Scleroderma
Scleroderma, also known as systemic sclerosis (SSc), is a complex autoimmune disease characterized by excessive collagen deposition, leading to fibrosis and thickening of the skin and internal organs. At our company, we specialize in the development of drugs and therapies for autoimmune diseases and inflammation, including scleroderma.
Overview of Scleroderma
Scleroderma is a rare and chronic connective tissue disease characterized by abnormal immune system activation, resulting in the production of excess collagen. This excessive collagen deposition leads to fibrosis in multiple organs, including the skin, joints, blood vessels, and internal organs such as the lungs, heart, and gastrointestinal tract. The symptoms and severity of scleroderma can vary widely among individuals, making it a complex and heterogenous disease.
Extensive research efforts have revealed key players in the pathogenesis of scleroderma, including cytokines, growth factors, and immune cells such as T and B lymphocytes. Dysregulated signaling pathways, such as the transforming growth factor-beta (TGF-β) pathway, have been identified as critical drivers of fibrosis. Targeting these pathways and modulating immune responses hold promise for developing therapeutics.
Fig.1 Hematoxylin-eosin staining analysis of scleroderma. (Rosendahl A.H., et al., 2022)
Drug Discovery and Development for Scleroderma
Several approaches are being explored and have shown promise in scleroderma therapeutics studies.
| Types of Therapies | Description |
|---|---|
| Immunosuppressive Therapy | Immunosuppressive therapies, such as methotrexate, mycophenolate mofetil (MMF), and cyclophosphamide, have been used in the therapeutic of scleroderma. |
| Biologic Therapy | Tocilizumab, an interleukin-6 receptor antagonist, has demonstrated potential in reducing skin thickening and preserving lung function in cases with systemic sclerosis-related cutaneous disease. |
| Antifibrotic Therapy | Antifibrotic therapies aim to directly inhibit the excessive production and deposition of collagen in scleroderma. Nintedanib and pirfenidone, two antifibrotic agents approved for the therapeutic of idiopathic pulmonary fibrosis. |
| Gene Therapy | The use of small interfering RNA (siRNA) molecules to inhibit the expression of specific genes implicated in fibrosis, such as connective tissue growth factor (CTGF), has shown potential in preclinical studies. |
We are at the forefront of advancing scleroderma therapy, conducting extensive research to identify potential targets for drug intervention and develop groundbreaking therapeutic strategies. Click on the provided links below to explore and discover your personalized solution.
Small Molecule Drug
Probiotics Therapy
Therapeutic Antibody
Cell Therapy
Gene Therapy
Therapeutics Development
Our Services
Through our extensive R&D capabilities, we strive to identify new therapeutic targets and develop diagnostics and innovative therapies for scleroderma. Our expertise lies in animal models and in vitro models to ensure rigorous evaluation of safety, efficacy, and tolerability.
Inducible Fibrosis Models
Our company specializes in creating inducible fibrosis models. These models involve the administration of fibrosis-inducing agents such as bleomycin or transforming growth factor-β (TGF-β) to trigger the development of fibrosis.
TbRIIDk-fib Mouse Models
The transgenic mouse strain TbRIIDk-fib, provided by our expert team, overexpresses kinase-deficient type II TGF-b receptor in fibroblasts. These mice exhibit progressive fibrosis in the skin, lung, and gut.
Stiff Skin Syndrome-Mutant Fibrillin-1 Knock-in Models
Another model we offer is the stiff skin syndrome-mutant fibrillin-1 knock-in mouse model. These mice exhibit cutaneous fibrosis and immunological phenotypes associated with SSc.
Fli1 and KLF5-Deficient Mouse Models
Our company provides Fli1 and KLF5-deficient mouse models, which spontaneously develop SSc-like features, including skin fibrosis, vasculopathy, B cell activation, and autoantibody production.
Through our cell-based models, including fibroblast, endothelial cell, and immune cell models, we can study key cellular processes underlying SSc pathogenesis and evaluate therapeutics in vitro.
Our organoid models, such as skin and lung organoids, provide a physiologically relevant platform to study complex organ-specific interactions and evaluate potential therapeutic strategies.
Thorough preclinical research is a crucial step in the drug development process. At our company, we offer comprehensive preclinical research services for scleroderma, ranging from in vitro studies to pharmacokinetic and safety evaluations. In addition to the aforementioned range of services and models, we also specialize in tailoring customized solutions and developing disease models that precisely align with your distinctive requirements. Should our array of services capture your interest, we warmly encourage you to reach out to us without hesitation.
References
- Rosendahl Ann-Helen, Katrin Schönborn, and Thomas Krieg. "Pathophysiology of systemic sclerosis (scleroderma)." The Kaohsiung journal of medical sciences 38.3 (2022): 187-195.
- Zhu Jane L., et al. "Emerging treatments for scleroderma/systemic sclerosis." Faculty Reviews 10 (2021).