New research suggests patients with Duchenne muscular dystrophy (DMD) could one day have access to a treatment capable of restoring nearly 70% of healthy dystrophin protein, a level linked to better muscle strength and function, according to a press release published recently by Tevard Biosciences.
Data presented by Tevard Biosciences show the company’s suppressor transfer RNA (tRNA) therapy may offer meaningful benefits by repairing genetic mistakes that cause the progressive disease.
These results were presented at the Federation of European Biochemical Societies Special Meeting in Dubrovnik, Croatia. In animal models, the treatment was able to restore full-length dystrophin (the protein that is missing or defective in people with DMD), and this improvement was tied to recovery of motor function. Importantly, the therapy did not show signs of toxicity or harmful off-target effects.
“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 Elisabeth Gardiner, Chief Scientific Officer of Tevard Biosciences, who presented the data.
Tevard’s approach aims to address nonsense mutations, which are responsible for 10% to 15% of cases of DMD, by using tRNA molecules to “read through” premature stop signals in the genetic code.
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In this study, Tevard researchers reported that their third-generation suppressor tRNA therapy produced robust and lasting protein restoration after a single intravenous dose in DMD models. Results showed sustained benefit for up to 12 weeks, suggesting durability of effect. In addition, similar strategies restored titin, another important muscle protein, in models of dilated cardiomyopathy caused by titin truncations.
For patients, the potential impact is significant. Restoring high levels of dystrophin could translate into preserved mobility, stronger muscles and improved heart function. The absence of toxicity in preclinical studies also raises optimism about safety at therapeutic doses. While clinical testing has not yet begun, the company expects to advance its DMD program toward development candidate nomination in early 2026.
Tevard’s findings suggest that suppressor tRNA therapy may one day offer a versatile option for patients with DMD, especially those who currently lack effective treatments. If future trials confirm these early results, patients could benefit from a therapy that not only repairs the underlying genetic error but also delivers lasting improvements in muscle and heart health.
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