Lipid-Based Drug Delivery Systems in Porphyria Treatment

Background Treatment Strategies Creative Biolabs' Solutions Workflow Related Services Resources

At Creative Biolabs, we specialize in cutting-edge lipid-based drug delivery solutions tailored for porphyria-related challenges. With our deep-seated expertise and a commitment to excellence, we provide tailored delivery platforms that can significantly advance your porphyria projects, helping you achieve remarkable results in porphyria research.

Background of Porphyria

Heme, an iron-containing compound that gives blood its red color, is synthesized in the bone marrow (80%) and liver (15%) through a complex process involving eight different enzymes. Porphyria is a group of metabolic disorders caused by specific enzyme deficiencies in the heme biosynthesis pathway. This results in the abnormal accumulation of porphyrins and heme precursors, including 5-aminolevulinic acid (ALA) and porphobilinogen (PBG), in the body's tissues and organs, subsequently causing cellular damage. The neurotoxic heme precursors can result in acute hepatic porphyrias, while the photoreactive porphyrins, when excess, cause skin porphyrias characterized by photosensitivity.

Porphyria-1. (Erwin, Angelika L., and Manisha Balwani, 2021) (OA Literature)Fig. 1 The heme biosynthetic pathway.1

Based on the primary source of excess porphyrins or heme precursors, porphyrias are classified as either hepatic or erythropoietic.

Porphyria Porphyrin Origin Related enzymes
Acute Hepatic Porphyrias (AHP)
ALA-dehydratase deficiency porphyria (ADP) Hepatic Delta-aminolevulinic acid dehydratase (ALAD)
Acute intermittent porphyria (AIP) Hepatic Hydroxymethylbilane synthase (HMBS)
Hereditary Coproporphyria (HCP) Hepatic Coproporphyrinogen oxidase (CPOX)
Variegate Porphyria (VP) Hepatic Protoporphyrinogen oxidase (PPOX)
Cutaneous Erythropoietic Porphyrias
Erythropoietic protoporphyria (EPP) Erythropoietic Ferrochelatase (FECH)
X-linked protoporphyria (XLP) Erythropoietic Delta-aminolevulinic acid synthase 2 (ALAS2)
Congenital erythropoietic porphyria (CEP) Erythropoietic Uroporphyrinogen III synthase (UROS)
Cutaneous Hepatic Porphyrias
Porphyria cutanea tarda (PCT) Hepatic Uroporphyrinogen decarboxylase (UROD)
Hepato-erythropoietic porphyria (HEP) Erythropoietic, hepatic Uroporphyrinogen decarboxylase (UROD)

Treatment Strategies for Porphyria

  • Heme Therapy: This is the first-line treatment for acute porphyria. However, frequent heme infusions can increase the activity of aminolevulinate synthase 1 (ALAS1), thereby maintaining disease activity.
  • Glucose Supplementation: Administering glucose can be used as a monotherapy for sporadic porphyria. It helps to inhibit the activity of ALAS1, reducing the synthesis of heme precursors.
  • GalNAc-siRNA Conjugates: Givosiran, which is conjugated with GalNAc, can downregulate the expression of ALAS1. This helps to prevent the accumulation of the neurotoxic heme precursors ALA and PBG that are associated with AHP attacks.
  • Phlebotomy: This procedure involves removing blood to gradually deplete excess iron from the body. As iron levels decrease, the activity of uroporphyrinogen decarboxylase in the liver can return to normal, and the levels of porphyrins in the liver and blood can gradually decrease. It is commonly used for PCT.
  • Chloroquine or Hydroxychloroquine: These drugs can increase the excretion of porphyrins through urine, thereby helping to clear excess porphyrins from the liver.

Creative Biolabs can utilize its advanced lipid-based drug delivery systems, including liposomes and lipid nanoparticles (LNPs), to enhance the therapeutic effects of these treatment strategies for porphyria.

How Creative Biolabs' Lipid-Based Drug Delivery Systems Can Assist Your Project

  • Reduced Administration Frequency: Our lipid-based delivery systems can encapsulate heme, enhancing its stability and bioavailability. This allows for sustained release, reducing infusion frequency and preventing ALAS1 activity rebound, thus improving heme therapy.
  • Enhanced Drug Stability: Our liposomes can increase the stability of drugs such as heme, protecting them from premature degradation. This ensures more active drug reaches the target site, improving therapeutic efficacy.
  • Targeted Nucleic Acid Drug Delivery: By combining GalNAc with LNPs, Creative Biolabs can target hepatocytes, improving the delivery efficiency of siRNA drugs like givosiran.
  • Improved Skin Targeting: For skin-related porphyria symptoms, our lipid-based carriers can enhance the skin-targeting ability of drugs. This increases local drug concentration, improving therapeutic effects and reducing photosensitivity.

Our advanced technology offers innovative solutions to improve drug delivery, enhance therapeutic outcomes, and overcome challenges associated with porphyria. By choosing Creative Biolabs, you can leverage our expertise to advance your porphyria research and develop more effective therapies.

Workflow for Lipid-Based Drug Delivery Systems Development for Porphyria

Contact & requirements One-to-one technical support Submit custom service form Project start Product delivery Optional Pharmacodynamic Study Analysis and Characterization

With our expertise, customized solutions, and collaborative approach, Creative Biolabs can help you overcome challenges and achieve breakthroughs in porphyria research. We ensure consistent quality and reliable data in every project, which is crucial for advancing porphyria therapies. If you're ready to overcome research obstacles and drive innovation in porphyria treatment, contact us today.

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Resources

Reference

  1. Erwin, Angelika L., and Manisha Balwani. "Porphyrias in the age of targeted therapies." Diagnostics 11.10 (2021): 1795. Distributed under Open Access license CC BY 4.0, without modification.
For Research Use Only. Not For Clinical Use

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