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Investigational Gene Therapy for Adults with CF

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Trial Objectives

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 Contact

For more information, contact:

Samantha Johnson

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Trial Location

National Jewish Main Campus, Denver, CO

Trial Sponsors

4D Molecular Therapeutics, Inc.

Principal Investigators

Jennifer L. Taylor-Cousar

Jennifer L. Taylor-Cousar, MD, MSCS


  • Jane E. Gross

    Jane E. Gross, MD, PhD

  • Jerry A. Nick

    Jerry A. Nick, MD

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