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Neurogene To Present Data Related To NGN-401 Gene Therapy Program For Rett Syndrome At ESGCT 33rd Annual Congress; New Preclinical Data Expected To Further Validate NGN-401’s Differentiated Design

Oct 27, 2026 · 07:32 AM ET· updated 2h ago

New data showing rapid and durable full-length MECP2 expression in vivo with single-stranded AAV support improvements observed in NGN-401 Phase 1/2 trialNew in vivo data demonstrate that miRNA-based auto-regulatory

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Why It Matters

Neurogene is showing new scientific information about their medicine for Rett Syndrome. When a company shares positive lab results, it can mean their treatment might work well.

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Source: Benzinga · Read the original report at the publisher. Headline and figures shown for context; data may be delayed.

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