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AGTC 401

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Drug Profile

AGTC 401

Alternative Names: AAV-CNB3; AAV-PR1.7-hCNGBco vector; AAV2/8; AAV2tYF-PR1.7-hCNGB3co; AAV9-PR1.7-hCNGB3co; ACHM B3; ACHM CNGB3; AGTC-401; CNGB3 gene-therapy-AGTC; rAAV2-hCNGB3; rAAV2tYF-PR1.7-hCNGB3

Latest Information Update: 29 Jun 2023

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At a glance

  • Originator Applied Genetic Technologies Corporation
  • Developer Beacon Therapeutics
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action CNGB3 protein replacements; 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

  • 08 Feb 2022 Efficacy and adverse events data from a phase I/II trial of in Colour vision defects caused by mutations in CNGB3 gene released by Applied Genetic Technologies Corporation
  • 09 Nov 2021 Interim adverse events data from a phase I/II trial in Colour vision defects released by Applied Genetic Technologies Corporation
  • 29 Sep 2021 Applied Genetic Technologies Corporation plans to file End-of-Phase 2 (EOP2) meeting packet with the US FDA and expects feedback in the first half of 2022

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