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.
2016 numbers: CNS orphan drugs growing
With 2016 numbers now available, the number of orphan drugs in development for neurological indications is looking quite positive. I have reviewed the numbers of orphan drug designations and approvals by FDA in 2016 to see how popular are neurological orphan drugs today and what the trend is for the near future.
How close are we to creating transgenic people?
The journal Nature just released one of the most anticipated breaking news of the last few years: CRISPR gene editing has been tested in a person for the first time. In my day-to-day work I interact with families that have a child with a genetic disease. I get one question a lot: how close are we to turn that discovery into a therapy for people with genetic diseases?