LTX-002

LTX-002 is an investigational antisense oligonucleotide (ASO) therapy developed by Leal Therapeutics for the treatment of neurodegenerative diseases, primarily amyotrophic lateral sclerosis (ALS), as well as Alzheimer’s disease, Parkinson’s disease, and peripheral nervous system diseases. The drug specifically targets the SPTLC1 gene transcript to inhibit production of serine palmitoyltransferase long chain base subunit 1 (SPTLC1), a key enzyme in sphingolipid biosynthesis. By reducing SPTLC1 expression, LTX-002 aims to lower toxic sphingolipid accumulation implicated in ALS and other neurological disorders. Preclinical studies have demonstrated target engagement, safety, and reduction of pathogenic lipid species in relevant models[1][2][3][4].