Neurogene Presents New Data at ESGCT Congress that Reinforce Key Differentiators of NGN-401 Gene Therapy for Rett Syndrome
Business Wire•27/10/2026•07:30 ET
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Key Highlights
- ➤NGN-401 achieved full-length MeCP2 expression across evaluated tissues by Day 7.
- ➤Phase 1/2 participants showed improvement at median two months, continuing through 30 months.
- ➤miRNA-based regulation produced comparable expression in MeCP2-null and MeCP2-expressing neurons.
- ➤ICV delivery showed broader CNS biodistribution than intrathecal lumbar administration.
- ➤ICV administration was well-tolerated in 10 participants receiving the 1E15 vg dose.
Expert Statements
Rachel McMinn, Founder and Chief Executive Officer of Neurogene
“Two widely held assumptions in Rett syndrome gene therapy are that single-stranded AAV leads to slower expression than self-complementary AAV and that miRNA-based transgene auto-regulatory technology can selectively regulate expression based on a cell’s MeCP2 levels.”
Rachel McMinn, Founder and Chief Executive Officer of Neurogene
“Our preclinical studies were designed to directly and rigorously test those assumptions.”
Rachel McMinn, Founder and Chief Executive Officer of Neurogene
“In these studies, we found that NGN-401's single-stranded design produced rapid and durable expression of the fully functional, full-length MECP2 gene, which we believe is consistent with the early and sustained clinical improvements observed in our Phase 1/2 trial.”
Rachel McMinn, Founder and Chief Executive Officer of Neurogene
“Additional evidence generated from the preclinical studies demonstrated that miRNA-based auto-regulatory technology is not responsive to endogenous MeCP2 levels on a cell-by-cell basis but rather dampens transgene expression regardless of MeCP2 levels.”
Neurogene Presents New Data at ESGCT Congress that Reinforce Key Differentiators of NGN-401 Gene Therapy for Rett Syndrome
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 trial
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