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.
Neurogene announced 11 November safety data from seven patients, including five who received the low dose of 1E15vg and two...