ION717 (Ionis Pharmaceuticals)

ION717 is a 2'-O-(2-methoxyethyl) (MOE) modified antisense oligonucleotide (ASO) developed by Ionis Pharmaceuticals for the treatment of Pelizaeus-Merzbacher disease (PMD). PMD is a rare, X-linked leukodystrophy typically caused by duplications of the *PLP1* gene, leading to toxic overexpression of the proteolipid protein 1, which is essential for myelin formation in the central nervous system. ION717 is designed to bind to *PLP1* pre-mRNA and trigger its degradation via an RNase H1-dependent mechanism. By reducing the levels of PLP1 protein, ION717 aims to prevent the accumulation of toxic protein aggregates and restore oligodendrocyte function, potentially halting or reversing the progression of the disease. It is currently being evaluated in a Phase 1/2 clinical trial via intrathecal administration.

Other names

2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting PLP1 pre-mRNA