Mapping AAV gene therapies to diseases

The pipeline of clinical-stage gene therapies based on AAV vectors now numbers at least 74, with trials spanning eight major disease areas. More than half the candidates concentrate in neurological and ophthalmic indications, though a few standout indications with growing competition extend to other disease areas.

A snapshot of today’s in vivo gene therapy pipeline, taken from BioCentury’s BCIQ database, shows a modality in flux, making both lateral moves into more rare, genetic diseases and larger leaps to into prevalent and non-genetic diseases. AAV delivery still dominates in vivo gene therapies and BioCentury’s analysis focused on this class...