EB-101 Treatment for New and Previously Treated Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

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 RDEB wounds in new and previously EB-101 treated patients 12 months and older. (Show More)
Development Status	Active
Indication	Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Disease Ontology Term	DOID:4959
Compound Name	EB-101
Compound Alias	Prademagene zamikeracel
Compound Description	LZRSE-Col7A1 gene-corrected keratinocyte sheets with type VII collagen expression
Sponsor	Abeona Therapeutics, Inc
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	12
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 35cm^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, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	BLA resubmission October 2024, new PDUFA date 4/29/25