Cystic Fibrosis Foundation Helps a Biotech Firm

A Jerusalem-based SpliSense took the wraps off a Series B round to bring its antisense oligonucleotide therapy into the clinic. The round includes up to $8.4 million from the Cystic Fibrosis Foundation, bringing SpliSense’s total raise to just over $30 million.

William Skach, Cystic Fibrosis Foundation executive VP and CSO said that it is an intriguing technology that the company hasn’t invested in with other companies and groups, so we thought this would be a very unique prospect. The Cystic Fibrosis Foundation was awarded $400,000 to get SpliSense going back in 2017, and Skach said it contributed again because it was very pleased with early results.

Cystic Fibrosis Foundation is caused by mutations in the CFTR gene, which is responsible for shuttling chloride into and out of cells. It is a weakening disease that leads to frequent lung infections, breathing difficulties, and a shorter lifespan. SpliSense’s approach uses ASOs to correct mutations in CFTR mRNA, thus driving the cell to produce functional CFTR proteins.

Gili Hart, CEO said that the technology addresses the underlying genetic cause, thereby offering, for the first time, the hope of restoring adequate lung function to CF patients. She was brought in about a year and a half ago to help Kerem, founding CSO, build the company from scratch.

The company’s preclinical candidate, SPL84-23, is designed to correct the 3849+10kb C->T CFTR mutation and is headed for a Phase I/IIa trial in 2022. The goal is to treat a small portion of patients with rare mutations who don’t benefit enough or at all from Vertex’s already approved CFTR modulators. Translate Bio has its inhalable mRNA candidate but faced a setback in March when it reported that the drug had virtually no effect on patients lung function.