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Porphyria Cutanea Tarda

Porphyria cutanea tarda is the most frequent variant of porphyria. It is often associated with an existing internal condition, which poses hepatologic concerns impacting life expectancy. Protheragen is committed to providing complete diagnostic, therapeutic, disease modeling, and preclinical research services to assist scientists and researchers in the understanding of iron metabolism diseases. We are particularly focused on the complex interplay of porphyria cutanea tarda and disorders of iron metabolism, offering tailored and thorough services for porphyria cutanea tarda therapeutic development.

Overview of Porphyria Cutanea Tarda

Porphyria cutanea tarda develops from the inhibition of uroporphyrinogen decarboxylase (UROD) in the presence of hepatic iron alongside oxidative stress. Most individuals suffering from porphyria cutanea tarda demonstrate signs of siderosis upon liver biopsy, and the condition improves with iron depletion. Porphyria cutanea tarda is characterized by the skin's fragility, blistering lesions on the skin that occur in areas exposed to sunlight, dark urine, and heightened levels of porphyrins in both plasma and urine.

Porphyria cutanea tarda is a unique iron-related disorder. Fig.1 A unique iron-related disorder, porphyria cutanea tarda. (Leaf, R. K., and Dickey, A. K., 2024)

Iron Homeostasis Abnormalities in Porphyria Cutanea Tarda

The development of porphyria cutanea tarda is associated with free oxygen radicals, which are a product of iron, that oxidize uroporphyrinogen to form a uroporphomethene, which then inhibits UROD. In murine models, the formation of uroporphomethene is directly linked to the concentration of iron in the liver, and in heterozygous UROD knockouts, there is a phenotypic expression of porphyria cutanea tarda with high iron levels. In addition, hepcidin, the master controller of iron regulation in the body, is thought to be involved in porphyria cutanea tarda's pathogenesis.

Porphyrin accumulation and PCT symptoms result from uroporphomethene inhibiting UROD. Fig.2 UROD inhibition by uroporphomethene leads to porphyrin accumulation and porphyria cutanea tarda. (Leaf, R. K., and Dickey, A. K., 2024)

Therapeutics Development for Porphyria Cutanea Tarda

Drug Name	Mechanism of Action	Targets	Research Phase
Deferoxamine	Binding extra iron, lowering hepatic iron deposits, and, as a consequence, reducing oxidative stress and inhibition of UROD activity.	Iron chelation	Approved
Hydroxychloroquine	With respect to hydroxychloroquine, at low doses, it helps in moving excess porphyrins out of the liver and alleviates symptoms.	Autophagy	Phase II
Deferasirox	Promoting the binding and excretion of excess iron helps in reducing the hepatic concentration of iron, which in turn decreases the accumulation of porphyrin.	CYP2C8	Phase III

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

At Protheragen, we are at the forefront of porphyria cutanea tarda drug research and development. We provide an all-in-one suite of solutions that include diagnostics, therapeutics, and even disease model development, which helps to accelerate your scientific work. In addition, we provide pharmacokinetics and drug safety evaluation services that thoroughly assess drug absorption, distribution, metabolism, excretion, and possible toxicities, which are vital to supporting promising therapeutic candidates.

Therapeutic Development Services

Animal Model Development Services for Porphyria Cutanea Tarda

For various researchers across the globe seeking comprehensive in vivo platforms, our services in animal model development stand out as the most reliable option. We create and provide tailor-made animal models of porphyria cutanea tarda, which accurately reflect the human disease pathology and are crucial for studying the mechanisms of the disease, assessing new diagnostic markers, and evaluating novel therapeutic targets.

Induced Animal Models

These models induce porphyria cutanea tarda-like symptoms by administering specific chemicals or diets that interfere with UROD activity or induce iron overload.

Iron/ALA/polychlorinated biphenyls (PCB)-induced model
And more

Genetically Engineered Models

These models involve the direct modification of the animal's genome to introduce or remove genes pertinent to porphyria cutanea tarda.

Urod knockout model
And more

Pharmacokinetics and Drug Safety Evaluation Services

Pharmacokinetic Research Services
Physiochemical Studies	Bioavailability Studies
In Vitro Metabolism Studies	Metabolite Profiling and Identification Studies
In Vitro Permeability and Transporter Studies	Mass Balance Studies
Drug Absorption Studies	Biliary Excretion Studies
Quantitative Tissue Distribution Studies
Drug Safety Evaluation Services
General Toxicology Evaluation	Phototoxicity Evaluation
Genetic Toxicology Evaluation	Tissue Cross-Reactivity Evaluation
Developmental and Reproductive Toxicity Evaluation	Immunogenicity and Immunotoxicity Evaluation
Safety Pharmacology Evaluation	Toxicokinetics Evaluation
Local Toxicity Evaluation

Protheragen's porphyria cutanea tarda research and development services include target identification and validation, lead compound discovery and optimization, and full-scale preclinical testing. Our proficiency in developing and applying such solutions guarantees that your studies are performed with utmost scientific precision. Reach out to us today so that we may discuss how our bespoke services can advance your projects.

Reference

Leaf, Rebecca K, and Amy K Dickey. "Porphyria cutanea tarda: a unique iron-related disorder." Hematology. American Society of Hematology. Education Program 2024.1 (2024): 450-456.

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

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