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Duchenne Muscular Dystrophy Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

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    Report

  • 366 Pages
  • April 2022
  • Region: Global
  • Global Markets Direct
  • ID: 5589949
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The latest Pharmaceutical and Healthcare disease pipeline guide Duchenne Muscular Dystrophy - Drugs In Development, 2022, provides an overview of the Duchenne Muscular Dystrophy (Genetic Disorders) pipeline landscape.

Duchenne muscular dystrophy (DMD) is a genetic disorder that causes muscles to gradually weaken over time. Signs and symptoms of DMD include fatigue, learning difficulties, intellectual disability, muscle weakness and progressive difficulty walking. Risk factors include gender and family history. Treatment includes steroid medication, respiratory therapy and surgery.

Report Highlights


The Pharmaceutical and Healthcare latest pipeline guide Duchenne Muscular Dystrophy - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Duchenne Muscular Dystrophy (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Duchenne Muscular Dystrophy (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Duchenne Muscular Dystrophy and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Filing rejected/Withdrawn, Phase III, Phase II, Phase I, IND/CTA Filed, Preclinical, Discovery and Unknown stages are 1, 1, 7, 12, 15, 2, 71, 34 and 1 respectively. Similarly, the Universities portfolio in Phase III, Phase II, Phase I, IND/CTA Filed, Preclinical and Discovery stages comprises 2, 1, 2, 1, 13 and 3 molecules, respectively.

Duchenne Muscular Dystrophy (Genetic Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

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

Scope


  • The pipeline guide provides a snapshot of the global therapeutic landscape of Duchenne Muscular Dystrophy (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Duchenne Muscular Dystrophy (Genetic Disorders) 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 Duchenne Muscular Dystrophy (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Duchenne Muscular Dystrophy (Genetic Disorders) 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 latest news related to pipeline therapeutics for Duchenne Muscular Dystrophy (Genetic Disorders)

Reasons to Buy


  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Duchenne Muscular Dystrophy (Genetic Disorders).
  • 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 it’s most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Duchenne Muscular Dystrophy (Genetic Disorders) 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 pipeline.

