4D Molecular Therapeutics disclosed encouraging interim data from its 4D‑710 gene‑therapy candidate in the Phase 1 AEROW trial for cystic fibrosis. The data, presented by investigators Jennifer L. Taylor‑Cousar and Felix Ratjen, show that the selected Phase 2 dose was well tolerated and achieved physiologically relevant levels of CFTR expression, with evidence of clinical benefit across multiple lung function and symptom measures.
The AEROW trial delivers the CFTR transgene via an aerosolized AAV vector and is the first known genetic medicine to demonstrate successful delivery and expression of CFTR throughout the lungs of people with cystic fibrosis. The interim update, based on a data cutoff of December 1, 2025, supports the program’s potential to provide a durable, variant‑agnostic therapy for the subset of CF patients who do not respond to existing modulators.
CEO David Kirn said the positive data reinforce the company’s strategy to advance 4D‑710 toward a Phase 2 study and ultimately a regulatory filing. The company plans to share additional updates in the second half of 2026 as the trial progresses toward pivotal milestones.
The announcement comes amid growing interest in gene‑therapy approaches for cystic fibrosis, with AAV vectors offering a low‑immunogenicity platform and the potential for sustained gene expression. 4D‑710’s ability to achieve physiologically relevant CFTR expression and early signs of clinical benefit positions it as a promising candidate to address the unmet need of patients who are ineligible for or intolerant of current CFTR modulators.
The data reinforce 4D‑710’s competitive positioning and support the company’s broader Therapeutic Vector Evolution platform, which aims to create targeted gene delivery vehicles. Positive interim results may accelerate the company’s path to regulatory filing and enhance its valuation among investors focused on gene‑therapy.
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