YOLT-201

YOLT-201 is a first-in-class, in vivo gene-editing therapy developed by YolTech Therapeutics for the treatment of hereditary transthyretin amyloidosis (ATTR), including both polyneuropathy (ATTR-PN) and cardiomyopathy (ATTR-CM) subtypes. The drug uses lipid nanoparticles (LNPs) to deliver mRNA encoding a CRISPR-Cas editor and a single-guide RNA targeting the TTR gene specifically to liver cells, where transthyretin protein is produced. Once inside hepatocytes, the CRISPR system edits the TTR gene, preventing its transcription and halting production of the pathogenic TTR protein. This approach aims for a one-time administration that results in durable reduction of serum TTR levels and potentially halts disease progression. Clinical trials have shown robust reductions (>90%) in circulating TTR after single-dose infusion with favorable safety profiles[1][2][4][5][6].