Table of Contents

IntroductionReport CoverageDuchenne Muscular Dystrophy - Overview
Duchenne Muscular Dystrophy - Therapeutics Development
  • Pipeline Overview
  • Pipeline by Companies
  • Pipeline by Universities/Institutes
  • Products under Development by Companies
  • Products under Development by Universities/Institutes
Duchenne Muscular Dystrophy - Therapeutics Assessment
  • Assessment by Target
  • Assessment by Mechanism of Action
  • Assessment by Route of Administration
  • Assessment by Molecule Type
Duchenne Muscular Dystrophy - Companies Involved in Therapeutics DevelopmentDuchenne Muscular Dystrophy - Drug ProfilesDuchenne Muscular Dystrophy - Dormant ProjectsDuchenne Muscular Dystrophy - Discontinued ProductsDuchenne Muscular Dystrophy - Product Development Milestones
Featured News & Press Releases
  • Apr 28, 2022: Pfizer to open first U.S. sites in phase 3 trial of investigational gene therapy for ambulatory patients with Duchenne muscular dystrophy
  • 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 29, 2022: Santhera and ReveraGen start rolling NDA submission to the FDA for Vamorolone 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 16, 2022: Stealth BioTherapeutics showcases new Duchenne muscular dystrophy nonclinical data at the 2022 Muscular Dystrophy Association and Clinical Scientific Conference
  • 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 15, 2022: Santhera and ReveraGen to present efficacy and safety data with vamorolone at 2022 Muscular Dystrophy Association Conference
  • Mar 14, 2022: Significant modulation of two bone morphogenetic proteins supports potential of ATL1102 for improving bone density in DMD
  • Mar 11, 2022: The Lancet publishes positive results from Capricor Therapeutics’ phase 2 study evaluating CAP-1002 in late-stage 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 09, 2022: Antisense Therapeutics to present poster at world’s largest NMD conference
  • Mar 08, 2022: Edgewise Therapeutics to present on EDG-5506 for Becker and Duchenne Muscular Dystrophy (BMD, DMD) at the 2022 Annual MDA Clinical and Scientific Conference
  • Mar 08, 2022: Roche to present new SRP-9001 data at MDA 2022 and highlight expanding neuromuscular disease portfolio
AppendixMethodologyCoverageSecondary ResearchPrimary ResearchExpert Panel ValidationContact the Analyst
List of Tables
  • Number of Products under Development for Duchenne Muscular Dystrophy, 2022
  • Number of Products under Development by Companies, 2022
  • Number of Products under Development by Universities/Institutes, 2022
  • Products under Development by Companies, 2022
  • Products under Development by Universities/Institutes, 2022
  • Number of Products by Stage and Target, 2022
  • Number of Products by Stage and Mechanism of Action, 2022
  • Number of Products by Stage and Route of Administration, 2022
  • Number of Products by Stage and Molecule Type, 2022
  • Duchenne Muscular Dystrophy - Pipeline by AAVogen Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Alpha Anomeric, 2022
  • Duchenne Muscular Dystrophy - Pipeline by American CryoStem Corp, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Anagenesis Biotechnologies SAS, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Antisense Therapeutics Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by ARMGO Pharma Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Astria Therapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by AUM LifeTech Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Autotac Bio Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Avidity Biosciences Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Axolo Pharma Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Bayer AG, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Biogen Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by BioIncept LLC, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Bioleaders Corp, 2022
  • Duchenne Muscular Dystrophy - Pipeline by BioMarin Pharmaceutical Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Biophytis SA, 2022
  • Duchenne Muscular Dystrophy - Pipeline by CANbridge Life Sciences Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Capricor Therapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Chengdu Fanxi Biopharma Co Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Code Biotherapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Consortium.AI, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Constant Therapeutics LLC, 2022
  • Duchenne Muscular Dystrophy - Pipeline by CRISPR Therapeutics AG, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Cumberland Pharmaceuticals Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Daiichi Sankyo Co Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by DepYmed Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by DMD Therapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by DTx Pharma Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Dyne Therapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Dystrogen Therapeutics SA, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Edgewise Therapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Editas Medicine Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Eli Lilly and Co, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Eloxx Pharmaceuticals Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Encell Co Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Entrada Therapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Epirium Bio Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by EryDel SpA, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Evox Therapeutics Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by FibroGen Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by FibroGenesis LLC, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Fulcrum Therapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by InnoBioscience LLC, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Italfarmaco SpA, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Ixchel Pharma LLC, 2022
  • Duchenne Muscular Dystrophy - Pipeline by J2H Biotech, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Keros Therapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by KSbitugen Co Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by LambdaGen Therapeutics, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Ludi Therapeutics, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Milo Biotechnology LLC, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Mitobridge Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Mitochon Pharmaceuticals Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by MitoRx Therapeutics Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by MyoGene Bio LLC, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Myos Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Myosana Therapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by MyoTherix Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Nippon Shinyaku Co Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by NS Pharma Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by OliPass Corporation, 2022
  • Duchenne Muscular Dystrophy - Pipeline by OMEICOS Therapeutics GmbH, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Oncocross Co Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Oncotelic Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Pepgen Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by PeptiDream Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Pfizer Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Pharmaxis Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by PhaseBio Pharmaceuticals Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Pliant Therapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Progenitor Therapeutics Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Prothelia Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by PTC Therapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by PYC Therapeutics Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by RASRx LLC, 2022
  • Duchenne Muscular Dystrophy - Pipeline by RegenxBio Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by ReoStem LLC, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Ridgeline Therapeutics LLC, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Santhera Pharmaceuticals Holding AG, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Sarcomed AB, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Sarepta Therapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Satellos Bioscience Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Solid Biosciences Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Stealth BioTherapeutics Corp, 2022
  • Duchenne Muscular Dystrophy - Pipeline by SteroTherapeutics LLC, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Sutura Therapeutics Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Suzhou GenAssist Therapeutics Co Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Taiho Pharmaceutical Co Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Tivorsan Pharmaceuticals Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Tolerion Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Triplex Therapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by UGISense AG, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Ultragenyx Pharmaceutical Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Vandria SA, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Vertex Pharmaceuticals Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Vita Therapeutics Inc, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Wave Life Sciences Ltd, 2022
  • Duchenne Muscular Dystrophy - Pipeline by Zata Pharmaceuticals Inc, 2022
  • Duchenne Muscular Dystrophy - Dormant Projects, 2022
  • Duchenne Muscular Dystrophy - Discontinued Products, 2022

