The results come from five patients enrolled in the biotech's ingoing phase 1/2 study of RGX-111 – which uses an adeno-associated viral vector (AAV9) to deliver a gene coding for an enzyme that ...
A viral vector is a carrier developed to inject genetic ... rare pediatric diseases and will produce adeno-associated virus (AAV9) vectors to provide to Child's Cure. Child's Cure plans to develop ...