Tevard Biosciences announced the presentation of new preclinical data showing potent restoration of full-length functional proteins in models of Duchenne muscular dystrophy (DMD) and dilated cardiomyopathy (DCM-TTNtv). Importantly, the findings include data showing on average 70% restoration of wild-type dystrophin protein in DMD models with the latest generation of suppressor tRNAs (suptRNA), supporting the potential for meaningful clinical outcomes at lower doses.
The data were presented by Elisabeth Gardiner, PhD, Chief Scientific Officer of Tevard Biosciences, 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.” The presentations took place at the 2025 Federation of European Biochemical Societies (FEBS) Special Meeting “Expanding Frontiers in Aminoacyl-tRNA Synthetase Research” in Dubrovnik, Croatia, being held September 28 to October 3, 2025.
“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. Our platform uses native cellular machinery to produce natural proteins that function the way the body expects.”
Key Findings Presented
- 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. 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, 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.
“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.”
Read More: Clinical Trials for Duchenne (List of All Researches)
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