DelveInsight’s, “Duchenne Muscular Dystrophy Pipeline Insight, 2022,” report provides comprehensive insights about 75+ companies and 75+ pipeline drugs in the Duchenne Muscular Dystrophy pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

Key takeaways from Duchenne Muscular Dystrophy Pipeline Insight Report

• DelveInsight’s Duchenne Muscular Dystrophy Pipeline report depicts a robust space with 75+ active players working to develop 75+ pipeline therapies for Duchenne Muscular Dystrophy.
• The leading 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.
• The companies and academics are working to assess challenges and seek opportunities that could influence R&D Duchenne Muscular Dystrophy. The therapies under development are focused on novel approaches to treat/improve Duchenne Muscular Dystrophy.
• In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Duchenne Muscular Dystrophy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

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

 

Duchenne Muscular Dystrophy Overview

Duchenne muscular dystrophy (DMD) is one of the most severe forms of inherited muscular dystrophies. It is the most common hereditary neuromuscular disease and does not exhibit a predilection for any race or ethnic group. Mutations in the dystrophin gene lead to progressive muscle fiber degeneration and weakness. This weakness may present initially with difficulty in ambulation but progressively advances to such an extent that affected patients are unable to carry out activities of daily living and must use wheelchairs. Cardiac and orthopedic complications are common, and death usually occurs in the twenties due to respiratory muscle weakness or cardiomyopathy. Current therapy is centered on treatment with glucocorticoids and physiotherapy to prevent orthopedic complications.

 

Latest Breakthroughs of Duchenne Muscular Dystrophy Treatment Landscape

• 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.

 

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

 

Duchenne Muscular Dystrophy Emerging Drugs

• Vamorolone: Santhera
• Givinostat: Italfarmaco
• Pamrevlumab: Fibrogen

 

DelveInsight’s Duchenne Muscular Dystrophy Pipeline Report covers around 75+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

 

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

 

Scope of the Duchenne Muscular Dystrophy Pipeline Report

• Coverage- Global
• Duchenne Muscular Dystrophy Therapeutic Assessment
• Assessment by Product Type
• Assessment by Stage and Product Type
• Assessment by Route of Administration
• Assessment by Stage and Route of Administration
• Assessment by Molecule Type
• Assessment by Stage and Molecule Type
• 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 BioTherapeutics, and others
• Duchenne Muscular Dystrophy 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 Companies

 

Table of Content

1. Introduction
2. Executive Summary
3. Duchenne Muscular Dystrophy: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Duchenne Muscular Dystrophy– DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. Delandistrogene moxeparvovec: Roche
9. Mid Stage Products (Phase II)
10. SRP 5051: Sarepta Therapeutics
11. Early Stage Products (Phase I/II)
12. WVE N531: Wave Life Sciences
13. Early Stage Products (Phase I)
14. EDG 5506: Edgewise Therapeutics
15. Inactive Products
16. Duchenne Muscular Dystrophy Key Companies
17. Duchenne Muscular Dystrophy Key Products
18. Duchenne Muscular Dystrophy- Unmet Needs
19. Duchenne Muscular Dystrophy- Market Drivers and Barriers
20. Duchenne Muscular Dystrophy- Future Perspectives and Conclusion
21. Duchenne Muscular Dystrophy Analyst Views
22. Duchenne Muscular Dystrophy Key Companies
23. Appendix

 

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

 

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