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Report Overview

Recessive dystrophic epidermolysis bullosa (RDEB) is a rare genetic skin disorder characterized by fragile skin and recurrent blistering, caused by mutations in the COL7A1 gene. According to Chan I Thien et al., 2024, out of 309 patients with hereditary epidermolysis bullosa, 31.7% were diagnosed with RDEB. The drug pipeline for RDEB includes small molecules, monoclonal antibodies, gene therapies, peptides, and protein-based therapies, reflecting a growing focus on targeted and personalized treatments. According to the recessive dystrophic epidermolysis bullosa (RDEB) pipeline analysis by Expert Market Research, rising prevalence, unmet medical needs, and advances in gene and protein therapies are expected to drive significant growth in the treatment landscape in the coming years.

  • Major companies involved in the recessive dystrophic epidermolysis bullosa (RDEB) pipeline analysis include Castle Creek Biosciences, LLC., Abeona Therapeutics, Inc., and others.

  • Leading drugs currently in the pipeline include FCX-007, EB-101, BVEC, and others.

  • The recessive dystrophic epidermolysis bullosa (RDEB) drug pipeline is expanding, driven by advances in gene therapies, protein-based treatments, and targeted small molecules, addressing unmet medical needs and supporting significant market growth in the coming years.

Report Coverage

The Recessive Dystrophic Epidermolysis Bullosa (RDEB) Pipeline Analysis Report by Expert Market Research gives comprehensive insights into recessive dystrophic epidermolysis bullosa (RDEB) therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for recessive dystrophic epidermolysis bullosa (RDEB). The recessive dystrophic epidermolysis bullosa (RDEB) report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The recessive dystrophic epidermolysis bullosa (RDEB) pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with recessive dystrophic epidermolysis bullosa (RDEB) treatment guidelines to ensure optimal care practices.

The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to recessive dystrophic epidermolysis bullosa (RDEB).

Recessive Dystrophic Epidermolysis Bullosa (RDEB) Pipeline Analysis by Drug Class

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Recessive Dystrophic Epidermolysis Bullosa (RDEB) Pipeline Outlook

Recessive dystrophic epidermolysis bullosa (RDEB) is a rare genetic disorder caused by mutations in the COL7A1 gene, leading to absent or dysfunctional Type VII collagen. This results in extremely fragile skin that blisters and tears easily, causing chronic wounds, high infection risk, and increased susceptibility to skin cancer. The disease primarily affects the epidermis and dermis junction, compromising skin integrity and overall quality of life.

Recessive dystrophic epidermolysis bullosa (RDEB) treatments are focusing on gene- and cell-based therapies, including topical gels and autologous cell therapies, alongside supportive wound care and pain management. In April 2025, the U.S. FDA approved Zevaskyn, an autologous, cell-based gene therapy for RDEB. The therapy uses genetically engineered keratinocyte sheets expressing functional COL7A1, grafted onto chronic wounds, promoting Type VII collagen production and improving skin integrity in patients.

Recessive Dystrophic Epidermolysis Bullosa (RDEB) Epidemiology

The recessive dystrophic epidermolysis bullosa (RDEB) drug pipeline is witnessing robust growth, driven by increasing focus on gene, protein, and cell-based therapies, alongside drug repurposing strategies. Chan I. Thien et al., 2024, observed that 31.7% of dystrophic EB cases are recessive. According to Stefanos A. Koutsoukos et al., 2024, the incidence of RDEB in the United States is 3.05 per one million live births, with a prevalence of 1.35 per one million. Eunice Jeffs et al., 2024, reported prevalence for all forms of epidermolysis bullosa ranging from 11.1 per million in the USA to 34.8 per million in England and Wales, while RDEB-specific prevalence in the UK is 1.4-3.3 per million, with incidence between 3.05-8.1 per million live births. This growing epidemiological awareness, combined with ongoing preclinical and clinical studies, is accelerating therapeutic innovation and supporting sustained market growth.

Recessive Dystrophic Epidermolysis Bullosa (RDEB) – Pipeline Therapeutic Assessment

This section of the report covers the analysis of recessive dystrophic epidermolysis bullosa (RDEB) drug candidates based on several segmentations, including:

By Phase

The pipeline assessment report covers 50+ drug analyses based on phase:

  • Late-Stage Products (Phase 3 and Phase 4)
  • Mid-Stage Products (Phase 2)
  • Early-Stage Products (Phase I)
  • Preclinical and Discovery Stage Products

By Drug Class

The recessive dystrophic epidermolysis bullosa (RDEB) pipeline analysis report covers 50+ drug analyses based on drug classes:

  • Small Molecules
  • Monoclonal Antibodies
  • Gene Therapies
  • Peptides
  • Protein

By Route of Administration

The pipeline assessment report covers 50+ drug analyses based on the route of administration.

  • Oral
  • Parenteral
  • Others

Recessive Dystrophic Epidermolysis Bullosa (RDEB) Pipeline Assessment Segmentation, By Phases

The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase II and I, with 35% each, cover a major share of the total recessive dystrophic epidermolysis bullosa (RDEB) clinical trials. It is followed by phase III at 21% and early phase I at 7%. These advanced-stage developments highlight strong clinical activity and innovation, positively impacting the RDEB market by enhancing the potential for effective therapies and supporting sustained growth across the forecast period.

