Therapeutic Development Services
Polycythemia vera (PV) is a myeloproliferative disease that seriously endangers human health. At Protheragen , we focus on developing novel therapeutics and building accurate animal models to accelerate preclinical studies of potential therapies for PV. Our expertise ensures that your research receives the most reliable and relevant support, accelerating your drug development journey.
Polycythemia vera (PV) is a less common form of chronic myeloproliferative neoplasm (MPN) and it is associated with excessive erythrocyte, leucocyte and thrombocyte production due to pathological changes in the bone marrow. This increases blood viscosity, which raises the risk of blood clots, strokes and heart attacks. Timely diagnosis and treatment can prevent life-threatening complications. Because of the infrequency of the condition and its variety of presentations, there are barriers to therapeutics.
Fig. 1 Molecular mechanisms of polycythemia vera (PV), essential thrombocythemia, and primary myelofibrosis. (Tashkandi H, Younes I E., 2024)
The primary cause of polycythemia vera (PV) is acquired somatic mutations in the JAK2 gene (either JAK2V617F or exon 12 mutations) which leads to abnormal activation of the JAK-STAT signaling cascade pathway. This results in the unchecked growth of erythroid, myeloid, and megakaryocytic lineages. Other possible mutations, such as TET2, ASXL1, or DNMT3A, could also contribute to the disease's progression. Dysregulation of the marrow microenvironment results in persistent inflammation and cytokine secretion, which in turn amplifies proliferation of the bone marrow.
Fig. 2 The JAK2 signaling pathway is closely related to the pathogenesis of polycythemia vera (PV). (Ginzburg Y Z, et al., 2018)
The therapeutic market for polycythemia vera (PV) is anticipated to reach $19.4 billion by 2024, supported by therapies such as JAK inhibitors, hydroxyurea, and interferon. Nevertheless, PV treatment poses considerable difficulties, particularly in managing the risks associated with thrombosis and bleeding, as well as the obvious limitations in current therapeutic approaches. In light of these difficulties, there is an urgent need for the emergence of transformative therapies that could reshape PV treatment paradigms.
Table. 1 Drug development pipeline for polycythemia vera (PV).
| Drug Names | Mechanism of Action | Targets | NCT Number | Research Phase |
|---|---|---|---|---|
| Ropeginterferon Alfa-2b | Suppresses JAK-STAT signaling, reduces erythrocytosis. | IFNAR | NCT06743035 | Approved |
| Rusfertide | Inhibits iron release, reducing erythropoiesis. | Ferroportin | NCT05210790 | Phase III |
| Bomedemstat | Blocks malignant hematopoietic growth. | LSD1 | NCT05558696 | Phase II |
| SLN124 | Enhances endogenous hepcidin expression and inhibits iron release. | TMPRSS6 | NCT05499013 | Phase I/II |
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.
Recognizing the complexity of diagnosing and treating polycythemia vera (PV), Protheragen is committed to building a team of experts to provide cutting-edge diagnostic and therapeutic development solutions. Our commitment lies in providing a variety of customized therapy development services to meet the diverse research needs of our customers. We also excel in generating precise disease models that are carefully engineered to replicate the unique features of PV. These models are valuable tools for validating the safety and efficacy of potential therapeutics.
Therapeutic Development Services
As a leading provider of therapeutic development services, Protheragen is focused on developing a variety of innovative therapies for polycythemia vera (PV) to address the complex challenges in this field.
Animal Model Development Services
Animal models are essential tools for understanding the biology of polycythemia vera (PV) and evaluating the efficacy and safety of potential therapies. Our models include but are not limited to the following:
At Protheragen, we are committed to validating and optimizing therapies for polycythemia vera (PV) through preclinical studies including pharmacodynamics (PD), pharmacokinetics (PK) and toxicology to ensure their successful regulatory approval. 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)