Duchenne Muscular Dystrophy (DMD) Clinical Trial Analysis Featuring 60+ Companies | DelveInsight

Duchenne Muscular Dystrophy pipeline constitutes 60+ key companies continuously working towards developing 75+ Duchenne Muscular Dystrophy treatment therapies, analyzes DelveInsight

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New York, USA, Sept. 07, 2022 (GLOBE NEWSWIRE) -- Duchenne Muscular Dystrophy (DMD) Clinical Trial Analysis Featuring 60+ Companies | DelveInsight

Duchenne Muscular Dystrophy pipeline constitutes 60+ key companies continuously working towards developing 75+ Duchenne Muscular Dystrophy treatment therapies, analyzes DelveInsight

DelveInsight’s 'Duchenne Muscular Dystrophy Pipeline Insight 2022' report provides comprehensive global coverage of available, marketed, and pipeline duchenne muscular dystrophy therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the duchenne muscular dystrophy pipeline domain.

Key Takeaways from the Duchenne Muscular Dystrophy Pipeline Report

  • DelveInsight’s duchenne muscular dystrophy pipeline report depicts a robust space with 60+ active players working to develop 75+ pipeline therapies for duchenne muscular dystrophy treatment. 
  • Key duchenne muscular dystrophy companies such as Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, and others are evaluating new drugs for duchenne muscular dystrophy to improve the treatment landscape.
  • Promising duchenne muscular dystrophy pipeline therapies in various stages of development include Vamorolone, Delandistrogene moxeparvovec, Givinostat, WVE N531, Pamrevlumab, EDG 5506, Fordadistrogene movaparvovec, Renadirsen, SRP 5051, EN 001, TAS-205, SGT 001, CAP 1002, NS 089/NCNP 02, ENTR-601-44, SRP-9001, GNT 0004, SRP-5051, UX810, SGT-003, Exon 53, Exon 45, AVGN7, PGN EDO51, PGN EDODM1, PGN EDO53, PGN EDO44, PGN EDO45, ALT1102, BMN 351, AOC 1044, SRP-9001, GNT 0004, SRP-5053, SRP-5045, SRP-5052, SRP-5044, SRP-5050, SRP-5051, DYNE-251, SGT-003, RGX-202, Elamipretide, and others.
  • In July 2022, Capricor Therapeutics dosed the first subject in Phase III HOPE-3 clinical trial of cell therapy, CAP-1002, to treat late-stage Duchenne muscular dystrophy (DMD).
  • In June 2022, FibroGen announced that it had completed the target enrollment for its ongoing Phase III LELANTOS-2 clinical trial (NCT04632940) of pamrevlumab, a first-in-class connective tissue growth factor (CTGF) inhibitor antibody, in patients with Duchenne muscular dystrophy (DMD). The trial is set to read out topline data in the second half of 2023 after completing the target enrollment of 73 patients with Duchenne muscular dystrophy aged 6 to 12 years.
  • In May 2022, Stealth BioTherapeutics announced that the FDA had granted orphan drug designation to elamipretide for the treatment of Duchenne muscular dystrophy.
  • In January 2022, The US Food and Drug Administration (FDA) approved Regenxbio’s request to launch a Phase I/II clinical trial in the U.S. evaluating the safety and efficacy of RGX-202, its experimental gene therapy for Duchenne muscular dystrophy (DMD).
  • In June 2020, Sarepta Therapeutics announced that they have entered into a Research License and Option agreement granting Sarepta an option to license the rights to develop and commercialize Selecta’s immune tolerance platform, ImmTOR, for use in Duchenne muscular dystrophy (DMD) and certain limb-girdle muscular dystrophies (LGMDs).
  • In November 2021, CANbridge Pharmaceuticals announced that it has entered into a two-year sponsored research agreement with the University of Washington School of Medicine in Seattle, Washington, for gene therapy research in Duchenne muscular dystrophy (DMD), a rare neuromuscular disease

Request a sample and discover the recent advances in duchenne muscular dystrophy treatment drugs @Duchenne Muscular Dystrophy Pipeline Report

The duchenne muscular dystrophy pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage duchenne muscular dystrophy drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the duchenne muscular dystrophy clinical trial landscape.

Duchenne Muscular Dystrophy Overview

Duchenne muscular dystrophy (DMD) is one of the most severe inherited muscular dystrophies. It is the most common hereditary neuromuscular disease and has no preference for any race or ethnic group. Duchenne muscular dystrophy symptoms include atrophy of the pelvic muscles, followed by involvement of the shoulder muscles. As the condition progresses, muscle weakness and atrophy move from the trunk and forearms to other muscles throughout the body. Mutations in the DMD gene on the X chromosome are among the Duchenne muscular dystrophy causes.

Furthermore, for duchenne muscular dystrophy diagnosis, a thorough clinical assessment, a detailed patient history, and a number of specialized diagnostics, including molecular genetic tests, are used.


