Recombinant AAV-based complement therapies in GA
| Therapeutic | Payload | Company | Target disease | Mechanism of action | Stage |
|---|---|---|---|---|---|
| 4D-175 | Optimized CFH | Aevitas Therapeutics/Molecular Therapeutics | GA | Express CFH to restore complement regulation | Phase 1 expected Q2 2024 |
| GT005 | CFI | Gyroscope Therapeutics/Novartis | GA | Express CFI to restore complement regulation | Phase 2 completed Development stopped |
| HMR59/JNK-1887 | Soluble CD59 | Hemera Biosciences/Johnson & Johnson | GA, wet AMD | Express CD59 to restore complement regulation | Phase 1 completed [255] |
AMD: age-related macular degeneration; CFH: complement factor H; CFI: complement factor I; GA: geographic atrophy