Investigational Gene Therapy for Adults with CF
Trial Objectives
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This study is designed to test the safety of 4D-710, an investigational gene therapy drug for adults with cystic fibrosis (CF) who are unable to use CFTR modulators. Adeno-associated viruses (AAV) are small viruses that are commonly found in nature and are not known to cause disease in humans. 4D-710 is an AAV that was changed in the laboratory to deliver a copy of the CFTR gene to the lungs, thereby providing a copy of instructions for making normal CFTR protein. Participants will use a nebulizer to inhale 4D-710 into the lungs to see if 4D-710 is safe for use in adults with cystic fibrosis and to see if 4D-710 can help lung function.
Who Can Participate
Adults with CF who are not eligible for CFTR modulatory therapy or discontinued CFTR modulator therapy due to side effects.
Age: 18+ Gender: Any
Estimated Time Commitment
24 months
Time Commitment Details
Year 1: 10 scheduled in-person visits. Year 2: Long-term follow up, phone check in.
Payment & Reimbursement
Payment: Provided
Travel Reimbursement: Available
Trial Location
National Jewish Main Campus, Denver, CO
Trial Sponsors
4D Molecular Therapeutics, Inc.
Principal Investigators
Jennifer L. Taylor-Cousar, MD, MSCS
Co-Investigators
Jerry A. Nick, MD