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TT-034
- Development stage
- Discontinued
- Lead developer
- Benitec Biopharma
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, 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, Recombinant Proteins and Enzymes, Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
TT-034 is an investigational gene therapy developed for the treatment of chronic hepatitis C virus (HCV) infection. It utilizes a recombinant adeno-associated virus serotype 8 (AAV8) vector to deliver three short hairpin RNA (shRNA) sequences into hepatocytes. These shRNAs are designed to target and cleave highly conserved regions of the HCV RNA genome via RNA interference, thereby inhibiting viral replication. The therapy is administered as a single intravenous infusion and aims to provide long-lasting suppression or potential cure of HCV with one dose. Once inside liver cells, TT-034 continuously expresses the anti-HCV shRNAs for the lifetime of those cells[1][5][6][7]. The drug was developed by Benitec Biopharma using its ddRNAi platform technology and was tested in Phase 1/2 clinical trials but development was discontinued after demonstrating safety and proof-of-concept[3][5].
- Other names
- AAV8-shRNA-HCVAAV-8-shRNA-HCVAAV 8-shRNA-HCV
02
Targets
HCV RNA (Hepatitis C virus RNA)
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