retinal penetrating adeno-associated virus

Gene delivery to the outer retina, i.e. the photoreceptors can generally only be achieved via the subretinal route of injection, i.e. a surgical procedure.

Millions of people receive intravitreal injection of drugs as an office procedure every 6 to 8 weeks and such injections are considered safe.

We have developed an adeno-associated virus that can delver genes to the outer retina via an intravitreal injection.

Development of this technology has the potential for wide scale application of retinal gene therapy as an office procedure.

In figure above, one can see GFP expression in the outer retina following intravitreal injection with a traditional AAV (AAV9CAGGFP) as well as our patented AAV-IKV and AAV-IKV in combination with our patented chaperone Nuc1