Lysogen

Lysogene was a clinical-stage biotechnology company that pioneered the development of gene therapies for rare neurodegenerative diseases, particularly those affecting the central nervous system. Founded in 2009 by Karen Aiach following her daughter's diagnosis with Sanfilippo syndrome, the company focused on using adeno-associated virus (AAV) vectors to deliver functional genes directly to the brain. Its lead program, LYS-SAF302, reached a pivotal Phase 2/3 trial for the treatment of Sanfilippo syndrome type A, while LYS-GM101 targeted GM1 gangliosidosis. Despite significant clinical progress and a high-profile partnership with Sarepta Therapeutics, the company faced financial insolvency after the partnership's termination and entered liquidation in 2023. Its assets and intellectual property were subsequently acquired by the biotech firm EG 427 and the non-profit Généthon.