HEM-9

HEM-9 is a preclinical non-viral gene therapy candidate developed by Copernicus Therapeutics for the treatment of Hemophilia B. The therapy utilizes Copernicus's proprietary DNA nanoparticle (NP) platform, which consists of single molecules of plasmid DNA compacted with polycations (such as PEG-substituted polylysine) to form stable, small-diameter particles. HEM-9 is designed to deliver the gene encoding human Coagulation Factor IX (FIX) to the liver. By using a non-viral delivery system, the approach aims to avoid the pre-existing immunity and inflammatory responses associated with viral vectors (like AAV), potentially allowing for redosing if necessary. The primary therapeutic goal is to achieve long-term, stable expression of Factor IX to restore blood clotting function and reduce the frequency of bleeding episodes.

Other names

Hemophilia B gene therapy (Copernicus)Copernicus Hemophilia B DNA nanoparticle