Loss of homeostasis of iron impacts over 2 billion individuals worldwide, from anemia to neurodegenerative conditions. It is crucial to characterize these diseases by studying their molecular bases, the targets for therapy, and the obstacles that prevent the development of drugs. At Protheragen, we are dedicated to advancing the understanding and therapy of these complex conditions, offering a comprehensive suite of drug research and development services, from diagnostics and therapeutics to preclinical studies and beyond.
Iron is essential for life and functions as a cofactor in hemoglobin, mitochondrial respiration, and enzymatic reactions. But its bifunctional roles as an essential nutrient and a potential toxin necessitate rigorous regulation. Iron homeostasis is maintained by the delicate balance among mechanisms of absorption, transport, storage, and utilization. The body is delicately balanced, where both iron deficiency and iron overload can cause serious pathologies. This complex interplay is central to any effective approach addressing iron metabolism disorders.
Metabolism-related disease is an umbrella term for various common but often severe diseases, highlighting disorders whose onsets range from excessive iron accumulation to iron deficiency, and disorders that are summarized by etiologies and their features are diverse.
Types | Diseases | |
Autoimmune Diseases | Myasthenia Gravis Rheumatoid Arthritis |
Systemic Lupus Erythematosus (SLE) |
Aging | ||
Blood Disorders | Anemia of Chronic Disease Anemia of Inflammation Congenital Dyserythropoietic Anemias Iron-deficiency Anemia Sickle-cell Anemia |
Sideroblastic Anemias Thalassaemia Atransferrinemia Myelodysplastic Syndromes Paroxysmal Nocturnal Hemoglobinuria (PNH) |
Cancers | Acute Myeloid Leukemia Colorectal Cancer (CRC) Endometrial Cancer Gallbladder Cancer (GBC) Head and Neck Cancer (HNC) |
Hepatocellular Carcinoma Lung Adenocarcinoma Melanoma Myelodysplastic Syndromes (MDS) Pancreatic Ductal Adenocarcinoma (PDAC) |
Cardiovascular Diseases | Aortic Dissection Atherosclerosis Coronary Heart Disease Dilated Cardiomyopathy Doxorubicin-Induced Cardiomyopathy (DIC) |
Hypertrophic Cardiomyopathy Inflammatory Infiltration of Myocardium Iron Overload Cardiomyopathy (IOC) Metabolic Cardiomyopathy Myocardial I/R Injury |
Neurodegenerative Diseases | Aceruloplasminemia Alzheimer's Disease Friedreich's Ataxia Huntington's disease Kufor-Rakeb Syndrome |
Neurodegeneration with Brain Iron Accumulation (NBIA) Neuroferritinopathy Pantothenate Kinase-associated Neurodegeneration (PKAN) Parkinson's Disease |
Cutaneous Abnormalities | Age Spots Hair Loss Ichthyosis Melasma Nail Abnormalities |
Polymorphous Light Eruption (PMLE) Psoriasis Skin Aging Vitiligo |
Digestive System Diseases | Acute and Chronic Liver Injury Acute liver failure (ALF) Chronic Hepatitis C Fatty Liver Hepatic Fibrosis Inflammatory Bowel Disease |
Liver Cirrhosis Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD) Non-alcoholic Steatohepatitis (NASH) Porphyria Cutanea Tarda Wilson Disease |
Metabolic Disorders | Adrenal Insufficiency Gestational Diabetes Mellitus Growth Hormone Deficiency Haemochromatosis Hyperlipidemia Hypogonadism |
Hypoparathyroidism Hypothyroidism Metabolic dysfunction-associated fatty liver disease (MAFLD) Obesity Type 2 Diabetes |
Musculoskeletal Diseases | Ankylosing Spondylitis Duchenne Muscular Dystrophy (DMD) Gout arthritis Hemophilic Arthropathy |
Osteoarthritis Osteoporosis Restless Legs Syndrome Sarcopenia |
Early and precise diagnosis is crucial for successful therapy of iron metabolism diseases, under which conditions early management of the iron metabolism disorder can prevent irreversible damage to the organ. Frequently, these require a series of tests, both in the lab, through imaging, and occasionally genetic testing.
The main diagnostic means for iron metabolism disorders are blood tests, providing valuable information in relation to the body’s iron levels and the way iron is handled. These tests look at the individual’s complete blood count (CBC), serum ferritin, serum iron, total iron-binding capacity (TIBC), and others.
In the case of an inherited disorder, such as Hemochromatosis, genetic testing is essential to establish the diagnosis. HFE gene mutations are the mutations most often linked to hereditary hemochromatosis. Other genetic causes of iron metabolism abnormalities can also be determined by genetic testing.
MRI is a non-invasive and powerful tool for the measurement of iron in the liver and heart. Other imaging modalities can be used to evaluate organ damage from iron overload, such as echocardiograms to reflect cardiac involvement, and joint X-rays to reflect arthropathy.
