Progress & Impact

Gene therapy saved Connie Burnett-West from a grim lung cancer diagnosis in 1999 – she had no other options. Today she remains cancer free.

Five years ago, another lung-cancer patient, also near the end of life, participated in a trial at the University of Miami using a gene therapy vaccine that boosts the impact of chemotherapy, without side effects, and she is still in remission.


1990 FDA approves first gene therapy study on a young patient treated for ADA-SCID.
2010 Three patients dying of leukemia, including a child, now in remission because of a clinical trial at the University of Pennsylvania, based on research funded by ACGT.
2012  First gene therapy drug, Glybera, approved by the European Medicines Agency, in review by FDA.
2013  Human Gene Therapy magazine names 4 ACGT scientists top pioneers in cell and gene therapies.
2014  Over 1,000 gene therapy trials completed or ongoing worldwide.