Tevard Biosciences, Inc., a privately held biotechnology company focused on tRNA-based therapies for genetic diseases, presented new preclinical data at the 2025 Federation of European Biochemical Societies (FEBS) Special Meeting in Dubrovnik, Croatia, demonstrating potent restoration of full-length functional proteins in models of Duchenne muscular dystrophy (DMD) and dilated cardiomyopathy caused by titin truncations (DCM-TTNtv). The findings, announced on September 29, 2025, highlight the potential of Tevard's engineered suppressor tRNAs (suptRNAs) to address the underlying genetic causes of these conditions.
The data, presented by Elisabeth Gardiner, PhD, Chief Scientific Officer, showed that AAV-delivered suptRNAs targeting Gln-TAA and Arg-TGA nonsense mutations restored on average 70% of full-length wild-type dystrophin protein levels in DMD models. This restoration correlated strongly with 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. In vivo, AAV-delivered Arg-TGA suptRNAs drove robust full-length titin production and restored proteomic homeostasis in the heart within six weeks in a TTNtv mouse model.
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, indicating durability of the therapeutic effect after a single intravenous dose. These results mark the first disclosure of data from Tevard's DCM-TTNtv program, underscoring the versatility of its suppressor tRNA platform across genetically distinct indications.
“These results show that our engineered suppressor tRNAs are capable of restoring full-length, native protein expression at levels that are not only biologically meaningful but clinically promising,” said Dr. Gardiner. “When dealing with structural proteins like dystrophin and titin, where proper folding, localization, and protein-protein interactions are essential, restoring a full-length protein makes all the difference.”
Tevard's suppressor tRNA platform has evolved from Gen 1 molecules 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 premature stop codons (UGA, UAA, UAG) that underlie 10–40% of all genetic diseases. Both the DMD and DCM programs are advancing toward development candidate nomination in Q1 2026.
“Being selected for a featured oral presentation and poster session at this world-class conference highlights the impact of our suppressor tRNA platform,” said Daniel Fischer, Co-Founder, President and CEO of Tevard Biosciences. “Recent breakthroughs in suppressor tRNA and vector design have achieved the levels of protein rescue needed to confidently advance our DMD and DCM-TTNtv programs into the clinic at safe, efficacious doses.”
For more information about Tevard Biosciences, visit www.tevard.com.
