Neurogene Shares Crumble After Adverse Event in Rett Syndrome Gene Therapy Trial

Neurogene experienced a dramatic 36% decrease in its share price following the announcement of a severe adverse event in its Phase I/II Rett syndrome gene therapy trial. The troubling incident involved a female patient who developed systemic hyperinflammatory syndrome after receiving a high dose of the NGN-401 therapy, a reaction associated with the adeno-associated virus (AAV) vector used in the gene delivery system[1]. Previously, the company's share price had already fallen by 35% when the adverse event was first mentioned without full details, with further declines after the revelation of the patient's critical condition[1]. Despite lower dose treatments showing promising results and being well-tolerated in initial patients, the adverse event at the higher dosage has raised significant safety concerns, overshadowing the potential therapeutic benefits[2].