Early data from Neurogene’s Phase I/II trial of NGN-401 in Rett syndrome look promising, but it remains unclear how differentiated they are from competitor Taysha Gene Therapy’s TSHA-102, being developed for the same disease. Meanwhile, a serious adverse event (SAE) occurred in the high-dose cohort of the NGN-401 trial, though the low-dose cohort seems to have dodged the issue while still showing encouraging efficacy.
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