Congenital Dyserythropoietic Anemias (CDAs)

Congenital dyserythropoietic anemias (CDAs) are a heterogeneous group of disorders characterized by ineffective erythropoiesis, which predominantly leads to monolinear cytopenia. At Protheragen, we are committed to expeditious research and therapeutic breakthroughs promoting iron metabolism-related diseases, such as the CDAs. We provide integrated, high-quality, application-oriented solutions for life sciences that allow researchers and scientists around the world to identify disease mechanisms, to develop and test strategies and therapies to change those mechanisms.

Overview of Congenital Dyserythropoietic Anemias (CDAs)

Congenital dyserythropoietic anemias (CDAs) are a heterogeneous group of inherited anemias that can disrupt the normal differentiation-proliferation pathways of the erythroid cell lineage. They are part of a broad spectrum of ineffective erythropoiesis disorders and predominantly present with monolinear cytopenia. CDAs can be divided into the 3 major types (I, II, III), transcription factor-related CDAs, and those of CDA variants, depending on the unique morphological and genetic characteristics.

Fig.1 The physical and genetic traits of CDA individuals. (Iolascon, A., et al., 2020)

Iron Metabolism Abnormalities in Congenital Dyserythropoietic Anemias (CDAs)

CDA syndromes are genetic conditions marked by persistent low-reticulocyte anemia, mild hemolysis, and excess iron deposition. To date, mutations in more than ten distinct genes have been linked to classical CDA, CDA variants, and syndromic presentations. Advances have primarily centered on CDA type II, where dissecting iron homeostasis has shed light on pathophysiology. Key to this progress has been the discovery that erythroferrone, secreted by stressed erythroblasts, selectively down-regulates hepcidin, thus driving excessive hepatic iron accumulation.

Fig.2 A schematic of the subcellular processes involved in CDA pathogenesis. (Iolascon, A., et al., 2020)

Therapeutics Development for Congenital Dyserythropoietic Anemias (CDAs)

Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a therapy plan recommendation. For guidance on therapy options, please visit a regular hospital.

Our Services

Protheragen provides complete solutions for the development of novel therapies for CDAs. Our capabilities encompass the full research and development continuum. Applying state-of-the-art omics technologies and bioinformatics, we help to identify new therapeutic targets with regard to iron metabolism and erythropoiesis in CDA. We also offer tailor-made disease model development services and perform extensively in vitro and in vivo testing to thoroughly evaluate the efficacy, pharmacokinetics, and safety profiles of the potential drug candidates in disease-relevant CDA models.

Therapeutic Development Services

Animal Model Development Services for CDAs

Valid animal models in preclinical research are necessary to comprehend the pathogenesis of CDA and to test therapeutic approaches. Our expertise lies in the development and characterization of cutting-edge animal models that closely resemble the human situation to support customers' research objectives.

Pharmacokinetics and Drug Safety Evaluation Services

At Protheragen, we are dedicated to advancing the science of iron metabolism and rare blood disorders. By working with us, investigators have the opportunity to benefit from our unique expertise, technology-rich environment, and one-stop approach to drive innovation around diagnostics and therapeutics for CDA. Get in touch with us today to find out how we can help you speed up your CDA research and therapy development efforts.