Gene Therapy Manufacturing: Process & Capabilities
Gene therapy represents the next wave of treatment innovation and holds tremendous 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.1,2,3 Currently, we are focusing on diseases caused by a single-gene alteration.2
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.4,5
Interactive Look at Gene Therapy Menufacturing
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.