Dysfibrinogenemia is a condition marked by impeded function of the fibrinogen molecule. To tackle the difficulties associated with managing dysfibrinogenemia, Protheragen has devoted resources to advanced technologies and specialists aiming at developing innovative therapeutic approaches. With our extensive support services, the entire process of developing a drug candidate to commercialization will be more efficient.
Dysfibrinogenemia is an infrequent type of coagulation disorder with its defining characteristic being inadequate functioning fibrinogen molecules, complemented by normal or near-normal fibrinogen antigen levels. This aberration is the result of alterations in the FGA, FGB, or FGG genes which code for the alpha, beta, and gamma components of fibrinogen, respectively. Dysfibrinogenemia manifests with a wide range of symptoms that may include absence of symptoms, severe bleeding diatheses, or paradoxical thrombotic phenomena that are uncharacteristic in nature.
Fig.1 Pathogenesis of thrombosis and bleeding in association with low fibrinogen activity. (Brunclikova M, et al., 2022)
Dysfibrinogenemia occurs due to modifications in the FGA, FGB, or FGG genes which change the structure and function of fibrinogen. These mutations can either interfere with thrombin's role in cleaving fibrinopeptide, polymerizing fibrin, or resisting fibrinolysis, thus leading to bleeding or thrombosis. Certain variants cause retention and subsequent degradation in the endoplasmic reticulum, which decreases the amount of functional fibrinogen in circulation.
Fig.2 Treatment algorithm for dysfibrinogenemia. (Casini A, de Moerloose P., 2021)
Due to the lack of mutation-specific therapies and the challenges of correcting the underlying fibrinogen dysfunction, current treatment of dysfibrinogenemia remains limited to symptomatic care, such as fibrinogen replacement therapy for bleeding and anticoagulation for thrombosis. There is an urgent need to develop targeted therapies to facilitate the management of this disease.
| Therapy | Types | Mechanism of Action | Phase |
|---|---|---|---|
| Fibrinogen Replacement Therapy | Plasma-derived fibrinogen concentrate or recombinant fibrinogen | Replenishes functional fibrinogen to support normal clot formation and hemostasis. | Approved |
| Aminocaproic Acid | Synthetic lysine analog | Inhibits fibrinolysis by blocking plasminogen activation and plasmin binding. | Approved |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
Protheragen develops comprehensive diagnostic and therapeutic solutions for the efficient management of dysfibrinogenemia. Addressing the myriadof molecular underpinnings of dysfibrinogenemia, we are commited to the development of targeted therapies which fulfill evolving medical necessities. Our professionals stand out in the construction of precise disease models which allow thorough testing for the safety and effectiveness of possible therapeutics.
Committed to supporting the development of innovative therapeutics, Protheragen offers comprehensive preclinical research services, including pharmacodynamics (PD), pharmacokinetic (PK) and toxicology studies. Our customized approach addresses the unique challenges of your studies and helps you optimize your drug candidates for commercial success. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
References
Myeloproliferative Disorders (MPDs)
Bone Marrow Failure Syndromes (BMFS)
