China-based HuidaGene (Shanghai) Biotechnology Co., Ltd has announced that it has received Rare Pediatric Disease Designation (RPDD) from the US FDA for its RNA editing therapy, HG204, targeting MECP2 duplication syndrome (MDS). This designation is a significant step forward in the development of novel treatments for this rare pediatric neurodevelopmental disorder.
HG204: A Promising RNA Editing Therapy
The HG204 program utilizes an RNA editing therapy based on CRISPR/hfCas13Y, independently developed by HuidaGene. It introduces a new high-fidelity RNA editor, hfCas13Y, and gRNA targeting the MECP2 gene using a single adeno-associated virus (AAV) vector. Preclinical research indicates that HG204 effectively reduces MECP2 expression levels, restores motor function, and significantly extends lifespan in MDS mouse models.
Understanding MECP2 Duplication Syndrome
MECP2 duplication syndrome (MDS) is a rare and severe neurodevelopmental disorder affecting children, with a prevalence rate of about 1 in 150,000 male newborns. Characterized by infant hypotonia, developmental delay, intellectual disability, and other symptoms, MDS currently has no effective treatment, with current methods focusing only on symptom management and supportive care.
Implications of RPDD Status
The RPDD status granted to HG204 means that the therapy is eligible for a Priority Review Voucher (PRV), which can be used for a 6-month accelerated review of any subsequent product launch application or can be sold to third parties. This designation not only highlights the potential of HG204 in treating MDS but also underscores the FDA’s commitment to encouraging the development of treatments for rare pediatric diseases.-Fineline Info & Tech
