Cell and Gene Therapy for Rare Diseases

Multiple gene therapies advancing: AMT-130 for Huntington (75% progression reduction), lonvo-z for HAE (87% attack reduction), deramiocel for DMD (PDUFA Aug 22), FLT201 for Gaucher type 1 (Phase 3 dosed), RP-A501 for Danon disease (safety update), and LCA5 gene therapy for retinal disease. In vivo CRISPR and epigenetic editing platforms gaining traction. New: St. Jude receives $28.5M ARPA-H grant for in vivo gene-editing in rare bone-marrow failure disorders.