Type of information: Series B financing round
Company: Splisense (IL)
Investors: Orbimed (IL) Israel Biotech Fund (IL) Biotel Limited (USA) Integra Holdings (IL) the Cystic Fibrosis Foundation (USA)
Amount: $28.5 million
Funding type: series B financing round
Planned used: Proceeds will be used to advance Splisense’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. 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’ 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.
Others: SpliSense, a biopharmaceutical company developing transformative mRNA-altering therapies for cystic fibrosis and other genetic pulmonary diseases, 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.
Therapeutic area: Rare diseases - Genetic diseases - Pulmonary diseases