TMPRSS6
GalNac Conjugated siRNA Targeting TMPRSS6 for Polythemia Vera (PV), Hereditary Hemochromatosis (HH) and hematologic indications as well as iron overload and anemia indications (β‑thalassemia, hereditary hemochromatosis) and other related iron‑loading anemias.
- VIAL-TMPRSS6 siRNA designed to knock down TMPRSS6.
- Completed IND-enabling studies.
- Phase I initiating in Q1 2026.
Key Data
Based on in vitro and in vivo studies, VIAL-INHBE effectively demonstrates functional potency and target-binding comparable to or better than clinical programs.
Genetically validated target
- TMPRSS6 loss of function is genetically validated to improve iron homeostasis.
- Reduced TMPRSS6 activity raises hepcidin, improving iron balance and protecting against overload.
- Loss-of-function variants in the TMPRSS6 gene are associated with low hemoglobin and iron deficiency anemia.
Key data highlights
- VIAL-TMPRSS6 is progressing to demonstrate an equal-or-better profile across potency, dosing frequency, and safety compared to other assets in development for PV.
- VIAL-TMPRSS6 demonstrated superior stability in NHP liver lysates (S9 fractions) suggesting improved knockdown durability and the potential for less frequent dosing.
