Gene therapy for Rett may be around the corner. https://rettsyndromenews.com/2021/02/12/novartis-seeking-approval-by-years-end-clinical-testing-rett-gene-therapy/ Novartis is trying to get an IND for OAV201, a gene therapy for Rett . I imagine the IND process for gene therapy is more complicated than for a small molecule. Some news from the https://rettnews.org/articles/outcomes-of-a-marathon-fda-meeting-on-the-safety-of-gene-replacement/ Rett Syndrome Research Trust. . They seem excited. Also another gene therapy is https://rettaustralia.org.au/blog/a-game-changer-for-gene-therapy-for-rett-syndrome/ also in the works . The Rett Society of Australia seems very excited.
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