Therapy Areas: Hereditary Disorders
SpliSense commences first-in-human, Phase 1/2 clinical trial of SPL84 to treat cystic fibrosis
15 December 2022 -

SpliSense, an Israel-based biopharmaceutical company, announced on Wednesday that it has started a first-in-human, Phase 1/2 clinical trial of SPL84, the company's lead anti sense oligonucleotide (ASO) product to treat subjects with cystic fibrosis (CF) carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the transmembrane conductance regulator (CFTR) gene.

The Phase 1/2 placebo-controlled, double–blind, randomised study is to assess the safety, tolerability, pharmacokinetics, and preliminary efficacy of SPL84, and includes two parts. The first part is a single ascending dose study in healthy volunteers, while the second part is a multiple ascending dose study in patients with CF carrying the 3849 +10 Kb C->T mutation. The product will be administrated through inhalation in a weekly or every other week regimen.

Gili Hart, PhD, SpliSense chief executive officer, 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. This program is our first-in-human and will serve as proof of concept of our pulmonary platform for our additional promising programs, including muco-obstructive diseases and IPF, the latter of which is expected to enter the clinic next year.'



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