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TT-033
- Development stage
- Discontinued
- Lead developer
- Benitec Biopharma
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies
- Administration
- Intravenous
01
Overview
TT-033 (also known as PF-0488818 or OBP-701) is a first-generation RNAi-based therapeutic developed for hepatitis C virus (HCV) infection. It consists of three distinct short hairpin RNA (shRNA) elements targeted directly against HCV, delivered via an adeno-associated virus (AAV) vector for hepatic expression. The mechanism involves RNAi-mediated silencing of HCV RNA in hepatocytes, demonstrating potent inhibition in HCV replicon models and efficient transduction in nonhuman primate livers. However, preclinical studies revealed dose-dependent hepatotoxicity, including elevated ALT levels and liver injury at doses ≥3.75 × 10^11 vg/kg, attributed to excessive shRNA expression from strong Pol III promoters, leading to its replacement by improved TT-034.[1][2][4][5]
- Other names
- ddRNAi-HCV
02
Targets
Hepatitis C virus genomic RNA internal ribosome entry site (IRES) site 3S1 (SARS-CoV-2 spike S1 subunit)
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