Jerusalem, Israel – May 13, 2021 – SpliSense, a biopharmaceutical company developing transformative mRNA-altering therapies for cystic fibrosis (CF) and other genetic pulmonary diseases, today announced that it has closed a $28.5 million Series B funding round. Orbimed, Israel Biotech Fund, Biotel Limited, Integra Holdings and the Cystic Fibrosis Foundation participated in the round.
Proceeds will be used to advance the Company’s pipeline, including its lead Anti Sense Oligonucleotide (ASO) product, SPL84-23. SPL84-23, designed to treat the 3849+10kb C->T CFTR mutation, has demonstrated that it is able to completely restore Cystic Fibrosis Transmembrane conductance Regulator (CFTR) channel function in patient-derived cell cultures. The Company plans to initiate a Phase 1/2a trial in 2022.
CF is a genetic multisystem disorder that originates from various mutations in the CFTR gene, which is responsible for the production of the CFTR protein, a chloride channel expressed, among other systems, in the lungs. SpliSense utilizes short, precisely targeted proprietary RNA stretches called ASOs to correct various mutations in the CFTR mRNA. In particular, the ASO binds to the mutated CFTR RNA in the desired spot, leading to the elimination of the mutated region from the mRNA and allowing the cell to produce functional CFTR proteins. The 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. SpliSense utilizes proprietary algorithms to support the design of optimized ASOs, thereby maximizing efficiency and reducing the potential for undesired effects.
SpliSense’ technology is based on the research of Prof. Batsheva Kerem, PhD, 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 of 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. We look forward to using this funding and the support of the Cystic Fibrosis Foundation to advance our pipeline for the benefit of CF patients.”
Dr. Nissim Darvish, MD, Chairman of the Board, SpliSense, “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.”
About Cystic Fibrosis
Cystic fibrosis (CF) is a genetic disease that leads to respiratory infections and disabilities and affects over 90,000 people worldwide. It is caused by mutations in the CFTR gene, leading to dysfunctional CFTR proteins. Disease-causing CFTR protein variants create irregularities in the transport of ions, generating mucus obstructions, chronic infection, inflammation and even respiratory failure.
Over 2000 variants in the CFTR gene have been identified, and over 400 are known to cause disease. The past decade has seen a dramatic change in CF care. However, approved CFTR modulators do not support all patients with CF or offer a cure for the disease. Thus, new strategies of medication development are essential to address non-responsive mutations.
SpliSense is a biopharmaceutical company focused on transformative mRNA-altering treatments targeting unmet needs in genetic pulmonary diseases. The Company’s pioneering platform harnesses ASOs for treatment of genetic diseases such as CF. SpliSense’ pipeline includes innovative therapies in various development stages, from discovery to IND enabling studies. For additional information, please visit our website at www.splisense.com.
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