The Duchenne Muscular Dystrophy Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.
“Duchenne Muscular Dystrophy Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Duchenne Muscular Dystrophy Market.
Some of the key takeaways from the Duchenne Muscular Dystrophy Pipeline Report:
- Companies across the globe are diligently working toward developing novel Duchenne Muscular Dystrophy treatment therapies with a considerable amount of success over the years.
- Duchenne Muscular Dystrophy companies working in the treatment market are Vertex Pharmaceuticals, Bioleaders Corporation, Wave Life Sciences Ltd., REGENXBIO Inc., Daiichi Sankyo, Sarepta Therapeutics, Taiho Pharmaceutical, FibroGen, Santhera, Italfarmaco, Roche/Sarepta Therapeutics, Edgewise Therapeutics, Sarepta Therapeutics, Wave Life Sciences Ltd, PepGen, Ultragenyx Pharmaceutical, and others, are developing therapies for the Duchenne Muscular Dystrophy treatment
- Emerging Duchenne Muscular Dystrophy therapies in the different phases of clinical trials are- CRISPR editing therapy, BLS-M22, WVE-N531, RGX-202, DS-5141, SRP-5051, TAS-205, Pamrevlumab, Vamorolone, Givinostat, Delandistrogene moxeparvovec, EDG 5506, SRP-5051, WVE N531, PGN EDO51, UX810, and others are expected to have a significant impact on the Duchenne Muscular Dystrophy market in the coming years.
- In July 2022, The Phase III HOPE-3 clinical trial of Capricor Therapeutics' cell treatment, CAP-1002, to treat late-stage Duchenne muscular dystrophy (DMD), dosed its first participant.
- In June 2022, For its current Phase III LELANTOS-2 clinical study (NCT04632940) of pamrevlumab, a first-in-class connective tissue growth factor (CTGF) inhibitor antibody, in patients with Duchenne muscular dystrophy (DMD), FibroGen said that it had reached its target enrollment goal. Once the trial has enrolled all 73 Duchenne muscular dystrophy patients between the ages of 6 and 12 who were intended to participate, topline data will be read out in the second half of 2023.
- In May 2022, Elamipretide has been designated as an orphan medication by the FDA for the treatment of Duchenne muscular dystrophy, according to a statement from Stealth BioTherapeutics.
- In January 2022, The US Food and Drug Administration (FDA) has given Regenxbio permission to begin a Phase I/II clinical trial in the US to assess the security and effectiveness of RGX-202, an investigational gene therapy for Duchenne muscular dystrophy (DMD).
Duchenne Muscular Dystrophy Overview
The most prevalent genetic neuromuscular condition that affects people of all racial and ethnic backgrounds is Duchenne muscular dystrophy. Only men are affected; it affects 1/3,600 live births of infant boys. Some affected neonates may exhibit moderate hypotonia, but further symptoms are rarely noticeable at birth or throughout the first few months of life.
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Emerging Duchenne Muscular Dystrophy Drugs Under Different Phases of Clinical Development Include:
- Pamrevlumab: FibroGen
- Vamorolone: Santhera
- Givinostat: Italfarmaco
- Delandistrogene moxeparvovec: Roche/Sarepta Therapeutics
- CRISPR editing therapy: Vertex Pharmaceuticals
- BLS-M22: Bioleaders Corporation
- WVE-N531: Wave Life Sciences Ltd.
- RGX-202: REGENXBIO Inc.
