Single Domain Antibody (SdAb) R&D in Hematological Disorders
Hematological disorders refer to diseases that primarily affect the blood & blood-forming organs. In general, hematological disorders include rare genetic disorders, anemia, lymphoma, leukemia, thrombocytopenia, etc.
Four Principal Components Affected by Hematological Disorders
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Red Blood Cells: Responsible for transporting oxygen to the body tissues.
Associated Diseases: Iron-deficiency anemia, anemia of chronic disease, pernicious anemia (B12 deficiency), aplastic anemia, autoimmune hemolytic anemia, thalassemia, sickle cell anemia, polycythemia vera, and malaria.
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White Blood Cells: Crucial for combating infections.
Associated Diseases: Lymphoma, leukemia, multiple myeloma, and myelodysplastic syndrome.
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Platelets: Essential for blood clotting.
Associated Diseases: Thrombocytopenia, idiopathic thrombocytopenic purpura, heparin-induced thrombocytopenia, thrombotic thrombocytopenic purpura, and essential thrombocytosis (primary thrombocythemia).
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Plasma: The liquid component of blood.
Associated Diseases: Hemophilia, von Willebrand disease, hypercoagulable state, deep venous thrombosis, and disseminated intravascular coagulation (DIC).
Single domain antibody (sdAb) is a specialized antibody fragment derived from the heavy-chain-only antibodies found in camelids or sharks, or from specifically developed human VH and VL domains. Compared to conventional antibody formats, sdAbs offer significant advantages, including:
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Small Size: Facilitates better tissue penetration.
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High Solubility: Enhances bioavailability.
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Great Stability: Increases shelf life and resistance to harsh conditions.
SdAbs can be efficiently produced in bacterial systems, and their modular nature allows for the creation of multivalent complexes. Recently, sdAbs have been investigated as potent therapeutics across various medical fields, including oncology, inflammatory diseases, neurological disorders, and hematological conditions.
Single Domain Antibody (SdAb) Discovered against Hematological Disorders
In recent years, several single domain antibodies (sdAbs) have been developed for hematological disorders. These sdAbs function by inhibiting platelet adhesion to vessel walls, thereby controlling platelet aggregation and clot formation. Measurements using the platelet aggregation biomarker (RICO) have demonstrated that sdAb injections are well tolerated and effectively inhibit von Willebrand factor (vWF)-mediated clotting. Additional research has focused on the treatment of thrombotic thrombocytopenic purpura (TTP), making sdAbs applicable to a broader patient population. In summary, sdAbs are versatile molecules with numerous advantageous properties for therapeutic and diagnostic applications, as well as for use as research tools.
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