Israel-based SpliSense said that it has initiated a Phase 1/2 trial of its SPL84 inhaled anti sense oligonucleotide for the treatment of cystic fibrosis associated with the 3849+10 kb C-to-T mutation. SPL84 has been granted Orphan Drug designation from both the EMA and the FDA. The company said that the SAD portion of the trial will evaluate SPL84 in healthy volunteers; the MAD portion of the trial will enroll CF patients with the 3849+10 kb C-to-T mutation.
SpliSense CEO Gili Hart said, “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, while we address the underlying genetic cause of the disease, thereby offering hope of restoring the defective protein and generating adequate lung function in patients suffering from CF with the benefit of an easy-to-use and less frequent treatment approach. Specifically, CF patients carrying the 3849 +10 Kb C->T mutation have no specific approved treatment. Our lead product, SPL84, has shown to have completely restored CFTR activity in the CF gold standard pharmacological model, suggesting potential cure for these patients.”
Read the SpliSense press release.
