nL-ASXL3-001

nL-ASXL3-001 is an experimental antisense oligonucleotide (ASO) developed by the n-Lorem Foundation for the treatment of Bainbridge-Ropers syndrome (BRS). BRS is an ultra-rare neurodevelopmental disorder caused by mutations in the *ASXL3* gene, which is involved in epigenetic regulation and chromatin remodeling. As a personalized "n-of-1" therapy, nL-ASXL3-001 is designed to target the *ASXL3* mRNA transcript specifically for a single patient's genetic profile. The mechanism typically involves the use of a chemically modified oligonucleotide to bind to the target mRNA via Watson-Crick base pairing, either to trigger RNase H-mediated degradation of a toxic mutant transcript or to modulate splicing/expression to restore functional protein levels. Developed in collaboration with technology partners like Ionis Pharmaceuticals, this drug represents a novel approach to treating nano-rare genetic conditions where traditional commercial development is not feasible.

Other names

ASXL3 ASOASXL-3 ASOASXL 3 ASO