Neurogene’s Rett Data Draw Optimism Despite Serious Adverse Event

Analysts also looked into how much differentiation there was between data for the gene therapy NGN-401 and what Taysha has shown with TSHA-102 in the same disease.

Rett syndrome
• Source: Shutterstock

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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