NGNE: NGN-401 gene therapy drove durable, multidomain gains and increased independence in Rett syndrome

NGN-401 gene therapy in Phase 1/2 for Rett syndrome showed durable, multidomain developmental gains in all participants, with rapid onset and deepening effects over time. The therapy was well-tolerated, with no new safety concerns, and led to increased independence and reduced caregiver burden. Embolden trial dosing is complete, with topline data expected in 2H 2027.

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