Therna Enters into Collaboration with Charles River to Advance Personalised RNA Therapeutics for Ultra-Rare Diseases
Friday, March 06, 2026
Therna Biosciences has announced a collaboration with Charles River to develop single-patient RNA medicines for individuals living with severe ultra-rare disorders.
The partnership will initially focus on two programmes. One programme targets an adult patient suffering from a rapidly progressing rare form of lung fibrosis, while the second focuses on a newborn diagnosed with Lamb-Shaffer syndrome, an ultra-rare central nervous system disorder.
Therna is using its artificial intelligence-enabled RNA design platform to develop highly personalised therapies. Through this platform, an mRNA therapeutic candidate for the lung fibrosis case was designed within three days, and its properties were validated in the laboratory in less than three months. The AI system also helped design an mRNA candidate with the potential for improved durability, tissue specificity and gene expression.
Charles River will conduct further preclinical testing for the lung fibrosis programme, with the aim of submitting a single-patient Investigational New Drug application and initiating treatment later this year. For the newborn patient with Lamb-Shaffer syndrome, Therna is developing small antisense oligonucleotides designed to increase gene expression and restore the function of the defective gene. Charles River will support the preclinical development of these candidate therapies.
The collaboration combines Therna’s expertise in RNA biology and artificial intelligence with Charles River’s capabilities in preclinical research and development. The companies aim to accelerate the development of personalised treatments for patients with life-threatening conditions where no approved therapies currently exist.
Therna’s platform integrates experimental RNA research with advanced AI models to design and optimise therapeutic RNA sequences. Data generated from each personalised programme will also contribute to improving the company’s AI models, supporting the future development of RNA medicines for a wider range of diseases.
For ultra-rare conditions where traditional clinical trials are often not feasible, Therna plans to pursue alternative development pathways. These approaches include close engagement with regulators, comprehensive preclinical validation and individualised clinical assessment to enable treatment for patients with urgent unmet medical needs.
Source: prnewswire.com
