WVE-003

WVE-003 is an experimental, allele-selective antisense oligonucleotide therapy developed to treat Huntington's disease. It is designed to selectively bind and degrade mutant huntingtin (mHTT) messenger RNA containing the SNP3 mutation, thereby reducing production of the toxic mHTT protein while preserving levels of normal wild-type huntingtin protein. This selectivity aims to slow disease progression by preventing the accumulation of harmful protein aggregates in nerve cells without disrupting normal cellular function. WVE-003 is administered via intrathecal injection and has demonstrated significant reductions in mHTT levels in cerebrospinal fluid with a favorable safety profile in early clinical trials[1][2][3][4][5][6][7][8].

Other names

antisense oligonucleotide targeting rs149109767