List of Figures
  • Number of Products under Development for Duchenne Muscular Dystrophy, 2022
  • Number of Products under Development by Companies, 2022
  • Number of Products under Development by Universities/Institutes, 2022
  • Number of Products by Top 10 Targets, 2022
  • Number of Products by Stage and Top 10 Targets, 2022
  • Number of Products by Top 10 Mechanism of Actions, 2022
  • Number of Products by Stage and Top 10 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 by Top 10 Molecule Types, 2022
  • Number of Products by Stage and Top 10 Molecule Types, 2022

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

  • AAVogen Inc
  • Alpha Anomeric
  • American CryoStem Corp
  • Anagenesis Biotechnologies SAS
  • Antisense Therapeutics Ltd
  • ARMGO Pharma Inc
  • Astria Therapeutics Inc
  • AUM LifeTech Inc
  • Autotac Bio Inc
  • Avidity Biosciences Inc
  • Axolo Pharma Inc
  • Bayer AG
  • Biogen Inc
  • BioIncept LLC
  • Bioleaders Corp
  • BioMarin Pharmaceutical Inc
  • Biophytis SA
  • CANbridge Life Sciences Ltd
  • Capricor Therapeutics Inc
  • Chengdu Fanxi Biopharma Co Ltd
  • Code Biotherapeutics Inc
  • Consortium.AI
  • Constant Therapeutics LLC
  • CRISPR Therapeutics AG
  • Cumberland Pharmaceuticals Inc
  • Daiichi Sankyo Co Ltd
  • DepYmed Inc
  • DMD Therapeutics Inc
  • DTx Pharma Inc
  • Dyne Therapeutics Inc
  • Dystrogen Therapeutics SA
  • Edgewise Therapeutics Inc
  • Editas Medicine Inc
  • Eli Lilly and Co
  • Eloxx Pharmaceuticals Inc
  • Encell Co Ltd
  • Entrada Therapeutics Inc
  • Epirium Bio Inc
  • EryDel SpA
  • Evox Therapeutics Ltd
  • FibroGen Inc
  • FibroGenesis LLC
  • Fulcrum Therapeutics Inc
  • InnoBioscience LLC
  • Italfarmaco SpA
  • Ixchel Pharma LLC
  • J2H Biotech
  • Keros Therapeutics Inc
  • KSbitugen Co Ltd
  • LambdaGen Therapeutics
  • Ludi Therapeutics
  • Milo Biotechnology LLC
  • Mitobridge Inc
  • Mitochon Pharmaceuticals Inc
  • MitoRx Therapeutics Ltd
  • MyoGene Bio LLC
  • Myos Inc
  • Myosana Therapeutics Inc
  • MyoTherix Inc
  • Nippon Shinyaku Co Ltd
  • NS Pharma Inc
  • OliPass Corporation
  • OMEICOS Therapeutics GmbH
  • Oncocross Co Ltd
  • Oncotelic Inc
  • Pepgen Ltd
  • PeptiDream Inc
  • Pfizer Inc
  • Pharmaxis Ltd
  • PhaseBio Pharmaceuticals Inc
  • Pliant Therapeutics Inc
  • Progenitor Therapeutics Ltd
  • Prothelia Inc
  • PTC Therapeutics Inc
  • PYC Therapeutics Ltd
  • RASRx LLC
  • RegenxBio Inc
  • ReoStem LLC
  • Ridgeline Therapeutics LLC
  • Santhera Pharmaceuticals Holding AG
  • Sarcomed AB
  • Sarepta Therapeutics Inc
  • Satellos Bioscience Inc
  • Solid Biosciences Inc
  • Stealth BioTherapeutics Corp
  • SteroTherapeutics LLC
  • Sutura Therapeutics Ltd
  • Suzhou GenAssist Therapeutics Co Ltd
  • Taiho Pharmaceutical Co Ltd
  • Tivorsan Pharmaceuticals Inc
  • Tolerion Inc
  • Triplex Therapeutics Inc
  • UGISense AG
  • Ultragenyx Pharmaceutical Inc
  • Vandria SA
  • Vertex Pharmaceuticals Inc
  • Vita Therapeutics Inc
  • Wave Life Sciences Ltd
  • Zata Pharmaceuticals Inc