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Also known as EB-101 Surgical application of RDEB wounds, LZRSE-Col7A1 Engineered Autologous Epidermal Sheets [LEAES], Prademagene Zamikeracel, Pz-cel
EB-101 · 2 trials · 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.
Proportion of RDEB wound sites with ≥50% healing from Baseline in treated versus untreated wounds
Associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds
| Arm | Type | Description |
|---|---|---|
| EB-101 Surgical application of RDEB wounds | EXPERIMENTAL | New or Previously Treated RDEB Patients |
| EB-101 | EXPERIMENTAL | One-time surgical application of EB-101 on up to 6 chronic, RDEB wounds |
| 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 \[LEAES\] |
| EB-101 | BIOLOGICAL | autologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7 |
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 therapy being developed for the treatment of epidermolysis bullosa, specifically recessive dystrophic epidermolysis bullosa (RDEB). It is applied surgically to RDEB wounds. Abeona Therapeutics is evaluating it in Phase 3 clinical trials for patients with this rare genetic skin condition.
EB-101 is being developed by Abeona Therapeutics Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol ABEO. The company is responsible for the clinical development of EB-101 for epidermolysis bullosa.
EB-101 is in Phase 3 clinical development. Two Phase 3 trials have been conducted: one completed trial (NCT04227106) and one active trial that is no longer recruiting (NCT05725018). The program has not been approved by the FDA and remains investigational.
EB-101 has been studied in two Phase 3 trials. NCT04227106 is a completed open-label trial in recessive dystrophic epidermolysis bullosa. NCT05725018 is an active Phase 3b study in new and previously EB-101 treated patients, currently not recruiting, with an estimated enrollment of 12 participants.
Yes, EB-101 is also referred to as EB-101 Surgical application of RDEB wounds. This alternative name describes the method of administration, in which EB-101 is surgically applied to wounds in patients with recessive dystrophic epidermolysis bullosa.
EB-101 has received four FDA designations: Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Rare Pediatric Disease. These designations are intended to support and expedite the development of therapies for serious rare conditions such as epidermolysis bullosa.