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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; rAAV8.hRKp.AIPL1

Latest Information Update: 26 Nov 2025

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At a glance

  • Originator MeiraGTx
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action AIPL1 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 - Cone rod dystrophies; Retinitis pigmentosa; Leber congenital amaurosis
  • New Molecular Entity No

Highest Development Phases

  • Clinical Phase Unknown Leber congenital amaurosis

Most Recent Events

  • 10 Nov 2025 AAV AIPL licensed to Eli Lilly and Company worldwide
  • 19 Aug 2025 MeiraGTX plans to submit a BLA for AAV-AIPL1 in Leber congenital amaurosis in USA
  • 21 Feb 2025 Efficacy and adverse events data from a clinical trial in Leber congenital amaurosis released by MeiraGTx

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