STEP-CRISPR

STEP-CRISPR is a preclinical gene editing therapy developed by Couragene for the treatment of Angelman Syndrome (AS). The therapy utilizes Couragene's proprietary Stimuli-Responsive Traceless Engineering Platform (STEP) to deliver CRISPR-Cas9 ribonucleoprotein (RNP) complexes directly to the central nervous system. The therapeutic target is the UBE3A antisense transcript (UBE3A-ATS), a long non-coding RNA that silences the paternal copy of the UBE3A gene in neurons. By using CRISPR-Cas9 to disrupt the genomic sequence of UBE3A-ATS, the therapy aims to permanently reactivate the expression of the paternal UBE3A gene, thereby restoring functional UBE3A protein levels which are deficient in AS patients. Preclinical studies in mouse models have demonstrated that a single administration of STEP-CRISPR achieves brain-wide genome editing and persistent reactivation of Ube3a, leading to improvements in neurobehavioral phenotypes without significant off-target toxicity.

Other names

STEP-RNPSTEP (CRISPR RNP delivered via STEP technology)STEP-CRISPR-UBE3A-ATSSTEP-CRISPR-UBE-3A-ATSSTEP-CRISPR-UBE 3A-ATS