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NCATS, FDA Co-Host Workshop on Gene Therapy for Rare Diseases

“We are now at the point of translating the potential of gene therapy — which has been around for a while — into a reality for patients,” said NCATS Director Christopher P. Austin, M.D.

These new advances in gene therapies have truly been a long time in coming. The first human study that involved gene therapy, a type of treatment to replace or fix a defective gene in a patient, began in 1990 at the NIH Clinical Center. Nearly 30 years later, in 2017, the Food and Drug Administration (FDA) approved the first three gene therapies for use in the U.S. One of those three is for an inherited disease; the others alter a patient’s own cells to fight two forms of cancer.

NCATS, FDA Co-Host Workshop On Gene Therapy For Rare Diseases