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Recessive Dystrophic Epidermolysis Bullosa

8
Pipeline Programs
6
Companies
9
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
3
2
0
2
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

6 companies ranked by most advanced pipeline stage

Abeona Therapeutics
Abeona TherapeuticsCLEVELAND, OH
2 programs
1
1
EB-101Phase 33 trials
Collagen VII (COL7A1 gene transfer)Phase 1/2
Active Trials
NCT05725018Active Not Recruiting12Est. Sep 2025
NCT05708677Enrolling By Invitation22Est. Aug 2036
NCT04227106Completed11Est. Oct 2022
Castle Creek Biosciences
1 program
1
FCX-007Phase 31 trial
Active Trials
NCT04213261Active Not Recruiting6Est. Jul 2037
Tissue Repair
Tissue RepairAustralia - Sydney
3 programs
1
2
PTR-01Phase 2
PTR-01Phase 21 trial
PTR-01Phase 1/21 trial
Active Trials
NCT03752905Completed12Est. Nov 2020
NCT04599881Completed6Est. Sep 2021
RHEACELL
RHEACELLGermany - Heidelberg
1 program
1
allo-APZ2-EBPhase 1/21 trial
Active Trials
NCT03529877Completed16Est. Nov 2021
Traws Pharma
Traws PharmaPA - Newtown
1 program
1
Rigosertib SodiumPhase 11 trial
Active Trials
NCT04177498Completed3Est. May 2025
BridgeBio Pharma
BridgeBio PharmaCA - Palo Alto
1 program
PTR-01PHASE_21 trial
Active Trials
NCT05143190Completed2Est. Aug 2022

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Abeona TherapeuticsEB-101
Castle Creek BiosciencesFCX-007
Abeona TherapeuticsEB-101
BridgeBio PharmaPTR-01
Tissue RepairPTR-01
RHEACELLallo-APZ2-EB
Tissue RepairPTR-01
Traws PharmaRigosertib Sodium
Abeona TherapeuticsEB-101

Clinical Trials (9)

Total enrollment: 90 patients across 9 trials

NCT05725018Abeona TherapeuticsEB-101

A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients

Start: Apr 2023Est. completion: Sep 202512 patients
Phase 3Active Not Recruiting
NCT04213261Castle Creek BiosciencesFCX-007

A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa

Start: Jun 2020Est. completion: Jul 20376 patients
Phase 3Active Not Recruiting
NCT04227106Abeona TherapeuticsEB-101

Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Start: Jan 2020Est. completion: Oct 202211 patients
Phase 3Completed
NCT05143190BridgeBio PharmaPTR-01

Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01)

Start: Dec 2021Est. completion: Aug 20222 patients
Phase 2Completed
NCT04599881Tissue RepairPTR-01

A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa

Start: Oct 2020Est. completion: Sep 20216 patients
Phase 2Completed
NCT03529877RHEACELLallo-APZ2-EB

Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa

Start: Feb 2019Est. completion: Nov 202116 patients
Phase 1/2Completed
NCT03752905Tissue RepairPTR-01

A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Start: Jan 2019Est. completion: Nov 202012 patients
Phase 1/2Completed
NCT04177498Traws PharmaRigosertib Sodium

Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC

Start: Aug 2021Est. completion: May 20253 patients
Phase 1Completed
NCT05708677Abeona TherapeuticsEB-101

A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEB

Start: Feb 2021Est. completion: Aug 203622 patients
N/AEnrolling By Invitation

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

2 late-stage (Phase 3) programs, potential near-term approvals
6 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.