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TTN suppressor tRNA program
- Development stage
- Preclinical
- Lead developer
- Tevard Biosciences
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
Tevard Biosciences is developing a suppressor tRNA-based gene therapy for the treatment of dilated cardiomyopathy (DCM) caused by nonsense mutations in the titin (*TTN*) gene. The therapy utilizes an adeno-associated virus (AAV) vector to deliver engineered suppressor transfer RNAs (tRNAs) that specifically recognize and 'read through' premature termination codons (PTCs) within the *TTN* mRNA. By suppressing these nonsense mutations, the therapy restores the translation of full-length, functional titin protein, which is essential for the structural integrity and contractile function of the cardiac sarcomere. As truncating mutations in *TTN* are the most common genetic cause of DCM, this approach aims to provide a disease-modifying treatment for a significant patient population currently lacking targeted therapies.
- Other names
- TTN suppressor tRNA programTevard TTN ProgramTevard TTN gene therapy
02
Targets
TTN (Titin)
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