SpliSense is at the forefront of developing innovative RNA-based therapies, specifically utilizing Antisense Oligonucleotides (ASOs) to address genetic and pulmonary diseases. Their approach focuses on restoring and modifying protein functionality to tackle untreatable conditions. The company is currently in the clinical stage, with a pipeline of transformative treatments aimed at unmet medical needs in various pulmonary diseases. Notably, their ASO technology is delivered non-invasively through inhalation, allowing for targeted therapy directly to the lungs. This method is particularly significant for patients with specific genetic mutations, such as the 3849+10 kb C->T mutation associated with cystic fibrosis, where existing treatments have shown limited efficacy.

The SpliSense platform is built on four distinct modalities that facilitate the creation of specific ASOs tailored for pulmonary diseases. One of their key products, SPL84, is designed to address the challenges posed by the 3849 mutation by preventing the inclusion of a cryptic exon in the CFTR RNA, which leads to the production of non-functional proteins. By effectively binding to the target region in the lungs, SPL84 aims to generate fully functional CFTR proteins, thereby offering a promising solution for patients who have not responded well to current therapies. With successful completion of Phase 1 trials and plans for Phase 2 targeting unmet cystic fibrosis patients, SpliSense is poised to make significant advancements in the treatment of genetic pulmonary diseases.

Israel

SpliSense Ltd., is a growing and exciting biotechnology company, developing novel therapies for genetic diseases by inhalation. The company is currently focusing on splicing mutations in the CFTR gene for treating Cystic Fibrosis (CF) patients.

SpliSense

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