Drug Profile

AAVB 039

Alternative Names: AAV.ABCA4.intein; AAV8.ABCA4; AAVB-039; Dual adeno-associated virus based Stargardt disease gene therapy - AAVantgarde

Latest Information Update: 21 Aug 2025

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At a glance

Originator AAVantgarde Bio
Class Eye disorder therapies; Gene therapies
Mechanism of Action ATP binding cassette transporter subfamily A member 4 replacements; Gene transference

Orphan Drug Status

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

No
New Molecular Entity No

Highest Development Phases

Phase I/II Stargardt disease

Most Recent Events

12 Aug 2025 AAVB 039 receives Fast Track designation for Stargardt disease [Subretinal] in USA
15 Jul 2025 Phase-I/II clinical trials in Stargardt disease in USA (Subretinal)
15 Jul 2025 The US FDA approves an IND application for AAVB 039 in Stargardt disease

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