RZ-005

RZ-005 is a preclinical RNA editing therapy developed by Rznomics for the treatment of Rett Syndrome, a rare neurodevelopmental disorder caused by mutations in the MECP2 gene. The drug utilizes Rznomics' proprietary trans-splicing ribozyme platform, which functions as an "RNA replacement enzyme." This technology is designed to specifically recognize and cleave mutant Methyl-CpG binding protein 2 (MECP2) mRNA and replace the defective sequence with a functional wild-type MECP2 transcript through a trans-splicing reaction. By editing the RNA rather than the genome, RZ-005 aims to restore functional MECP2 protein levels in a regulated manner, potentially avoiding the toxicity associated with MECP2 overexpression while addressing the underlying genetic cause of the disease.

Other names

RZ-005-RznomicsRZ005-RznomicsRZ 005-Rznomics