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)
Indication	Cystic Fibrosis, Modulator-Ineligible/Intolerant
Compound Name	4D-710 (A101-CMV173-coCFTRΔR)
Sponsor	4D Molecular Therapeutics
Funder Type	Industry
Status	Recruiting
Enrollment Count	40

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
Dose 1	2.5 x 10^14 vg
Dose 2	5 x 10^14 vg
Dose 3	1x10^15 vg
Dose 4	2 x 10^15 vg (discontinued due to high transduction to interstitium)
Dose 5	MTD: 1 x 10^15 vg

No.of Trial Sites	16
Locations	United States

Has US IND	True
Recent Updates	Phase III Study Initiation planned H2 2025