Early Data Show Potential of Tevard’s tRNA Therapy in Duchenne Muscular Dystrophy

Transfer RNA (abbreviated tRNA and formerly referred to as sRNA, for soluble RNA with RNA strand in the cell 3d rendering | Image Credit: © Love Employee - stock.adobe.com

In an oral presentation on day three of the 2025 American Society of Gene & Cell Therapy Annual Meeting (ASGCT 2025)—held May 13-17 in New Orleans—the privately held biotechnology company Tevard Biosciences presented preclinical data indicating that the company’s suppressor transfer RNA (tRNA) therapy rescued full-length dystrophin protein in a nonsense mutation Duchenne muscular dystrophy (DMD) disease model (1). With no evidence of adverse effects, the tRNA therapy also appeared to restore motor function in the model.

"To our knowledge, this is the first demonstration of sustained restoration of full-length dystrophin accompanied by functional improvement in a DMD in-vivo model. We are advancing this candidate through our own development efforts, while exploring potential collaborations that could expand its reach and impact patients," said Daniel Fischer, co-founder, president, and CEO of Tevard Biosciences. "In addition to its potential as a highly effective treatment for DMD patients with nonsense mutations, these results establish our suppressor tRNAs as a viable therapeutic platform for a range of neuromuscular disorders and cardiomyopathies. We anticipate sharing additional preclinical data from our lead program in dilated cardiomyopathy caused by nonsense mutations in the TTN gene in the coming months."

According to the Muscular Dystrophy Association, DMD is “a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact” (2). About 6 in 100,000 people in North America and Europe have the condition, which usually has an onset between ages 2 and 3 and can cause difficulty walking, running, and jumping. Progressive weakness associated with DMD can eventually impair pulmonary function and even cause acute respiratory failure.

During the oral presentation at ASGCT 2025, titled “Rescue of Full-Length Dystrophin Protein and Motor Performance in a Mouse Model of Duchenne Muscular Dystrophy Using an AAV-tRNA Therapeutic,” Tevard Associate Director, Julien Oury, PhD, provided preclinical study results based on the D2-mdx animal model, which recapitulates key features of DMD pathology observed in humans. Important study findings included by Dr. Oury included (1):

• Treated animals showed dose-dependent expression of full-length dystrophin protein in muscle tissue, sustained through 12 weeks after treatment, with early indications of continued expression up to 24 weeks.
• The structure and placement of the restored dystrophin protein closely resembled that found in healthy, wild-type tissue.
• Notable improvements in motor abilities were observed, including increased time on the rotarod test and significant gains in both forelimb and hindlimb grip strength.
• Treatment led to dose-related correction of abnormal protein expression patterns.
• No signs of treatment-related toxicity were detected, based on behavioral assessments, tissue analyses (including liver), and blood chemistry evaluations across all dosage levels.

“Restoring full-length dystrophin in DMD patients has been the goal guiding therapeutic development for more than 25 years,” said Tevard Co-Founder and Board Member Harvey Lodish, PhD, who is also chair of the Scientific Advisory Board. “Other approaches are unable to achieve this, and the clinical benefits remain subject to debate. We are able to restore full-length dystrophin expression using our suppressor tRNAs. We are currently working on the next generation of suppressor tRNA therapies, with preliminary data showing that it more than doubled the potency seen in earlier versions.”

The news signals that gene-based approaches for monogenic diseases like DMD are maturing—and that the role of pharmaceutical manufacturing scientists in ensuring reproducibility, quality, and scalability is becoming more critical than ever.

References

1. Tevard Biosciences. Tevard Reports Data on Suppressor tRNA Therapy Restoring Full-Length Dystrophin in Duchenne Muscular Dystrophy Model. Press Release. May 15, 2025.
2. Muscular Dystrophy Association. Duchenne Muscular Dystrophy. MDA.org (accessed May 15, 2025).