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Research programme: gene therapies - Neurogene Inc

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Drug Profile

Research programme: gene therapies - Neurogene Inc

Alternative Names: AAV gene therapies - Neurogene Inc; AAV-based gene therapies - Neurogene Inc; Adeno-associated virus gene therapies - Neurogene Inc; AGU gene therapy - Neurogene Inc; CMT4J gene therapy - Neurogene Inc; Neurological disorders - Neurogene Inc

Latest Information Update: 28 Aug 2023

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At a glance

  • Originator Neurogene Inc
  • Developer Neurogene Inc; The Jackson Laboratory; University of Texas Southwestern Medical Center
  • Class Gene therapies
  • Mechanism of Action Aspartylglucosylaminase replacements; Gene transference; MFSD8 protein replacements; Phosphoinositide 5-phosphatase replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Neuronal ceroid lipofuscinosis; Aspartylglucosaminuria
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Preclinical Globoid cell leukodystrophy
  • No development reported Aspartylglucosaminuria; Charcot-Marie-Tooth disease; Lysosomal storage diseases; Neuronal ceroid lipofuscinosis

Most Recent Events

  • 28 Aug 2023 No recent reports of development identified for preclinical development in Neuronal ceroid lipofuscinosis in USA (Parenteral)
  • 28 Mar 2023 No recent reports of development identified for preclinical development in Aspartylglucosaminuria in USA (Intrathecal)
  • 28 Mar 2023 No recent reports of development identified for preclinical development in Aspartylglucosaminuria in USA (IV)

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