Severe Toxicity Reported in High-Dose AAV Gene Therapy in Animals

ISTOCK, EUGENEONLINE

Researchers reported severe, life-threatening toxicity in nonhuman primates and piglets treated with high-dose, adeno-associated virus (AAV9) gene therapy this Monday (January 29) in Human Gene Therapy. The news caused stocks of several leading biotech companies to tumble.

James Wilson, a molecular biologist at the University of Pennsylvania, and his colleagues found that an AAV9 variant (AAVhu68) used to deliver a human gene to spinal cord motor neurons in rhesus monkeys and piglets resulted in severe toxicity affecting the liver and motor neurons. Symptoms began appearing within five days of administering the gene therapy. One monkey and three piglets had to ...