Industry News Archives | International Rett Syndrome Foundation

January 12, 2026

Neurogene Announces Recent Achievements and Anticipated 2026 Key Milestones for NGN-401, a Potential Best-in-Class Gene Therapy for Rett Syndrome

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Neurogene

Neurogene Announces Recent Achievements and Anticipated 2026 Key Milestones for NGN-401, a Potential Best-in-Class Gene Therapy for Rett Syndrome

Taysha

Taysha Gene Therapies Updates on REVEAL & ASPIRE Studies (Community Letter)

Taysha

Taysha Gene Therapies Announces Progress Across TSHA-102 Pivotal Gene Therapy Program in Rett Syndrome

Acadia

Acadia Pharmaceuticals Announces FDA Approval of DAYBUE® STIX (trofinetide) for Oral Solution, a New Powder Formulation of Trofinetide for the Treatment of Rett Syndrome

Unravel

Unravel Biosciences Announces Approval by Colombian Health Regulatory Authority to Initiate RVL-001 Clinical Studies for Rett Syndrome and Pitt Hopkins Syndrome

Neurogene

Neurogene Reports Positive Interim Data in Pediatric Cohort from NGN-401 Gene Therapy Trial for Rett Syndrome

Neurogene

Neurogene Shares Enrollment Locations and Information for Embolden™ Registrational Trial of NGN-401 Gene Therapy for Rett Syndrome (Community Letter)

Neurogene

Neurogene Announces First Participant Dosed in Embolden™ Registrational Trial of NGN-401 Gene Therapy for Rett Syndrome

Neurogene

Neurogene Announces Positive Regulatory Update for NGN-401 Gene Therapy in Rett Syndrome with Plans to Initiate Dosing in Embolden™ Registrational Trial in Q4 2025