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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
Clinically meaningful improvements in ppFEV1 at 12 months observed in 2 of 3 participants with mild to moderate baseline lung function impairment (ppFEV1
About this update from 4d Molecular Therapeutics, Inc.
[{"type":"text","content":"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 datePre-existing AAV immunity cross-reactive with A101 did not affect transgene expression, biological activity or safety1E15 vg dose cleared for evaluation in Phase 2 Dose Expansion stage; enrollment expected to begin in H2 2024 and next interim data update expected in mid-2025Company to host live webcast today at 8:00 a.m. ET with Dr. Jennifer L. Taylor-Cousar, lead Principal Investigator in the AEROW clinical trial EMERYVILLE, Calif., June 06, 2024 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company), a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today announced positive interim clinical data from the 4D-710 Phase 1/2 AEROW clinical trial for the treatment of cystic fibrosis (CF) lung disease. Interim data from the study will be presented by Jennifer L. Taylor-Cousar, M.D., MSCS, in an oral presentation titled, “CFTR transgene expression in airway epithelial cells following aerosolized administration of the AAV-based gene therapy 4D-710 to adults with cystic fibrosis lung disease,” at the 47th European Cystic Fibrosis Conference held in Glasgow, UK on Thursday, June 6 at 5:00 p.m. BST. “We are pleased with the widespread CFTR transgene and protein expression in airway cells from all participants at all doses in the AEROW clinical trial,” said Jennifer L. Taylor-Cousar, M.D., MSCS, Professor, Departments of Medicine and Pediatrics, and Co-Director, Adult Cystic Fibrosis Program, Director, Cystic Fibrosis Therapeutics Development Center, National Jewish Health and lead Principal Investigator in the AEROW clinical trial. “The consistent widespread 4D-710–mediated CFTR expression and initial clinical activity in Phase 1 are encouraging and suggest that 4D-710 has the potential to be the first treatment option for those living with cystic fibrosis who do not currently benefit from available disease-modifying ...