Therna Biosciences, Charles River Partner to Advance Single-Patient RNA Medicines

Therna Biosciences, a biotechnology company pioneering programmable RNA therapeutics, has joined forces 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 brings together Therna’s 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.

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. In addition to speed, Therna’s AI platform enabled the creation of an mRNA candidate with potential enhanced durability, tissue specificity, and expression. 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.

“Therna was founded on the belief that RNA can be programmed with precision, speed, and predictability, making it possible to design therapies tailored to the unique genetic drivers of each patient’s disease,” said Nazli Azimi, Ph.D., CEO of Therna. “This first single-patient program demonstrates the power of our integrated RNA biology and AI platform to rapidly generate superior therapeutic candidates and therefore validates our approach for RNA design. Partnering with Charles River enables us to efficiently advance these highly personalized medicines for patients with urgent, unmet needs.”

“We are proud to work with Therna in advancing innovative RNA therapies for patients with rare and ultra-rare diseases,” said Roxana Redis, Ph.D., Science Director, Advanced Modalities at Charles River. “The ability to move a personalized therapeutic concept into preclinical testing with such speed and precision reflects the strength of Therna’s platform and enables us to further compress the development timelines for these individualized therapies.”

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