Shixi Biologics Announces First-in-Human Dosing of RC001, a Novel ADAR-Recruiting RNA Editing Therapy for Dravet Syndrome

2025Year12Month25Day,Small Nucleotide Developed by RecoRNARNAEdit DrugRC001AlreadyCompletion of First Patient Dosing.This marks the beginning of endogenous-basedADARRecruited Small NucleotideRNAEditing therapeutic technology has taken a crucial step on the path to clinical translation, while also providingDravetBrings new hope to patients with syndromes. Currently, the patient's overall condition is stable after the first dosing, and they will subsequently undergo close monitoring and follow-up. The initiation of this early-stage clinical study providesRC001Laid an important foundation for subsequent larger-scale clinical trials.

AboutRC001Drug

Developed by RecoRNARC001The drug, through a small nucleotide drug strategy, recruits endogenousADARAchieve Single BaseRNAEditor, inRNAPrecise targeted intervention at the level of causation has the potential to improve disease progression and offer patients more effective treatment options.RC001Is the world's first to recruit endogenousADARSmall nucleotide RNA Editing drug strategies for application in neurological diseases and advancing to human trials. In earlier preclinical studies,RC001Demonstrated good efficacy and safety, laying the foundation for subsequent research and development progress.2025Year4Month，RC001Has obtained the approval from the U.S. Food and Drug Administration (FDA) Orphan Drug Designation (ODD）, which reflectsFDAYesRC001Used for treatmentDravetThe recognition of the syndrome's potential clinical value also suggestsRECORNA inRC001Subsequent clinical trial design, R&D advancement, regulatory aspects, etc., can all be obtainedFDA More scientific guidance and regulatory support.

AboutDravetSyndrome

DravetSyndrome(Dravet Syndrome，DS)It is a rare and refractory epilepsy syndrome, belonging to developmental and epileptic encephalopathy.(Developmental and Epileptic Encephalopathy，DEE)Category. Its clinical features mainly include onset in infancy, various types of epileptic seizures, drug-resistant epilepsy, psychomotor developmental delay, and a high risk of sudden death. Currently, there is no curative treatment for this disease. Existing anti-epileptic drugs can reduce the frequency of seizures to a certain extent, but there are still significant limitations in improving the course of the disease and comorbidities.

About RecoRNA

RecoRNA is a small nucleic acid drug research and development company thatRNARECORNA develops innovative drugs through technical platforms such as editing, aiming to meet unmet clinical needs including neurological disorders and metabolic diseases. Relying on the technology transfer from Professor Rui Zhang's lab at Sun Yat-sen University's School of Life Sciences, RECORNA has established its own intellectual property rights based on the world's largest...ADARSubstrate Editing LibraryRNASingle-Base Editing Design PlatformMIRROR、RNAFragment Clipping ToolSISSORAndAIScreeningATOMICPlatform. By leveraging these innovative technologies, the company is able to overcome the limitations of traditional small molecules and antibody drugs, addressing critical therapeutic challenges such as frameshift mutation diseases, single-point mutation genetic disorders, major indications like metabolism-related conditions, and tumor immunotherapy, while avoiding...DNAIrreversible off-target safety risks caused by layered gene editing therapy.

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