The startup has partnered with Eli Lilly and enjoys the backing of some of Silicon Valley's most influential VCs.

Nature Medicine, Published online: 16 January 2026; doi:10.1038/s41591-025-04073-zAnalyses of liver biopsies from a child with spinal muscular atrophy treated with adeno-associated virus gene therapy who developed hepatitis reveal contaminating manufacturing plasmids and disrupted vector genomes, possibly resulting from recombination events.