Second Human Tumor Links to Integrated AAV Vector Sequence

Company FBX-101 investigators (NEJM correspondence) Event Type Peer-Reviewed Safety Report (NEJM) Modality AAV gene therapy (recombinant AAV vector; with cord-blood transplantation) Asset FBX-101 Target GALC (transgene); GLI1 activation by integrated vector sequence Disease Area Krabbe disease; AAV gene therapy safety Summary A peer-reviewed New England…