| Complement factor B |
IONIS-FB-LRx
|
Roche/Ionis |
Subcutaneous ligand-conjugated (LICA) antisense therapy |
Alternative pathway |
Phase II GOLDEN trial ongoing |
| Complement factor I |
GT005 |
Gyroscope |
Subretinal AAV2 vector; gene therapy designed to induce expression of CFI |
Alternative pathway |
Phase I/II FOCUS trial showed GT005 to be well tolerated; phase II EXPLORE and HORIZON studies are ongoing |
| Complement factor H |
GEM103 |
Gemini |
IVT recombinant human CFH |
Alternative pathway |
Phase 2a ReGAtta trial enrollment complete |
| Complement factor I |
GEM104 |
Gemini |
IVT full-length recombinant human CFI |
Alternative pathway |
In preclinical development |
| Complement factor D |
ALXN2040 |
Alexion/AstraZeneca |
Oral factor D inhibitor |
Alternative pathway |
IND application submitted; phase II study planned |
| Complement C3 |
NGM621 |
NGM Bio |
IVT humanized IgG1 monoclonal antibody |
Classical pathway |
Phase II CATALINA study currently recruiting |
| Complement C3 |
Pegcetacoplan |
Apellis |
IVT cyclic peptide-bound polyethylene glycol polymer |
Classical pathway |
Phase II FILLY trial showed reduction of GA growth; phase III DERBY and OAKS trials ongoing |
| Complement C5 |
Avacincaptad pegol |
Iveric Bio |
IVT pegylated RNA aptamer |
Classical pathway |
Phase II GATHER1 trial showed decrease in GA lesion size; phase III GATHER2 trial ongoing |
