inc, H2 2019
Pipeline by Wave Life Sciences Ltd, H2 2019
Dormant Projects, H2 2019
Discontinued Products, H2 2019
List of Figures
Number of Products under Development by Stage of Development, H2 2019
Number of Products under Development by Therapy Areas, H2 2019
Number of Products under Development by Top 10 Indications, H2 2019
Number of Products by Stage and Mechanism of Actions, H2 2019
Number of Products by Routes of Administration, H2 2019
Number of Products by Stage and Routes of Administration, H2 2019
Number of Products by Molecule Types, H2 2019
Number of Products by Stage and Molecule Types, H2 2019

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