Researchers have obtained the first signs of clinical benefit in an early-stage gene therapy trial for Tay-Sachs disease, according to a presentation at the European Society of Gene & Cell Therapy (ESGCT) annual conference last week.
Terence R. Flotte, MD, executive deputy chancellor, provost, and dean of the University of Massachusetts (UMass) School of Medicine, presented the results in Barcelona. Flotte is also the editor-in-chief of Human Gene Therapy (a sister journal of GEN).
“Buckle your seatbelts,” commented Fyodor Urnov, PhD, a gene therapy expert at the Innovative Genomics Institute, UC Berkeley, on Twitter. “A gene therapy early-stage success for Tay-Sachs!!!”
Urnov said: “Flotte has long been an inspiration and a leader for the field, and this is just MAGNIFICENT. Tay-Sachs is devastating—but perhaps for not much longer?”
Tay-Sachs is an incurable recessively inherited pediatric genetic disease, a member of a group of lysosomal storage diseases, which is particularly common in individuals of Ashkenazi Jewish descent. Patients have a median life expectancy of approximately three to four years.
