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Customized Solutions for Iron Metabolism-related Disease Research and Drug Development

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.

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Overview of Iron Metabolism-related Diseases

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.

Types of Iron Metabolism-related Diseases

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

Diagnosis of Iron Metabolism-related Diseases

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.

Blood Tests

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.

Genetic Testing

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.

Imaging Techniques

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.

Targets for Iron Metabolism-related Diseases

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.

  • Hepcidin: The master regulator of systemic iron homeostasis. Modulating hepcidin levels or its activity (e.g., hepcidin agonists for iron overload, hepcidin antagonists for iron deficiency) is a major therapeutic strategy.
  • Ferroportin: The only known iron exporter. Targeting ferroportin offers avenues for controlling cellular iron efflux.
  • Transferrin Receptor: Involved in cellular iron uptake.
  • Divalent Metal Transporter 1 (DMT1): Crucial for dietary iron absorption.
  • Others: Steap3 and other proteins are involved in erythropoiesis and iron recycling.

Challenges in Iron Metabolism Drug Development

  • Specificity and Off-Target Effects: Targeting fundamental iron pathways can lead to unintended consequences on other physiological processes.
  • Bioavailability and Delivery: Ensuring sufficient drug concentration at the target site.
  • Long-Term Safety: For chronic conditions, the long-term safety profiles of new drugs are particularly crucial.
  • Translational Gaps: Bridging the gap between promising preclinical findings and successful outcomes.
  • Diagnostic Precision: Developing more accurate and accessible diagnostic tools for early detection and disease stratification.

Customized Services

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.

Therapeutic Development Services

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.

Disease Model Development Services

Cell-based Models Development

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.

Cell-based Models Development
Organoid Models Development

Organoid Models Development

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.

  • Adult Stem Cell (ASC)-derived Organoid
  • Embryonic Stem Cell-derived Organoid
  • iPSC-derived Organoid Model
  • Patient-derived Organoid (POD) Model

Animal Models Development

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.

Drug Metabolism and Pharmacokinetics (DMPK) Studies

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.

  • Pharmacokinetic studies
  • Bioanalytical method development and validation
  • Metabolite identification and profiling
  • Drug-drug interaction studies

Drug Safety and Toxicology Research

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.

Iron Metabolism-related Nutraceutical and Cosmetics Development Services

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 Metabolism-related Nutraceutical Development Services

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 Types

  • Iron Supplements Development
  • Iron Absorption Enhancers Development
  • Iron Chelators Development
  • Hepcidin Modulators Development
  • Anti-oxidants Development

By Process By Process

  • Nutraceutical Formulation Development
  • Nutraceutical Safety Testing
  • Nutraceutical Efficacy Testing
  • Bioavailability Studies of Nutraceuticals
  • Nutraceutical Stability Testing

Iron Metabolism-related Cosmetics Development Services

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 Types

  • Anti-Aging Products Development
  • Anti-Inflammatory Skincare Development
  • Color Cosmetics Development
  • Hair Care Products Development
  • Barrier Repair Skincare Development
  • Sun Care Products Development
  • Scar Repair Products Development

By Process By Process

  • Cosmetic Formulation Development
  • Cosmetic Safety Testing
  • Cosmetic Efficacy Testing
  • Stability Testing for Cosmetics
  • Microbiology Testing for Cosmetics

Advanced Technology Platform

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.

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All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.