Tevard Biosciences to Present Data Demonstrating tRNA-based Therapy Rescued Full-Length Dystrophin and Motor Function in Duchenne Muscular Dystrophy Model

Preclinical results demonstrating the potential of Tevard Biosciences’ tRNA therapy for Duchenne Muscular Dystrophy (DMD) will be presented.

Tevard Biosciences is a privately held biotechnology firm that is leading the way in developing tRNA-based medicines to treat a variety of genetic illnesses.

In a DMD disease model, the study showed that Tevard’s suppressor tRNA preserved full-length dystrophin protein and restored motor function while demonstrating no negative therapeutic effects.

Details of the study’s findings will be presented orally by the company at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, which will take place from May 13–17, 2025.

“We are looking forward to sharing the results of preclinical studies from our tRNA program in DMD, which demonstrated rescue of full-length dystrophin protein and restoration of motor function in a disease model with no evidence of toxicity,” said Daniel Fischer, Co-Founder, President and CEO of Tevard Biosciences. “These early data support the potential of our suppressor tRNA approach to deliver a highly effective treatment for DMD patients with nonsense mutations. We believe these results also bode well for applying our tRNA platform to other diseases including cardiomyopathies where our lead has demonstrated the ability to restore full-length Titin protein in a model of Dilated Cardiomyopathy.”

Highlights:

• Preclinical results from studies using the D2-mdx mouse model, which contains a nonsense mutation in the DMD gene and recapitulates key aspects of DMD pathology in humans
• Muscles of treated animals expressed full-length dystrophin protein in a dose-dependent manner at 6 weeks post-dosing
• Rescued protein is organized in a fashion similar to wild-type dystrophin protein at 6 weeks
• At 12 weeks post-dosing, there was a significant restoration of motor function as demonstrated by an increase in latency time in the rotarod performance test and significantly increased forelimb and hindlimb grip strength
• There was no evidence of adverse treatment effects as measured by behavioral or histologic changes in major organs or in blood chemistry

Nonsense mutations, in which a premature termination codon prevents translation of full-length dystrophin, are found in approximately 15% of patients with muscular dystrophy and are associated with more severe forms of the disorder. Suppressor tRNAs are tRNA molecules in which the anticodon has been altered to read through nonsense mutations and restore full-length, functional protein. Since there are only three premature termination codons (TGA, TAA and TAG), a limited number of suppressor tRNAs may allow for treatment of all DMD patients with nonsense mutations.

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