RARE Daily

CF Foundation Invests Up to $8.5 Million in SpliSense

April 3, 2024

Rare Daily Staff

The Cystic Fibrosis Foundation said it is investing up to $8.5 million in additional funds in SpliSense to continue clinical trials for its inhaled antisense oligonucleotide therapy for people with rare pulmonary disease cystic fibrosis who have certain splicing 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 therapy in people with the splicing mutation 3849+10Kb C-to-T.

A recent phase 1a study indicated the drug was safe and well tolerated.

Splicing mutations disrupt the production of normal cystic fibrosis transmembrane conductance regulator (CFTR) RNA, resulting in dysfunctional CFTR proteins. SpliSense’s drug would work by binding to the RNA molecule and correcting the instructions to create healthy, full-length CFTR proteins, helping restore the proper balance of salt and fluids in the lungs of people with CF.

The objective of the clinical trial is to generate a proof-of-concept for ASO technology in cystic fibrosis. If the clinical trial is successful, it could validate other similar therapeutic approaches in SpliSense’s pipeline that address rare CF mutations.

“We continue to pursue diverse strategies to develop potential treatments for people with CF who can’t benefit from existing modulator therapies,” said Steven Rowe, executive vice president and chief scientific officer at the Cystic Fibrosis 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.”

In 2021, the Foundation invested $8.4 million in SpliSense to develop a therapy for splicing mutations, in addition to $400,000 the Foundation provided in 2017. All these investments are part of the Foundation’s $500 million Path to a Cure, an ambitious research initiative to accelerate treatments for everyone with CF and ultimately deliver a cure.

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