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

NCTID	NCT05725018 (View at clinicaltrials.gov)
Description	To evaluate and further characterize the safety of EB-101 (LZRSE-Col7A1 gene-corrected keratinocyte sheets with type VII collagen \[C7\] expression) for the treatment of large, chronic DEB wounds in new and previously EB-101 treated patients 12 months and older. (Show More)
Development Status	Approved
Indication	Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Disease Ontology Term	DOID:4959
Compound Name	ZEVASKYN
Compound Alias	Prademagene zamikeracel, EB-101
Compound Description	ex-vivo-expanded autologous keratinocytes transduced with LZRSE retroviral vector containing the human COL7A1 gene
Sponsor	Abeona Therapeutics, Inc
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	12 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	COL7A1
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Skin graft
Drug Product Type	Autologous cells
Target Tissue/Cell	Keratinocytes
Delivery System	Viral transduction
Vector Type	LZRSE
Editor Type	none
Dose 1	Transduced 41.25cm^2 keratinocyte sheets, up to 6 wound sites
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
FDA Designations	Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	FDA approved on 4/29/25, list price $3.1M USD