Drug Profile


Alternative Names: AAV-PR1.7-hCNGBco vector; AAV2tYF-PR1.7-hCNGB3co; AAV5-PR2.1-hCNGB3; AAV5-PR2.1-hCNGB3co; AAV9-PR1.7-hCNGB3co; ACHM B3; ACHM CNGB3

Latest Information Update: 07 Oct 2016

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At a glance

  • Originator Applied Genetic Technologies Corporation
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action CNBG3 gene expression stimulants; Gene transference
  • Orphan Drug Status

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

    Yes - Colour vision defects
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Phase I/II Colour vision defects

Most Recent Events

  • 01 Feb 2016 Phase-I/II clinical trials in Colour vision defects in USA (Subconjunctival) (NCT02599922)
  • 07 Jan 2016 Pharmacodynamics data from a preclinical trial in Achromatopsia released by AGTC
  • 06 Jan 2016 Applied Genetic Technologies Corporation enter into collaboration with BCM Families Foundation to develop Adeno-associated virus-based gene therapies for the treatment of X-linked achromatopsia
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