Tevard Reports Data on Suppressor tRNA Therapy Restoring Full-Length Dystrophin in Duchenne Muscular Dystrophy Model

May 15, 2025 -- Boston, MA -- Tevard Biosciences, Inc., a privately held biotechnology company pioneering tRNA-based therapies to cure a broad range of genetic diseases, presented preclinical data demonstrating the potential of its tRNA therapy in Duchenne Muscular Dystrophy (DMD). The study showed that Tevard’s suppressor tRNA rescued full-length dystrophin protein and restored motor function in a nonsense mutation DMD disease model with no evidence of adverse effects. Detailed results of the study were shared in an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.

"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."

In the oral presentation titled “Rescue of Full-Length Dystrophin Protein and Motor Performance in a Mouse Model of Duchenne Muscular Dystrophy Using an AAV-tRNA Therapeutic,” Tevard provided results of preclinical studies using the D2-mdx animal model, which contains a nonsense mutation in the DMD gene and recapitulates key aspects of DMD pathology in humans.

Key study results included:

• Muscles of treated animals expressed full-length dystrophin protein in a dose-dependent manner through 12 weeks post-dosing with preliminary data showing expression through 24 weeks
• Organization and localization of the rescued protein is indistinguishable from protein in wild type tissue
• 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
• Dose-dependent normalization of dysregulated protein expression in treated animals
• No evidence of adverse treatment effects as measured by behavioral, histopathologic (including liver) or serum chemistries across dose groups

“Restoring full length dystrophin in DMD patients has been the goal guiding therapeutic development for more than 25 years. 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,” added Harvey Lodish, Ph.D., Co-Founder, Chair of the Scientific Advisory Board and Board Member of Tevard Biosciences. “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.”

Nonsense mutations, which introduce premature termination codons (PTCs), occur in about 15% of DMD patients and are often linked to more severe disease. These mutations prevent the production of full-length functional dystrophin protein. Suppressor tRNAs are engineered to recognize these PTCs to restore translation and enable production of full-length protein. Because there are only three PTCs (TGA, TAA, and TAG), a limited set of suppressor tRNAs could potentially treat all DMD patients with nonsense mutations.

Tevard’s tRNA-based platform enables precise, durable therapies that can target genes of any size without the risk of overexpression and restore normal protein function. Using molecular dynamics modeling and proprietary cell-based assays, Tevard identifies and optimizes next-generation suppressor tRNAs for efficient and sustained readthrough without affecting the reading of normal stop codons. Due to their compact nature, DNAs encoding Tevard’s tRNAs are packaged into muscle-targeting AAV vectors, enabling delivery of therapies that can reach affected tissues with minimal off-target effects to organs such as the liver, as supported by extensive toxicology studies. This is especially important for targeting large structural proteins like dystrophin, which cannot be replaced using standard AAV-based therapies.