BEHIND-THE-SCENES OF GENE THERAPY
Gene therapy represents the next wave of treatment innovation and holds promise for patients living with rare genetic diseases.
We are developing highly specialized treatments that use custom-made recombinant adeno-associated virus (AAV) vectors to deliver gene therapy directly to targeted cells. Currently, we are focusing on diseases caused by a single-gene alteration.
When the vector reaches the targeted cell, the functioning gene is transferred and used as a blueprint to produce the missing or non-functioning protein.
Interactive Look at Gene Therapy Manufacturing
Take a journey with us as we tour our Sanford, North Carolina gene therapy manufacturing facility and explore how gene therapies are made.
End-To-End Gene Therapy Capabilities
Please click on the image below to learn more about manufacturing.