Therna Biosciences announced a collaboration with Charles River to advance single-patient RNA medicines for individuals with severe, ultra-rare disorders. The first two programs under the collaboration focus on an adult patient with a rapidly progressive, rare form of lung fibrosis and a newborn with an ultra-rare central nervous system disorder called Lamb-Shaffer Syndrome. The collaboration is being unveiled at the Personalized Medicine World Conference in Silicon Valley on March 6, 2026.

Using Therna's AI-enabled RNA design platform, the mRNA therapeutic candidate for lung fibrosis was generated in just three days and its properties were validated in the laboratory in less than three months. Charles River is advancing the preclinical program with further testing, with the goal of submitting a single patient Investigational New Drug application and dosing the patient later this year. For the newborn patient, Therna is designing small antisense oligonucleotides (ASOs) to increase gene expression, which is intended to restore the function of a defective gene, and Charles River will undertake the preclinical development of the candidate ASOs.

The collaboration brings together Therna's deep expertise at the intersection of RNA biology and generative AI with Charles River's capabilities in preclinical development. Through this collaboration, the companies aim to accelerate the path from diagnosis to treatment for patients with individualized, life-threatening conditions where no existing therapies are available. Therna's platform combines experimental RNA biology with advanced foundation models to create, optimize, and predict the behavior of therapeutic RNA sequences.

Each single-patient program not only serves an individual patient but also generates new biological and functional data that continuously strengthens Therna's AI models, helping accelerate the development of the Company's pipeline of RNA medicines across broader disease areas. For ultra-rare conditions where randomized clinical trials are not feasible, Therna is pursuing development paths in collaboration with industry leaders such as Charles River, informed by close regulatory engagement, rigorous preclinical validation, and individualized clinical assessment, as approaches increasingly recognized as appropriate for serious diseases with no available treatment options.