Spirovant Sciences Doses First Patient in Phase 1/2 Trial of SP-101 Gene Therapy for Cystic Fibrosis
核心洞察
Spirovant Sciences (搜索) has initiated dosing in a Phase 1/2 clinical trial (SAAVe) evaluating SP-101, an inhaled gene therapy for cystic fibrosis (搜索) (CF), in adults aged 18-65.
The SAAVe trial (NCT06526923) aims to enroll 15 CF patients with stable disease and a ppFEV1 between 50% and 100% to assess the safety and efficacy of SP-101.
SP-101 delivers a functional version of the _CFTR (搜索)_ gene to lung cells, potentially treating a range of CF patients, including those ineligible or insufficiently treated by CFTR modulators.
Spirovant Sciences (搜索) has announced the dosing of the first patient in its Phase 1/2 clinical trial, SAAVe (NCT06526923), which is evaluating SP-101, an inhaled gene therapy for the treatment of cystic fibrosis (搜索) (CF). The trial is currently recruiting participants aged 18 to 65 with stable CF and a percent predicted forced expiratory volume in one second (ppFEV1) between 50% and 100% at sites in Kansas City, Kansas, and New York City. Additional sites in Philadelphia and Boston are expected to join the study.
SAAVe Trial Details
The SAAVe trial aims to enroll 15 participants who will receive a single administration of SP-101 in combination with one of two doses of doxorubicin, a chemotherapy molecule intended to enhance the activity of the CF gene therapy candidate. The trial's design focuses on assessing the safety and potential efficacy of SP-101 in improving lung function and addressing the underlying genetic cause of CF.
Addressing Unmet Needs in CF Treatment
Cystic fibrosis (搜索) is caused by mutations in the CFTR (搜索) gene, leading to the production of thick, sticky mucus that damages organs and causes various disease symptoms. SP-101 is designed to deliver a functional version of the CFTR gene to lung cells, enabling them to produce a working CFTR protein. This approach is intended to be mutation-agnostic, potentially benefiting a broader range of CF patients, including those who are not eligible for or do not respond adequately to CFTR modulators, which address specific CF-causing mutations. Approximately 10% of people with CF cannot benefit from modulator therapy.
Expert Commentary
"The dosing of the first patient in the SAAVe Phase 1/2 clinical trial is a major milestone for Spirovant and for the cystic fibrosis (搜索) patient community," said Roland Kolbeck, PhD, chief scientific officer of Spirovant. Claire Keating, MD, a study investigator at Columbia University Irving Medical Center in New York, added that this trial is "advancing a novel potential treatment pathway that moves us towards our goal of effectively treating people with CF who currently are not eligible for other highly effective therapies."