TMPRSS6
GalNac Conjugated siRNA Targeting TMPRSS6 for Polycythemia Vera (PV)
- VIAL-TMPRSS6 siRNA designed to knock down TMPRSS6.
- Entering IND-enabling studies in Q3 2025.
- 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.
