Breathing new hope: inhaled gene therapy trial for cystic fibrosis begins

NCT ID NCT05504837

Recruiting now Disease control Sponsor: Krystal Biotech, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage study tests a new gene therapy called KB407, given as a mist through inhalation, in 20 adults with cystic fibrosis. The goal is to see if it is safe and can improve lung function by delivering a working copy of the CF gene to lung cells. Participants will receive increasing doses to find the best balance of safety and effect.

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Conditions

The condition(s) this trial relates to.

cystic fibrosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CYSTIC FIBROSIS

Contacts and locations

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Locations

Atrium Health Wake Forest Baptist Medical Center
RECRUITING

Winston-Salem, North Carolina, 27157, United States
New York Medical College/ Boston Children's Health Physicians
RECRUITING

Hawthorne, New York, 10532, United States
Northwell Health Physicians
RECRUITING

New York, New York, 10028, United States
St. Lukes CF Center of Idaho
RECRUITING

Boise, Idaho, 83712, United States
The Cystic Fibrosis Institute
RECRUITING

Northfield, Illinois, 60093, United States

Contact Email: •••••@•••••
University of Florida, Gainesville
RECRUITING

Gainesville, Florida, 32610, United States

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