Amicus Therapeutics Gets Orphan Drug Designation for Treatment of CLN3 Batten Disease - (Fidelity via NewsPoints Desk)

  • Amicus Therapeutics' adeno-associated virus (AAV) serotype 9 containing the human CLN3 gene for the treatment of CLN3 Batten disease has been granted orphan drug status by the FDA, as reported by Fidelity.

  • Early this year, the company initiated a Phase I/II trial testing a single intrathecal administration of serotype 9 AAV9-CLN3 gene therapy in children with CLN3 Batten disease.

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