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Lysosomal storage disease gene therapy - Avigen

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Drug Profile

Lysosomal storage disease gene therapy - Avigen

Latest Information Update: 07 Sep 2005

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At a glance

  • Originator Avigen
  • Developer Avigen; Johns Hopkins University
  • Class Gene therapies
  • Mechanism of Action Enzyme replacements; Gene transference
  • Orphan Drug Status

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

    No

Highest Development Phases

  • Discontinued Fabry's disease; Gaucher's disease; Tay-Sachs disease

Most Recent Events

  • 27 Jun 2005 Discontinued - Preclinical for Fabry's disease in USA (unspecified route)
  • 27 Jun 2005 Discontinued - Preclinical for Gaucher's disease in USA (unspecified route)
  • 27 Jun 2005 Discontinued - Preclinical for Tay-Sachs disease in USA (unspecified route)

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