4D Molecular Therapeutics has reported positive interim clinical data from the phase 1/2 AEROW study on aerosolized 4D-710 for treating cystic fibrosis (CF) lung disease.

The data, reported via news release, demonstrated meaningful improvements in CTFR expression and cystic fibrosis-related quality of life.

“The widespread CFTR transgene expression demonstrated in all lung samples from participants in cohort one is a critical first step for the development of 4D-710, especially given the signals of clinical activity in this CF population with the highest unmet medical need,” said David Kirn, MD, cofounder and chief executive officer of 4DMT. “These interim results also demonstrate the potential of our proprietary aerosolized A101 vector for other lung diseases such as alpha-1 antitrypsin deficiency lung disease and other highly prevalent lung diseases.”

The data reported are from cohort 1, which comprises 3 patients with CF who were ineligible for or intolerant of standard CFTR modulator therapy. They received a single aerosolized dose of 4D-710 administered via a nebulizer.

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4D-710 is composed of A101, a targeted and evolved vector, and a codon-optimized CFTR∆R transgene. This 4D-710 dose-exploration stage (1E15 and 2E15 vg doses) will be followed by a phase 2 dose expansion stage for up to 12 patients.

Biomarker analyses of the patients in cohort 1 revealed widespread and consistent CFTR DNA and RNA expression in all lung biopsy samples. CFTR protein expression was identified in all major airway epithelial cells, and the percentage of CFTR-positive cells was significantly higher than in control lung samples and in CF lung-derived samples.

Furthermore, the data revealed meaningfully improved quality of life scores and stable or improved percent predicted forced expiratory volume in one second in the 3 participants. The phase 2 dose expansion stage is expected to be launched in the second half of 2023.


4DMT presents positive interim data from aerosolized 4D-710 phase 1/2 AEROW clinical trial in patients with cystic fibrosis at the ECFS 46th annual meeting. News release. 4D Molecular Therapeutics, Inc.; June 7, 2023.