Investigational Gene Therapy for Adults with CF
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.