Neurogene Announces Upcoming Presentations Highlighting Key Differentiators of NGN-401 for Rett Syndrome at the ESGCT 33rd Annual Congress
Business Wire•20/10/2026•07:30 ET
0
Key Highlights
- ➤ESGCT presentations scheduled October 27-30, 2026, in Hamburg, Germany
- ➤NGN-401 data will examine rapid full-length MECP2 expression after single-stranded AAV delivery
- ➤Preclinical studies will compare miRNA-based approaches regulating MeCP2 expression cell by cell
- ➤Phase 1/2 data showed early clinical benefit signals and durable multidomain gains
- ➤NGN-401 at 1E15 vg was generally well-tolerated as of June 16, 2026
Neurogene Announces Upcoming Presentations Highlighting Key Differentiators of NGN-401 for Rett Syndrome at the ESGCT 33(rd) Annual Congress
New presentations to highlight rapid transgene expression of full-length MECP2 following single-stranded AAV delivery and evaluation of miRNA-based approaches to regulating MeCP2 expression on a cell-by-cell basis
Get started
Create a free account to read the full story.