Sionna Therapeutics, a life sciences company focused on advancing treatments for cystic fibrosis (CF), has initiated a Phase 1 clinical trial for SION-109 after receiving approval for its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA). The Phase 1 trial has successfully dosed its first healthy subject.

SION-109 is a small molecule specifically designed to target the interaction between the intracellular loop 4 (ICL4) region and the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This approach complements the company's existing portfolio of compounds directly targeting NBD1, including SION-638, a first-in-class NBD1 stabilizer currently in Phase 1.

Mike Cloonan, President and Chief Executive Officer of Sionna, highlighted the company's innovative strategy in targeting NBD1 correction. With SION-109 entering clinical development, there is an opportunity to explore proprietary combination treatments that could potentially achieve full CFTR correction. Cloonan acknowledged the dedication of the research and development teams, emphasizing their expertise in CF and commitment to advancing these programs swiftly to provide new options for individuals with CF and their families.

Cystic fibrosis is caused by genetic mutations in the CFTR protein, a crucial epithelial ion channel responsible for generating healthy, unobstructed mucus in various organs, including the airways and digestive system. The most prevalent mutation, ΔF508, leads to the unfolding of NBD1 at body temperature, severely compromising CFTR function. The inclusion of complementary targets such as ICL4 opens up the possibility of combination treatments with the potential for achieving complete CFTR correction. Approximately 90 percent of individuals with CF carry the ΔF508 genetic mutation.

 

Source: prnewswire.com

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