Neurogene Inc. announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation to its lead gene‑therapy candidate, NGN‑401, for the treatment of Rett syndrome.
The designation was based on interim Phase 1/2 data with a data cutoff of October 30 2025, showing clinically meaningful, durable, and multidomain functional improvements in patients and a favorable safety profile at the 1 × 10^15 vector genomes dose.
NGN‑401 delivers the full‑length human MECP2 gene using Neurogene’s proprietary EXACT™ transgene regulation technology and is administered via intracerebroventricular delivery, a strategy designed to address the underlying genetic defect in Rett syndrome.
The company is on track to complete dosing in its Embolden™ registrational trial in the second quarter of 2026, with additional interim Phase 1/2 data planned for presentation in mid‑2026.
NGN‑401 has also received Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA, and equivalent designations from the EMA and UK regulators, further supporting an expedited development pathway.
Neurogene is a clinical‑stage biotechnology company with no current revenue and significant net losses, but it maintains a strong balance sheet with substantial cash reserves that provide runway for ongoing clinical trials and development activities.
"The FDA's decision to grant Breakthrough Therapy designation validates the clinically meaningful, durable and multidomain functional improvements observed with NGN‑401, including continued skill acquisition observed over time, and underscores the significant unmet medical need of those living with Rett syndrome," said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene.
"We appreciate the Agency's continued engagement, including through our participation in the START Pilot Program and now our Breakthrough Therapy designation, to support the rapid advancement of NGN‑401," added McMinn.
"We are pleased to share that multiple participants have been dosed in our Embolden registrational trial of NGN‑401 gene therapy for Rett syndrome, an important step toward completing dosing in the second quarter," she added.
The announcement was well received by investors, reflecting confidence in the accelerated regulatory pathway and the potential of NGN‑401 to address a critical unmet need in Rett syndrome.
The content on EveryTicker is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.