December 25, 2025 — RecoRNA today announced that RC001, its proprietary oligonucleotide RNA editing therapy, has successfully completed first-patient dosing in an investigator-initiated clinical trial (IIT) study. This milestone marks a critical step forward in the clinical translation of oligonucleotide RNA base-editing therapeutics based on endogenous ADAR recruitment and brings new hope to patients with Dravet syndrome.
The patient remains in stable condition following the initial dose and will continue to undergo close monitoring and follow-up. The initiation of this early-stage clinical study lays an important foundation for larger-scale clinical trials of RC001 in the future.
About RC001
RC001, developed by RecoRNA, employs a chemically modified oligonucleotide therapeutic strategy to recruit endogenous ADAR enzymes for single-base RNA editing, enabling precise, RNA-level targeted intervention. By addressing the disease at its root cause, it has the potential to offer patients a more fundamental disease-modifying treatment option.
RC001 is the first therapeutic worldwide to apply a chemically synthesized oligonucleotide RNA editing strategy that recruits endogenous ADAR to a neurological disorder and to advance into human clinical testing. In preclinical studies, RC001 demonstrated favorable efficacy and safety profiles, providing a solid foundation for its continued development. In April 2025, RC001 received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA), reflecting the agency’s recognition of its potential clinical value for treating Dravet syndrome. This designation also indicates that RecoRNA is expected to receive enhanced scientific guidance and regulatory support from the FDA in areas such as future clinical trial design, development planning, and regulatory strategy for RC001.
About Dravet Syndrome
Dravet syndrome (DS) is a rare and drug-resistant epileptic syndrome and is classified as a developmental and epileptic encephalopathy (DEE). It is characterized by onset in infancy, multiple seizure types, pharmacoresistant epilepsy, psychomotor developmental delay, and an increased risk of sudden unexpected death.
At present, there is no curative treatment for Dravet syndrome. Existing anti-seizure medications can reduce seizure frequency to some extent; however, they show limited efficacy in modifying disease progression or improving associated comorbidities.
About RecoRNA
RecoRNA is an RNA therapeutics company that develops innovative medicines using platforms such as RNA editing, with a focus on addressing unmet clinical needs in neurological, metabolic and other disorders. Building on the translational research from Professor Rui Zhang’s laboratory at the School of Life Sciences, Sun Yat-sen University, RecoRNA has established a suite of proprietary platforms, including MIRROR, an RNA single-base editing design platform based on the world’s largest ADAR substrate libraries, along with the RNA excision tool SCISSOR and the AI-driven ATOMIC screening platform. Leveraging these innovative technologies, the company can overcome the limitations of traditional small-molecule and antibody drugs, addressing key therapeutic challenges such as frameshift- mutation disorders, single-nucleotide genetic diseases, major metabolic indications, and tumor immunity-related conditions, while avoiding irreversible off-target safety risks associated with DNA-level gene editing therapies.
For more information, please contact: info@recornabio.com