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GTB-MASH-siRNA
- Development stage
- Preclinical
- Lead developer
- Madrigal 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
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Overview
GTB-MASH-siRNA is an investigational small interfering RNA (siRNA) therapy being developed by GeneToBe for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). The drug utilizes RNA interference (RNAi) to reduce the expression of specific, though currently undisclosed, disease-relevant genes in the liver. By targeting the underlying genetic drivers of MASH, the therapy aims to mitigate liver inflammation and fibrosis. As of early 2024, the program is in the IND-enabling stage of development, focusing on leveraging GeneToBe's gene therapy and gene editing expertise to address chronic liver diseases.
- Other names
- Metabolic dysfunction-associated steatohepatitis programGeneToBe MASH siRNA program
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