Neurogene jumps as FDA approves rare gene therapy trial design

Shares of drug developer Neurogene NGNE.O rise 35% to $20.23

Late on Monday, co said it received FDA approval for the design of its trial testing the NGN-401 gene therapy for Rett syndrome in females aged 3 and older

Rett syndrome is a rare genetic disorder that causes the brain to stop developing normally after early infancy, leading to a gradual loss of movement, speech, and coordination skills

The trial will be a single-arm, open-label study using patients as their own controls

Co said the trial will enroll about 18 participants, with the main results measured 12 months after treatment

Co has extended its cash runway into early 2028 with $292.6 million available as of March 2025

Including session's moves, stock down 12% YTD