ExoRNA Bio's siRNA Therapy ER2001 Receives IND Approval for Huntington’s Disease

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According to the CDE official website, ExoRNA BioExoRNA Bio (Nanjing) Co., Ltd. (hereinafter referred to as "ExoRNA") has received acceptance for the IND application of its "ER2001 Injection".

ER2001, the first product from ExoRNA Bio's In-Vivo Self-Assembled Exosome Delivery (IVSAED) technology platform, has demonstrated exciting preliminary efficacy in preclinical animal studies and shown superior safety.May 2023,ER2001 Receives FDA Orphan Drug Designation,Used for the treatment of Huntington's Disease (HD).

Animal experiments have shown that ER2001 utilizes the body's own organs and tissues as bioreactors to "process" siRNA into its active form in vivo. The siRNA is then assembled into endogenous exosomes targeting the central nervous system, which are simultaneously produced by the "bioreactor." These exosomes are secreted to "transport" the siRNA to neuronal cells, where it degrades the mRNA of mutant huntingtin protein (mHTT), thereby exerting its therapeutic effect. Exosomes not only protect the siRNA during transport across cellular and biological barriers but also exhibit excellent biocompatibility. ExoRNA Bio’s third-generation nucleic acid delivery technology platform ensures the safety of nucleic acid drug delivery and achieves a breakthrough in the challenge of "lack of effective non-liver delivery systems for nucleic acid drugs." It also avoids issues associated with the use of exosomes as carriers in vitro, such as high production costs, complex quality control, instability and susceptibility to degradation, and the need for high doses to take effect. Even at extremely low dosing concentrations, ER2001 can exert a powerful effect in vivo. Currently, ER2001 is still under development and research and has not yet been approved for marketing.

Source: ExoRNA Bio