Therna Partners with Charles River to Develop AI-Designed Single-Patient RNA Therapeutics for Ultra-Rare Diseases
核心洞察
Therna Biosciences (搜索) and Charles River have announced a collaboration to advance single-patient RNA medicines for individuals with severe, ultra-rare disorders, focusing initially on lung fibrosis and Lamb-Shaffer Syndrome.
Using Therna's AI-enabled RNA design platform, the mRNA therapeutic candidate for lung fibrosis was generated in just three days and validated in the laboratory in less than three months.
The collaboration aims to submit a single patient Investigational New Drug application and dose the first patient later this year, demonstrating an accelerated development pathway for personalized therapies.
Therna Biosciences (搜索) has partnered with Charles River to advance single-patient RNA medicines for individuals with severe, ultra-rare disorders, marking a significant step forward in personalized therapeutic development. The collaboration, unveiled at the Personalized Medicine World Conference on March 6, 2026, focuses on two initial programs: an adult patient with rapidly progressive, rare lung fibrosis and a newborn with Lamb-Shaffer Syndrome, an ultra-rare central nervous system disorder.
AI-Accelerated Drug Design Platform
The collaboration showcases the remarkable speed of Therna's AI-enabled RNA design platform. The mRNA therapeutic candidate for lung fibrosis was generated in just three days, with laboratory validation completed in less than three months. Beyond speed, Therna's AI platform enabled the creation of an mRNA candidate with potential enhanced durability, tissue specificity, and expression compared to conventional approaches.
"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., Chief Executive Officer 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."
Dual Therapeutic Approach
The collaboration encompasses two distinct RNA therapeutic modalities. For the lung fibrosis patient, 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 with Lamb-Shaffer Syndrome, Therna is designing small antisense oligonucleotides (ASOs) to increase gene expression, intended to restore the function of a defective gene, while Charles River will undertake the preclinical development of the candidate ASOs.
Strategic Partnership Benefits
The collaboration brings together Therna's expertise at the intersection of RNA biology and generative AI with Charles River's industry-leading capabilities in preclinical development. This partnership aims 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."
Platform Technology and Future Applications
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.
The platform enables rapid, end-to-end design of precisely engineered mRNA molecules optimized for enhanced translation, stability, immune evasion, and tissue-specific expression. It also efficiently identifies optimal target sites within mRNA for maximally effective ASO/siRNA design, allowing gene expression to be upregulated, downregulated, or finely tuned.
Regulatory Pathway for Ultra-Rare Diseases
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. These approaches are increasingly recognized as appropriate for serious diseases with no available treatment options.