Our proprietary portfolio includes NBD1 stabilizers and complementary modulators. We believe the synergistic approach of combining NBD1 stabilizers with complementary modulators provides the highest probability of normalizing CFTR function for CF patients. Our programs target the NBD1, ICL4, and TMD1 regions of CFTR.
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.