Genetic Engineering & CRISPR

Gene editing has crossed into the clinic with Casgevy (exa-cel) approved for sickle cell disease and beta-thalassemia, and in vivo programs from Verve Therapeutics (VERVE-102 for hypercholesterolemia) and Intellia (NTLA-2001 for ATTR amyloidosis, NTLA-2002 for HAE) reporting durable knockdown with single LNP-delivered doses. The track will dissect base editing (David Liu lab, Beam Therapeutics), prime editing (Prime Medicine), epigenetic editing, and the next-generation delivery toolbox spanning lipid nanoparticles, engineered AAV capsids, and virus-like particles. Off-target detection, immunogenicity, and regulatory frameworks for heritable edits will receive dedicated sessions.