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AAV AQP1

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Drug Profile

AAV AQP1

Alternative Names: AAV-hAQP1; A00X; AAV-AQP1; AAV2-hAQP1

Latest Information Update: 03 Dec 2025

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At a glance

  • Originator Kadmon Gene Therapy Holdings
  • Developer MeiraGTx
  • Class Gene therapies
  • Mechanism of Action Aquaporin 1 replacements; Gene transference
  • Orphan Drug Status

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

    Yes - Xerostomia
  • New Molecular Entity No

Highest Development Phases

  • Phase III Xerostomia
  • Phase I/II Sjogren's syndrome

Most Recent Events

  • 03 Dec 2025 Phase-III clinical trials in Xerostomia (Intraglandular), prior to December 2025 (MeiraGTx pipeline, December 2025 )
  • 13 Nov 2025 MeiraGTx expect to received approval in 2027
  • 13 Nov 2025 MeiraGTx plans to file biological license application (BLA) for Xerostomia in early 2027

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