Report cover image

Dystrophin (DMD) Drugs in Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players, 2022 Update

Publisher Global Markets Direct
Published Apr 29, 2022
Length 122 Pages
SKU # GMD17181980

Description

Dystrophin (DMD) Drugs in Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players, 2022 Update

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 acts 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 67 molecules. Out of which approximately 60 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 The molecules developed by companies in Pre-Registration, Filing rejected/Withdrawn, Phase III, Phase II, Phase I, IND/CTA Filed, Preclinical and Discovery stages are 1, 1, 3, 6, 2, 1, 31 and 15 respectively. Similarly, the universities portfolio in Phase I and Preclinical stages comprises 1 and 6 molecules, respectively. Report covers products from therapy areas Genetic Disorders and Musculoskeletal Disorders which include indications Duchenne Muscular Dystrophy, Alport Syndrome and Muscular Dystrophy.

The latest report Dystrophin - Drugs In Development, 2022, 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.

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

Table of Contents

122 Pages
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
Astellas Gene Therapies
Avidity Biosciences Inc
Code Biotherapeutics Inc
Daiichi Sankyo Co Ltd
Dystrogen Therapeutics SA
Editas Medicine Inc
Eli Lilly and Co
Evox Therapeutics Ltd
FibroGenesis LLC
MyoGene Bio LLC
Myosana Therapeutics Inc
Nippon Shinyaku Co Ltd
NS Pharma Inc
OliPass Corporation
Pepgen Ltd
Pfizer Inc
RegenxBio Inc
Sarepta Therapeutics Inc
Solid Biosciences Inc
Sutura Therapeutics Ltd
Suzhou GenAssist Therapeutics Co Ltd
Tolerion Inc
Ultragenyx Pharmaceutical Inc
Vertex Pharmaceuticals Inc
Wave Life Sciences Ltd
Dystrophin (DMD) – Drug Profiles
Antisense Oligonucleotide 1 to Activate Dystrophin for Duchenne Muscular Dystrophy – Drug Profile
Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy – Drug Profile
Antisense Oligonucleotides to Activate Dystrophin for Duchenne Muscular Dystrophy – Drug Profile
AOC-1044 – Drug Profile
AT-702 – Drug Profile
AT-751 – Drug Profile
AT-753 – Drug Profile
CRISPR-Based Gene Therapy for Duchenne Muscular Dystrophy – Drug Profile
CYMD-201 – Drug Profile
delandistrogene moxeparvovec – Drug Profile
DS-5144 – Drug Profile
DS-5150 – Drug Profile
DS-5151 – Drug Profile
DS-5153 – Drug Profile
DTDEC-01 – Drug Profile
eteplirsen – Drug Profile
fordadistrogene movaparvovec – Drug Profile
GEN-6045 – Drug Profile
GEN-6050 – Drug Profile
GEN-6051 – Drug Profile
Gene Therapies to Activate Dystrophin for Duchenne Muscular Dystrophy – Drug Profile
Gene Therapy 1 to Activate Dystrophin for Duchenne Muscular Dystrophy – Drug Profile
Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy – Drug Profile
Gene Therapy to Activate Dystrophin for Muscular Dystrophy – Drug Profile
GNT-0004 – Drug Profile
golodirsen – Drug Profile
Next AOC DMD Programs-Exon 45 Dystrophin – Drug Profile
Next AOC DMD Programs-Exon 51 Dystrophin – Drug Profile
NS-045 – Drug Profile
NS-051 – Drug Profile
NS-055 – Drug Profile
NS-089 – Drug Profile
Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy – Drug Profile
PBGENE-DMD – Drug Profile
PGN-EDO45 – Drug Profile
PGNEDO-44 – Drug Profile
PGNEDO-51 – Drug Profile
Protein to Target Dystrophin for Duchenne Muscular Dystrophy – Drug Profile
renadirsen – Drug Profile
RGX-202 – Drug Profile
SGT-001 – Drug Profile
SGT-003 – Drug Profile
SRP-4044 – Drug Profile
SRP-4050 – Drug Profile
SRP-4052 – Drug Profile
SRP-4055 – Drug Profile
SRP-5044 – Drug Profile
SRP-5045 – Drug Profile
SRP-5050 – Drug Profile
SRP-5051 – Drug Profile
SRP-5052 – Drug Profile
SRP-5053 – Drug Profile
Stem Cell Therapy 1 to Activate Dystrophin for Duchenne Muscular Dystrophy – Drug Profile
Stem Cell Therapy 2 to Activate Dystrophin for Duchenne Muscular Dystrophy – Drug Profile
Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy – Drug Profile
SUT-001 – Drug Profile
UX-810 – Drug Profile
viltolarsen – Drug Profile
WVEN-531 – Drug Profile
Dystrophin (DMD) – Dormant Products
Dystrophin (DMD) – Discontinued Products
Dystrophin (DMD) – Product Development Milestones
Featured News & Press Releases
Apr 07, 2022: Dystrogen Therapeutics investigational chimeric cell therapy DT-DEC01 for the treatment of Duchene Muscular Dystrophy shows safety and functional improvements
Apr 06, 2022: PepGen announces first participant dosed in a phase 1 clinical trial of PGN-EDO51 for the treatment of Duchenne Muscular Dystrophy
