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

Recently, the IND application for ExoRNA's "ER2001 Injection" has been accepted.

ER2001 Injection is the first product from its In Vivo Self-Assembled Exosome Delivery (IVSAED) technology platform. ER2001 utilizes the body's own organs and tissues as bioreactors, being "processed" in vivo into the active form of siRNA, and assembled into endogenous exosomes targeting the central nervous system that are simultaneously produced by the "bioreactor." These are then "transported" via exosome secretion to neuronal cells to degrade the mRNA of mutant huntingtin protein (mHTT), thereby exerting its therapeutic effect.

Exosomes not only protect siRNA to complete transportation across cells and biological barriers but also possess excellent biocompatibility. The third-generation nucleic acid delivery technology platform developed by ExoRNA Bio ensures the safety of nucleic acid drug delivery while avoiding the high cost, quality control difficulties, and susceptibility to degradation associated with the in vitro production of exosomes as carriers. In preclinical studies, the candidate product has already demonstrated promising therapeutic potential and safety.

Last year, ER2001 injection was granted Orphan Drug Designation by the FDA for the treatment of Huntington's disease (HD).

Editor: Liuli

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