HuidaGene (Shanghai) Biotechnology Co., Ltd, a biotech company based in China, has announced that it has received positive opinions from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) regarding its orphan drug filing for HG204. HG204 is an RNA editing therapy developed independently by HuidaGene, utilizing CRISPR/hfCas13Y technology.
The HG204 program is designed to treat MECP2 duplication syndrome (MDS), a rare pediatric neurodevelopmental disorder. MDS arises from a two or three-fold duplication of the MECP2 gene, which can result in hypotonia, developmental delay, intellectual disability, anxiety, motor and language loss, epilepsy, recurrent respiratory infections, and even premature death in infants. Currently, there is no effective treatment for MDS. HG204 delivers a high-fidelity RNA editor hfCas13Y and gRNA targeting the MECP2 gene using a single adeno-associated virus (AAV) vector. Pre-clinical trials have shown that HG204 can effectively reduce MECP2 expression levels, restore motor function, and significantly extend the lifespan in MDS mouse models. The therapy has been granted orphan drug designation (ODD) and Rare Pediatric Disease Designation (RPDD) in the US.- Flcube.com
