Israeli biotech startup SpliSense, a pre-clinical developer of mRNA-altering therapies for cystic fibrosis (CF) and other genetic pulmonary diseases, has announced the closure of a $28.5 million Series B funding round with investors that include Orbimed, the Israel Biotech Fund, Biotel Limited, Integra Holdings, a major shareholder, and the US Cystic Fibrosis Foundation.
Founded in 2016, SpliSense created a platform that harnesses Anti Sense Oligonucleotides (ASO), small pieces of DNA or RNA that can bind to specific molecules of RNA, for the treatment of genetic diseases such as CF, a genetic disease that leads to respiratory infections and disabilities. CF is caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene, leading to dysfunctional CFTR proteins.
The company says it hopes to initiate a Phase 1/2a trial in 2022 for its ASO product, SPL84-23 which is designed to correct various mutations in the CFTR mRNA. The proprietary RNA stretches, or ASOs, are administered directly and preferentially to the lungs via inhalation where it is taken up by the lung cells and drive the production of corrected CFTR mRNA and eventually functional CFTR proteins, the company explained in a statement.
SpliSense’ technology is based on the research of Professor Batsheva Kerem, a renowned geneticist from the Hebrew University of Jerusalem, who was part of the research team that identified and cloned the CFTR gene. The technology was licensed from Yissum, the technology transfer office of Hebrew University.
“This successful funding round is a vote of confidence in our platform technology and early-stage therapeutic pipeline,” said Gili Hart, PhD, Executive Officer, SpliSense. “CF is a debilitating disease, leading to frequent lung infections, breathing difficulties, and reduced life expectancy. Currently available treatments focus on treating the symptoms of the disease, such as channel function and protein modulators, antibiotics to treat lung infections, or mucus thinning drugs. However, they do not treat the root cause of the disease. Our technology addresses the underlying genetic cause, thereby offering, for the first time, hope of restoring adequate lung function to CF patients.”
Dr. Nissim Darvish, MD, and chairman of the Board at SpliSense said, “SpliSense couples Prof. Kerem’s pioneering research on the genetic underpinnings of CF together with a strong track record in developing ASO therapies and inhaled treatments.”
“We have great faith in SpliSense’s transformative technology for the genetic treatment of CF and await with interest the advancement of its pipeline into the clinic, as well as its expansion to additional indications,” he added.
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