4DMT Presents Positive Interim Data from Phase 1/2 AEROW Clinical Trial of Aerosolized 4D-710 for Modulator-Ineligible/-Intolerant Cystic Fibrosis at 47th European Cystic Fibrosis Conference
FDMT4D Molecular Therapeutics(FDMT) Newsfilter·2024-06-06 19:30

Clinically meaningful improvements in ppFEV1 at 12 months observed in 2 of 3 participants with mild to moderate baseline lung function impairment (ppFEV1 50-80%) and >6 months follow up Aerosolized 4D-710 was well tolerated at doses up to 1E15 vg (n=6) Dose-dependent and widespread 4D-710-mediated CFTR transgene RNA and protein expression observed in all lung biopsies from all participants evaluated to date Pre-existing AAV immunity cross-reactive with A101 did not affect transgene expression, biological ac ...