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pp-215

Solution for injection

Dossier type
US CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Rett syndrome — severe X-linked neurodevelopmental disorder caused by MECP2 loss-of-function mutations Clinical stage: Phase 2/3 Modality: Biological (AAV9 gene therapy delivering MECP2 gene with auto-regulatory miRNA — NBE) Mechanism / Target: AAV9 intrathecal vector delivering functional MECP2 transgene with embedded auto-regulatory miRNA sequence; miRNA senses endogenous MECP2 levels and self-limits transgene expression to prevent overexpression toxicity; restores MECP2 protein function in neurons; addresses root genetic cause of Rett syndrome Route / form: Intrathecal injection (single-dose, one-time treatment) Differentiation: Only MECP2 gene therapy with built-in auto-regulatory dosage control (prevents overexpression-related toxicity seen in other MECP2 gene therapy attempts);
Manufacturer #39351
US-based clinical-stage gene therapy company developing Rx treatments for serious neurological disorders with high unmet need. Lead program uses a novel regulated gene-therapy construct and is advancing through clinical trials. Focused on pediatric and adult neurological conditions.

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