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VG801 Gene Therapy in Biallelic ABCA4 Mutation-Associated Retinal Dystrophy
2024-520425-37-00Phase 2Authorised
- Sponsor
- VeonGen Therapeutics
- Start
- Sep 2025
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Engineered AAV → AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 3
01
Overview
Phase 2, open-label, single-arm trial of VG801 in Fundus dystrophy and ABCA4-related macular dystrophy (ABCA4-MD), sponsored by VeonGen Therapeutics. Primary endpoints: Number of adverse events (ocular and systemic), Humoral immune responses against the AAV capsid and 8 more. Planned start Sep 2025; status: authorised.
02
Drugs
VG801
03
Conditions
Fundus dystrophyABCA4-related macular dystrophy (ABCA4-MD)
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