
miRNA Nucleic Acid Drug Developer

Huntington's disease currently has only symptomatic relief drugs available globally, while other potential disease-modifying drugs are still in clinical or preclinical stages. In previously conducted IIT studies, ER2001 demonstrated good safety and preliminary clinical efficacy.
In the favorable environment of NMPA encouraging innovation and supporting the development of orphan drugs, ExoRNA Bio, after obtaining clinical trial approval by default, will actively promote subsequent clinical research and other studies of ER2001 injection, making a positive contribution to improving the survival period and quality of life for patients with Huntington's disease in China and globally.
Huntington's disease is a rare and fatal neurodegenerative disorder caused by the abnormal expansion of CAG triplet codon 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 global prevalence of Huntington's disease is approximately 2.7/100,000.
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 brand-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-type nucleic acid innovative drug company.
E.N.D

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