Lead mRNA program targeting an ultra-rare lung fibrosis and lead ASO program for ultra-rare central nervous system disorder
Collaboration unveiled at the Personalized Medicine World Conference
SAN FRANCISCO, March 5, 2026 /PRNewswire/ -- Therna Biosciences, a biotechnology company pioneering programmable RNA therapeutics, today 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, at 9:45 a.m. PT, where Nazli Azimi, Ph.D., Chief Executive Officer of Therna, will present how precision medicine and AI-driven innovation are converging to enable individualized treatments for patients with serious diseases.
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 Dr. Azimi. "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."
The collaboration brings together Therna's deep expertise at the intersection of RNA biology and generative AI with Charles River's industry-leading 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.
"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."
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.
About Therna Biosciences
Therna is a biotechnology company redefining RNA therapeutics by uniting deep RNA biology with state-of-the-art RNA foundation models and generative AI. Its biology-guided platform enables rapid, end-to-end design of precisely engineered mRNA molecules that are optimized for enhanced translation, stability, immune evasion, and tissue-specific expression. The platform also efficiently identifies optimal target sites within mRNA for maximally effective ASO/siRNA design. Therna's technology allows gene expression to be upregulated, downregulated, or finely tuned, enabling broad applications across a range of therapeutic areas, including genetic, metabolic, immunological, and cardiovascular diseases, in addition to ultra-rare, individualized treatments. Headquartered in San Francisco, Therna is backed by leading investors and scientific advisors at the forefront of RNA and AI innovation. Please visit therna.com for more information and follow us on LinkedIn.
View original content to download multimedia:https://www.prnewswire.com/news-releases/therna-announces-collaboration-with-charles-river-to-advance-single-patient-personalized-rna-therapeutics-302704703.html
SOURCE Therna Biosciences
