Last reviewed · How we verify
EB-101
EB-101 is a gene therapy that delivers a functional copy of the ABCD1 gene to restore adrenoleukodystrophy protein function in patients with cerebral adrenoleukodystrophy.
EB-101 is a gene therapy that delivers a functional copy of the ABCD1 gene to restore adrenoleukodystrophy protein function in patients with cerebral adrenoleukodystrophy. Used for Cerebral adrenoleukodystrophy (CALD).
At a glance
| Generic name | EB-101 |
|---|---|
| Also known as | pz-cel, LZRSE-Col7A1 Engineered Autologous Epidermal Sheets [LEAES] |
| Sponsor | Abeona Therapeutics, Inc |
| Drug class | Gene therapy |
| Target | ABCD1 gene |
| Modality | Biologic |
| Therapeutic area | Neurology |
| Phase | Phase 3 |
Mechanism of action
EB-101 uses a lentiviral vector to transduce patient hematopoietic stem cells ex vivo with the ABCD1 gene, which encodes the adrenoleukodystrophy protein responsible for transporting very long-chain fatty acids. The modified cells are then reinfused into the patient, where they engraft and produce functional protein to prevent or slow neurological deterioration in cerebral adrenoleukodystrophy.
Approved indications
- Cerebral adrenoleukodystrophy (CALD)
Common side effects
- Infusion-related reactions
- Hematologic toxicity
- Infection
Key clinical trials
- A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients (PHASE3)
- A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEB
- A Follow-up Study to Evaluate the Efficacy and Safety of ALLO-ASC-DFU in ALLO-ASC-EB-101 Clinical Trial
- Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- EB-101 CI brief — competitive landscape report
- EB-101 updates RSS · CI watch RSS
- Abeona Therapeutics, Inc portfolio CI