AAVB 039
Alternative Names: AAV.ABCA4.intein; AAVB-039; Dual adeno-associated virus based Stargardt disease gene therapy - AAVantgardeLatest Information Update: 25 Apr 2024
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
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Preclinical Stargardt disease
Most Recent Events
- 23 Apr 2024 Pharmacodynamics and adverse events data from a preclinical study in Stargardt disease released by AAVantgarde Bio
- 06 Jun 2023 AAVantgarde plans clinical trial for Stargardt disease
- 06 Jun 2023 Preclinical trials in Stargardt disease in Italy (Opthalmic) (AAVantgarde pipeline, June 2023)