Find out more about duchenne muscular dystrophy treatment drugs @Drugs for Duchenne Muscular Dystrophy Treatment

A snapshot of the Duchenne Muscular Dystrophy Pipeline Drugs mentioned in the report:

DrugsCompanyPhase MoARoA
VamoroloneSantheraPre-registrationGlucocorticoid receptor agonist; Mineralocorticoid receptor antagonistOral
GivinostatItalfarmacoPhase IIIHistone deacetylase inhibitorOral
PamrevlumabFibrogenPhase IIIConnective tissue growth factor inhibitorIntravenous
Delandistrogene moxeparvovecRoche/Sarepta TherapeuticsPhase IIIDystrophin replacement; Gene transferenceIntravenous
EDG 5506Edgewise TherapeuticsPhase IICreatine kinase inhibitor; Myosin inhibitorOral
SRP-5051Sarepta TherapeuticsPhase IIDystrophin expression modulatorIntravenous
WVE N531Wave Life Sciences LtdPhase I/IIDystrophin expression stimulants; RNA interferenceIntravenous
PGN EDO51PepGenPhase IDystrophin expression stimulantIntravenous
UX810Ultragenyx PharmaceuticalPreclinicalDystrophin replacement; Gene transferenceParenteral

Learn more about the emerging duchenne muscular dystrophy pipeline therapies @Duchenne Muscular Dystrophy Clinical Trials

Duchenne Muscular Dystrophy Therapeutics Assessment

The duchenne muscular dystrophy pipeline report proffers an integral view of the duchenne muscular dystrophy emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.

Scope of the Duchenne Muscular Dystrophy Pipeline Report 

  • Coverage: Global 
  • Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
  • Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
  • Therapeutics Assessment By Route of Administration: Intravenous, Oral, Subcutaneous
  • Therapeutics Assessment By Molecule Type: Cell therapy, Peptide, Polymer, Small molecule, Gene therapy
  • Therapeutics Assessment By Mechanism of Action: Glucocorticoid receptor agonists, Mineralocorticoid receptor antagonists, Histone deacetylase inhibitors, Connective tissue growth factor inhibitors, Dystrophin expression stimulants, RNA interference, Dystrophin replacements, Gene transference, Creatine kinase inhibitors, Myosin inhibitors, Protein synthesis modulators, Prostaglandin synthase inhibitors
  • Key Duchenne Muscular Dystrophy Companies: Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutic, and others.
  • Key Duchenne Muscular Dystrophy Pipeline Therapies: Vamorolone, Delandistrogene moxeparvovec, Givinostat, WVE N531, Pamrevlumab, EDG 5506, Fordadistrogene movaparvovec, Renadirsen, SRP 5051, EN 001, TAS-205, SGT 001, CAP 1002, NS 089/NCNP 02, ENTR-601-44, SRP-9001, GNT 0004, SRP-5051, UX810, SGT-003, Exon 53, Exon 45, AVGN7, PGN EDO51, PGN EDODM1, PGN EDO53, PGN EDO44, PGN EDO45, ALT1102, BMN 351, AOC 1044, SRP-9001, GNT 0004, SRP-5053, SRP-5045, SRP-5052, SRP-5044, SRP-5050, SRP-5051, DYNE-251, SGT-003, RGX-202, Elamipretide, and others.

Dive deep into rich insights for new drugs for duchenne muscular dystrophy treatment, visit @Duchenne Muscular Dystrophy Gene Therapy

Table of Contents

1.Duchenne Muscular Dystrophy Pipeline Report Introduction
2.Duchenne Muscular Dystrophy Pipeline Report Executive Summary
3.Duchenne Muscular Dystrophy Pipeline: Overview
4.Analytical Perspective In-depth Commercial Assessment
5.Duchenne Muscular Dystrophy Clinical Trial Therapeutics
6.Duchenne Muscular Dystrophy Pipeline: Late Stage Products (Pre-registration)
6.1Vamorolone: Santhera
7.Duchenne Muscular Dystrophy Pipeline: Late Stage Products (Phase III)
7.1Givinostat: Italfarmaco
8.Duchenne Muscular Dystrophy Pipeline: Mid Stage Products (Phase II)
8.1EDG 5506: Edgewise Therapeutics
9.Duchenne Muscular Dystrophy Pipeline: Early Stage Products (Phase I)
9.1PGN EDO51: PepGen
10.Duchenne Muscular Dystrophy Pipeline Therapeutics Assessment
11.Inactive Products in the Duchenne Muscular Dystrophy Pipeline
12.Company-University Collaborations (Licensing/Partnering) Analysis
13.Key Companies
14.Key Products in the Duchenne Muscular Dystrophy Pipeline
15.Unmet Needs
16.Market Drivers and Barriers
17.Future Perspectives and Conclusion
18.Analyst Views
19.Appendix

For further information on the duchenne muscular dystrophy pipeline therapeutics, reach out @Duchenne Muscular Dystrophy Treatment Drugs

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