Complement C5-IN-1 is a small-molecule inhibitor of complement component 5 protein (C5).

Avacincaptad pegol, a pegylated aptamer, stands out as an effective C5 complement inhibitor capable of mitigating inflammation-induced damage to the retinal pigment epithelium (RPE). This compound is under investigation for its potential application in treating Stargardt macular dystrophy (STGD1) and geographic atrophy (GA) [1] [2].

Eculizumab is a recombinant humanized monoclonal antibody against the complement protein C5

Zilucoplan (RA101495), a 15-amino acid macrocyclic peptide and potent complement component 5 (C5) inhibitor, is utilized in the study of immune-mediated necrotising myopathy (IMNM) [1] [2].

Ravulizumab (ALXN1210) is a humanized monoclonal antibody targeting complement factor 5 that specifically binds to human complement protein C5 with high affinity to block complement activation. Ravulizumab can be used for the prevention and treatment of paroxysmal sleep hemoglobinuria, atypical hemolytic uremic syndrome and myasthenia gravis.

Cemdisiran, an investigational N-acetylgalactosamine-conjugated RNAi (RNA interference) agent with terminal sugar modification, is a small interfering RNA (siRNA) designed to specifically target and suppress the C5 mRNA, thereby reducing the liver's production of the complement component C5 [1].

ARC186 is a highly potent aptamer that serves as a complement inhibitor by blocking the convertase-catalyzed activation of C5.

Gefurulimab (ALXN-1720) is a bispecific antibody derived from humans, targeting complement C5 and albumin. It specifically binds to C5, inhibiting its activation [1].

Crovalimab (SKY59; RO7112689) is a novel humanized antibody designed to target C5 in a pH-dependent manner, demonstrating binding affinities (KD) of 15.2 nM and 16.8 μM at pH levels of 7.4 and 5.8, respectively. Additionally, it shows high affinity for human FcRn at pH 6.0, with a KD of 17 μM. By blocking C5 cleavage by C5 convertase and inhibiting the activity of a C5 variant (p.Arg885His), Crovalimab effectively prevents the formation of C5b-9 across the classical (CP), lectin (LP), and alternative (AP) complement pathways. This characteristic highlights its potential utility in research concerning paroxysmal nocturnal hemoglobinuria (PNH) and other complement-mediated diseases.

Cemdisiran, a conjugated siRNA with N-acetylgalactosamine (GalNAc), targets the liver to inhibit the production of complement 5 (C5) protein, serving as a potential therapeutic agent for the study of complement-mediated diseases.

ARC1905, a non-PEGylated/naked anti-C5 RNA aptamer, functions by inhibiting the cleavage of complement factor 5 (C5) into its components, C5a and C5b, without PEG conjugation. This compound is currently being researched for its potential application in the treatment of age-related macular degeneration (AMD).

Pexelizumab (h5G1.1-SC), a humanized scFv monoclonal antibody, targets the C5 complement component, thereby inhibiting apoptosis and leukocyte infiltration. It is utilized in researching cerebral ischemia-reperfusion (IR) injury and myocardial infarction [1] [2].

NH2-C6-ARC186 is a derivative of the aptamer ARC186, featuring an NH2-C6 moiety that enables coupling to various peptides or molecules. As a potent complement inhibitor, it operates by inhibiting convertase-mediated C5 activation [1].

Avacincaptad pegol (ARC1905), an anti-C5 RNA aptamer, prevents the cleavage of complement factor 5 (C5) into fragments C5a and C5b. It is currently under investigation for the treatment of age-related macular degeneration (AMD).

Pozelimab (REGN3918), a fully human IgG4 monoclonal antibody, is designed to target and bind with high affinity to both C5 and its variants, effectively inhibiting complement-mediated hemolysis. It holds potential for research into complement-mediated diseases [1].

Zilucoplan TFA (RA101495), a 15-amino acid macrocyclic peptide, serves as a potent inhibitor of complement component 5 (C5). It has applications in the study of immune-mediated necrotising myopathy (IMNM) [1] [2].