ExoRNA Bio Submits IND for ER2001, a First-in-Class In Vivo Self-Assembled siRNA Therapy for Huntington’s Disease

On March 13, 2024, the official website of the Center for Drug Evaluation (CDE) under the National Medical Products Administration (NMPA) showed that the "ER2001 Injection" submitted by ExoRNA Bio (Nanjing) Co., Ltd. (hereinafter referred to as ExoRNA) has been accepted.

According to ExoRNA Bio, ER2001 is the first candidate product developed based on its In-Vivo Self-Assembled Exosome Delivery (IVSAED) technology platform and is being developed for the treatment ofHuntington's Disease（Huntington's Disease, HD）。

ER2001 is a nucleic acid drug, firstUsing the body's own organs or tissuesAs a bioreactor, it is "processed" into the active form of siRNA in vivo and assembled into endogenous exosomes targeting the central nervous system that are simultaneously produced by the "bioreactor." These are then "transported" to neurons via exosome secretion.Cells degrade the mRNA of mutant huntingtin protein (mHTT), thereby producing therapeutic effects.

ER2001 is delivered through ExoRNA Bio's third-generation nucleic acid delivery technology platform for the delivery of nucleic acid drugs.ER2001 Delivered to body tissues and cells; then processed into "active" forms within the cells.After siRNA, specific endogenous exosome targeting produced by cells is utilized for the delivery of therapeutic molecules.

Using endogenous exosomes as delivery vehicles can leverage all the advantages of exosomes as drug carriers, including excellent biocompatibility, transport across cellular and biological barriers, while avoiding the obstacles associated with exosome production in vitro, such as high costs and challenges in quality control.Unstable, prone to degradation, high effective dosage, etc.

In preclinical studies, ER2001 demonstrated positive preliminary efficacy and good safety. According to reports, ER2001 was able to function in vivo even at extremely low administered concentrations.

Notably, ER2001 received orphan drug designation from the U.S. FDA in May 2023 for the treatment of Huntington's disease.

In fact, several companies in China have already submitted clinical applications for siRNA drugs, including Junshi Biosciences, Zhengdatianqing, and Fosun Pharma. However, these siRNA drugs are classified as chemical drugs, with the acceptance number starting with "CXHL".

ExoRNA Bio's new siRNA product is classified as a "biological product," which is determined by its unique mechanism of action. However, compared with other siRNA drugs, ExoRNA Bio's ER2001 has a more complex and precise mechanism of action.

This also leads to higher unpredictability of ER2001 after entering the clinical stage, compared to the preclinical stage.ER2001Whether it can function as expected in the complex human body environment.

1.ExoRNAExoRNA BioExoRNA Bio's Pipeline Product ER2001 Granted FDA Orphan Drug Designation