Lysogene's lead gene therapy flunks phase 2/3 trial almost a year after Sarepta walked out

The company’s top pipeline candidate, LYS-SAF302, failed its phase 2/3 trial among patients with mucopolysaccharidosis type IIIA (also known as Sanfilippo syndrome), a neurodegenerative disease that can spur a wide range of bodily dysfunction, including infections, dementia, sleep disturbances and propensity to chew on objects. Much of the symptoms begin to appear in early childhood.