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FDA approval
Neurogene's NGN-401 Rett Syndrome therapy selected for FDA's START program
Cell & Gene Therapy
Jun 3, 2024
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Cell & Gene Therapy

Cell & Gene Therapy

Jun 3, 2024

Neurogene's NGN-401 Rett Syndrome therapy selected for FDA's START program

Product updates
FDA approval

  • Neurology-focused gene therapy company Neurogene has been selected to participate in the FDA's Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program with its gene therapy for Rett Syndrome, known as NGN-401. The program is designed to facilitate constant communication with the FDA to expedite the development process of NGN-401.

  • NGN-401 is an investigational gene therapy developed as a single treatment for Rett Syndrome, a rare neurological disorder currently being studied in a Phase I/II clinical trial.

  • Analyst QuickTake : Neurogene’s NGN-401 joins the FDA’s recent line of interest in treatments for Rett Syndrome. Since Acadia Pharmaceuticals’ DAYBUE (trofinetide) was approved by the FDA in March 2023 —marking the first-ever FDA-approved treatment for the disease—the regulatory progress for potential treatments for Rett Syndrome has seen an upward trajectory. Last week, Unravel Biosciences received Orphan Drug designation for RVL-001 (Vorinostat), which is expected to enter clinical trials in Q4 2024. Similarly, in August 2023 , Taysha Gene Therapies’ Phase I/II gene therapy, TSHA-102, received Fast Track designation. 

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