Cryopyrin-Associated Periodic Syndrome (CAPS)
Cryopyrin-associated periodic syndrome (CAPS) is a group of unusual heterogeneous self-inflammation syndromes. Here at the company, we focus on efficient CAPS management by providing novel diagnostics and therapeutics. As your partner in CAPS-related studies, we afford high quality, comprehensive services to meet the scientific research scope.
Overview of CAPS
CAPS is a nomenclature for a set of rare genetic disorders that have overlapping phenotypes, which are all due to a single underlying mutation. CAPS consists of three separate conditions, including familial cold autoinflammatory syndrome type 1 (FCAS1), Muckle-Wells syndrome (MWS), and neonatal onset multisystem inflammatory disorder (NOMID).
| Disease Types | Symptoms and Manifestations | Severity | Incidence | ||
| FCAS1 |
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Mildest | 1-2/1,000,000 |
| MWS |
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Intermediate Severity | |
| NOMID |
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Most Severe | |
Pathogenesis of CAPS
Novel cryopyrin-associated periodic syndrome or CAPS results exclusively from NLRP3 gene mutations. The NLRP3 gene is responsible for the production of a protein called cryopyrin. NLR proteins, which are a part of the immune system, have a role in controlling inflammation. NLRPs constitute the large family of intracellular proteins and are often referred to as a “NOD-like” receptor. The cryopyrin protein is responsible for assembling a macromolecular structure called an inflammasome, which activates inflammation.
Fig. 1 Two steps in NLRP3 inflammasome activation. (Matsuda, Tomoko, et al., 2023)Targets of CAPS Therapy
Interleukin-1β (IL-1β)
Caps is brought about by mutations of the NLRP3 Gene which results in the overproduction of hot and cold proteins and consequent overproduction of IL-1β. Blocking agents such as canakinumab and anakinra may ease the symptoms of CAPS.
Inflammasome
The formation of the inflammasome complex leads to the secretion of IL-1β. Investigational new drugs are being developed that target allosteric regulation of the inflammasome complex to prevent hypersecretion of IL-1β and decrease the inflammation response in CAPS.
Our Services
Our company provides a full solution for the diagnostics and therapies for rare diseases and drug development such as small molecule drug, cell therapy, gene therapy, therapeutic antibody, therapeutic peptide, and therapeutic protein. In particular, we focus on developing diagnostic and therapeutic methods for Cryopyrin Associated Periodic Syndrome CAPS.
Recognizing the significance of animal disease models in the therapy development for CAPS, we offer our expertise in establishing animal models specifically tailored for CAPS. These models serve as invaluable tools to facilitate the safety evaluation and pharmacokinetics study of your drug candidates.
Animal Models of CAPS
| Xenograft Models | ||
| Xenograft models involve the transplantation of human cells or tissues into immunodeficient mice. In the context of CAPS, researchers can transplant immune cells or tissues obtained from CAPS individuals into mice to study the immune dysregulation and inflammatory responses characteristic of the disease. Cell lines for CAPS xenografts include THP-1 cells, peripheral blood mononuclear cells (PBMCs), NLRP3-Mutant patient-derived cells, cytokine-stimulated cells, etc. | ||
| Genetically Engineered Models | ||
| Our scientists have introduced Nlrp3 gene mutations into animal genomes via knock-in or transgenic techniques to replicate the dysregulated immune response and inflammation seen in CAPS. These models enable studying disease progression, evaluating potential therapeutics, and exploring underlying molecular mechanisms. | ||
| Optional Models |
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| Optional Species | Mice, Rats, Rabbits, Guinea Pigs, and Zebrafish, Non-Human Primates (Macaques), Others | |
In supporting the efforts in developing CAPS specific animal disease models, we provide services in the creation of disease animal models for CAPS which are essential for the safety evaluation and pharmacokinetics study of your drugs.
Regardless of your current stage of research, we offer comprehensive research services tailored to your needs. If you are interested in our services, please don't hesitate to contact us for more information and a detailed quotation regarding the specific services you require.
References
- Matsuda, Tomoko, et al. "Similarities and differences in autoinflammatory diseases with urticarial rash, cryopyrin-associated periodic syndrome and Schnitzler syndrome." Allergology International 72.3 (2023): 385-393.
- Brydges, Susannah D., et al. "Animal models of Inflammasomopathies reveal roles for innate but not adaptive immunity." Immunity 30.6 (2009): 875.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.