Recessive Dystrophic Epidermolysis Bullosa (RDEB) Pipeline Assessment Segmentation, By Drug Classes

The drug molecule categories covered under the recessive dystrophic epidermolysis bullosa (RDEB) pipeline analysis include small molecules, monoclonal antibodies, gene therapies, peptides, and proteins. The recessive dystrophic epidermolysis bullosa (RDEB) report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for recessive dystrophic epidermolysis bullosa (RDEB). Mesenchymal stromal cell-based therapies are emerging as promising treatment options for recessive dystrophic epidermolysis bullosa (RDEB). For instance, CORDStrom, developed by INmune Bio, is an allogeneic, pooled umbilical cord-derived MSC therapy under investigation in the MissionEB study. CORDStrom is designed to modulate inflammation, reduce skin blistering, and improve wound healing in pediatric patients with intermediate and severe RDEB, offering a potential systemic therapy that could significantly enhance clinical outcomes in the RDEB market.

Recessive Dystrophic Epidermolysis Bullosa (RDEB) Clinical Trials – Key Players

The EMR report for the recessive dystrophic epidermolysis bullosa (RDEB) pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed recessive dystrophic epidermolysis bullosa (RDEB) therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in recessive dystrophic epidermolysis bullosa (RDEB) clinical trials:

  • Castle Creek Biosciences, LLC.
  • Abeona Therapeutics, Inc.
  • Aegle Therapeutics
  • Krystal Biotech, Inc.
  • Eliksa Therapeutics, Inc.
  • Argenx
  • Daewoong Pharmaceutical Co. Ltd.
  • Phoenix Tissue Repair, Inc.
  • Traws Pharma, Inc.

Recessive Dystrophic Epidermolysis Bullosa (RDEB) – Emerging Drugs Profile

This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for recessive dystrophic epidermolysis bullosa (RDEB). It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of recessive dystrophic epidermolysis bullosa (RDEB) drug candidates.

Biological: FCX-007

FCX-007 is a genetically modified autologous human dermal fibroblast therapy, designed to deliver functional collagen VII protein to the skin of patients with recessive dystrophic epidermolysis bullosa (RDEB). Sponsored by Castle Creek Biosciences, LLC., this Phase 3 DEFI-RDEB study is examining whether intradermal injections of FCX-007, in addition to standard care, can enhance wound healing compared to untreated control wounds. Subjects are receiving multiple treatment sessions, with safety and efficacy monitored through Week 48, followed by a long-term follow-up period, supporting evaluation of both therapeutic benefits and safety outcomes.

Biological: EB-101

EB-101 is an autologous, gene-corrected keratinocyte sheet therapy designed to treat recessive dystrophic epidermolysis bullosa (RDEB) by restoring functional Type VII collagen in the skin. Sponsored by Abeona Therapeutics, Inc., this Phase 3b study is examining the safety and efficacy of a one-time surgical application of up to 12 EB-101 sheets on large, chronic DEB wounds in both new and previously treated patients aged 12 months and older. Patients are being monitored through 24 weeks post-treatment to assess wound healing, safety outcomes, and long-term therapeutic potential.

Drug: BVEC

BVEC is a topical, herpes simplex virus type 1-based gene therapy designed to deliver functional COL7A1 to restore Type VII collagen in recessive dystrophic epidermolysis bullosa (RDEB) skin. Sponsored by Stanford University, this Phase 2 study is examining whether BVEC-induced C7 expression after squamous cell carcinoma (SCC) excision can normalize the tumor microenvironment and reduce recurrence. The therapy is being applied weekly in gel droplet form to targeted wounds, aiming to improve skin integrity, enhance wound healing, and potentially extend patient lifespan.

*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*

Key Questions Answered in the Recessive Dystrophic Epidermolysis Bullosa (RDEB) Pipeline Insight Report

  • Which companies/institutions are leading the recessive dystrophic epidermolysis bullosa (RDEB) drug development?
  • Which company is leading the recessive dystrophic epidermolysis bullosa (RDEB) pipeline development activities?
  • What is the current recessive dystrophic epidermolysis bullosa (RDEB) commercial assessment?
  • What are the opportunities and challenges present in the recessive dystrophic epidermolysis bullosa (RDEB) pipeline landscape?
  • What is the efficacy and safety profile of recessive dystrophic epidermolysis bullosa (RDEB) pipeline drugs?
  • Which company is conducting major trials for recessive dystrophic epidermolysis bullosa (RDEB) drugs?
  • Which companies/institutions are involved in recessive dystrophic epidermolysis bullosa (RDEB) collaborations aimed at providing enhanced therapeutic alternatives for patients?
  • What are the geographies covered for clinical trials in recessive dystrophic bullosa epidermolysis (RDEB)?

Reasons To Buy This Report

The Recessive Dystrophic Epidermolysis Bullosa (RDEB) Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for recessive dystrophic epidermolysis bullosa (RDEB). It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into recessive dystrophic epidermolysis bullosa (RDEB) collaborations, regulatory environments, and potential growth opportunities.

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*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*

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Key Highlights of the Report

Please note that the figures mentioned in the description serve as estimates and may vary from the actual figures presented in the final report.

Scope of the Report

Details

Drug Pipeline by Clinical Trial Phase

  • Late-Stage Products (Phase III and Phase IV)
  • Mid-Stage Products (Phase II)
  • Early-Stage Products (Phase I)
  • Preclinical and Discovery Stage Products

Route of Administration

  • Oral
  • Parenteral
  • Others

Drug Classes

  • Small Molecules
  • Monoclonal Antibodies
  • Gene Therapies
  • Peptides
  • Protein

Leading Sponsors Covered

  • Castle Creek Biosciences, LLC.
  • Abeona Therapeutics, Inc.
  • Aegle Therapeutics
  • Krystal Biotech, Inc.
  • Eliksa Therapeutics, Inc.
  • Argenx
  • Daewoong Pharmaceutical Co. Ltd.
  • Phoenix Tissue Repair, Inc.
  • Traws Pharma, Inc.

Geographies Covered

  • North America
  • Europe
  • Asia Pacific
  • Others

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