nL-TARDB-006

nL-TARDB-006 is a personalized antisense oligonucleotide (ASO) developed by the n-Lorem Foundation for the treatment of amyotrophic lateral sclerosis (ALS) caused by a pathogenic variant in the TARDBP gene, which encodes the TDP-43 protein. As an individualized therapy, it is designed for a single patient whose ALS is driven by a specific TARDBP mutation. The drug is an experimental compound being evaluated in an open-label Phase 1/2 study to assess its safety and efficacy. It functions by targeting the mutant TARDBP mRNA to modulate the production of the TDP-43 protein, which is central to the pathology of certain forms of ALS.