• Neurogene said it has fully enrolled the Embolden registrational trial for NGN-401 in Rett syndrome, positioning the program for a potential path toward a BLA submission.
• The company reported that more than 50% of trial participants have been dosed, with completion of dosing targeted for Q2 2026.
• Neurogene said the FDA granted Breakthrough Therapy designation for NGN-401, which could support a faster regulatory review timeline if development milestones are met.
• The company said its commercial manufacturing scale matches its current clinical manufacturing scale, removing the need for comparability studies and potentially reducing execution risk ahead of commercialization.
• Neurogene reported USD 269.0 million in cash, cash equivalents and short-term investments, with an expected runway through Q1 2028.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Neurogene Inc. published the original content used to generate this news brief via EDGAR, the Electronic Data Gathering, Analysis, and Retrieval system operated by the U.S. Securities and Exchange Commission (Ref. ID: 0001404644-26-000012), on March 24, 2026, and is solely responsible for the information contained therein.