Amyloidosis
Amyloidosis is an umbrella term that relates to a group of diseases that are marked by the deposition of abnormal proteins in tissues. Our company focuses on enabling development of diagnostics and therapeutics targeted towards management of amyloidosis. Thus, as your partner in amyloidosis research, we provide a collaborative approach to meet all the requirements of your scientific research.
Introduction to Amyloidosis
Amyloidosis is a set of rare and intricately connected disorders that affects many organs and tissues of the body. It is marked by the deposition of abnormally shaped proteins called amyloid fibrils that disturb normal functioning of the organ and can result in organ failure. The range of amyloidosis is thought to be around 30 in every 100,000 people.
Fig. 1 Molecular mechanisms of cardiotoxicity in AL amyloidosis. (Ikura, Hidehiko, et al., 2022)Pathogenesis of Amyloidosis
The process of Protein misfolding leads to the formation of amyloid fibrils, which get accumulated in different organs and result in organ dysfunction, progressively leading to the development of amyloidosis. The exact process of amyloid fibril formation remains unclear, though there are many prominent proteins that are common in many simple types of amyloidosis that have been discovered.
| Disease Type | Fibril Protein | Affected Organs | Incidence |
|---|---|---|---|
| AL amyloidosis | Immunoglobulin light chains (AL) | Heart, kidneys, and other organs | 12/1,000,000 |
| AA amyloidosis | Serum A protein | Kidneys, liver, and gastrointestinal tract | 1-2/1,000,000 |
| ATTR amyloidosis | Transthyretin (TTR) protein | heart and nervous system | 1/5,800 |
Strategies of Amyloidosis Therapy Development
Reduce Abnormal Protein Production
Amyloidosis stems from relatively misunderstood abnormal production of certain proteins. In AL amyloidosis for example, chemotherapy protocols, immunomodulatory agents, or proteasome inhibitors can be utilized to suppress the synthesis of abnormal light chains.
Inhibit Protein Misfolding and Aggregation
Another focal point in developing AL amyloidosis therapeutics is blocking the formation of amyloid fibrils. For instance, Tafamidis is a small molecular stabilizer of ΤΤR that washes out in oligomeric form and sets to TTR to prevent its misfolding and conversion into amyloid fibrils.
Clear Amyloid Deposits
One more therapeutic option is to actively try removing the amyloid deposits from the affected tissues. A few monoclonal antibodies being tested, such as birtimab, NEOD001, and daratumumab, are designed to identify and clear amyloid deposits while modulating immune responses.
Our Services
With a solid understanding of the biotechnology field and years of experience in the industry, our company provides complete approaches to diagnostic and therapeutic studies of amyloidosis related to biotechnology.
- Diagnostic Development Services: For rare diseases such as amyloidosis, our company offers diagnostic development services. We are dedicated to assisting you in the development of rapid and point-of-care diagnostic tests for amyloidosis, ensuring accurate and timely detection.
- Therapeutic Development Services: Our company provides a wide range of services for the development of small molecule drug, cell therapy, gene therapy, therapeutic antibody, therapeutic peptide, and therapeutic protein. We specialize in the development of monoclonal antibody drugs, a highly promising avenue for amyloidosis therapeutics.
- Animal Model Development Service: To support the preclinical research and development of amyloidosis therapeutics, we offer amyloidosis animal models development services to facilitate your pharmacokinetics study and drug safety evaluation.
| Xenograft Models | ||
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| Xenograft models involve the transplantation of human tissue or cells into immunodeficient animals. These models are particularly useful for studying localized forms of amyloidosis, in which deposition of amyloid fibrils occurs in specific organs or tissues. | ||
| Genetically Engineered Models | ||
| Our company is dedicated to the development of genetically engineered models for amyloidosis research. Our scientists have successfully integrated mutant human genes associated with amyloidosis into the mouse genome, effectively replicating the key characteristics of human amyloidosis. These transgenic models play a crucial role in assessing disease progression and evaluating the efficacy of therapeutic interventions. | ||
| Optional Models |
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| Optional Species | Mice, Rats, Nematodes, Drosophila, Non-Human Primates (Monkeys), Others | |
No matter what research stage you are at, we can provide you with corresponding research services. If you are interested in our services, please feel free to contact us for more details and quotation information for related services.
References
- Ikura, Hidehiko, et al. "Molecular mechanism of pathogenesis and treatment strategies for AL amyloidosis." International journal of molecular sciences 23.11 (2022): 6336.
- Picken, Maria M. "The pathology of amyloidosis in classification: a review." Acta haematologica 143.4 (2020): 322-334.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.