YOLT-206

YOLT-206 is an investigational in vivo gene editing therapy developed by Yoltech Therapeutics for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). The therapy utilizes a lipid nanoparticle (LNP) delivery system to transport CRISPR/Cas9 gene-editing components to the liver. Once in the hepatocytes, the therapy is designed to permanently knock out or silence the *TTR* gene, thereby reducing the systemic production of transthyretin (TTR) protein. By lowering circulating TTR levels, YOLT-206 aims to prevent the formation and deposition of amyloid fibrils in cardiac tissue, potentially slowing or reversing the progression of cardiomyopathy in patients with both hereditary and wild-type ATTR.

Other names

YOLT 206YOLT206YOLT-206