Drug Profile

AAV AQP1

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

Latest Information Update: 21 Mar 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 II Xerostomia
Phase I/II Sjogren's syndrome

Most Recent Events

17 Mar 2025 MeiraGTx plans to file biological license application (BLA) for Xerostomia in 2026
09 Dec 2024 AAV AQP1 receives Regenerative Medicine Advanced Therapy (RMAT) status for Xerostomia in USA
09 Dec 2024 MeiraGTx plans to file BLA for treatment of xerostomia in USA

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