Addressing the intricate nature of anemia demands the creation of avant-garde therapies and tailored therapeutic approaches. At our company, we uphold a dedication to excellence and stand as a premier provider of services for anemia diagnostic and therapeutic development. Specializing in the establishment of state-of-the-art in vitro and in vivo models, we lead the way in conducting preclinical drug studies to drive forward the progress of therapeutic solutions.
Anemia is a group of blood disorders that affects millions of people worldwide and poses a major healthcare challenge. Characterized by decreased oxygen-carrying capacity in the blood, anemia stems from a variety of causes, ranging from nutritional deficiencies to genetic conditions. Anemia symptoms such as tiredness, paleness, weakness, and difficulty breathing can significantly affect the quality of life for individuals.
Fig. 1 Regulatory mechanisms of hepcidin synthesis in cancer-related anemia. (Madeddu, Clelia, et al., 2018)The pathogenesis of anemia is multifaceted, involving various factors such as iron deficiency, genetic elements, and hemolytic process. Understanding the underlying mechanisms of anemia is crucial for accurate diagnosis, targeted therapeutic strategies, and improving the prognosis of individuals affected by this intricate blood disorder.
Iron Deficiency Anemia
Iron-deficiency anemia, a prevalent form of anemia, results from insufficient iron levels crucial for synthesizing hemoglobin. Iron is pivotal in hemoglobin production; its scarcity hinders the body from generating an ample amount of red blood cells, ultimately culminating in anemia.
Genetic Anemia
Genetic mutations that affect hemoglobin structure or red blood cell function are the root causes of inherited anemias. Take sickle cell anemia as an illustration: it arises from a specific point mutation in the β-globin gene, prompting red blood cells to adopt a sickle shape when exposed to low oxygen levels.
Hemolytic Anemia
Hemolytic anemia is a form of anemia distinguished by the premature breakdown of red blood cells, resulting in a deficiency of these cells in the bloodstream. For example, autoimmune hemolytic anemia (AIHA), in which the immune system mistakenly targets and destroys red blood cells, causes anemia.
The development of therapeutics for anemia encompasses a spectrum of interventions targeting the root causes and alleviating symptoms. Oral and injectable iron supplements are standard for managing iron deficiency anemia. Emerging strategies like erythropoiesis-stimulating agents and gene therapy hold potential for addressing refractory anemia.
Table. 1 Development pipeline of anemia therapies.
| Drugs | Types of Therapy | Targeted Diseases | Developmental Stage |
|---|---|---|---|
| Epoetin Alfa | Erythropoiesis-stimulating Agent | Anemia of Chronic Kidney Disease, Anemia of Cancer | Approved |
| Darbepoetin Alfa | Erythropoiesis-stimulating Agent | Anemia of Chronic Kidney Disease, Anemia of Cancer | Approved |
| Ferumoxytol | Iron Replacement Therapy | Iron Deficiency Anemia | Approved |
| Hemapro | Erythroid Maturation Agent | Beta-Thalassemia | Approved |
| GBT440 | Hemoglobin Modulator | Sickle Cell Disease, Hemolytic Anemia | Approved |
| Pegcetacoplan | Complement C3 Inhibitor | Paroxysmal Nocturnal Hemoglobinuria, Autoimmune Hemolytic Anemia | Clinical Research |
| IMR-687 | PDE9 Inhibitor/Fetal Hemoglobin Inducer | Sickle Cell Disease, Beta-Thalassemia | Clinical Research |
| CTX001 | CRISPR/Cas9-based Gene Editing Therapy | Beta-Thalassemia, Sickle Cell Disease | Clinical Research |
| HGB-206 | Gene Therapy | Sickle Cell Disease | Clinical Research |
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.
With years of dedicated involvement in rare blood disease research, our company is steadfast in pioneering the exploration of state-of-the-art diagnostic and therapeutic approaches for anemia through advanced preclinical research methods. Our focus lies in the development of sophisticated in vitro and animal models, aimed at in-depth exploration of anemia's pathogenesis and thorough validation of therapeutic impacts.
| A-D | |
|---|---|
| Alpha-Thalassemia (A-THAL) Aplastic Anemia (AA) Atypical Hemolytic Uremic Syndrome (AHUS) Autoimmune Hemolytic Anemia (AIHA) |
Beta-Thalassemia (B-THAL) Blackfan-Diamond Anemia Congenital Dyserythropoietic Anemia Diamond-Blackfan Anemia (DBA) |
| E-Z | |
| Fanconi Anemia (FA) Hemolytic Anemia Hereditary Spherocytosis Iron Deficiency Anemia Megaloblastic Anemia |
Normocytic Anemia Paroxysmal Nocturnal Hemoglobinuria (PNH) Pure Red Cell Aplasia Sickle Cell Disease (SCD) Sideroblastic Anemia |
By establishing precise animal models, we are dedicated to the pharmacodynamics, pharmacokinetics (PK), and toxicology research of anemia therapies, empowering the progression of innovative therapeutics to market. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
Reference
Myeloproliferative Disorders (MPDs)
Bone Marrow Failure Syndromes (BMFS)
