Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
EB-101 Surgical application of RDEB wounds · 1 trial · 3 indications
The number of treatment-related and treatment-emergent adverse events (TEAEs), treatment-emergent wound adverse events (TEWAEs), and serious adverse events (SAEs), including systemic and wound-specific adverse events.
The number of patients and wounds that have an infection or any related adverse event (AE).
The number of patients and wounds that result in hospitalization (SAE).
The incidence of squamous cell carcinoma (SCC).
Replication-competent retrovirus (RCR) status.
| Arm | Type | Description |
|---|---|---|
| EB-101 Surgical application of RDEB wounds | EXPERIMENTAL | New or Previously Treated RDEB Patients |
| Name | Type | Description |
|---|---|---|
| EB-101 Surgical application of RDEB wounds | BIOLOGICAL | EB-101 autologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7 Other Names: • LZRSE-Col7A1 Engineered Autologous Epidermal Sheets \[LEAES\] |
Inclusion Criteria: 1. Clinical diagnosis of DEB. 2. Age 12 months and older. 3. Willing and able to give consent/assent; if under the age of 18 years, guardian(s) is/are willing and able to give consent. 4. (This inclusion criterion was deleted as of Amendment 1.) 5. Confirmation of DEB diagnosis ...
EB-101 is an investigational gene-corrected cell therapy being developed for epidermolysis bullosa, specifically recessive dystrophic epidermolysis bullosa (RDEB). It is applied surgically to RDEB wounds. The drug is in Phase 3 clinical development and is not yet approved by the FDA.
EB-101 is being developed by Abeona Therapeutics Inc., a biopharmaceutical company traded on the NASDAQ under the ticker ABEO. The company is conducting a Phase 3b clinical trial for the treatment of dystrophic epidermolysis bullosa in new and previously EB-101 treated patients.
EB-101 is currently in Phase 3 clinical development. The active trial is a Phase 3b study (NCT05725018) that is enrolling patients with epidermolysis bullosa, including recessive dystrophic epidermolysis bullosa. The study is active but not recruiting participants.
EB-101 is being studied in one active clinical trial, NCT05725018, a Phase 3b study for the treatment of dystrophic epidermolysis bullosa in new and previously EB-101 treated patients. The trial is enrolling 12 participants in the United States and is open to patients as young as 12 months.
EB-101 has received several FDA designations, including Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Rare Pediatric Disease designations. These designations are intended to expedite the development and review of the drug for epidermolysis bullosa.
EB-101 is a gene-corrected cell therapy applied surgically to wounds in patients with recessive dystrophic epidermolysis bullosa. It is being studied in a Phase 3b trial and has received FDA designations including Breakthrough Therapy and RMAT, reflecting its potential as a regenerative medicine.