HG204

HG204 is an experimental CRISPR RNA-editing therapy developed for the treatment of MECP2 duplication syndrome. The drug uses a single adeno-associated virus (AAV) vector to deliver HuidaGene’s proprietary high-fidelity Cas13Y (hfCas13Y) RNA editor and a guide RNA targeting MECP2. Its mechanism is to reduce overexpressed MECP2 mRNA and protein, correcting the underlying pathological gene overdosage of MECP2 duplication syndrome. Preclinical studies demonstrated stable and durable expression in animal brain tissues and improvement in disease phenotypes in mouse models. HG204 is the subject of the HERO clinical trial (NCT06615206), the first-in-human trial of an RNA-editing therapy for MDS, and has been granted both Orphan Drug and Rare Pediatric Disease designations by the FDA, as well as Orphan Drug Designation by the EMA[1][2][3][4][5][6][7].

Other names

HG204HG-204HG 204