Neurogene Provides Update on NGN-401 Gene Therapy Clinical Trial for Rett Syndrome
Neurogene Inc. (NGNE)
Company Research
Source: Business Wire
NEW YORK--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced an update on its ongoing Phase 1/2 open-label clinical trial evaluating NGN-401 gene therapy for the treatment of Rett syndrome.As previously disclosed, on November 11, 2024, Neurogene became aware of an emerging treatment-related serious adverse event (SAE) in a trial participant who received NGN-401 at a dose of 3E15 vg (high-dose cohort). This participant, who was dosed on November 5, subsequently experienced signs of a systemic hyperinflammatory syndrome, a rare and life-threatening immune response that has been reported with systemic exposure to high doses of AAV. Hyperinflammatory syndromes are associated with aberrant cytokine release and include hemophagocytic lymphohistiocytosis (HLH) and multisystem inflammatory syndrome. The participant is in critical condition
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- Neurogene Inc. (NASDAQ: NGNE) had its price target lowered by analysts at BMO Capital Markets from $60.00 to $45.00. They now have an "outperform" rating on the stock.MarketBeat
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