4DMT Announces Positive Interim Clinical Data from 4D-710 AEROW Phase 1 Clinical Trial in Cystic Fibrosis Lung Disease

Core Insights - 4D Molecular Therapeutics announced positive interim clinical data from the 4D-710 Phase 1 AEROW clinical trial for cystic fibrosis lung disease, indicating potential for transformative treatment options for patients [1][2] Clinical Trial Data - The AEROW trial demonstrated that the selected Phase 2 dose of 4D-710 was well tolerated and achieved significant CFTR expression levels, showing clinical benefits across various lung function and symptom measures [2] - Clinically meaningful lung function activity was observed, measured by ppFEV1 and LCI2.5, with follow-up through one year at the selected Phase 2 dose [4] - The trial enrolled 16 participants with CF lung disease who were ineligible for or intolerant of CFTR modulator therapy, with follow-up durations ranging from 4 months to 3.5 years [5] Safety and Efficacy - No new pulmonary or safety events were reported in higher-dose cohorts, and adverse events in lower-dose cohorts were generally mild and transient [5] - Airway biopsy results showed consistent CFTR transgene RNA levels at or above physiologically relevant levels, with the 2.5E14 vg dose cohort meeting target expression profiles [5] Future Developments - The company plans to complete enrollment of the AEROW Phase 2 Dose-Expansion cohort in the first half of 2026 and provide program updates in the second half of 2026 [11] - 4D-710 is positioned as a durable, redosable, and variant-agnostic genetic medicine that could serve as a foundational therapy for cystic fibrosis [8] Company Overview - 4D Molecular Therapeutics is a late-stage biotechnology company focused on developing durable and disease-targeted therapeutics, with 4D-710 being a key product candidate aimed at addressing the underlying causes of cystic fibrosis [9]