Abeona Therapeutics has received permission to test out its gene therapy on a small group of patients with a fatal rare disease.
The trial will involve six-to-nine patients with Sanfilippo Syndrome Type A. Children with the condition have a genetic defect that prevents them from properly breaking down certain sugars, which leads to muscular and cognitive decline.
Abeona aims to deliver a functional version of that gene into their bodies in one dose, through an IV. The gene would then be carried into their cells via a common virus.
Articles originally published February 29 and March 1, 2016.