Cryopyrin-Associated Periodic Syndrome (CAPS)
Cryopyrin-associated periodic syndrome (CAPS) is a group of rare heterogeneous autoinflammatory diseases. At our company, we are dedicated to advancing the forefront of CAPS management through state-of-the-art diagnostics and therapeutics. As your trusted ally in CAPS research, we provide efficient and all-encompassing solutions to cater to your every scientific research need.
Overview of CAPS
Cryopyrin-associated periodic syndrome (CAPS) is a group of rare genetic conditions characterized by overlapping signs and symptoms, all stemming from the same underlying genetic cause. CAPS encompasses three distinct conditions: 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
CAPS is driven by genetic mutations in the NLRP3 gene, which encodes the cryopyrin protein. Cryopyrin belongs to a family of proteins known as intracellular "NOD-like" receptor (NLR) proteins, crucial players in the immune system's regulation of inflammation. The cryopyrin protein plays a pivotal role in assembling a molecular complex called an inflammasome, which triggers the inflammatory process.
Fig. 1 Two steps in NLRP3 inflammasome activation. (Matsuda, Tomoko, et al., 2023)Targets of CAPS Therapy
Interleukin-1β (IL-1β)
Mutations in the NLRP3 gene lead to overactivity of hot and cold proteins and overproduction of IL-1β, thereby causing CAPS. IL-1β blockers, such as canakinumab and anakinra, may be helpful in relieving CAPS symptoms.
Inflammasome
Formation of the inflammasome results in the release of IL-1β. Small molecule inhibitors targeting the inflammasome complex are currently being investigated to disrupt excessive activation of IL-1β and attenuate the inflammatory response in CAPS.
Our Services
Our company has established comprehensive platforms for developing rare disease diagnostics and therapies, encompassing small molecule drug, cell therapy, gene therapy, therapeutic antibody, therapeutic peptide, and therapeutic protein. Through our dedicated platforms, we are fully devoted to advancing the development of innovative diagnostic tools and therapies 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 | |
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.