A familiar theme re-emerged at the 2026 J.P. Morgan Healthcare Conference with new urgency: RNA is no longer just a messenger—it’s becoming medicine. Across interviews with leaders from Korro Bio, Trace Neuroscience, and CAMP4 Therapeutics, a picture came into focus of an RNA “middle ground” in genetic medicine, one that sits between permanent DNA editing and conventional small molecules, offering precision with flexibility. Together, these companies are pushing RNA therapeutics beyond silencing genes toward editing, repairing, and even amplifying gene function—often in ways that would be difficult or risky at the DNA level.
Ram Aiyar, PhD (CEO, Korro Bio)
Korro Bio CEO Ram Aiyar, PhD, presents RNA editing as both biologically natural and clinically practical. Human cells already edit RNA via enzymes like ADAR, which convert adenosine to inosine. Korro leverages this machinery with proprietary antisense oligonucleotides that direct precise RNA edits without altering DNA. The advantage, Aiyar argues, is reversibility and choice: while permanent DNA edits may suit severe pediatric diseases, transient RNA editing offers a lower-risk option for late-onset or chronic conditions. As Aiyar put it, alternatives especially when lifelong DNA changes may feel disproportionate.
Korro’s path reflects both promise and growing pains. Its first program in alpha-1 antitrypsin deficiency showed RNA correction and protein repair in humans but missed therapeutic thresholds, leading the company to halt development and refocus as a leaner organization.
That focus may soon pay off. Korro plans to advance KRRO-121 into the clinic in the second half of 2026 for urea cycle disorders and hepatic encephalopathy. By editing a single amino acid to stabilize a metabolic enzyme, the therapy could lower ammonia with dosing every two to four weeks—far simpler than today’s regimens. Preclinical data showing near-100% RNA editing in animals suggest that large swaths of the transcriptome are now editable, even if most diseases require far less.
Eric Green, MD, PhD (Co-founder and CEO, Trace Neuroscience)
If Korro is rewriting RNA letters, Trace Neuroscience is correcting RNA grammar. Trace’s CEO Eric Green described a therapeutic strategy rooted in human genetics and splicing biology for amyotrophic lateral sclerosis (ALS). Nearly all ALS patients lose a protein called UNC13A, essential for synaptic communication. The loss isn’t due to a faulty gene sequence but improper RNA splicing triggered by dysfunction of TDP-43, a protein implicated across multiple neurodegenerative diseases.
Trace’s solution is an ASO designed to bind UNC13A RNA and restore correct splicing—an approach inspired by the success of ASOs like Spinraza in spinal muscular atrophy. In preclinical models, the molecule potently restores UNC13A protein, addressing a pathology seen in roughly 97% of ALS patients.
Delivery, long a hurdle for CNS drugs, is well defined: intrathecal injection into cerebrospinal fluid, with strong distribution to the spinal cord and cortex. An essential consideration for regulators, patients, and payers is the frequency of dosing, and Green highlighted that the molecule’s durability may permit just a few doses per year. The implications extend beyond ALS. The same TDP-43–driven mis-splicing appears in frontotemporal dementia and subsets of Alzheimer’s disease, opening the door to broader applications with the same therapeutic construct.
Josh Mendel Brehm (CEO, CAMP4 Therapeutics) and Kelly Gold (CFO, CAMP4 Therapeutics)
CAMP4 Therapeutics is taking yet another RNA path: increasing gene expression rather than suppressing it. CEO Josh Mendel Brehm and CFO Kelly Gold explained how CAMP4 targets regulatory RNAs—non-coding RNAs arising from enhancers and promoters—to boost output from healthy genes. Using antisense oligonucleotides, the company can increase expression roughly twofold, which is precisely what’s needed in haploinsufficiency disorders, where patients produce only 50% of a critical protein.
Their lead program focuses on SYNGAP1-related disorders, a rare but severe neurodevelopmental epilepsy with no disease-modifying therapies. Like Trace, CAMP4 benefits from a de-risked CNS delivery pathway via intrathecal dosing and established oligonucleotide chemistry. With partnerships outside the CNS and plans to enter the clinic for SYNGAP1 this year, the company positions itself as a “product company with a platform,” shaped by years of capital discipline.
RNA’s expanding role in precision medicine
Taken together, these JPM conversations highlight how RNA medicines are diversifying. Editing can fine-tune protein function. Splicing correction can rescue lost biology. Regulatory RNAs can turn gene expression back on. None require permanent DNA alteration, yet all are grounded in human genetics and increasingly validated delivery technologies.
For a field once defined largely by gene silencing, RNA therapeutics are now writing, editing, and amplifying biology itself—and, as JPM 2026 made clear, they’re doing so with patients very much in mind.
