UBE3A antisense oligonucleotide (gapmer)

UBE3A antisense oligonucleotide (gapmer) is a preclinical-stage antisense oligonucleotide (ASO) therapeutic designed to target and knock down the overexpression of the UBE3A gene (encoding ubiquitin-protein ligase E3A) for the treatment of chromosome 15q duplication (Dup15q) syndrome. Developed by Quiver Bioscience (formerly Q-State Biosciences), the therapeutic utilizes a gapmer design—a central DNA core flanked by modified RNA wings—to hybridize with UBE3A pre-mRNA or mRNA, recruiting endogenous RNase H to selectively degrade the target transcript. By reducing excess UBE3A protein levels, the ASO aims to rescue patient-specific cellular phenotypes, alleviate symptoms such as hypotonia, developmental delays, intellectual disability, and autism spectrum disorder, and control intractable seizures associated with Dup15q syndrome.

Other names

UBE3A ASOUBE-3A ASOUBE 3A ASOUBE3A antisense oligonucleotideUBE-3A antisense oligonucleotideUBE 3A antisense oligonucleotideUBE3A gapmer ASOUBE-3A gapmer ASOUBE 3A gapmer ASOUBE3A-targeting ASOUBE-3A-targeting ASOUBE 3A-targeting ASOanti-UBE3A gapmeranti-UBE-3A gapmeranti-UBE 3A gapmer