Drug intelligence / Profile preview

tomligisiran (GSK)

Development stage
Unknown
Lead developer
GSK
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

Tomligisiran (GSK3965193) is an investigational small interfering RNA (siRNA) therapeutic developed by GSK for the treatment of chronic hepatitis B (CHB). It is designed to target and degrade hepatitis B virus (HBV) RNA transcripts through the RNA interference (RNAi) pathway, specifically aiming to silence the expression of all HBV proteins, including the hepatitis B surface antigen (HBsAg). By significantly reducing the viral antigen burden, tomligisiran is intended to alleviate the immune suppression typically caused by high levels of HBsAg, thereby potentially restoring the host's immune response and contributing to a functional cure. It is currently in Phase 2b clinical development, frequently evaluated as part of a fixed-dose combination with daplusiran (another siRNA) known as DAP/TOM (GSK4388067), and in sequential regimens with the antisense oligonucleotide bepirovirsen.

02

Targets

HBV (Hepatitis B virus)HBV X RNA (Hepatitis B virus X region RNA transcript)

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