Mar 18, 2022: Exon 44-targeted DMD drug hits goals in PI/II: NCNP/Nippon Shinyaku
Mar 17, 2022: The result of an investigator-initiated clinical trial (First In Human trial) of NS-089/NCNP-02 for the treatment of Duchenne muscular dystrophy
Mar 15, 2022: PepGen announces approval by Health Canada of CTA to begin first in human trials of PGN-EDO51 to treat Duchenne Muscular Dystrophy
Mar 11, 2022: The result of an investigator-initiated clinical trial (First In Human trial) of NS-089/NCNP-02 for the treatment of Duchenne muscular dystrophy
Mar 08, 2022: Roche to present new SRP-9001 data at MDA 2022 and highlight expanding neuromuscular disease portfolio
Jan 10, 2022: Sarepta Therapeutics’ Gene Therapy SRP-9001 shows statistically significant functional improvements compared to pre-specified matched external control in Part 2 of study SRP-9001-102 for the treatment of Duchenne Muscular Dystrophy
Jan 07, 2022: FDA grants IND clearance for REGENXBIO’s gene therapy trial for Duchenne
Jan 06, 2022: Sarepta Therapeutics to present at the 40th Annual J.P. Morgan Healthcare Conference
Nov 29, 2021: Nippon Shinyaku implements VILTEPSO managed access program
Nov 25, 2021: Addition of Viltepso to the list of Injection Drugs that physicians providing health insurance treatment can administer
Nov 22, 2021: REGENXBIO announces Orphan Drug Designation granted to RGX-202, a novel gene therapy candidate for the treatment of Duchenne Muscular Dystrophy
Oct 11, 2021: Sarepta Therapeutics’ SRP-9001 shows sustained functional improvements in multiple studies of patients with Duchenne
Oct 04, 2021: Solid Biosciences and Forge Biologics announce viral vector contract development and cGMP manufacturing partnership
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Table 1: Number of Products under Development by Stage of Development, 2022
Table 2: Number of Products under Development by Therapy Areas, 2022
Table 3: Number of Products under Development by Indication, 2022
Table 4: Number of Products under Development by Companies, 2022
Table 5: Products under Development by Companies, 2022
Table 6: Products under Development by Companies, 2022 (Contd..1)
Table 7: Products under Development by Companies, 2022 (Contd..2)
Table 8: Products under Development by Companies, 2022 (Contd..3)
Table 9: Number of Products under Investigation by Universities/Institutes, 2022
Table 10: Products under Investigation by Universities/Institutes, 2022
Table 11: Number of Products by Stage and Mechanism of Actions, 2022
Table 12: Number of Products by Stage and Route of Administration, 2022
Table 13: Number of Products by Stage and Molecule Type, 2022
Table 14: Pipeline by Alpha Anomeric, 2022
Table 15: Pipeline by Astellas Gene Therapies, 2022
Table 16: Pipeline by Avidity Biosciences Inc, 2022
Table 17: Pipeline by Code Biotherapeutics Inc, 2022
Table 18: Pipeline by Daiichi Sankyo Co Ltd, 2022
Table 19: Pipeline by Dystrogen Therapeutics SA, 2022
Table 20: Pipeline by Editas Medicine Inc, 2022
Table 21: Pipeline by Eli Lilly and Co, 2022
Table 22: Pipeline by Evox Therapeutics Ltd, 2022
Table 23: Pipeline by FibroGenesis LLC, 2022
Table 24: Pipeline by MyoGene Bio LLC, 2022
Table 25: Pipeline by Myosana Therapeutics Inc, 2022
Table 26: Pipeline by Nippon Shinyaku Co Ltd, 2022
Table 27: Pipeline by NS Pharma Inc, 2022
Table 28: Pipeline by OliPass Corporation, 2022
Table 29: Pipeline by Pepgen Ltd, 2022
Table 30: Pipeline by Pfizer Inc, 2022
Table 31: Pipeline by RegenxBio Inc, 2022
Table 32: Pipeline by Sarepta Therapeutics Inc, 2022
Table 33: Pipeline by Solid Biosciences Inc, 2022
Table 34: Pipeline by Sutura Therapeutics Ltd, 2022
Table 35: Pipeline by Suzhou GenAssist Therapeutics Co Ltd, 2022
Table 36: Pipeline by Tolerion Inc, 2022
Table 37: Pipeline by Ultragenyx Pharmaceutical Inc, 2022
Table 38: Pipeline by Vertex Pharmaceuticals Inc, 2022
Table 39: Pipeline by Wave Life Sciences Ltd, 2022
Table 40: Dormant Projects, 2022
Table 41: Discontinued Products, 2022
List of Figures
Figure 1: Number of Products under Development by Stage of Development, 2022
Figure 2: Number of Products under Development by Therapy Areas, 2022
Figure 3: Number of Products under Development by Top 10 Indications, 2022
Figure 4: Number of Products by Stage and Mechanism of Actions, 2022
Figure 5: Number of Products by Routes of Administration, 2022
Figure 6: Number of Products by Stage and Routes of Administration, 2022
Figure 7: Number of Products Molecule Types, 2022
Figure 8: Number of Products by Stage and Molecule Types, 2022

