Research programme: eye disorder gene therapies - Applied Genetic Technologies Corporation/Biogen

Drug Profile

Research programme: eye disorder gene therapies - Applied Genetic Technologies Corporation/Biogen

Alternative Names: Age-related macular degeneration gene therapy- Applied Genetic Technologies Corporation; X-linked retinitis pigmentosa gene therapy - Applied Genetic Technologies Corporation; XLRP gene therapy - Applied Genetic Technologies Corporation

Latest Information Update: 16 Aug 2017

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At a glance

  • Originator Applied Genetic Technologies Corporation
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    Yes - Retinitis pigmentosa
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Age-related macular degeneration; Retinitis pigmentosa

Most Recent Events

  • 13 Sep 2017 AGTC plans a clinical study to evaluate the safety and efficacy of a proprietary gene therapy for X-linked retinitis pigmentosa (caused by mutations in the RPGR gene) in USA in first quarter of 2018
  • 10 Aug 2017 Applied Genetic Technologies Corporation files an IND application with the FDA in USA for Retinitis pigmentosa
  • 10 Aug 2017 Applied Genetic Technologies Corporation plans a phase I/II trial for Retinitis pigmentosa in USA
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