New preclinical data from Tevard Biosciences demonstrates compelling restoration of full-length functional proteins in models of Duchenne muscular dystrophy and dilated cardiomyopathy caused by titin truncations. The findings, presented at the 2025 Federation of European Biochemical Societies Special Meeting in Dubrovnik, Croatia, show on average 70% restoration of wild-type dystrophin protein in DMD models using the latest generation of suppressor tRNAs, supporting potential for meaningful clinical outcomes at lower doses.
Elisabeth Gardiner, PhD, Chief Scientific Officer of Tevard Biosciences, presented the data during an invited oral presentation and poster session titled "The Use of Therapeutic Suppressor tRNAs for the Treatment of Duchenne Muscular Dystrophy and Dilated Cardiomyopathy." Dr. Gardiner stated, "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. 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."
In the DMD program, AAV-delivered suppressor tRNAs 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, suppressor tRNA treatment restored full-length titin protein expression and contractility in iPSC-derived human cardiomyocytes within four days. In vivo studies showed AAV-delivered Arg-TGA suppressor tRNAs 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, suppressor tRNA 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, further highlighting the versatility and maturity of its suppressor tRNA platform across genetically distinct indications.
Daniel Fischer, Co-Founder, President and CEO of Tevard Biosciences, commented, "Being selected for a featured oral presentation and poster session at this world-class conference highlights the impact of our suppressor tRNA platform. 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."
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 suppressor tRNAs achieve efficient and codon-specific readthrough of the most common 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. For more information about the company's research, visit https://www.tevard.com.
