Tevard Biosciences Demonstrates Breakthrough Protein Restoration in Genetic Muscle and Heart Diseases
Tevard Biosciences presented preclinical data showing its tRNA-based therapy can restore 70% of functional proteins in Duchenne muscular dystrophy and dilated cardiomyopathy models, potentially enabling clinical advancement for genetic diseases affecting millions worldwide.
Tevard Biosciences, Inc. announced new preclinical data demonstrating potent 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, showed 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.
Chief Scientific Officer Elisabeth Gardiner, PhD, emphasized the significance of restoring full-length native proteins for structural proteins like dystrophin and titin, where proper folding and protein interactions are essential. The platform uses native cellular machinery to produce natural proteins that function as the body expects, representing a fundamental shift in genetic disease treatment approaches.
In the DMD program, AAV-delivered suppressor tRNAs targeting Gln-TAA and Arg-TGA nonsense mutations restored 70% of full-length wild-type dystrophin levels in vivo, with strong correlation to motor function recovery and normalization of proteomic biomarkers. The DCM-TTNtv program showed suptRNA treatment restored full-length titin protein expression and contractility in iPSC-derived human cardiomyocytes within four days, with robust full-length titin production and restored proteomic homeostasis in the heart within six weeks in mouse models.
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 disclosure of results from Tevard's DCM-TTNtv program, one of its lead development efforts.
Tevard's platform has evolved from Gen 1 molecules to Gen 3 candidates optimized through high-throughput screening of over 80,000 variants. These engineered suppressor tRNAs achieve efficient codon-specific readthrough of premature stop codons that underlie 10-40% of all genetic diseases. The company's technology addresses a critical gap in genetic medicine by enabling restoration of full-length functional proteins rather than partial or modified versions. Both the DMD and DCM programs are advancing toward development candidate nomination in Q1 2026, positioning the company to address multiple genetic disorders through its versatile platform. For more information about the company's approach to genetic disease treatment, visit https://www.tevard.com.