A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa

NCTID	NCT04917874 (View at clinicaltrials.gov)
Description	This is a 112-week (approximately two-year) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 2 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB). Participants will be dosed weekly with the topical B-VEC therapy. The primary endpoint will be to assess long term safety and tolerability of the topical gene therapy. The study is for those who participated in Phase 3 study, as well as, new participants who were unable to participate in the Phase 3 study, who meet all enrollment criteria. (Show More)
Development Status	Approved
Indication	Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Disease Ontology Term	DOID:4959
Compound Name	VYJUVEK
Compound Alias	beremagene geperpavec
Sponsor	Krystal Biotech, Inc.
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	47
Results Posted	Not Available

Target Gene/Variant	COL7A1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Topical
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	HSV1
Editor Type	none
Dose 1	Maximum weekly dose: 1.6E9 PFU (6 months to < 3 years)
Dose 2	Maximum weekly dose: 3.2E9 PFU (over 3 years old)
Dose 3
Dose 4
Dose 5

No.of Trial Sites	6
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Priority Review, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	FDA approved 5/19/23, estimated price $600K/year