Exploration of Immunology

Table 4. 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]

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AMD: age-related macular degeneration; CFH: complement factor H; CFI: complement factor I; GA: geographic atrophy

Declarations

Acknowledgments

Authors want to thank Matt Ferenc for his suggestions in improving the manuscript.

Author contributions

MK: Conceptualization. MK and KNR: Writing—original draft. MY, AP, and PD: Writing—review & editing. All authors agree with the contents of the manuscript.

Conflict of interest

KNR, MY, AP, PD and MK are employees and shareholders at Apellis Pharmaceuticals Inc. Apellis researches, produces, and markets products that inhibit the complement system.

Ethical approval

Not applicable.

Consent to participate

Not applicable.

Consent to publication

Not applicable.

Availability of data and materials

Not applicable.

Funding

Not applicable.

Copyright

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