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NCT05248230

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-710 in Adults With Cystic Fibrosis

Study Summary

This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.

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Interventions

4D-710BIOLOGICAL
4D-710 is an adeno-associated virus (AAV) gene therapy comprised of an AAV capsid variant (4D-A101) carrying a transgene cassette encoding human cystic fibrosis transmembrane conductance regulator with a deletion in the regulatory domain (CFTRΔR).

Study Locations

FacilityCityStateCountry
University of Alabama Child Health Research UnitBirminghamAlabamaUnited States
The University of ArizonaTucsonArizonaUnited States
University of California San FranciscoSan FranciscoCaliforniaUnited States
National Jewish HealthDenverColoradoUnited States
University of FloridaGainesvilleFloridaUnited States
University of Miami HospitalMiamiFloridaUnited States
Northwestern Memorial HospitalChicagoIllinoisUnited States
University of Kansas Medical CenterKansas CityKansasUnited States
Johns Hopkins HospitalBaltimoreMarylandUnited States
Massachusetts General HospitalBostonMassachusettsUnited States
Boston Children's HospitalBostonMassachusettsUnited States
University of North Carolina at Chapel HillChapel HillNorth CarolinaUnited States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical CenterClevelandOhioUnited States
Nationwide Children's HospitalColumbusOhioUnited States
Penn State HealthHersheyPennsylvaniaUnited States
The Hospital of the University of PennsylvaniaPhiladelphiaPennsylvaniaUnited States
Medical University of South CarolinaCharlestonSouth CarolinaUnited States
The University of Texas Southwestern Medical CenterDallasTexasUnited States
Virginia Commonwealth University Health SystemRichmondVirginiaUnited States
University of Washington Medical CenterSeattleWashingtonUnited States

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026