Israeli company wins $ 28.5 million for restorative cystic fibrosis treatment

Jerusalem-based SpliSense, with a focus on cystic fibrosis and other hereditary lung diseases Closed In a $ 28.5 million Series B funding round.Participated in the round OrbiMed, Israel Biotech Fund, Integra Holdings, and Cystic Fibrosis Foundation..

The company has a pipeline containing SPL84-23, an antisense oligonucleotide (ASO) product designed to fully restore cystic fibrosis transmembrane conductance regulator (CFTR) channel function in patient-derived cell cultures. We plan to use the funds to move forward. SpliSense plans to begin Phase I / IIa trials in 2022.

Cystic fibrosis (CF) is caused by mutations in the CFTR gene, which encodes the CFTR protein, a chloride channel expressed in many systems, especially the lungs. CF can be life-threatening. The body produces thick, sticky mucus that can clog the lungs and occlude the pancreas. If the CFTR protein does not function properly, the chloride component of the salt cannot be transferred to the cell surface. Due to the lack of chloride, water is not attracted to the cell surface, resulting in a thick and sticky mucus.

In the lungs, this can lead to bacterial capture, leading to infections, inflammation and respiratory failure. In the pancreas, mucus buildup can block the bile ducts and cause liver disease.

A Detailed report More than 30,000 people in the United States live with the disease, according to a report in the CF market by BioSpace. There are some failures and program terminations, but there are also promising signs.

SpliSense’s technology is based on the work of Batsheva Kerem, a geneticist at the Hebrew University of Jerusalem. He was part of a research group that first identified and cloned the CFTR gene. SpliSense has licensed the technology from Yissum, the Hebrew University technology transfer office.

Their technique leverages a unique, short and accurately targeted RNA segment called ASO to correct mutations in CFTR mRNA. ASO binds to the mutant CFTRRNA at the appropriate location, eliminating the mutant region from the mRNA and allowing the cell to produce a functional CFTR protein.

“This successful funding round is a vote of confidence in our platform technology and early-stage treatment pipeline,” said Gili Hart, Executive Officer at SpliSense. “CF is a debilitating illness that leads to frequent lung infections, dyspnea, and reduced life expectancy. Currently available treatments include channel function, protein modulators, and antibiotics to treat lung infections. Focuses on treating the symptoms of the disease, such as substances, mucilage diluents, but they do not treat the root cause of the disease. Our technology addresses the root genetic cause. For the first time, it provides CF patients with the hope of restoring proper lung function. “

One Large investor In the Series B round, there was the Cystic Fibrosis Foundation based in Bethesda, Maryland. The foundation has invested $ 8.4 million in rounds as part of Path to a Cure, a research strategy to accelerate treatment of the root cause of CF.

The fund will support the company’s splicing mutation modification program in both preclinical development and ASO phase I / IIa trials in people with splicing mutations 3849 + 10KbC-to-T. They believe that if the trial is successful, it may validate other similar approaches in the SpliSense pipeline that focus on rare mutations in CF that do not respond to current therapies.

“This agreement will enable the search for synthetic antisense oligonucleotides as potential therapies for CF in the clinic, opening up exciting new opportunities for therapeutic development and as part of the path to treatment. The approach we are pursuing will be even more diverse, “says William Skach. Executive Vice President and Chief Scientific Officer of the CF Foundation. “Therefore, these studies provide important information for the future development of these potential therapies for additional people with rare mutations.”