ExoRNA Bio's First-in-Class In-Vivo Self-Assembled Exosome-Delivered siRNA Therapy ER2001 Receives IND Acceptance from China CDE for Huntington’s Disease
ExoRNA
miRNA Nucleic Acid Drug Developer
On March 13, the IND application for "ER2001 Injection" by ExoRNA Bio (Nanjing) Co., Ltd. ("ExoRNA Bio") was accepted by the CDE. It is reported that,ER2001 is the first product from ExoRNA Bio's In-Vivo Self-Assembled Exosome Delivery (IVSAED) technology platform.Last year, it was granted Orphan Drug Designation by the FDA for the treatment of Huntington's Disease (HD).
According toExoRNA Bio Official Report,ER2001 Demonstrates Promising Preliminary Efficacy and Superior Safety in Preclinical Animal Studies. Animal experiments show that ER2001 utilizes the body’s own organs and tissues as bioreactors to "process" the compound into its active form of siRNA, which is then assembled into endogenous exosomes targeting the central nervous system that are simultaneously produced by the "bioreactor." These exosomes are secreted to "transport" the siRNA to neuronal cells, degrading the mRNA of mutant huntingtin protein (mHTT) and thereby exerting therapeutic effects. Exosomes not only protect the siRNA during transport across cellular and biological barriers but also exhibit excellent biocompatibility. The third-generation nucleic acid delivery technology platform developed by ExoRNA Bio ensures the safety of nucleic acid drug delivery and achieves a breakthrough in the challenge of “no effective non-liver delivery system for nucleic acid drugs.” It also avoids issues associated with exosome carriers produced in vitro, such as high cost, challenging quality control, instability, susceptibility to degradation, and high effective dosage requirements. Even at extremely low dosing concentrations, ER2001 can exert powerful effects in vivo. Currently, ER2001 is still under development and research and has not yet been approved for marketing.
Recommended reading article link:ExoRNA Breaks Through the Blood-Brain Barrier Delivery Bottleneck for Nucleic Acid Drugs with Its Unique Third-Generation In Vivo Self-Assembled Exosome Delivery Technology, Completing Multi-Million Angel+ Round Financing
About Huntington's Disease
Huntington's disease is a rare autosomal dominant genetic disorder. Patients with this condition often experience neurological dysfunction, as well as motor, cognitive, and psychiatric abnormalities. Current medications and surgical treatments can only alleviate some symptoms but cannot achieve a complete cure. The pathogenic mechanism of Huntington's disease involves the abnormal aggregation of mutated huntingtin protein (mHTT) into large molecular complexes within cells, leading to neuronal death. Therefore, reducing mHTT protein is the fundamental and optimal strategy for treating this disease.
About ExoRNA Bio
ExoRNA Bio, founded in September 2021, is committed to developing more products that can benefit patients with unmet clinical needs in actual treatments, especially therapeutic drugs for central nervous system diseases, aiming to build a platform-type RNA innovative drug enterprise. So far, ExoRNA Bio has received investments from several well-known investment institutions in China and is actively advancing its product pipeline.
E.N.D
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