DelveInsight’s, “Duchenne Muscular Dystrophy Pipeline Insight 2023,” report provides comprehensive insights about 75+ companies and 75+ pipeline drugs in the Duchenne Muscular Dystrophy pipeline landscape. It covers the Duchenne Muscular Dystrophy pipeline drug profiles, including Duchenne Muscular Dystrophy clinical trials 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 the Duchenne Muscular Dystrophy Pipeline 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 treatment.
- The leading Duchenne Muscular Dystrophy Companies include 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
- Promising Duchenne Muscular Dystrophy pipeline therapies 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.
- October 2023: Sarepta Therapeutics Inc. announced a study of Phase 3 clinical trials for Delandistrogene Moxeparvovec. The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
- November 2023: Dyne Therapeutics announced a study of Phase 1 & 2 clinical trials for DYNE-251. The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension period (96 weeks).
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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.
For further information, refer to the detailed Duchenne Muscular Dystrophy Drugs Launch, Duchenne Muscular Dystrophy Developmental Activities, and Duchenne Muscular Dystrophy News, click here for Duchenne Muscular Dystrophy Ongoing Clinical Trial Analysis
Duchenne Muscular Dystrophy Emerging Drugs Profile
- Vamorolone: Santhera
- Givinostat: Italfarmaco
- Pamrevlumab: Fibrogen
Duchenne Muscular Dystrophy Pipeline Therapeutics Assessment
There are approx. 75+ key companies which are developing the therapies Duchenne Muscular Dystrophy. The companies which have their Duchenne Muscular Dystrophy drug candidates in the most advanced stage, i.e phase III include Roche
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
Duchenne Muscular Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Oral
- Intravenous
- Subcutaneous
Duchenne Muscular Dystrophy Pipeline Products have been categorized under various Molecule types such as
- Small molecule
- Cell Therapy
- Peptides
- Polymer
- Small molecule
- Gene therapy
Find out more about the Duchenne Muscular Dystrophy Pipeline Segmentation, Therapeutics Assessment, and Duchenne Muscular Dystrophy Emerging Drugs @ Duchenne Muscular Dystrophy Treatment Landscape
Scope of the Duchenne Muscular Dystrophy Pipeline Report
- Coverage- Global
- 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 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.
- Duchenne Muscular Dystrophy Segmentation: Molecule Type, Mechanism of Action, Route of Administration, Product Type
Dive deep into rich insights for drugs for Duchenne Muscular Dystrophy Pipeline Companies and Therapies, click here @ Duchenne Muscular Dystrophy Unmet Needs and Analyst Views
Table of Content
- Introduction
- Executive Summary
- Duchenne Muscular Dystrophy: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Duchenne Muscular Dystrophy– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Delandistrogene moxeparvovec: Roche
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- SRP 5051: Sarepta Therapeutics
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I/II)
- WVE N531: Wave Life Sciences
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- EDG 5506: Edgewise Therapeutics
- Drug profiles in the detailed report…..
- Inactive Products
- Duchenne Muscular Dystrophy Key Companies
- Duchenne Muscular Dystrophy Key Products
- Duchenne Muscular Dystrophy- Unmet Needs
- Duchenne Muscular Dystrophy- Market Drivers and Barriers
- Duchenne Muscular Dystrophy- Future Perspectives and Conclusion
- Duchenne Muscular Dystrophy Analyst Views
- Duchenne Muscular Dystrophy Key Companies
- Appendix
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