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
Discovery
DC / IND-Enabling
Phase 1
Phase 2
Mechanism:
NBD1
SION‑638
SION‑719
SION‑451
Additional Candidates
Mechanism:
ICL4
SION-109
Mechanism:
TMD1
Galicaftor* SION-2222
SION-2851*
Additional Candidate
Mechanism:
TMD2 (Potentiator)
Navocaftor* SION-3067
*Licensed compounds from AbbVie: Galicaftor (FKA ABBV-2222), SION-2851 (FKA ABBV-2851), and Navocaftor (FKA ABBV-3067)
DC – Development Candidate, ICL4 – Intracellular Loop 4 of CFTR, IND – Investigational New Drug application, NBD1 – Nucleotide Binding Domain 1 of CFTR, TMD1 – Transmembrane Domain 1 of CFTR.