This article is a summary of potential gene therapies for cystic fibrosis, written for non-specialists and the lay community.
Cystic fibrosis (CF) is a genetic disease that affects the lungs and other organs. New treatments, called CFTR modulator therapies, have recently become available for people with CF. However, not all people with CF are able to benefit from CFTR modulator therapies, so additional new treatments are urgently needed. Gene therapy is a potential treatment to address the unmet need in CF. This article summarises what CF is, and how gene therapy could be used to treat it. It also includes some information on gene therapies for CF that are currently being developed.
The purpose of this plain language summary is to help readers understand the findings from recent research. None of the potential treatments mentioned in this plain language summary (4D-710, SP-101, KB407 and BI 3720931) are yet approved to treat CF.
