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BEBT-507
- Development stage
- Phase 1
- Lead developer
- BeBetter Med
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long 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
BEBT-507 is a **GalNAc-conjugated small interfering RNA (siRNA) therapeutic** developed by BeBetter Med. It targets **TMPRSS6 (Transmembrane Protease Serine 6, also known as matriptase-2)**, a serine protease expressed in the liver that negatively regulates the iron-regulatory hormone **hepcidin**. By silencing TMPRSS6 expression, BEBT-507 increases hepcidin levels, thereby reducing iron absorption and mobilization—a strategy intended to treat iron overload disorders such as **polycythemia vera** and **hemochromatosis**, as well as other iron metabolism disorders like β-thalassemia. It is the first RNA interference (RNAi) therapy from BeBetter Med to reach clinical development. As of mid-2025, BEBT-507 is in **Phase I clinical trials** in China for polycythemia vera and hemochromatosis, with administration by subcutaneous injection. The drug is not an orphan drug and is classified as a new molecular entity[1][2][3][5][7].
- Brand names
- BEBT-507BEBT507BEBT 507
- Other names
- BEBT-507BEBT507BEBT 507
02
Targets
TMPRSS6 (Transmembrane Serine Protease 6)
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