Systemic Sclerosis (SSc) or scleroderma is a chronic autoimmune connective tissue disease that is rare and characterized by vascular injury, immune response activation, and widespread fibrosis of the skin and internal organs. For Systemic Sclerosis, Protheragen provides comprehensive preclinical drug and therapy development systems which will make your research processes easier while providing innovative solutions.
Systemic Sclerosis is a chronic autoimmune condition which is extremely disabling and affects multiple organ systems. microvessels are damaged along with the immune system, resulting in immune dysregulation. SSc affects approximately 7 to 50 individuals for every 100,000 people, and more women are affected by the disease. The need for this type of therapy is often overlooked, which indicates the wide gap that there is for better focused and effective therapies for the condition.
Systemic Sclerosis involves an intricate interplay of three core processes: immune activation, vascular damage, and fibrosis. In the case of Systemic Sclerosis, an autoimmune response is triggered early in the disease which activates different immune cells such as T and B lymphocytes capable of producing autoantibodies. The immune activation causes damage to the endothelial cells and results in a type of vasculopathy with narrowing of the blood vessels and reduced circulation. The chronic immune activation in the body and persistent damage to the vascular system ultimately leads to the activation of fibroblasts, which then becomes myofibroblasts. These myofibroblasts that are now activated produce collagen and other extracellular matrix components in excess, leading to the aforementioned progressive fibrosis and tissue hardening in the skin and internal organs. Some important mediators involved are: transforming growth factor-beta (TGF-β), connective tissue growth factor (CTGF) and certain profibrotic cytokines.
| Category | Drug Name | Target / Intervention | Mechanistic Validation/Model Data | Development Stage |
| Biologics | Belimumab | BAFF inhibitor | Anti-Scl-70 antibody titer reduction at 48 weeks | Phase III |
| Anifrolumab | IFNAR blockade | Improved skin ulcer healing; reduced ISG expression in fibroblasts | Phase II | |
| Imsidolimab | IL-36R antagonism | Inhibition of TGF-β-driven fibroblast transformation (3D skin models) | Phase II | |
| Small Molecule | AXA-1125 | PPARγ agonism | Reduced lung collagen density (HRCT); suppression of fibrotic gene signatures | Phase II |
| Cell Therapy | Autologous HSCT | Immune reset | ASTIS trial: Improved 5-year survival in early diffuse SSc; T-cell clonal clearance | Approved |
| FAP-CAR-T | Fibroblast activation protein | Significant skin thickness improvement in Tsk-1 mice; ECM remodeling | Preclinical |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
Protheragen provides a full testing service offering research services to advance development for systemic sclerosis. Our team of scientists, dermatologists, rheumatologists, and geneticists use and develop cutting-edge technology for you to expedite the adoption of new therapeutic options for this complex disease; and provide specialized, focused therapeutic development and disease model development.
Protheragen offers customized in vitro (2D cell models and 3D skin models) allowing to study unique molecular pathogenic processes associated with systemic sclerosis, including excessive collagen deposition, vascular damage, and immune cell activation. Our animal models faithfully reproduce classical disease phenotypes that resemble the fibrosis and vascular abnormalities seen in SSc, allowing for strong in vivo validation of both targeted therapies and small molecule therapeutics.
In Vivo Pharmacokinetics Services
Protheragen is a one-stop preclinical research services provider who is committed to advancing therapeutics for complex autoimmune and rare skin diseases, including Systemic Sclerosis. Our end-to-end solutions range from target discovery, disease modeling, and comprehensive drug safety evaluation and DMPK services. If you are interested in our services, please feel free to contact us.
References
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.
