YOLT-202

YOLT-202 is an investigational in vivo gene-editing therapy developed by Yoltech Therapeutics for the treatment of alpha-1 antitrypsin deficiency (AATD). It utilizes a proprietary adenine base editor (YolBE) delivered via lipid nanoparticles (LNPs). The therapy is designed to precisely correct the E342K (PiZ) mutation in the SERPINA1 gene, which is the most common cause of AATD. By performing a precise adenine-to-guanine conversion at the target site, YOLT-202 aims to restore the production of functional alpha-1 antitrypsin (AAT) protein while simultaneously reducing the accumulation of misfolded mutant Z-AAT protein aggregates in the liver. This approach addresses both the lung manifestations (emphysema) and liver manifestations (cirrhosis) of the disease, potentially offering a one-time treatment for lifelong benefit.