SP-101 is an inhaled adeno-associated virus (AAV) gene therapy intended to provide a new, correct copy of the CFTR gene to lung cells. This would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation.

Status

A Phase 1/2 study to test the safety and tolerability of SP-101 is currently underway. The study is for adults with CF who are not eligible for CFTR modulators or have stopped taking a modulator. Some people who are taking a CFTR modulator but have experienced a less than 5 percentage point improvement in lung function may also be eligible later in the study.

Sponsor

This program is sponsored by Spirovant Sciences and partially funded by the Cystic Fibrosis Foundation. It is being conducted through the Therapeutics Development Network.