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AI-enabled RNA Design Fast Tracks Two N=1 Therapies
Two Urgent Programs, One Integrated AI Approach
At the Personalized Medicine World Conference, Therna and Charles River announced a collaboration to accelerate single-patient (N=1) RNA medicines from diagnosis to preclinical testing at an uncommon speed. The relationship combines Therna’s expertise at the intersection of RNA biology and generative AI with Charles River’s industry-leading capabilities in preclinical development, focused on advancing patient care for those with life-threatening conditions who lack treatment options, because every moment matters. The collaboration’s initial focus spans two high-need cases:
- mRNA lead program for an adult with rapidly progressive, ultra-rare lung fibrosis
- ASO lead program for a newborn with Lamb Shaffer Syndrome, an ultra-rare neurodevelopmental disorder Shaffer Syndrome
From AI-guided Design to Preclinical Momentum
Using Therna’s AI-enabled RNA design platform, the team generated a lead mRNA therapeutic candidate for lung fibrosis in three days, followed by laboratory validation of key properties within three months. With the goal of dosing a patient later this year, Charles River is advancing the preclinical program with additional testing to sustain momentum and derisk next steps.
In parallel, Therna and Charles River are designing antisense oligonucleotides (ASOs) to increase gene expression and restore the function of the defective gene in the newborn patient. Charles River will test both Therna’s ASO designs alongside our own and lead the preclinical development of the candidate ASOs through to IND submission. This includes aligning study design, characterization, and downstream readiness to ensure decisions and handoffs remain speedy, coordinated, and clear.
We are proud to work with Therna in advancing innovative RNA therapies for patients with rare and ultra-rare diseases. 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.”
A Scalable Model for Personalized Medicine: What AI Makes Possible for N=1
By pairing AI at the front end with end-to-end preclinical depth, Therna and Charles River offer a reproducible blueprint for future N=1 and ultra-personalized programs, especially where compressed timelines and complex therapeutic decisions are the norm. The result is a collaboration engineered to deliver what matters most to patients and their care teams: forward movement, without delay, because every moment matters.