New preclinical data from Tevard Biosciences, Inc. demonstrated potent restoration of full-length functional proteins in models of Duchenne muscular dystrophy and dilated cardiomyopathy caused by titin truncations. The findings showed on average 70% restoration of wild-type dystrophin protein in DMD models with the latest generation of suppressor tRNAs, supporting potential for meaningful clinical outcomes at lower doses. These results demonstrate that engineered suppressor tRNAs can restore full-length, native protein expression at levels that are biologically meaningful and clinically promising.
In the DMD program, AAV-delivered suptRNAs targeting Gln-TAA and Arg-TGA nonsense mutations restored on average 70% of full-length wild-type dystrophin levels in vivo, with strong correlation to motor function recovery and normalization of proteomic biomarkers. In the DCM-TTNtv program, suptRNA treatment restored full-length titin protein expression and contractility in iPSC-derived human cardiomyocytes within four days. Both programs demonstrated dose-dependent transduction, protein rescue, and functional improvement following systemic administration, with no detectable toxicity or off-target effects.
Notably, suptRNA expression and protein rescue were sustained up to 12 weeks post-treatment, highlighting the durability of the therapeutic effect following a single intravenous dose. These data mark the first time Tevard is disclosing results from its DCM-TTNtv program, one of its lead development efforts. Tevard's suppressor tRNA platform has evolved from Gen 1 molecules with a single anticodon edit to Gen 3 candidates optimized through high-throughput screening of over 80,000 variants.
These rationally engineered suptRNAs achieve efficient and codon-specific readthrough of the most common premature stop codons that underlie 10-40% of all genetic diseases. Both the DMD and DCM programs are advancing toward development candidate nomination in Q1 2026. The data were presented by Elisabeth Gardiner, PhD, Chief Scientific Officer of Tevard Biosciences, during an invited oral presentation and poster session at the 2025 Federation of European Biochemical Societies Special Meeting in Dubrovnik, Croatia. For more information about the company's research, visit https://www.tevard.com.
