An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease

NCTID	NCT04519749 (View at clinicaltrials.gov)
Description	This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease. (Show More)
Development Status	Active
Indication	Fabry Disease
Disease Ontology Term	DOID:14499
Compound Name	4D-310
Sponsor	4D Molecular Therapeutics
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	18
Results Posted	Not Available

Target Gene/Variant	GLA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell	Cardiomyocyte
Delivery System	Viral transduction
Vector Type	AAV C102
Editor Type	none
Dose 1	1E13 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	4
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation
Recent Updates	No further significant investment is expected on this program, pending additional financing or partnerships