REGENXBIO 1 article

The genetic-medicine profile of REGENXBIO combines AAV gene therapy with NAV technology platform, emphasizing gene addition or replacement rather than direct genome editing. Its platform also uses AAV vector engineering and microdystrophin gene delivery, while retinal gene therapy development explains how vector design, route of administration or tissue tropism supports expression of the therapeutic gene. GenoMethods maps vectors, transgenes and individual gene therapies separately; the genetic-medicine profile of REGENXBIO also includes rare genetic disease therapeutics.