4D-710 in Adult Patients With Cystic Fibrosis

NCTID	NCT05248230 (View at clinicaltrials.gov)
Description	This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis. (Show More)
Development Status	Active
Indication	Cystic Fibrosis
Disease Ontology Term	DOID:1485
Compound Name	4D-710
Compound Description	A101-CMV173-coCFTRΔR
Sponsor	4D Molecular Therapeutics
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	40
Results Posted	Not Available

Target Gene/Variant	CFTR
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Inhalational
Drug Product Type	Viral vector
Target Tissue/Cell	Airway epithelial cells
Delivery System	Viral transduction
Vector Type	AAV A101
Editor Type	none
Dose 1	2.5E14 vg
Dose 2	5E14 vg
Dose 3	1E15 vg (maximum tolerated dose)
Dose 4	2E15 vg (discontinued due to high transduction to interstitium)
Dose 5

No.of Trial Sites	17
Locations	United States

Has US IND	True
FDA Designations	Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Phase 1 AEROW enrollment completed in November 2024 (Cohorts 3 & 4 fully enrolled with n=3 each), follow-up ongoing; Interim data update expected to be presented in mid-2025