nL-CHCHD-001

nL-CHCHD-001 is a personalized antisense oligonucleotide (ASO) drug developed for the treatment of amyotrophic lateral sclerosis (ALS) caused by ultra-rare, pathogenic mutations in the CHCHD10 gene. The CHCHD10 gene encodes a protein critical for mitochondrial stability and function; mutations result in toxic protein accumulation that damages neurons, leading to ALS and related disorders such as frontotemporal dementia. nL-CHCHD-001 works by binding to the messenger RNA of CHCHD10, promoting its degradation and thereby reducing levels of the mutant protein. This approach aims to slow or halt disease progression in patients with these specific genetic forms of ALS. The drug is being developed under the Silence ALS initiative led by n-Lorem Foundation, with support from Columbia University and funding from NIH's URGenT program[1][6][7][9].

Other names

ASO for CHCHD10