SpliSense Initiates Phase 1/2 Study of SPL84, RNA-Based Therapy, for the Treatment of Cystic Fibrosis
- Category: DNA RNA and Cells
- Published on Thursday, 15 December 2022 15:58
- Hits: 2612
JERUSALEM, Israel I December 14, 2022 I SpliSense, a biopharmaceutical company developing transformative RNA-based therapies for pulmonary diseases including cystic fibrosis (CF), muco-obstructive diseases and idiopathic pulmonary fibrosis (IPF), today announced that it has initiated a first-in-human, Phase 1/2 clinical trial of SPL84, the Company's lead anti sense oligonucleotide (ASO) product for the treatment of patients with 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, randomized study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of SPL84 consists of two parts. The first part is a single ascending dose study in healthy volunteers and the second part is a multiple ascending dose study in subjects with CF carrying the 3849 +10 Kb C->T mutation. SPL84 will be administrated via inhalation in a weekly or every other week regimen.
"CF is a debilitating disease, leading to frequent lung infections, breathing difficulties and reduced life expectancy," said Gili Hart, PhD, Chief Executive Officer, SpliSense. "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," added Dr. Hart.
CF is a genetic multisystem disorder that originates from various mutations in the CFTR gene, which is responsible for the production of the CFTR protein, a chloride channel expressed in the lungs as well as in other tissues. The past decade has seen a dramatic change in CF care with the approval of new therapies (CFTR modulators). However, approved CFTR modulators do not support all patients with CF and do not offer a cure for the disease. Thus, new strategies of medication development are essential to address non-responsive CF patients, and specifically patients carrying the 3849 +10 Kb C->T splicing mutation.
SpliSense utilizes short, precisely targeted proprietary RNA stretches called ASOs to correct various mutations in the CFTR mRNA. In particular, the ASO binds specifically to the mutated CFTR RNA in the targeted sequence, leading to the modulation of the mutated region in the mRNA, allowing the cell to produce fully functional CFTR proteins. The ASOs are administered directly and preferentially to the lungs via inhalation where they are taken up by the lung cells and drive the production of corrected CFTR mRNA and eventually fully functional CFTR proteins.
SpliSense is a biopharmaceutical company focused on RNA-based treatments for pulmonary diseases. The Company's pioneering platform harnesses ASOs for treatment of unmet cystic fibrosis mutations and large pulmonary diseases including muco-obstructive diseases and IPF. Through its novel ASO pulmonary approach, SpliSense aims to overcome specific genetic mutations, by restoring or reducing protein function, thus targeting the root cause of the disease. 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. Investors in the Company include Orbimed, Israel Biotech Fund, Biotel Limited, Integra Holdings and the Cystic Fibrosis Foundation.
For additional information, please visit our website at SpliSense.com.