Gene therapy has demonstrated enormous potential for
changing how
we combat disease. By directly engineering the genetic composition
of cells, it provides a broad range of options for improving human
health. Adeno-associated viruses (AAVs) represent a leading gene therapy
vector and are expected to address a wide range of conditions in the
coming decade. Three AAV therapies have already been approved by the
FDA to treat Leber’s congenital amaurosis, spinal muscular
atrophy, and hemophilia B. Yet these therapies cost around $850,000,
$2,100,000, and $3,500,000, respectively. Such prices limit the broad
applicability of AAV gene therapy and make it inaccessible to most
patients. Much of this problem arises from the high manufacturing
costs of AAVs. At the same time, the field of synthetic biology has
grown rapidly and has displayed a special aptitude for addressing
biomanufacturing problems. Here, we discuss emerging efforts to apply
synthetic biology design to decrease the price of AAV production,
and we propose that such efforts could play a major role in making
gene therapy much more widely accessible.