There is no doubt that AAVs will support the development of many gene therapies for genetic diseases, but for many diseases AAVs are too small to carry a copy of the gene that patients need. There is a strong need to find non-AAV alternatives that can provide a suitable gene therapy option for those diseases caused by mutations in large genes. Within Dravet syndrome, these next-generation therapies are being led not by companies but by academic groups with the support of patient organizations. This is a review of these programs and how they are attempting to develop a gene therapy for treating Dravet syndrome.

we expect in 2018 the first approval of a treatment for Dravet syndrome in the US, the results of clinical trials with three new therapeutics (ZX008, ataluren and OV935/TAK935), and the initiation of the first clinical trial ever done with a disease-modifying therapy designed to treat Dravet syndrome (OPK88001). 2018 is going to be a good year.