SpliSense has commenced a Phase I/II clinical trial of its lead anti sense oligonucleotide (ASO) product, SPL84, to treat patients with cystic fibrosis (CF) carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the transmembrane conductance regulator (CFTR) gene.
Comprising two portions, the first-in-human, double–blind, placebo-controlled, randomised trial has been designed to assess the tolerability, safety, pharmacokinetics, and preliminary efficacy of SPL84.
The first single ascending dose study part will be carried out in healthy volunteers and the second part is a multiple ascending dose trial in participants with CF carrying the 3849 +10 Kb C->T mutation.
As part of the trial, SPL84 will be given through inhalation in a weekly or every other week regimen.
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.
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“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.”
SpliSense corrects various mutations in the CFTR mRNA by using short, precisely targeted RNA stretches, known as ASOs, administered directly and preferentially to the lungs through inhalation.