We’re developing novel small molecule therapies targeting the most common genetic mutation affecting cystic fibrosis (CF) patients, ΔF508, with the goal of normalizing CFTR function. Our programs targeting the NBD1, ICL4, and TMD1 regions of CFTR are complementary, and together they have the potential to achieve best in class efficacy by normalizing CFTR function.
Mechanism
Program
Preclinical
Phase 1
Phase 2
Mechanism:
NBD1
SION‑719
SION‑451
Mechanism:
TMD1
SION-2851
Galicaftor SION-2222
Mechanism:
ICL4
SION-109
Mechanism:
TMD2 (Potentiator)
Navocaftor SION-3067
ICL4 – Intracellular Loop 4 of CFTR, NBD1 – Nucleotide Binding Domain 1 of CFTR, TMD1 – Transmembrane Domain 1 of CFTR.