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, 2023“ 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, Santhera, Sarepta Therapeutics, Taiho Pharmaceutical, FibroGen, Italfarmaco, and others, are developing therapies for the Duchenne Muscular Dystrophy treatment 

• Emerging Duchenne Muscular Dystrophy therapies such as – CRISPR editing therapy, BLS-M22, WVE-N531, RGX-202, DS-5141, Vamorolone, SRP-5051, TAS-205, Pamrevlumab, Givinostat, 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 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), FibroGen reported that it had reached its target enrollment goal. After reaching its goal of enrolling 73 individuals with Duchenne muscular dystrophy between the ages of 6 and 12 years, the trial is scheduled to read out topline data 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 April 2021, FibroGen, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the company’s anti-CTGF antibody, pamrevlumab, for the treatment of patients with Duchenne muscular dystrophy (DMD). This designation follows review of the Phase II clinical data from a single-arm trial in non-ambulatory patients with DMD, and represents recognition by the FDA that pamrevlumab has the potential to address an unmet medical need for this disease. Pamrevlumab is currently being evaluated in two Phase III trials for the treatment of DMD
• In December 2020, FibroGen initated a Phase III, randomized, double-blind, trial of Pamrevlumab or Placebo in combination with systemic corticosteroids in ambulatory subjects with Duchenne muscular Dystrophy (DMD). Approximately 70subjects will be enrolled at a 1:1 ratio to Arm A or Arm B respectively

Duchenne Muscular Dystrophy Overview

Duchenne muscular dystrophy is the most common inherited neuromuscular disorder that affects all races and ethnicities. Affecting only males, it occurs in 1/3,600 live-born infant boys and some affected newborns may have some mild hypotonia, other symptoms are rarely present at birth or in early infancy.

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Emerging Duchenne Muscular Dystrophy Drugs Under Different Phases of Clinical Development Include:

• CRISPR editing therapy: Vertex Pharmaceuticals
• BLS-M22: Bioleaders Corporation
• WVE-N531: Wave Life Sciences Ltd.
• RGX-202: REGENXBIO Inc.
• DS-5141: Daiichi Sankyo
• Vamorolone: Santhera
• SRP-5051: Sarepta Therapeutics
• TAS-205: Taiho Pharmaceutical
• Pamrevlumab: FibroGen
• Givinostat: Italfarmaco

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

Molecule Type

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 BioTherapeutic, 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 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 DMD 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, Santhera, Sarepta Therapeutics, Taiho Pharmaceutical, FibroGen, Italfarmaco, and others
• Key Duchenne Muscular Dystrophy Therapies: CRISPR editing therapy, BLS-M22, WVE-N531, RGX-202, DS-5141, Vamorolone, SRP-5051, TAS-205, Pamrevlumab, Givinostat, 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

*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

About DelveInsight

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