Recessive Dystrophic Epidermolysis Bullosa - Pipeline Insight, 2025

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:

• Global coverage

Recessive Dystrophic Epidermolysis Bullosa: Understanding

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-B 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.

Recessive Dystrophic Epidermolysis Bullosa Emerging Drugs Chapters

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

Prademagene zamikeracel (pz-cel) is an autologous, engineered cell therapy for RDEB, a rare connective tissue disorder in which patients suffer with severe epidermal wounds that impact the length and quality of their lives. Treatment with pz-cel involves using gene transfer to deliver COL7A1 genes into an RDEB patient's own skin cells (keratinocytes) and transplanting them back to the patient to enable normal Type VII collagen expression and skin function. pz-cel has the potential to be the only durable treatment to address large chronic wounds, which are the most painful and debilitating. pz-cel has shown to be well-tolerated with no serious treatment-related adverse events observed, consistent with past clinical experience. pz-cel has also been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Rare Pediatric Disease designations and Orphan Drug designation in both the U.S. and EU. Currently, the drug is in Registration stage of its development for the treatment of Recessive Dystrophic Epidermolysis Bullosa.

• FCX-007: Castle Creek Biosciences, LLC.

D-Fi, also known as FCX-007, (dabocemagene autoficel), is being developed as an autologous cell-based gene therapy to address the deficiency of functional COL7 in patients with dystrophic epidermolysis bullosa (DEB). The U. S. Food and Drug Administration has granted Orphan Drug designation to D-Fi for the treatment of Dystrophic Epidermolysis Bullosa, which includes RDEB. In addition, D-Fi has been granted Rare Pediatric Disease designation, Fast Track designation and Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA for treatment of RDEB. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Recessive Dystrophic Epidermolysis Bullosa.

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

DelveInsight's report covers around 3+ 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
• Route of Administration

Recessive Dystrophic Epidermolysis Bullosa 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
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

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

Recessive Dystrophic Epidermolysis Bullosa Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions:

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?

Key Players

• Abeona Therapeutics, Inc
• Castle Creek Biosciences, LLC.
• RHEACELL GmbH & Co. KG

Key Products

• Prademagene zamikeracel
• FCX-007
• allo-APZ2-EB