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Accelerating Complement-Mediated Disease Drug Development

Complement-mediated diseases present significant therapeutic challenges due to the complexity of the complement system and its role in immunological disorders. Protheragen is a specialized partner in the development of novel therapeutics targeting complement-mediated pathologies, offering end-to-end preclinical solutions from target validation through IND-enabling studies. Leveraging deep scientific expertise and advanced technology platforms, Protheragen delivers rigorous in vitro and in vivo models, robust biomarker strategies, and comprehensive pharmacology and toxicology assessments. Our integrated approach ensures seamless progression of drug candidates while maintaining strict adherence to global regulatory standards. With a proven track record in complement biology and preclinical drug development, Protheragen is dedicated to accelerating the advancement of innovative therapies, empowering our partners to address unmet medical needs in complement-mediated diseases.

What is Complement-Mediated DiseaseTargets for Complement-Mediated DiseaseDrug Discovery and Development ServicesWhy Choose Us

What is Complement-Mediated Disease

Complement-mediated diseases are a group of rare disorders characterized by abnormal activation or regulation of the complement system, an essential part of innate immunity. Etiologies include inherited mutations in complement regulatory proteins, acquired factors, or the development of autoantibodies that disrupt normal complement control. Pathophysiologically, this dysregulation leads to excessive or misdirected complement activity, resulting in tissue injury through membrane attack complex formation, inflammation, and opsonization of host cells. These processes can cause acute or chronic damage to organs such as the kidneys, blood vessels, central nervous system, and bone marrow. Clinically, complement-mediated diseases present with a wide range of symptoms depending on the specific disorder and organs involved. Common manifestations include hemolytic anemia, thrombocytopenia, renal dysfunction, neurological deficits, and fatigue. Diagnosis relies on a combination of clinical evaluation, laboratory tests for hemolysis and renal function, assessment of complement activity (C3, C4, CH50), and specialized studies such as genetic testing, autoantibody screening, tissue biopsy, or flow cytometry. Treatment strategies are disease-specific and may involve complement inhibitors (such as eculizumab), immunosuppressive therapies, plasma exchange, or supportive care to manage complications. Early recognition and targeted therapy are crucial to reduce morbidity and improve patient outcomes.

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Targets for Complement-Mediated Disease

Targets in Clinical or Later Phases of Development

Target Name Gene Symbol
complement C1s C1S
complement C3 C3
complement C5 C5
complement C5a receptor 1 C5AR1
Complement Complex
complement factor D CFD

Complement-mediated diseases arise from dysregulation or overactivation of the complement system, a central component of innate immunity. Key therapeutic targets include complement proteins such as C1s, C3, C5, and Factor D, which are integral to the classical, alternative, and terminal pathways of complement activation. C1s and C1r initiate the classical pathway, while C3 serves as the central amplification hub, and Factor D is critical in the alternative pathway's activation loop. Downstream, C5 and its receptor C5aR1 mediate potent inflammatory and cytolytic effects, with C5 cleavage leading to both membrane attack complex (MAC) formation and release of the pro-inflammatory peptide C5a. Regulatory proteins like Factor H, Factor I, and properdin act as essential checks, ensuring complement activation is appropriately controlled and directed away from host tissues. The therapeutic potential of targeting these molecules is well established, with several agents already approved or in advanced development. Monoclonal antibodies against C5 (eculizumab, ravulizumab) and C1s (sutimlimab) have transformed the treatment of diseases such as PNH and cold agglutinin disease. Inhibitors of C3 (pegcetacoplan) and Factor D (danicopan) are in clinical use or late-stage trials, reflecting the centrality of these nodes. Agents targeting C5aR1 (avacopan) are approved for vasculitis, and recombinant or gene therapies for regulatory proteins are under investigation. These advances highlight the promise of precise, pathway-specific complement inhibition to control disease activity while minimizing infection risk.

