CF Foundation backs a new round for a biotech upstart out to rival the mighty Vertex
As a postdoc fellow in 1989, Batsheva Kerem was part of the dogged team that worked with current NIH director Francis Collins to identify the faulty gene behind cystic fibrosis. Now, more than three decades later, she’s leading one company’s efforts to alter a key mutation in that gene — and she just got $28.5 million to do it.
On Thursday morning, Jerusalem-based SpliSense took the wraps off a Series B round to bring its antisense oligonucleotide (ASO) 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.
“It’s an intriguing technology that we really haven’t invested in with other companies and groups, so we thought this would be a very unique prospect,” CF Foundation executive VP and CSO William Skach told Endpoints News. The CF Foundation awarded $400,000 to get SpliSense going back in 2017, and Skach said it contributed again because it was “very pleased” with early results.
CF is caused by mutations in the CFTR gene, which is responsible for shuttling chloride into and out of cells. It’s a debilitating disease that leads to frequent lung infections, breathing difficulties, and shorter lifespan. SpliSense’s approach uses ASOs (short sequences of RNA administered by inhalation) to correct mutations in CFTR mRNA, thus driving the cell to produce functional CFTR proteins.
“Our technology addresses the underlying genetic cause, thereby offering, for the first time, hope of restoring adequate lung function to CF patients,” CEO Gili Hart said in a statement. She was brought in about a year and a half ago to help Kerem, founding CSO, build the company.
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 the small portion of patients with rare mutations who don’t benefit enough or at all from Vertex’s already approved CFTR modulators.
“The program we’re funding right now is looking at a mutation which is partially responsive to Vertex drugs, but we still think there could be more response, and a better response for people that could carry those mutations,” Skach said.
There are a couple other RNA-focused biotechs in the CF space, including Arrowhead, which is using RNA interference as opposed to antisense. Ionis scrapped its own early-stage effort this week, after a long-term toxicology in animals turned up concerning results. Translate Bio has its own inhalable mRNA candidate, but faced a setback in March when it reported that the drug had virtually no effect on patients’ lung function.
“ASO is very stable … within the body, within the cells, so you can give it less frequently. It’s small, it can easily penetrate,” Hart told Endpoints. “We believe our technology is very clean, simple and unique.”