WVE-120102

WVE-120102 is a stereopure antisense oligonucleotide (ASO) developed as an investigational, allele-selective therapy for Huntington's disease. It was designed to selectively bind and degrade the mutant huntingtin (mHTT) mRNA transcript associated with the single nucleotide polymorphism rs362331, thereby reducing production of the toxic mutant huntingtin protein while sparing the normal wild-type protein. This selective targeting aimed to slow disease progression by lowering levels of mHTT in patients carrying this specific SNP mutation, which is present in about 40% of Huntington’s patients. The drug was administered intrathecally and demonstrated dose-dependent exposure in plasma and cerebrospinal fluid. Clinical development was discontinued after Phase 1/2 trials due to lack of efficacy[1][3][4][5][6].