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Neurogene to share clinical insights on AAV9-linked HLH and Rett Syndrome Therapy at ASGCT 2025
Neurogene Inc. , a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced an oral presentation and participation in a fireside chat and panel discussion on critical gene therapy topics during the American Society for Gene and Cell Therapy (ASGCT) 28th Annual Meeting on May 13-17, 2025 in New Orleans. The oral presentation will include approaches for monitoring and managing hemophagocytic lymphohistiocytosis (HLH), a rare, severe hyperinflammatory syndrome that has been associated with systemic exposure to high doses of adeno-associated virus (AAV) gene therapy.
“Given the limited published research on HLH related to gene therapy, we believe it is important to provide information to the AAV gene therapy community on effective approaches to monitor, treat and reverse cases of HLH,” said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “In our ongoing clinical trial of NGN-401 gene therapy for Rett syndrome, we have implemented early monitoring and a treatment plan into our protocol, and we encourage others to do the same.”
The 1E15 vg dose level Neurogene is moving forward in its Phase 1/II trial of NGN-401 gene therapy for Rett syndrome translates into the E13 vg/kg range, and the Company is not aware of any case of HLH ever being reported at this dose level.
Oral Presentation Details
- Title: Hemophagocytic Lymphohistiocytosis (HLH)/Hyperinflammatory Syndrome Following High Dose AAV9 Therapy
- Session: Clinical Trial Spotlight Symposium
- Date: Friday, May 16
- Abstract Presentation Time: 3:00 p.m. CT
- Location: NOLA Theater C
Dr. McMinn will also participate in the following ASGCT sessions:
- Title: Fireside Chat: Reviving Hope in Deprioritized Cell and Gene Therapy Programs
- Date: Wednesday, May 14
- Time: 3:45 p.m. CT
- Location: Room 393-396
- Title: FDA’s START Pilot Program in Action: Insights from Year One
- Date: Thursday, May 15
- Time: 3:45 p.m. CT
Location: Room 393-396
