SRNA-001

SRNA-001 is an investigational, RNA-targeted small molecule being developed by Serna Bio (formerly Ladder Therapeutics) for the treatment of amyotrophic lateral sclerosis (ALS). SRNA-001 is designed to target and modulate the splicing of UNC13A pre-mRNA. In sporadic ALS, the loss of nuclear TDP-43 leads to aberrant splicing of UNC13A, resulting in the inclusion of a cryptic exon, nonsense-mediated decay of the transcript, and subsequent loss of UNC13A protein, which is critical for synaptic transmission. By binding directly to the target RNA, SRNA-001 aims to correct this splicing defect, restore functional UNC13A expression, and preserve motor neuron function. The program is currently in preclinical development.