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