SEN-401研究更新后，Neurogene股价暴跌36%

Neurogene (NASDAQ:NGNE) shares plunged 36% Monday after the company issued an update on a Phase 1/2 study of its gene therapy NGN-401 for Rhett syndrome.

Neurogene (NGNE) said that a patient who had been previously identified as developing a treatment-related serious adverse event, or SAE, is now in critical condition. The patient, who had received a 3E15 vg dose of NGN-401, experienced systemic hyperinflammatory syndrome, a rare and life-threatening immune response that has been associated with high doses of AAV.

As a result, Neurogene (NGNE) is pausing further use of NGN-401 at the higher 3E15 vg dose. After conducting a safety review, the FDA is allowing the company to proceed with the study using the lower 1E15 vg dose, according to a statement.

The company added that it no longer expects to complete enrollment in the 1E15 vg cohort in Q4 as it updates the protocol to reflect the discontinuation of the 3E15 vg dose.