Pricing

Currency Rates
How Do Licenses Work?

How Do Licenses Work?

The primary function of a report license is to define how many people within a company are authorized to use the purchased report. This is separate to how a report might be delivered. Unless specifically identified otherwise, the purchase option is Single User. Below is a helpful description of that license, along with a sampling of others most commonly offered.

  • This license allows only one user to have access to and to use/read the report. It is not one user at a time or one user group; it is an individual who will be reading and utilizing the report.

  • This license allows for the entire purchasing company to read and use the report. This is the license level that would allow for a report to be placed on an internal company shared drive for access by all employees of that direct company. This license does not extend to parent or sister company use.

  • This license is designed to allow an entire department within a company full access to a report. The departmental license is a great option for companies that have more than one location and the department is spread out all over the country/world

  • This license is for companies that have one location where they want the report to be accessible by all employees within that physical location.

  • This is the most flexible of the licensing options. Some publishers will allow you to get a license for a preset number of people (typically 5). These licenses are ideal for small work groups.

License options are at the discretion of each publisher. As such, not all licenses indicated above are available for every product. If you are uncertain of the best license for your needs or would like a license that is not proactively available, please contact us and we will be happy to assist.

Head shot

Questions or Comments?

Our team has the ability to search within reports to verify it suits your needs. We can also help maximize your budget by finding sections of reports you can purchase.
Chat Now
Contact Us