Preclinical data on TAVT-135, a cell-penetrating peptide conjugate intended to restore chloride ion balance in patients with cystic fibrosis (CF), are promising. Tavanta Therapeutics will present the data at the Cystic Fibrosis Foundation’s North American Cystic Fibrosis Conference, being held November 3-5, 2022, in Philadelphia, Pennsylvania.
“While CF transmembrane conductance regulator (CFTR) modulators have revolutionized the treatment of CF, a significant unmet need remains for patients with ineligible genotypes, inadequate responses, or intolerance,” the company said in a press release.“Therefore, new alternative agents, including those capable of mediating chloride ion transport, are needed.”
Cystic fibrosis is a hereditary, progressive disease in which secretions of various exocrine glands in organs are thickened due to impaired chloride transport to the cell surface, resulting in an accumulation of mucus. The thickening and accumulation of mucus in the lungs of patients with CF lead to progressive lung function deterioration, and agents that can restore chloride ion levels in the airways can slow this process.
A limitation of the revolutionary CFTR modulator treatments, which have significantly improved the prognosis of people with CF, is that they are not effective in patients with ineligible genotypes. Therefore, chloride-mediating agents that are effective regardless of CFTR mutations are an attractive alternative.
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The authors conducted a series of in vitro and ex vivo studies to evaluate the electrophysical and mucus-penetrating characteristics of TAVT-135. The in vitro data suggest that TAVT-135 readily facilitates the passive transport of chloride ions and penetrates mucus in a sustained manner. This, in turn, could increase electrolyte levels in the liquid layer of airway surfaces in patients, thereby speeding water transport out of epithelial cells and reducing mucus thickness and accumulation.
Further investigation of this new, artificial chloride ion transporter is ongoing.
Tavanta Therapeutics to present preclinical data for TAVT-135 at the North American Cystic Fibrosis Conference. News release. Tavanta Therapeutics; October 28, 2022.