Clinical Trial SuccessTSHA's clinical data showed a 100% responder rate in developmental milestones, suggesting potential positive outcomes for Neurogene Inc.'s similar trials.
Regulatory SupportThe FDA remains flexible on trial designs, which positively affects Neurogene Inc.'s approach to their own pivotal trial.
Safety ProfileThe selected low dose of NGN-401 is significantly safer compared to the high dose, offering a favorable safety profile.