PTC and Odylia join for gene therapy

Abstract

PTC Therapeutics, a rare-disease drug company best known for small-molecule therapies that alter gene expression, is partnering with Odylia Therapeutics to develop gene therapies for inherited eye diseases. Odylia is a nonprofit founded in 2017 to launch clinical trials based on the dozens of preclinical studies suggesting gene therapies can partially restore vision in inherited forms of blindness. In the partnership, PTC will gain access to a class of adeno-associated viruses called Anc80, which were made in Luk H. Vandenberghe’s lab at Massachusetts Eye and Ear.