Therna Biosciences Collaborates with Charles River on Personalized RNA Therapeutics

• Therna Biosciences has partnered with Charles River to advance single-patient RNA medicines targeting severe ultra-rare diseases, including lung fibrosis and Lamb-Shaffer Syndrome.

• Charles River will support the preclinical development of therapeutic candidates generated using Therna’s AI-enabled RNA design platform.

Therna Biosciences has entered a collaboration with Charles River to advance personalized RNA medicines designed for individual patients with severe, ultra-rare disorders. The partnership focuses on developing therapeutic candidates tailored to a single patient’s genetic condition. 

The companies said the first two programs under the collaboration will target an adult patient with a rapidly progressive form of lung fibrosis and a newborn diagnosed with Lamb-Shaffer syndrome, an ultra-rare central nervous system disorder. Therna is using its AI-enabled RNA design platform to develop therapeutic candidates, while Charles River will lead preclinical testing and development activities. 

According to the companies, the mRNA candidate for lung fibrosis was generated in three days using Therna’s platform and validated in laboratory testing in less than three months. Charles River is continuing preclinical work with the goal of submitting a single-patient Investigational New Drug application and potentially dosing the patient later this year. 

For the second program, Therna is designing antisense oligonucleotides intended to increase gene expression and restore the function of a defective gene associated with Lamb-Shaffer syndrome. Charles River will conduct the preclinical development for the ASO candidate. 

“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.”

Nazli Azimi, Ph.D., chief executive officer of Therna

The collaboration combines Therna’s RNA design capabilities with Charles River’s preclinical development expertise to support the advancement of individualized therapies for conditions where no treatments currently exist.