Neurogene Presents New Data at ESGCT Congress that Reinforce Key Differentiators of NGN-401 Gene Therapy for Rett Syndrome
Neurogene presented new preclinical data at the ESGCT Congress showing that NGN-401's single-stranded AAV9 design achieved rapid, durable full-length MECP2 expression within seven days, sustained through three months in vivo. The data also challenge a competitor design assumption: miRNA-based auto-regulatory technology dampened transgene expression globally rather than selectively responding to a cell's endogenous MeCP2 levels, supporting NGN-401's EXACT-regulation differentiation.
Key figures
- Drug Name
- NGN-401
- Phase Of Trial
- Phase 1/2 (plus Embolden registrational trial)
- Dose
- 1E15 vg
- Data cutoff
- June 16, 2026
- Durability followup
- 30 months
- Median time to improvement
- two months post-treatment
- Phase12 participants at dose
- 10
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