nL-UBTF-001

nL-UBTF-001 is a personalized antisense oligonucleotide (ASO) developed by the n-Lorem Foundation for the treatment of Childhood-Onset Neurodegeneration with Brain Atrophy (CONDBA). CONDBA is an ultra-rare, progressive neurological disorder caused by pathogenic gain-of-function mutations in the UBTF gene, most commonly the c.628G>A (p.Glu210Lys) variant. The UBTF gene encodes the Upstream Binding Transcription Factor (UBF), which is essential for RNA Polymerase I-mediated ribosomal RNA (rRNA) transcription. nL-UBTF-001 is designed to specifically target and reduce the expression of the mutant UBTF mRNA to mitigate the neurodegenerative effects of the gain-of-function mutation. It is currently being evaluated in a single-participant (n-of-1) Phase 1/2 clinical trial.