Neurogene Maps NGN-401 Pivotal Rett Timeline, Mid-2026 Data Update at Guggenheim Summit

Neurogene's lead gene therapy program NGN-401 for Rett syndrome has entered a pivotal study, with data expected in mid-2026.

Published on Feb. 14, 2026

Got story updates? Submit your updates here. ›

Neurogene (NASDAQ:NGNE) is advancing a genetic medicines pipeline focused on rare neurologic diseases, with its lead program NGN-401 aimed at treating Rett syndrome. The company's President and CFO, Christine Mikail, provided an update on the NGN-401 pivotal study timeline and upcoming data readout at the Guggenheim Emerging Outlook Biotech Summit.

Why it matters

Rett syndrome is a devastating neurological disorder that primarily affects young girls, leading to profound impairments in hand use, communication, and motor function. Neurogene's NGN-401 gene therapy aims to address the underlying genetic cause of Rett syndrome and potentially restore some of these lost capabilities in patients.

The details

Mikail said NGN-401 has entered a pivotal study, with dosing beginning in Q4 2025 and enrollment and dosing expected to be completed in Q2 2026. Neurogene also plans to present updated Phase I/II results across 10 dosed patients with at least 12 months of follow-up in the middle of 2026. The company has highlighted the multi-domain and durable improvements seen in its earlier Phase I/II data, which showed an average of 4 skills gained per patient. Neurogene's 'biology-first' approach includes targeting key brain regions, using the full-length human MECP2 gene, and regulating transgene expression to avoid toxicity.

  • Dosing for the NGN-401 pivotal study began in Q4 2025.
  • Enrollment and dosing for the pivotal study is expected to be completed in Q2 2026.
  • Neurogene plans to present updated Phase I/II results across 10 dosed patients with at least 12 months of follow-up in the middle of 2026.

The players

Neurogene

A clinical-stage biotechnology company specializing in the development of gene therapies for rare neurological diseases.

Christine Mikail

The President and CFO of Neurogene.

Got photos? Submit your photos here. ›

What they’re saying

“Rett syndrome is a 'devastating neurologic disease' that primarily affects girls.”

— Christine Mikail, President and CFO, Neurogene (Guggenheim Emerging Outlook Biotech Summit)

“We must not let individuals continue to damage private property in San Francisco.”

— Robert Jenkins, San Francisco resident (San Francisco Chronicle)

What’s next

The judge in the case will decide on Tuesday whether or not to allow Walker Reed Quinn out on bail.

The takeaway

Neurogene's 'biology-first' approach to its Rett syndrome gene therapy program, including targeting key brain regions and regulating transgene expression, aims to deliver a best-in-class profile as the company advances NGN-401 into a pivotal study with data expected in mid-2026.