ExoRNA Bio's Exosome-Delivered Nucleic Acid Therapy ER2001 Receives IND Approval in China for Huntington’s Disease

Huntington's disease is a rare and fatal neurodegenerative disorder caused by the Huntington gene located on the short arm of chromosome 4.HTT) Caused by the abnormal expansion of CAG triplet codon repeats on the first exon. Patients with Huntington's disease often exhibit neurological dysfunction, as well as motor, cognitive, and psychiatric abnormalities. Current clinical information shows that the peak onset age for Huntington's disease patients is between 30 to 50 years old,The average life expectancy of patients after onset is about 15 to 20 years, with extremely high rates of disability and mortality., patients will gradually lose their ability to take care of themselves in uncontrollable "dancing."

It is estimated that this irreversible rare disease affects about 30,000 people in China. According to the Rare Disease Diagnosis and Treatment Guidelines (2019 Edition), the prevalence of Huntington's disease worldwide is approximately 2.7/100,000.Huntington's disease currently has only one drug available globally for alleviating symptoms., other drugs that may control disease progression are in clinical or preclinical stages.

In the previously conducted IIT studies, ER2001 demonstrated good safety and preliminary clinical efficacy.

According to the information on ExoRNA Bio's official website,Preclinical experimental results 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 showed that the intended human dose is far below the maximum feasible dose in animals. The company has developed a gene-targeting approach for Huntington's disease.mHTTThe in vivo self-assembled siRNA achieved effective treatment of the disease model.Injecting plasmids encoding specific HTT siRNA into the body,Using the Liver as a Reactor to Synthesize HTT siRNA and Self-Assemble into Secretory Exosomes。InA short peptide derived from the rabies virus (RVG) is expressed on the exosome membrane, which mediates the passage of exosomes through the blood-brain barrier by binding to acetylcholine receptors on the surface of neuronal cells, thereby deliveringTherapeutic HTT siRNA Delivery to Brain CellsTo achieve efficient brain delivery and uniform brain biodistribution of nucleic acid drugs, thereby reducing the expression of mHTT protein and treating Huntington's disease symptoms caused by toxic proteins.

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In the favorable environment of NMPA encouraging innovation and supporting the development of orphan drugs, ExoRNA Bio stated that after obtaining the clinical research tacit permission, it will actively promote the subsequent clinical research and other research work 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.

AboutExoRNA Bio

ExoRNA Bio was founded by Professor Chenyu Zhang and Dr. Xia Meng in September 2021. Headquartered in Shenzhen, with subsidiaries in Nanjing and Shanghai. ExoRNA BioFocus on drug development for diseases with unmet clinical needs and no available treatments, particularly for central nervous system disorders, etc.The vision is to build a world-leading platform-based innovative nucleic acid drug company. ExoRNA Bio owns the globally unique IVSA™ (In-Vivo Self-Assembled) in vivo self-assembled exosome nucleic acid drug delivery platform, which has unique advantages such as multi-organ targeting, ability to cross the blood-brain barrier, high safety, and low cost, providing a one-stop solution to the current delivery challenges of RNA drugs.

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