Neurogene

Neurogene disclosed a patient death in its Phase I/II Rett syndrome trial due to systemic hyperinflammatory syndrome from a high-dose gene therapy. The FDA allowed the trial to continue with a lower dose, and Neurogene plans to advance the therapy based on positive results at this dose. The company also suspended its Batten disease program.

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A clinical trial participant who received a high dose of Neurogene's experimental Rett syndrome gene therapy has died. The FDA is allowing the study to continue with the low dose, and Neurogene plans to incorporate this dose in future trial designs. The high dose has been paused due to immune response risks associated with AAV delivery.

A patient in Neurogene’s Rett syndrome trial died due to a rare hyperinflammatory syndrome linked to AAV-based therapies. The company paused the 3E15 vg dose and plans to use 1E15 vg in future trials.

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Neurogene paused high-dose NGN-401 AAV gene therapy for Rett syndrome after a participant experienced systemic hyperinflammatory syndrome. The FDA allowed continued low-dose cohort dosing, with mild AEs. Neurogene plans to update the protocol and no longer expects to complete low-dose enrollment by Q4 2024.

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A patient treated with Neurogene’s NGN-401 for Rett syndrome developed a life-threatening immune response, prompting Neurogene to halt high-dose treatments. The FDA allowed continued low-dose trials, where initial results showed meaningful improvements in patients. Neurogene is updating the trial protocol and adjusting enrollment expectations.

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