Cell and Gene Therapy: In Vivo Editing and Approvals Accelerate

Multiple gene therapies advancing: AMT-130 for Huntington (75% progression reduction), lonvo-z for HAE (87% attack reduction), deramiocel for DMD (PDUFA Aug 22), TSHA-102 for Rett (Phase 3 enrolled). In vivo gene editing scaling with 140+ trials; CRISPR-armed phage therapy shows success. New: PTC acquires Fabry gene therapy ST-920, CRISPR screen identifies roadblocks to nonviral editing. Safety concerns highlighted by Stanford expert, warning of two deaths in China trials and need for rigorous preclinical work.