- DS-5141: Daiichi Sankyo
- SRP-5051: Sarepta Therapeutics
- TAS-205: Taiho Pharmaceutical
- EDG 5506: Edgewise Therapeutics
- SRP-5051: Sarepta Therapeutics
- WVE N531: Wave Life Sciences Ltd
- PGN EDO51: PepGen
- UX810: Ultragenyx Pharmaceutical
Duchenne Muscular Dystrophy Route of Administration
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 Molecule Type
Duchenne Muscular Dystrophy Products have been categorized under various Molecule types, such as
- Cell Therapy
- Peptides
- Polymer
- Small molecule
- Gene therapy
Duchenne Muscular Dystrophy Pipeline Therapeutics Assessment
- Duchenne Muscular Dystrophy Assessment by Product Type
- Duchenne Muscular Dystrophy By Stage and Product Type
- Duchenne Muscular Dystrophy Assessment by Route of Administration
- Duchenne Muscular Dystrophy By Stage and Route of Administration
- Duchenne Muscular Dystrophy Assessment by Molecule Type
- Duchenne Muscular Dystrophy by Stage and Molecule Type
DelveInsight's Duchenne Muscular Dystrophy 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)
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
Further Duchenne Muscular Dystrophy product details are provided in the report. Download the Duchenne Muscular Dystrophy pipeline report to learn more about the emerging Duchenne Muscular Dystrophy therapies
Some of the key companies in the Duchenne Muscular Dystrophy Therapeutics Market include:
Key companies developing therapies for Duchenne Muscular Dystrophy are - 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 Analysis:
The Duchenne Muscular Dystrophy pipeline report provides insights into
- The report provides detailed insights about companies that are developing therapies for the treatment of Duchenne Muscular Dystrophy with aggregate therapies developed by each company for the same.
- It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Duchenne Muscular Dystrophy Treatment.
- Duchenne Muscular Dystrophy key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
- Duchenne Muscular Dystrophy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
- Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Duchenne Muscular Dystrophy market.
The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.
Download Sample PDF Report to know more about Duchenne Muscular Dystrophy drugs and therapies
Duchenne Muscular Dystrophy Pipeline Market Drivers
- Robust Duchenne Muscular Dystrophy pipeline, development of Mutation Suppression therapies are some of the important factors that are fueling the Duchenne Muscular Dystrophy Market.
Duchenne Muscular Dystrophy Pipeline Market Barriers
- However, high cost of Duchenne Muscular Dystrophy treatments, regulatory procedure & and lack of standardized protocol and other factors are creating obstacles in the Duchenne Muscular Dystrophy Market growth.
Scope of Duchenne Muscular Dystrophy Pipeline Drug Insight
- Coverage: Global
- Key Duchenne Muscular Dystrophy Companies: Vertex Pharmaceuticals, Bioleaders Corporation, Wave Life Sciences Ltd., REGENXBIO Inc., Daiichi Sankyo, Sarepta Therapeutics, Taiho Pharmaceutical, FibroGen, Santhera, Italfarmaco, Roche/Sarepta Therapeutics, Edgewise Therapeutics, Sarepta Therapeutics, Wave Life Sciences Ltd, PepGen, Ultragenyx Pharmaceutical, and others
- Key Duchenne Muscular Dystrophy Therapies: CRISPR editing therapy, BLS-M22, WVE-N531, RGX-202, DS-5141, SRP-5051, TAS-205, Pamrevlumab, Vamorolone, Givinostat, Delandistrogene moxeparvovec, EDG 5506, SRP-5051, WVE N531, PGN EDO51, UX810, and others
- Duchenne Muscular Dystrophy Therapeutic Assessment: Duchenne Muscular Dystrophy current marketed and Duchenne Muscular Dystrophy emerging therapies
- Duchenne Muscular Dystrophy Market Dynamics: Duchenne Muscular Dystrophy market drivers and Duchenne Muscular Dystrophy market barriers
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Table of Contents
1. Duchenne Muscular Dystrophy Report Introduction
2. Duchenne Muscular Dystrophy Executive Summary
3. Duchenne Muscular Dystrophy Overview
4. Duchenne Muscular Dystrophy- Analytical Perspective In-depth Commercial Assessment
5. Duchenne Muscular Dystrophy Pipeline Therapeutics
6. Duchenne Muscular Dystrophy Late Stage Products (Phase II/III)
7. Duchenne Muscular Dystrophy Mid Stage Products (Phase II)
8. Duchenne Muscular Dystrophy Early Stage Products (Phase I)
9. Duchenne Muscular Dystrophy Preclinical Stage Products
10. Duchenne Muscular Dystrophy Therapeutics Assessment
11. Duchenne Muscular Dystrophy Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Duchenne Muscular Dystrophy Key Companies
14. Duchenne Muscular Dystrophy Key Products
15. Duchenne Muscular Dystrophy Unmet Needs
16 . Duchenne Muscular Dystrophy Market Drivers and Barriers
17. Duchenne Muscular Dystrophy Future Perspectives and Conclusion
18. Duchenne Muscular Dystrophy Analyst Views
19. Appendix
20. About DelveInsight
About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.
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