April 29, 2025 -- Boston, MA -- Tevard Biosciences, Inc., a privately held biotechnology company pioneering tRNA-based therapies to cure a broad range of genetic diseases, will present preclinical data supporting the potential of its tRNA therapy in Duchenne Muscular Dystrophy (DMD). The study demonstrated that Tevard’s suppressor tRNA rescued full-length dystrophin protein and restored motor function in a DMD disease model and showed no evidence of adverse treatment effects. The Company plans to share details of the study results in an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting to be held 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.”
Details of the oral presentation is as follows:
Title: Rescue of Full-Length Dystrophin Protein and Motor Performance in a Mouse Model of Duchenne Muscular Dystrophy Using an AAV-tRNA Therapeutic
Abstract #: 206
Date/Time: Thursday, May 15, 1:45PM, Rm 288-290
Presenter: Paul Kaplan (for Julien Oury)
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.
Duchenne muscular dystrophy (DMD) is a fatal X-linked disorder caused by the absence of functional dystrophin, a large structural protein critical to the integrity of skeletal and cardiac muscle cells. DMD is characterized by progressive loss of muscle, respiratory insufficiency, and dilated cardiomyopathy. Existing therapeutic strategies are unable to restore full-length dystrophin due to the large size of the protein (427 kDa) and the diversity of unique mutations.
Tevard Biosciences is pioneering tRNA-based and other mRNA-modulating therapies to cure a broad range of genetic diseases. The privately held biotechnology company was founded by renowned scientists along with life science executives and entrepreneurs who are also fathers of children with Dravet Syndrome, a rare genetic disease. Tevard is advancing the use of its novel Suppressor tRNA platform in neurological disorders, heart disease, and muscular dystrophies including Duchenne muscular dystrophy (DMD). For more information, please visit www.tevard.com.
