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

April 2022 | 366 pages | ID: D45CCD58851CEN
Global Markets Direct

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

SUMMARY

Global Markets Direct's 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

Global Markets Direct's 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by Companies/Universities/Institutes, the molecules developed by Companies in Pre-Registration, Filing rejected/Withdrawn, Phase III, Phase II, Phase I, IND/CTA Filed, Preclinical, Discovery and Unknown stages are 1, 1, 7, 12, 15, 2, 71, 34 and 1 respectively.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 Global Markets Direct’s 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.
Introduction
Global Markets Direct Report Coverage
Duchenne 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 Development
Duchenne Muscular Dystrophy - Drug Profiles
Duchenne Muscular Dystrophy - Dormant Projects
Duchenne Muscular Dystrophy - Discontinued Products
Duchenne 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
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

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 Target, 2022 (Contd..1)
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Mechanism of Action, 2022 (Contd..1)
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
Duchenne Muscular Dystrophy - Discontinued Products, 2022 (Contd..1)

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


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