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Gen4 suppressor tRNA adeno-associated virus
- Development stage
- Preclinical
- Lead developer
- Tevard Biosciences
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intramuscular, Intravenous
01
Overview
Gen4 suppressor tRNA adeno-associated virus is an investigational gene therapy developed by Tevard Biosciences in collaboration with Alnylam Pharmaceuticals for the treatment of Duchenne muscular dystrophy (DMD) caused by nonsense mutations. This fourth-generation (Gen4) platform utilizes engineered suppressor tRNAs (sup-tRNAs) delivered via muscle-tropic adeno-associated virus (AAV) vectors, such as MyoAAV2A. The mechanism involves the sup-tRNAs recognizing and enabling ribosomal readthrough of premature termination codons (PTCs), which allows for the translation of full-length, functional dystrophin protein rather than the truncated versions produced by current micro-dystrophin therapies. Preclinical data presented at ASGCT 2026 demonstrated that Gen4 sup-tRNAs achieved near-complete restoration of dystrophin in target muscles and significant improvements in motor function in the D2.mdx mouse model at clinically relevant doses.
- Other names
- Gen4 AAV-sup-tRNAGen-4 AAV-sup-tRNAGen 4 AAV-sup-tRNA
02
Targets
dC (Deoxycytidine)PTC (50S ribosomal peptidyl transferase center)
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