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Sideroblastic anemia (SA) is a form of anemia where ringed sideroblasts are found in the bone marrow. At Protheragen, we emphasize the creation of innovative therapeutics as well as the precise animal model to expedite the preclinical studies of prospective therapy for SA. Our capabilities guarantee that your investigation will receive the optimal support, accelerating the timeline for your therapeutic development.
Sideroblastic anemia (SA) describes a heterogeneous group of hematologic disorders which exhibit ineffective erythropoiesis and abnormal deposition of iron within mitochondria of erythroblasts, causing ringed sideroblast production. These disorders are categorized into hereditary and acquired types with specific and distinct genetic and environmental causative factors.
Fig.1 Morphological features of sideroblastic anemia (SA). (Ducamp S, Fleming M D., 2019)
The pathogenesis of sideroblastic anemia (SA) involves defective heme synthesis and mitochondrial iron accumulation.
Fig.2 Main causes of acquired sideroblastic anemia (SA). (Rodriguez-Sevilla J J, Calvo X, Arenillas L., 2022)
| Drug Names | Mechanism of Action | Targets | Phase |
|---|---|---|---|
| Deferoxamine | Chelates free iron and aluminum, forming stable complexes excreted renally. | Free iron (Fe3+), ferritin, hemosiderin | Approved |
| Deferasirox | Binds iron with high affinity, promoting fecal excretion via iron-chelate complexes. | Free iron (Fe3+), labile iron pool | Approved |
| Luspatercept | Binds TGF-β superfamily ligands, inhibiting SMAD2/3 signaling to enhance late-stage erythropoiesis. | Activin receptor type IIB (ACVR2B) | Approved |
| Pyridoxine | Converts to pyridoxal 5′-phosphate (PLP), a cofactor for ALAS2 in heme biosynthesis. | δ-Aminolevulinic acid synthase 2 (ALAS2) | 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 harnesses its knowledge and technologies for the complete diagnostic and therapeutic development of sideroblastic anemia (SA) to contribute towards its effective management. We focus on the governing molecular pathways of SA to develop novel and more precise therapies aimed at improving patients' lives. Our team develops highly representative disease models which allow for the thorough assessment of safety, efficacy, and mechanism of action of drug candidates.
Integrated preclinical services for sideroblastic anemia (SA) are a point of pride for Protheragen. These services cover all aspects of drug research including pharmacodynamics (PD), pharmacokinetics (PK), and safety. We observe the highest quality and ethics in the execution of all our research services to make certain the outcome is dependable. 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)