Tides Express | First Patient Dosed in Phase I Clinical Trial of ExoRNA Bio's Novel siRNA Therapy ER2001 for Huntington’s Disease in China

ER2001 Injection is a novel nucleic acid drug developed by ExoRNA Bio (Nanjing) Co., Ltd. It delivers siRNA targeting the central nervous system to degrade the Huntington (HTT) gene, thereby reducing the expression of the pathogenic protein in Huntington's disease and achieving therapeutic effects for patients with Huntington's disease.

"Phase I Clinical Study of ER2001 Injection for the Treatment of Early Manifest Huntington's Disease: A Randomized, Double-blind, Placebo-controlled Trial" received approval from the National Medical Products Administration (NMPA) in early June 2024 and successfully passed the ethical review of the First Affiliated Hospital of Sun Yat-sen University to proceed with clinical research. The study employs a two-phase design, including dose escalation and dose expansion, with a planned enrollment of 15-27 participants, approximately 10 of whom will be recruited from the Department of Neurology at the hospital to evaluate the safety and preliminary efficacy of ER2001 Injection.

ExoRNA Bio's official website shows that preclinical experimental results have confirmed that after intravenous administration of ER2001 injection, it can cross the blood-brain barrier to reach the affected area and exert a therapeutic effect. Safety evaluation studies indicate that the intended human dose is far below the maximum feasible dose in animals. The company has developed an in vivo self-assembling siRNA targeting the Huntington's disease gene mHTT, achieving effective treatment in the disease model. Plasmids encoding specific HTT siRNA are injected into the body, using the liver as a reactor to synthesize HTT siRNA and self-assemble into secretory exosomes. A short peptide derived from the rabies virus (RVG) is expressed on the exosome membrane. This peptide mediates the crossing of exosomes through the blood-brain barrier by binding to acetylcholine receptors on the surface of nerve cells, thereby delivering therapeutic HTT siRNA to brain cells. This achieves efficient brain delivery and uniform biodistribution of nucleic acid drugs in the brain, thereby reducing the expression of mHTT protein.To treat the symptoms of Huntington's disease caused by toxic proteins.

AboutHuntington's Disease

Huntington's disease is a rare and fatal neurodegenerative disorder caused by the abnormal expansion of CAG trinucleotide repeats in the first exon of the huntingtin (HTT) gene located on the short arm of chromosome 4. The Rare Disease Diagnosis and Treatment Guidelines (2019 Edition) indicate that the worldwide prevalence of Huntington's disease is approximately 2.7/100,000.

AboutExoRNAThing

ExoRNA Bio focuses on the development of drugs for diseases with unmet clinical needs, particularly in the field of synthetic biology self-assembled exosome nucleic acid drugs for central nervous system diseases, infections, and tumors. The company's all-new third-generation small nucleic acid delivery system is expected to overcome the delivery bottleneck of nucleic acid drugs, enabling targeted delivery of novel multifunctional genetic components, with the goal of becoming a globally leading platform-based nucleic acid innovative drug company.