After the initial approvals in monogenic inherited diseases, the scope of gene therapy is widening, with new delivery routes, novel vectors, cell-specific targeting and products aiming to treat chronic disorders, all making headway in 2023.

Sania Therapeutics Inc. is setting out its stall at the American Society of Gene & Cell Therapy (ASGCT) conference in Los Angeles this week, after generating proof of concept for its chemogenetics approach to treating motor disorders. The company has engineered adeno-associated viral vectors that can be targeted to specific cell types. It will use these to deliver well-characterized ion channels to dysfunctional motor neurons. The ion channels will then be selectively controlled by an activating drug, which is taken orally.