The FDA has awarded U.S. orphan drug designation and rare pediatric disease designation to Huidagene Therapeutics Co. Ltd.’s HG-204 (AAV-hfCas13Y-gMECP2), an RNA editing therapy based on CRISPR/Cas13Y, for the treatment of methyl-CpG binding protein 2 (MeCP2) duplication syndrome (MDS).

Ionis Pharmaceuticals Inc. has announced its intention to advance four new wholly owned neurology medicines into the clinic by the end of next year.

The neurodevelopmental disorder MECP2 duplication syndrome (MDS) is caused by duplications spanning the methyl-CpG binding protein 2 gene (MECP2) locus, and researchers have shown that the MDS-like phenotype can be reversed in adult symptomatic mice using MECP2-specific antisense oligonucleotides (MECP2-ASOs).