BEHIND-THE-SCENES OF GENE THERAPY
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 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.
1. Roberts SA, Dong B, Firrman JA, Moore AR, Sang N, Xiao W. Engineering Factor VIII for Hemophilia Gene Therapy. J Genet Syndr Gene Ther. 2011;1:S1-006.
2. Data on file. Pfizer Inc, New York. NY.
3. Thomas CE, Ehrhardt A, Kay MA. Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet. 2003;4(5):346-358.
4. NIH Genetics Home Reference (GHR). Help Me Understand Genetics: Gene Therapy. https://ghr.nlm.nih.gov/primer/therapy.pdf. Accessed February 10, 2020.
5. National Institutes of Health (NIH). How Does Gene Therapy Work? https://ghr.nlm.nih.gov/primer/therapy/procedures. Accessed February 10, 2020.