Exploration of Immunology

Table 2. Non-exhaustive list of disclosed C3 targeting therapeutics

Drug name	Company	Modality	Highest clinical stage achieved	Target indications
Pegcetacoplan (APL-2, POT-4)	Apellis	PEGylated cyclic peptide	Marketed	GA: marketed, PNH: marketed, C3G: phase 3
AMY-101	Amyndas	Cyclic peptide	Phase 2	Periodontitis: phase 2, COVID-19: phase 2 ongoing, C3G: phase 2 planned, PNH: phase 2 planned, kidney transplantation: phase 2 planned
ARO-C3	Arrowhead	siRNA	Phase 1	G3G: phase 1/2, IgAN: phase 1/2, development stopped?
ALXN2030	Alexion/AstraZeneca/Novo Nordisk	siRNA	Phase 1	Chronic active AMR: phase 1
APL-3007	Apellis	GalNAc conjugated siRNA	Phase 1	Healthy volunteers: phase 1
CB2782	Catalyst Biosciences	PEGylated C3-protease	Preclinical	GA: planned, development stopped?
ALN-CC3	Alnylam	GalNAc conjugated siRNA	Preclinical	Development stopped?
SLN-501	Silence/Mallinckrodt	GalNAc conjugated siRNA	Preclinical	Not disclosed
CB-101/501/601/801	Cascade Biotech	Not disclosed	Preclinical	Not disclosed
STP146G	Sirnaomics	GalNAc conjugated siRNA	Preclinical	Not disclosed
KNP-301	Kanaph/Samsung Biologics	Bi-specific C3b and VEGF mAb	Preclinical	Retinal disease
KNP-302	Kanaph	Bi-specific C3b and CD59 mAb	Preclinical	Not disclosed

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GalNAc: N-acetylgalactosamine; aHUS: atypical haemolytic uremic syndrome; AMR: antibody-mediated rejection; C3G: C3  glomerulopathy; COVID-19: coronavirus disease 2019; IgAN: IgA nephropathy; mAb: monoclonal antibody; PNH: paroxysmal nocturnal haemoglobinuria; siRNA: silencing ribonucleic acid; GA: geographic atrophy; VEGF: vascular endothelial growth factor

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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