In vivo gene editing with YOLT-201 for transthyretin amyloid cardiomyopathy – an open-label, first-in-human clinical trial
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive and life-threatening disease driven by continuous hepatic production of misfolded transthyretin. Existing therapies slow progression but require chronic administration. YOLT-201 is an in vivo CRISPR-Cas9 gene-editing therapy delivered by lipid nanopa...