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anti-Tmprss6 siRNA
- Development stage
- Unknown
- Lead developer
- Agios Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Anti-TMPRSS6 siRNA refers to a class of small interfering RNA (siRNA) therapeutics designed to target and silence the expression of transmembrane protease serine 6 (TMPRSS6, also known as matriptase-2) in the liver. TMPRSS6 is a negative regulator of hepcidin, the master hormone regulating systemic iron homeostasis, which it suppresses by cleaving the bone morphogenetic protein (BMP) co-receptor hemojuvelin (HJV). By degrading TMPRSS6 mRNA, these siRNA therapeutics promote BMP/SMAD signaling, leading to an upregulation of endogenous hepcidin. Elevated hepcidin levels restrict iron absorption and macrophage iron release, thereby limiting iron availability for erythropoiesis. This mechanism is being clinically investigated for the treatment of iron overload disorders, such as beta-thalassemia, and myeloproliferative neoplasms like polycythemia vera (PV), where restricting red blood cell production can reduce hematocrit levels and the need for therapeutic phlebotomy. Key candidates in this class include divesiran (SLN124) and AG-236 (ALN-TMP).
- Other names
- TMPRSS6 siRNATMPRSS-6 siRNATMPRSS 6 siRNAanti-TMPRSS6 siRNAanti-TMPRSS-6 siRNAanti-TMPRSS 6 siRNAmatriptase-2 siRNAmatriptase2 siRNAmatriptase 2 siRNA
02
Targets
TMPRSS6 (Transmembrane Serine Protease 6)
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