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AAV AIPL

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Drug Profile

AAV AIPL

Alternative Names: AAV-AIPL; AAV-AIPL1; AAV8-RK-AIPL1; adenovirus associated viral vector serotype 8 containing the human AIPL1 gene; rAAV8-hRKp-AIPL1

Latest Information Update: 21 Mar 2025

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At a glance

  • Originator MeiraGTx
  • 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 - Cone rod dystrophies; Retinitis pigmentosa; Leber congenital amaurosis
  • New Molecular Entity No

Highest Development Phases

  • Clinical Phase Unknown Leber congenital amaurosis

Most Recent Events

  • 21 Feb 2025 Efficacy and adverse events data from a clinical trial in Leber congenital amaurosis released by MeiraGTx
  • 31 Dec 2024 AAV AIPL receives Rare Pediatric Disease Designation (RPDD) from the US FDA for the treatment of Leber's congenital amaurosis prior to December 2024
  • 21 Nov 2024 MieraGTX receives Rare Pediatric Disease Designations from the FDA for AAV AIPL in Leber congenital amaurosis

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