Iron metabolism is a topic that continues to be a rapidly moving field with much new understanding of both molecular pathways and potential new therapeutics.
At Protheragen, we meet these challenges head-on and provide a one-stop service for your iron metabolism-related disease-specific drugs research and development needs. As a research service provider, we straddle the entire drug discovery pipeline, from early research to preclinical development.
Protheragen leverages a deep understanding of iron metabolism to design, synthesize, and optimize novel therapeutic candidates. Our services include target identification and validation, high-throughput screening and lead optimization, mechanism of action studies, and preclinical efficacy testing in relevant disease models.
Cell-based models are foundational for high-throughput screening and detailed mechanistic studies in a controlled environment. We leverage diverse cell types to create robust in vitro systems that mimic aspects of human iron metabolism.
Organoid models offer three-dimensional (3D) structures that better recapitulate the complex architecture, cellular diversity, and physiological functions of human organs. We offer customized organoid model development services for research on iron metabolism.
Animal models are essential for preclinical research, providing insights into disease pathogenesis and testing drug efficacy and safety. We custom model development to meet specific research needs, and develop and validate robust and predictive animal models that accurately reflect human iron metabolism disorders.
DMPK Studies are equally as critical for learning the fate of your potential drug in your body and also for analyzing drug absorption, distribution, metabolism, excretion (ADME), and compound effects on iron biomarkers. This provides guidance on the best doses and their effectiveness as a therapy.
The safety of your drug candidates is also a very important issue. We offer comprehensive drug safety and toxicology research services as part of a total preclinical development package developed to provide a detailed evaluation of the potential toxicity of new drugs and to ensure regulatory compliance.
Services | Description |
Acute and sub-acute toxicity studies | Assessment of possible toxic effects of the drugs after single or repeated doses, including general observations, hematology, organ weights, and gross pathology. |
Genotoxicity and Carcinogenicity Screening | Determine whether a compound can harm genetic material or cause cancer. |
Reproductive and Developmental Toxicology Studies | Assessing the potential of drug effects on fertility, embryos, fetuses, and subsequently during postnatal development. |
Safety Pharmacology Studies | Assess the risk of a drug to produce effects on essential organ systems (e.g., cardiovascular, respiratory effects, CNS effects) at exposure levels near or above the anticipated therapeutic range. |
Histopathology Analysis | All tissues and organs from toxicology studies were examined microscopically to seek out morphological changes caused by the drug and evaluate the degree of damage. |
Biomarker Analysis | Identification and validation of novel safety biomarkers, including some that might specifically relate to iron-related organ damage or iron dysregulation, to enable earlier detection of adverse effects. |
Outside of our work in drugs, our dedication is to iron metabolism beyond pharmaceuticals. According to our in-depth scientific knowledge of iron's functions in the human body and its complicated biological pathways, we provide specialized services for the development of nutraceuticals and cosmetics for the improvement of iron metabolism. We are the connection between the latest research and products that benefit consumers, meaning what we test works and has the science behind it proving that it’s safe.
Iron, being a fundamental micronutrient, is at the core of many health concerns. We provide comprehensive development services for a range of iron-related health supplements and other nutraceuticals.
By Types
By Process
Iron plays a crucial role in skin and hair health. Dysregulation of iron can contribute to various dermatological and hair issues. Our cosmetic development services harness this understanding to create and develop innovative cosmetics.
By Types
By Process
Protheragen maintains a broad suite of drug-development platforms that permit systematic exploration of varied therapeutic strategies, ranging from innovative small molecules to cutting-edge biologics.
Diseases of iron metabolism derive from a dysregulation of iron status in the body. Both too little and too much iron can lead to serious health conditions. These conditions are important because they are common and produce a significant effect on the quality of life of individuals.
We are particularly interested in a range of diseases, such as Iron Deficiency Anemia (IDA), Hemochromatosis, Thalassemia Syndromes, and Anemia of Chronic Disease (ACD). We also focus on diseases such as Aceruloplasminemia and Neuroferritinopathy, addressing both deficiency and excess iron.
Animal models are essential for understanding disease and preclinical drug testing. We develop genetic models, dietary models, chemically induced models, and humanized animal models to assess drug efficacy, pharmacokinetics, and safety before human trials.
We offer one-stop services in drug discovery and preclinical development, including facets of the drug discovery pipeline (e.g., diagnostic development, therapeutic development, disease model development, DMPK studies, and safety and toxicology research).
DMPK studies are crucial for understanding how drugs are absorbed, distributed, metabolized, and eliminated (ADME). For drugs that mediate iron metabolism, such knowledge is important to:
Dose schedule to maximize effectiveness and minimize side effects.
Identify potential drug-drug interactions.
Predict human pharmacokinetic behavior, which is important for approval by regulatory agencies.
Yes, we offer services for the development of health supplements and cosmetics related to iron metabolism as well. We hold these nutraceutical and cosmetics developments to the same tough standards and are diligent in scientific rigor and professional manufacturing standards.