BETHESDA, Md.--(BUSINESS WIRE)--The Cystic Fibrosis Foundation is investing up to $8.5 million in additional funds in SpliSense to continue clinical trials for its inhaled antisense oligonucleotide (ASO) drug for people with cystic fibrosis who have certain splicing mutations and potentially other rare mutations.
The Foundation's funding will support a planned Phase 2 clinical trial to test the efficacy of SpliSense’s inhaled ASO drug as a potential treatment for the lungs of people with the splicing mutation 3849+10Kb C-to-T. A recent Phase 1a study indicated the drug was safe and well tolerated.
“We continue to pursue diverse strategies to develop potential treatments for people with CF who can’t benefit from existing modulator therapies,” said Steven M. Rowe, MD, executive vice president and chief scientific officer at the Foundation. “Information from this study is key to advancing those efforts with a novel technology and will also provide valuable insight into the development of therapies for people with rare mutations.”