VivoSeq
Dermatology

Recessive Dystrophic Epidermolysis Bullosa

13

Pipeline Programs↗

2

Active Trials↗

10

Companies Involved↗

Recessive Dystrophic Epidermolysis Bullosa is an emerging landscape with 13 programs across 10 companies. The most active players are Sanofi, Veloxis Pharmaceuticals, Akero Therapeutics. 1 program is in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

41/100

Pipeline Velocity

6/100

Mech. Concentration

17/100

Completion Rate

90/100

Attrition Rate

6/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

Sanofi logo

Sanofi

1 program

Phase 1V

Veloxis Pharmaceuticals

1 program

Phase 1Akero Therapeutics logo

Akero Therapeutics

1 program

Phase 1G

Genzyme, a Sanofi Company

1 program

Phase 1T

Traws Pharma, Inc.

1 program

Phase 1P

Phoenicis Therapeutics

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

79%

Small molecule

11 of 14 classified programs — the dominant modality here.

21%ALL OTHERSMonoclonal antibody17%Protein / enzyme17%Cell therapy17%

Small molecule leads; the ring shows all 14 classified programs, each share of the total. Modality classified for 100% of 18 pipeline programs.

Analysis

Trial Status

16

clinical trials

Completed9 · 56%
Unknown status4 · 25%
Active, not recruiting1 · 6%
Terminated1 · 6%
Not yet recruiting1 · 6%

Clinical Trials

Trial Activity

16 trials

A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa
Active, not recruiting·CCastle Creek Biosciences, LLC.·Phase 3·NCT04213261·6 enrolled
Treatment of Dysphagia in Patients With Dystrophic Epidermolysis Bullosa With Budesonide: a Retrospective Bicentric Study Nice - Paris St Louis / Necker (EBUDE Study)
Not yet recruiting·CCentre Hospitalier Universitaire de Nice·Preclinical·NCT07684105·15 enrolled
Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Completed·AAbeona Therapeutics, Inc·Phase 3·NCT04227106·11 enrolled
Phase I Study of Isotretinoin in Patients With Recessive Dystrophic Epidermolysis Bullosa
Completed·UUniversity of North Carolina·Phase 1·NCT00014729·20 enrolled
A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa
Completed·PPhoenix Tissue Repair, Inc.·Phase 2·NCT04599881·6 enrolled
Safety Study of Gene-modified Autologous Fibroblasts in Recessive Dystrophic Epidermolysis Bullosa
Completed·KKing's College London·Phase 1·NCT02493816·5 enrolled
Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01)
Completed·PPhoenix Tissue Repair, Inc.·Phase 2·NCT05143190·2 enrolled
A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Completed·PPhoenix Tissue Repair, Inc.·Phase 1/2·NCT03752905·12 enrolled
Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC
Completed·TThomas Jefferson University·Phase 1·NCT04177498·3 enrolled
Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients
Completed·UUniversity of Southern California·Phase 1/2·NCT02698735·5 enrolled
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
Completed·AAbeona Therapeutics, Inc·Phase 1/2·NCT01263379·12 enrolled
Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Terminated·—·Phase 1·NCT00881556·3 enrolled
Safety Study and Preliminary Efficacy of Infusion Haploidentical Mesenchymal Stem Cells Derived From Bone Marrow for Treating Recessive Dystrophic Epidermolysis Bullosa
Unknown status·IInstituto de Investigación Hospital Universitario La Paz·Phase 1/2·NCT04153630·9 enrolled
Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Unknown status·UUniversity of Southern California·Phase 1/2·NCT03392909·9 enrolled
Study of PTW-002 in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa Due to Mutation(s) in Exon 73 of the COL7A1 Gene
Unknown status·PPhoenicis Therapeutics·Phase 1/2·NCT05529134·8 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)