
Recessive Dystrophic Epidermolysis Bullosa - Pipeline Insight, 2025
Description
DelveInsight’s, “Recessive Dystrophic Epidermolysis Bullosa - Pipeline Insight, 2025” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Recessive Dystrophic Epidermolysis Bullosa 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.
Geography Covered
Recessive Dystrophic Epidermolysis Bullosa: Overview
Recessive dystrophic epidermolysis bullosa (RDEB) is a severe form of epidermolysis bullosa (EB), a group of inherited skin disorders caused by mutations in genes that encode structural proteins in the skin. RDEB is characterized by mutations in the COL7A1 gene, which leads to defective type VII collagen, impairing the attachment between the epidermis and dermis. This results in skin blistering and ulceration following minor trauma. RDEB is inherited in an autosomal recessive pattern and typically presents with more severe clinical features compared to its dominant counterpart, dominant dystrophic epidermolysis bullosa (DDEB). RDEB is one of the most devastating forms of EB, with significant morbidity and a wide spectrum of clinical severity depending on the type of COL7A1 mutation present.
RDEB is caused by mutations in the COL7A1 gene, which encodes type VII collagen (C7), a critical structural protein in the skin. C7 forms anchoring fibrils that help bind the epidermis to the dermis, providing mechanical stability. In RDEB, mutations in both alleles of COL7A1 result in defective or absent C7, leading to poor epidermal-dermal adherence and skin fragility. These mutations can be missense, nonsense, or involve compound heterozygosity, where one mutation is inherited from each parent, resulting in a wide spectrum of clinical severity. The most severe form, RDEB-generalized severe (GS), is caused by premature termination codons that lead to truncated C7 polypeptides, while the milder RDEB-generalized other (GO) variant is associated with one premature termination codon and one missense mutation, resulting in partially functional C7.
Clinically, RDEB presents with widespread blistering and skin erosion, often involving mucous membranes and leading to complications such as esophageal strictures and chronic malnutrition. This can necessitate feeding tube placement. Patients may also experience scarring, pseudosyndactyly (fusion of fingers and toes), joint contractures, and visual impairment due to eye inflammation. RDEB-GS is the most severe form and often results in early death, typically by the fourth decade, due to aggressive squamous cell carcinoma arising from chronic scarring. In contrast, RDEB-GO tends to present with a milder phenotype and a better prognosis, with median survival extending into the 50s or 60s. The clinical course of RDEB can be influenced by genetic variability and other factors, such as differential activation of the TGF-β pathway, which may contribute to the phenotypic diversity observed in patients with identical mutations.
Current management of RDEB focuses on wound care, minimizing trauma, and multidisciplinary support to address complications such as dental issues, esophageal strictures, and squamous cell carcinoma (SCC) screenings. Innovative therapies under investigation include gene therapy, fibroblast cell therapy, bone marrow stem cell therapy, and protein therapy. Gene therapy aims to correct COL7A1 mutations in patients’ skin cells, while fibroblast cell therapy involves injecting healthy fibroblasts to boost C7 production. Bone marrow stem cell therapy has shown promise in enhancing wound healing and C7 production, although risks remain. Protein therapy, including C7 injections, is being explored to improve dermal-epidermal adherence. While these therapies show potential, clinical application is still in early stages, and ongoing research is crucial for developing effective, safe treatments for RDEB.
""Recessive Dystrophic Epidermolysis Bullosa- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Recessive Dystrophic Epidermolysis Bullosa pipeline landscape is provided which includes the disease overview and Recessive Dystrophic Epidermolysis Bullosa treatment guidelines. The assessment part of the report embraces, in depth Recessive Dystrophic Epidermolysis Bullosa commercial assessment and clinical assessment of the pipeline products under development. 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, Recessive Dystrophic Epidermolysis Bullosa collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
This segment of the Recessive Dystrophic Epidermolysis Bullosa report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Recessive Dystrophic Epidermolysis Bullosa Emerging Drugs
Further product details are provided in the report……..
Recessive Dystrophic Epidermolysis Bullosa: Therapeutic Assessment
This segment of the report provides insights about the different Recessive Dystrophic Epidermolysis Bullosa drugs segregated based on following parameters that define the scope of the report, such as:
Recessive Dystrophic Epidermolysis Bullosa: Pipeline Development Activities
The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Recessive Dystrophic Epidermolysis Bullosa therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Recessive Dystrophic Epidermolysis Bullosa drugs.
Recessive Dystrophic Epidermolysis Bullosa Report Insights
Current Treatment Scenario and Emerging Therapies:
Geography Covered
- Global coverage
Recessive Dystrophic Epidermolysis Bullosa: Overview
Recessive dystrophic epidermolysis bullosa (RDEB) is a severe form of epidermolysis bullosa (EB), a group of inherited skin disorders caused by mutations in genes that encode structural proteins in the skin. RDEB is characterized by mutations in the COL7A1 gene, which leads to defective type VII collagen, impairing the attachment between the epidermis and dermis. This results in skin blistering and ulceration following minor trauma. RDEB is inherited in an autosomal recessive pattern and typically presents with more severe clinical features compared to its dominant counterpart, dominant dystrophic epidermolysis bullosa (DDEB). RDEB is one of the most devastating forms of EB, with significant morbidity and a wide spectrum of clinical severity depending on the type of COL7A1 mutation present.
RDEB is caused by mutations in the COL7A1 gene, which encodes type VII collagen (C7), a critical structural protein in the skin. C7 forms anchoring fibrils that help bind the epidermis to the dermis, providing mechanical stability. In RDEB, mutations in both alleles of COL7A1 result in defective or absent C7, leading to poor epidermal-dermal adherence and skin fragility. These mutations can be missense, nonsense, or involve compound heterozygosity, where one mutation is inherited from each parent, resulting in a wide spectrum of clinical severity. The most severe form, RDEB-generalized severe (GS), is caused by premature termination codons that lead to truncated C7 polypeptides, while the milder RDEB-generalized other (GO) variant is associated with one premature termination codon and one missense mutation, resulting in partially functional C7.
