Codone Biotech to Present Data on its Suppressor-tRNA Platform in Disease Models with Nonsense Mutations (Premature Termination Codons)

Codone Biotechnology, which develops transformative medicines based on Suppressor-tRNAs for patients with severe diseases caused by nonsense mutations (aka, Premature Termination Codons), will present data on its lead tRNA candidates at the poster session for Late Breaking Abstract of the 27th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting on Wednesday, May 8, 2024.

Mutations in over 4000 genes are known to be involved in human diseases, and 11% of these mutations are nonsense mutations leading to protein truncations. These diseases can be potentially treated by Suppressor-tRNAs, which are novel tRNAs engineered at its anti-codon and other positions to readthrough nonsense mutations in disease-causing genes to rescue full-length protein production and functions, regardless of disease, genes or locations of nonsense mutations. A Suppressor-tRNA for one type of nonsense mutations has the potential to treat all diseases caused by such mutations.

"tRNA is the critical link between mRNA and protein, and engineering tRNA into a therapy opens up a new front of modifying protein translation to treat diseases," said Lingjie Kong, Ph.D., Co-founder. "Our proprietary tRNA candidates have been validated in multiple disease models with nonsense mutations in vitro and in vivo, and we continue to make exciting progress in optimizing leading molecules packaged in AAV."

"Suppressor-tRNAs have the unique potential to become universal treatment for a broad range of diseases with nonsense mutations, and we focus on diseases with major symptoms in target organs including CNS and kidney," said Dong Wei, Ph.D., Co-founder. "With support from our Scientific Advisory Board and collaborators, we are dedicated to advancing lead candidates into clinics in two years, bringing hope to patients in need."

Details of the presentation:

Title: Suppressor-tRNA Delayed Disease Progression in an Alport Syndrome Mouse Model

Date: 12:00 PM, May 8, 2024, EDT

Session Name: Wednesday Posters: Late-Breaking Abstracts

Late-Breaking Abstract Number: LBA-16

About Codone Biotechnology

Founded in 2022, Codone Biotechnology focuses on developing transformative medicines based on suppressor-tRNAs for patients with severe diseases caused by premature termination codons. The Company has established proprietary platforms in suppressor-tRNA screening and optimization as well as AAV delivery, for broad range of indications in target organs including Central Nervous System, kidney, heart and retina.