Jennifer Taylor-Cousar, MD, MSCS, on Early Signals of Efficacy With Cystic Fibrosis Gene Therapy

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The professor of internal medicine and pediatrics at National Jewish Health discussed updated data from the phase 1/2 AEROW trial of 4D-710.

“All 3 doses showed good expression in the airway. In those cohorts that were dosed at the lower doses, we also showed that people tolerated the drug, and that we saw at least at the 1x1015 vector genome dose, at least 1 person who had a clinically meaningful improvement in their percent predicted lung function, so we think that's very promising for looking at that dose or other lower doses in further participants.”

4D-710, 4D Molecular Therapeutics’ (4DMT) gene therapy candidate for treating cystic fibrosis, showcased CFTR transgene RNA and protein expression in all 10 participants treated at the higher and lower dose levels with follow-up up to 12 months in the phase 1/2 AEROW clinical trial (NCT05248230).1 The data come a couple of months after 4DMT shared that it had aligned on a pivotal phase 3 trial and registration path for the 4D-710 therapy.

Data from the trial were presented at the 47th European Cystic Fibrosis Conference, held in Glasgow, United Kingdom, in June 2024, by lead Principal Investigator Jennifer L. Taylor-Cousar, MD, MSCS, Professor, Departments of Medicine and Pediatrics, and codirector, Adult Cystic Fibrosis Program, and Director, Cystic Fibrosis Foundation Therapeutics Development Center, National Jewish Health.

CGTLive spoke with Taylor-Cousar to learn more about the progress of the AEROW trial, the new data, and next steps with the trial. She noted the higher-than-expected expression profile of the 2x1015 vg cohort and shared that the 1x1015 vg dose has been selected going forward. She shared cautious optimism for the therapy’s efficacy, having seen at least 1 participant experience clinically meaningful improvements in lung function.

REFERENCES
1. 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. News release. 4DMT. June 6, 2024. https://www.cgtlive.com/view/4dmt-aligns-path-forward-cystic-fibrosis-gene-therapy-4d-710
2. 4DMT Announces Update on Regulatory Interactions and Development Path for 4D-710 for Treatment of Cystic Fibrosis. News release. 4DMT. March 28, 2024. https://ir.4dmoleculartherapeutics.com/news-releases/news-release-details/4dmt-announces-update-regulatory-interactions-and-development
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