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

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Dystrophin Pipeline Drugs Market Report Overview

Dystrophin is a cytoplasmic protein, which anchors the extracellular matrix to the cytoskeleton via F-actin. It acts as ligand for dystroglycan. It also 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.

The Dystrophin – Drugs in Development research report provides a comprehensive overview on the therapeutics under development for Dystrophin, 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 Dystrophin and features dormant and discontinued projects.

Key Therapy Areas Genetic Disorders, and Musculoskeletal Disorders
Key Mechanisms of action Dystrophin Activator
Key Routes of Administration Intravenous, Subcutaneous, Intramuscular, and Parenteral
Key molecule types Antisense Oligonucleotide, Gene Therapy, Cell Therapy, Antisense Gene Therapy, Monoclonal Antibody Conjugated, Gene-Modified Cell Therapy, Oligonucleotide, and Protein
Major companies Sarepta Therapeutics Inc, Astellas Gene Therapies, Avidity Biosciences Inc, Daiichi Sankyo Co Ltd, Dystrogen Therapeutics SA, Editas Medicine Inc, Nippon Shinyaku Co Ltd, NS Pharma Inc, OliPass Corporation, Pepgen Ltd, Pfizer Inc, and RegenxBio Inc

Key Therapy Areas in the Dystrophin Pipeline Drugs Market

In the Dystrophin pipeline drugs market the key therapy areas are Genetic Disorders and Musculoskeletal Disorders. Genetic Disorders led the market in 2022.

Dystrophin Pipeline Drugs Market, by Therapy Areas, 2022 (%)

Dystrophin Pipeline Drugs Market, by Therapy Areas

For more therapy area insights, download a free report sample

Key MoA in the Dystrophin Pipeline Drugs Market

In the Dystrophin pipeline drugs market the key MoA is Dystrophin Activator.

Dystrophin Pipeline Drugs Market Segmentation by RoA

The key routes of administration in the Dystrophin pipeline drugs market are intravenous, subcutaneous, intramuscular, and parenteral. Intravenous has the maximum number of pipeline products in 2022.

Dystrophin Pipeline Drugs Market Analysis, by RoA, 2022 (%)

Dystrophin Pipeline Drugs Market Analysis, by RoA

To get more insights on key RoA, download a free sample report

Key Molecule Types in the Dystrophin Pipeline Drugs Market

The key molecule types in the Dystrophin pipeline drugs market are Antisense Oligonucleotide, Gene Therapy, Cell Therapy, Antisense Gene Therapy, Monoclonal Antibody Conjugated, Gene-Modified Cell Therapy, Oligonucleotide, and Protein.

Dystrophin Pipeline Drugs Market, by Molecule Type, 2022 (%)

Dystrophin Pipeline Drugs Market, by Molecule Type

To get more insights on key molecule types, download a free sample report

Major Companies in the Dystrophin Pipeline Drugs Market

The major companies in the Dystrophin pipeline drugs market are Sarepta Therapeutics Inc, Astellas Gene Therapies, Avidity Biosciences Inc, Daiichi Sankyo Co Ltd, Dystrogen Therapeutics SA, Editas Medicine Inc, Nippon Shinyaku Co Ltd, NS Pharma Inc, OliPass Corporation, Pepgen Ltd, Pfizer Inc, and RegenxBio Inc.

Sarepta Therapeutics Inc: Sarepta Therapeutics Inc (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. The company’s platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. Its commercial products include Exondys 51, Vyondys 53 and Amondys 45 indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene.

Dystrophin Pipeline Drugs Market, by Major Companies, 2022 (%)

Dystrophin Pipeline Drugs Market, by Major Companies

For more company insights, download a free sample report

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Dystrophin
  • The pipeline guide reviews pipeline therapeutics for Dystrophin by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Dystrophin therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Dystrophin therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA), and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews the latest news related to pipeline therapeutics for Dystrophin

Reasons to Buy

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with a potentially strong product portfolio and create effective countercstrategies to gain a competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Dystrophin
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and their most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Dystrophin 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.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from the pipeline.

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

Table of Contents

Table of Contents

List of Tables

List of Figures

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

Dystrophin (DMD) – Drug Profiles

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

Table

List of Tables

Number of Products under Development by Stage of Development, 2022

Number of Products under Development by Therapy Areas, 2022

Number of Products under Development by Indication, 2022

Number of Products under Development by Companies, 2022

Products under Development by Companies, 2022

Products under Development by Companies, 2022 (Contd..1)

Products under Development by Companies, 2022 (Contd..2)

Products under Development by Companies, 2022 (Contd..3)

Number of Products under Investigation by Universities/Institutes, 2022

Products under Investigation by Universities/Institutes, 2022

Number of Products by Stage and Mechanism of Actions, 2022

Number of Products by Stage and Route of Administration, 2022

Number of Products by Stage and Molecule Type, 2022

Pipeline by Alpha Anomeric, 2022

Pipeline by Astellas Gene Therapies, 2022

Pipeline by Avidity Biosciences Inc, 2022

Pipeline by Code Biotherapeutics Inc, 2022

Pipeline by Daiichi Sankyo Co Ltd, 2022

Pipeline by Dystrogen Therapeutics SA, 2022

Pipeline by Editas Medicine Inc, 2022

Pipeline by Eli Lilly and Co, 2022

Pipeline by Evox Therapeutics Ltd, 2022

Pipeline by FibroGenesis LLC, 2022

Pipeline by MyoGene Bio LLC, 2022

Pipeline by Myosana Therapeutics Inc, 2022

Pipeline by Nippon Shinyaku Co Ltd, 2022

Pipeline by NS Pharma Inc, 2022

Pipeline by OliPass Corporation, 2022

Pipeline by Pepgen Ltd, 2022

Pipeline by Pfizer Inc, 2022

Pipeline by RegenxBio Inc, 2022

Pipeline by Sarepta Therapeutics Inc, 2022

Pipeline by Solid Biosciences Inc, 2022

Pipeline by Sutura Therapeutics Ltd, 2022

Pipeline by Suzhou GenAssist Therapeutics Co Ltd, 2022

Pipeline by Tolerion Inc, 2022

Pipeline by Ultragenyx Pharmaceutical Inc, 2022

Pipeline by Vertex Pharmaceuticals Inc, 2022

Pipeline by Wave Life Sciences Ltd, 2022

Dormant Projects, 2022

Discontinued Products, 2022

Figures

List of Figures

Number of Products under Development by Stage of Development, 2022

Number of Products under Development by Therapy Areas, 2022

Number of Products under Development by Top 10 Indications, 2022

Number of Products by Stage and Mechanism of Actions, 2022

Number of Products by Routes of Administration, 2022

Number of Products by Stage and Routes of Administration, 2022

Number of Products Molecule Types, 2022

Number of Products by Stage and Molecule Types, 2022

Frequently asked questions

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