Gene Therapy, Rare Diseases, and Transfer RNA

Rare diseases, defined by FDA as “a disease or condition that affects less than 200,000 people in the United States” (1), may have the unique challenge of not being easily applied to multiple disease conditions; therefore, pharmaceutical companies may find it challenging to invest the time and resources into developing these important medicines.

Transfer RNA (tRNA)-based therapies are one option for treating rare diseases. At this year’s J.P. Morgan Healthcare Conference, being held in San Francsico from January 12–15, Tevard Biosciences will be highlighting preclinical data on the company’s suppressor tRNA platform, which has been shown to restore full-length, native full-length, native dystrophin, and titin in models of Duchenne muscular dystrophy and dilated cardiomyopathy caused by titin truncations (2).

PharmTech spoke with Elisabeth Gardiner, PhD, chief scientific officer of Tevard Biosciences, about what makes tRNA unique and how it may benefit rare diseases and other conditions.

“Existing gene therapy approaches can be especially limited when the goal is to fix large structural proteins. So, the size of the gene matters. The size of the protein matters, the number of unique mutations make it really difficult to do at scale,” Dr. Gardiner explains. “So Tevard’s tRNA therapeutic approach offers…a scalable platform that's capable of correcting disease-causing premature termination codons. These are also called nonsense mutations. Essentially, what happens in normal protein production is you reach the end of the protein, you have this thing called the ribosomal assembly, and it drives along, it reaches what's called a stop codon, and then the protein is released and can work effectively.

Dr. Gardiner further explains that, in the case of the company’s suppressor tRNA technology, team members can go in and fix these nonsense mutations at any point throughout the protein and create a full-length version of that protein. Previously, this action may have only been possible to due to the large size of the genetic material, in which a researcher would only be able to make part of the protein and repair that part, she continues. However, with tRNA suppressor technology, the researcher can go in and fix just those specific codons—those nonsense mutation codons—and create a full length protein that's completely functional, that can interact with other proteins, and that can provide contractility. This can result in a fully functional protein that can promote muscular repair, Dr. Gardiner notes.

References

1. FDA. Rare Diseases at FDA. FDA.gov. Nov. 21, 2024. https://www.fda.gov/patients/rare-diseases-fda
2. Tevard. Tevard Biosciences Presents Data Showing Compelling Full-Length Protein Rescue with tRNA-based Treatment for Duchenne Muscular Dystrophy and Dilated Cardiomyopathy at the 2025 FEBS Special Meeting. Press Release. Sept. 29, 2025.

About the speaker

Dr. Elisabeth Gardiner is chief scientific officer at Tevard Biosciences, joining the leadership team in 2025 to spearhead the company’s pioneering tRNA-based gene therapies for rare diseases with high unmet need. With more than 25 years in the biopharmaceutical industry, she has directed and managed drug discovery and development efforts yielding 11 investigational new drug filings, four Phase I/II trial candidates, and one Phase III candidate. Prior to Tevard, Dr. Gardiner held senior R&D leadership roles at Tactile Therapeutics, Alterome Therapeutics, Aravive, and Kinnate Biopharma, where she directed multidisciplinary teams and advanced programs from discovery to clinical-stage development across neurology, oncology, and rare disease. Dr. Gardiner gained experience in tRNA biology while working at aTyr Pharma and at Scripps Research Institute in the lab of Paul Schimmel. Dr. Gardiner’s commitment to the ethical development of effective and accessible medicines is her key focus in life. In addition to her professional work, Dr. Gardiner acts as a patient advocate in the rare disease and oncology space. She earned her PhD from the University of Wisconsin-Madison and holds a B.S. and an M.S. from Texas A&M University.