Clinical trials

Gene therapy for Dravet syndrome – 2020 update

Gene therapy for Dravet syndrome – 2020 update

There are multiple gene therapy and oligonucleotide programs in development for Dravet syndrome including those that supply and extra copy of the SCN1A gene and those that boost expression from the healthy SCN1A gene copy. Clinical trials have already started, with Stoke Therapeutics initiating the first clinical trial with a disease-targeting therapy in Dravet syndrome in summer 2020. Behind Stoke, gene therapies are approaching the clinic with Encoded Therapeutics having the most advanced clinical candidate and preparing for trials in 2021.

What marihuana teaches us about drug discovery

What marihuana teaches us about drug discovery

There was one clear star at the American Epilepsy Society Annual meeting this year, Epidiolex (cannabidiol). Yet four years ago no one would have thought that the cannabis-derived drug would take over the field of epilepsy in such a big way.