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Drug Discovery and Development Services

In Vitro Efficacy Testing ServicesIn Vivo Model DevelopmentPK/PD Study ServicesIn Vivo Toxicity Assessment ServicesBiomarker Analysis Services
In Vitro Efficacy Testing Services

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Our In Vitro Efficacy Testing Service accelerates discovery of therapies for complement-mediated diseases by providing robust, sensitive screening and characterization platforms. We offer comprehensive biochemical, biophysical, and functional assays targeting key complement proteins (C1S, C2, C3, C4B, C5, C5aR1, C7, Factor D) and pathways. Methods include ELISA, hemolysis, biolayer interferometry, surface plasmon resonance, and DTNB reduction assays. We deliver critical pharmacological parameters such as EC-50, IC-50, and Kd, enabling precise evaluation of potency, inhibition, and binding affinity. Our data-driven approach supports informed decision-making and optimizes complement-targeted drug development.

Complement C1S Complement C2
Complement C3 Complement C4B (Chido/Rodgers Blood Group)
Complement C5 Complement C5A Receptor 1
Complement C7 Complement Factor D

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Why Choose Us

Choosing Protheragen as your partner in the development of new therapeutics for Complement-mediated diseases means entrusting your project to a team with specialized expertise and a deep commitment to scientific excellence. At Protheragen, we have built a reputation for innovation in the field of Complement-mediated disease research and drug development, supported by our highly skilled professionals and state-of-the-art technology platforms. Our teams are dedicated to providing comprehensive preclinical drug development services, ensuring each project benefits from our extensive experience and proven track record. Reliability and quality are at the core of our operations, and we adhere strictly to the highest industry standards and regulatory requirements throughout every stage of development. Protheragen’s rigorous quality control processes and regulatory compliance ensure that your therapeutic candidates are advanced with the utmost precision and care. We are committed to advancing the field of Complement-mediated disease therapeutics, working collaboratively with our clients to bring new and effective treatments closer to patients in need. By choosing Protheragen, you gain a trusted partner dedicated to professionalism, reliability, and the pursuit of better health outcomes.

FAQs for Our Services

Q: What are the main preclinical research challenges specific to developing drugs for Complement-mediated diseases?

A: Preclinical research for Complement-mediated diseases presents unique challenges, including the complexity and redundancy of the complement cascade, species differences in complement system components, and the need for disease-relevant animal models. At our company, we address these challenges by utilizing advanced in vitro and in vivo models, including humanized mouse models and ex vivo assays with patient-derived samples, to ensure translational relevance and robust evaluation of therapeutic candidates.

Q: What regulatory considerations are important in the preclinical development of drugs targeting the complement system?

A: Regulatory agencies require comprehensive safety and efficacy data, particularly due to the immunological role of the complement system. Key considerations include demonstrating target specificity, minimizing off-target effects, and providing evidence that complement inhibition does not predispose to infection or other immunological complications. Our team has extensive experience in designing regulatory-compliant preclinical studies and preparing data packages for IND submissions, ensuring alignment with FDA and EMA guidelines.

Q: What are the key technical aspects to consider in preclinical research for Complement-mediated diseases?

A: Technical aspects include the selection of appropriate biomarkers for pharmacodynamic and safety assessments, development of validated assays for complement activity, and the establishment of reliable disease models. Our company offers a suite of state-of-the-art analytical platforms, including ELISA, flow cytometry, and multiplex assays, as well as customized animal models, to support comprehensive evaluation of complement-targeted therapeutics.

Q: What are the typical timeline and cost considerations for preclinical development of drugs for Complement-mediated diseases?

A: Preclinical development timelines for complement-targeted drugs typically range from 12 to 24 months, depending on the complexity of the program and the number of required studies. Costs can vary significantly based on the need for specialized models and assays, but early planning and risk assessment can help optimize resource allocation. Our company provides detailed project planning and transparent budgeting to help clients manage timelines and costs effectively.

Q: What are the critical success factors in preclinical drug development for Complement-mediated diseases?

A: Success in this field depends on a deep understanding of disease mechanisms, robust target validation, and the use of translationally relevant models. Early identification of safety liabilities and the ability to generate high-quality, reproducible data are also essential. Our integrated approach combines scientific expertise, advanced technologies, and regulatory experience to maximize the likelihood of successful progression to clinical development.

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