Sionna Therapeutics is developing small molecule therapies that target specific genetic mutations responsible for cystic fibrosis, aiming to enhance lung function and decrease the incidence of pulmonary exacerbations. These therapies focus on addressing the root causes of the disease rather than just its symptoms.
Funding
$182M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
ESFounders
Product
Problem
Cystic fibrosis (CF) results from mutations in the CFTR protein, most commonly the F508del mutation, leading to impaired chloride transport and causing severe lung disease and other complications. Current treatments often address the symptoms of CF without fully correcting the underlying protein defect, leaving room for improved therapies that normalize CFTR function.
Solution
Sionna Therapeutics is developing a pipeline of small molecule therapies designed to normalize CFTR function in patients with cystic fibrosis. Their programs target key domains of the CFTR protein, including NBD1, ICL4, and TMD1, to correct defects caused by the F508del mutation and other mutations. By directly targeting NBD1, Sionna aims to fully correct CFTR, addressing the root cause of the disease. These differentiated treatments have the potential to become best-in-class options for CF patients, setting a new standard of care.
Target Audience
The primary target audience includes cystic fibrosis patients with the F508del mutation and other CFTR mutations, as well as healthcare providers specializing in cystic fibrosis treatment.
Features
- Small molecule therapeutics targeting the NBD1 domain of the CFTR protein
- Programs designed to correct defects caused by the F508del mutation
- Complementary programs targeting ICL4 and TMD1 domains of CFTR
- Clinical-stage pipeline with ongoing Phase 1 clinical trials for SION-719 and SION-451