Clinically, RDEB presents with widespread blistering and skin erosion, often involving mucous membranes and leading to complications such as esophageal strictures and chronic malnutrition. This can necessitate feeding tube placement. Patients may also experience scarring, pseudosyndactyly (fusion of fingers and toes), joint contractures, and visual impairment due to eye inflammation. RDEB-GS is the most severe form and often results in early death, typically by the fourth decade, due to aggressive squamous cell carcinoma arising from chronic scarring. In contrast, RDEB-GO tends to present with a milder phenotype and a better prognosis, with median survival extending into the 50s or 60s. The clinical course of RDEB can be influenced by genetic variability and other factors, such as differential activation of the TGF-β pathway, which may contribute to the phenotypic diversity observed in patients with identical mutations.
Current management of RDEB focuses on wound care, minimizing trauma, and multidisciplinary support to address complications such as dental issues, esophageal strictures, and squamous cell carcinoma (SCC) screenings. Innovative therapies under investigation include gene therapy, fibroblast cell therapy, bone marrow stem cell therapy, and protein therapy. Gene therapy aims to correct COL7A1 mutations in patients’ skin cells, while fibroblast cell therapy involves injecting healthy fibroblasts to boost C7 production. Bone marrow stem cell therapy has shown promise in enhancing wound healing and C7 production, although risks remain. Protein therapy, including C7 injections, is being explored to improve dermal-epidermal adherence. While these therapies show potential, clinical application is still in early stages, and ongoing research is crucial for developing effective, safe treatments for RDEB.
""Recessive Dystrophic Epidermolysis Bullosa- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Recessive Dystrophic Epidermolysis Bullosa pipeline landscape is provided which includes the disease overview and Recessive Dystrophic Epidermolysis Bullosa treatment guidelines. The assessment part of the report embraces, in depth Recessive Dystrophic Epidermolysis Bullosa commercial assessment and clinical assessment of the pipeline products under development. 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, Recessive Dystrophic Epidermolysis Bullosa collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Recessive Dystrophic Epidermolysis Bullosa R&D. The therapies under development are focused on novel approaches to treat/improve Recessive Dystrophic Epidermolysis Bullosa.
This segment of the Recessive Dystrophic Epidermolysis Bullosa report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Recessive Dystrophic Epidermolysis Bullosa Emerging Drugs
- Prademagene zamikeracel: Abeona Therapeutics, Inc
- FCX-007: Castle Creek Biosciences, LLC.
Further product details are provided in the report……..
Recessive Dystrophic Epidermolysis Bullosa: Therapeutic Assessment
This segment of the report provides insights about the different Recessive Dystrophic Epidermolysis Bullosa drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Recessive Dystrophic Epidermolysis Bullosa
- There are approx. 3+ key companies which are developing the therapies for Recessive Dystrophic Epidermolysis Bullosa. The companies which have their Recessive Dystrophic Epidermolysis Bullosa drug candidates in the most advanced stage, i.e. Registration include, Abeona Therapeutics, Inc.
- Phases
- 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
- Route of Administration
- Oral
- Intravenous
- Subcutaneous
- Parenteral
- Topical
- Molecule Type
- Recombinant fusion proteins
- Small molecule
- Monoclonal antibody
- Peptide
- Polymer
- Gene therapy
- Product Type
Recessive Dystrophic Epidermolysis Bullosa: Pipeline Development Activities
The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Recessive Dystrophic Epidermolysis Bullosa therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Recessive Dystrophic Epidermolysis Bullosa drugs.
Recessive Dystrophic Epidermolysis Bullosa Report Insights
- Recessive Dystrophic Epidermolysis Bullosa Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Recessive Dystrophic Epidermolysis Bullosa drugs?
- How many Recessive Dystrophic Epidermolysis Bullosa drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Recessive Dystrophic Epidermolysis Bullosa?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Recessive Dystrophic Epidermolysis Bullosa therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Recessive Dystrophic Epidermolysis Bullosa and their status?
- What are the key designations that have been granted to the emerging drugs?
- Abeona Therapeutics, Inc
- Castle Creek Biosciences, LLC.
- RHEACELL GmbH & Co. KG
- Prademagene zamikeracel
- FCX-007
- allo-APZ2-EB
Table of Contents
60 Pages
- Introduction
- Executive Summary
- Recessive Dystrophic Epidermolysis Bullosa: Overview
- Introduction
- Causes
- Pathophysiology
- Signs and Symptoms
- Diagnosis
- Treatment
- Pipeline Therapeutics
- Comparative Analysis
- 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
- Recessive Dystrophic Epidermolysis Bullosa– DelveInsight’s Analytical Perspective
- Late Stage Products (Registration)
- Comparative Analysis
- Prademagene zamikeracel: Abeona Therapeutics, Inc
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Comparative Analysis
- Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Comparative Analysis
- Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Comparative Analysis
- Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Recessive Dystrophic Epidermolysis Bullosa Key Companies
- Recessive Dystrophic Epidermolysis Bullosa Key Products
- Recessive Dystrophic Epidermolysis Bullosa- Unmet Needs
- Recessive Dystrophic Epidermolysis Bullosa- Market Drivers and Barriers
- Recessive Dystrophic Epidermolysis Bullosa- Future Perspectives and Conclusion
- Recessive Dystrophic Epidermolysis Bullosa Analyst Views
- Recessive Dystrophic Epidermolysis Bullosa Key Companies
- Appendix
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