AAV Gene Therapies

We are accelerating the delivery of AAV gene therapies for central nervous system (CNS) disorders using next-generation science

The promise of gene therapy lies in its elegance: the ability to treat genetic diseases simply by replacing a defective gene with a working copy. It takes world-class thinkers, state-of-the-art science, and time. We have the thinkers and the science—time is the one component that our patients don’t have. So our mission is to responsibly accelerate delivery of these medicines.

Our next-generation science uses the leading platform for in vivo treatment of neurodegenerative diseases—vectors known as adeno-associated viruses (AAVs)—to deliver a functional copy of the gene to cells in both the central nervous system and the periphery. The gene delivered encodes for proteins that are secreted. The uptake of secreted protein in neighboring cells is referred to as cross-correction. This provides a potential expansion of benefits to cells beyond those directly transduced by gene therapy. There are potential risks associated with all gene therapies, including AAV-based therapies.