Its current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies.

Sio, which is headquartered in New York City and is incorporated in Basel, Switzerland, is also expanding the reach of gene therapy to highly prevalent conditions such as Parkinson’s disease.