Focal Segmental Glomerulosclerosis (FSGS)
Focal segmental glomerulosclerosis (FSGS) is a complex kidney disease characterized by the presence of sclerosis in certain glomeruli, leading to impaired kidney function. This condition affects the tiny filters in the kidneys, known as glomeruli, which are essential for filtering waste and excess fluids from the blood. Our company stands out in the field of rare diseases and is committed to providing comprehensive services that cater to the needs of the majority of researchers.
Overview of FSGS
FSGS is a kidney disease with the incidence varying from 1.4 to 21 cases per million population. The pathology of FSGS involves a variable degree of podocyte foot process effacement and gaps in the coverage of the glomerular basement membrane. Podocytes are specialized cells that play a crucial role in maintaining the structural integrity of the glomeruli and in regulating kidney function. In FSGS, scar tissue forms on the glomeruli, leading to impaired kidney function and potential progression to end-stage renal disease.
Fig.1 Current therapy of FSGS. (de Cos, M., et al., 2022)Pathogenesis of FSGS
The pathogenesis of FSGS involves a combination of genetic predisposition, immune system abnormalities, and environmental triggers. While genetic factors play a significant role in some cases, immune dysregulation and external stimuli can also precipitate the onset of FSGS. Additionally, conditions such as hypertension, diabetes, HIV, obesity, and certain medications have been identified as potential triggers for the disease. The formation of scar tissue on the glomeruli leads to characteristic focal and segmental damage, which results in proteinuria, edema, and decreased kidney function in FSGS individuals
Fig.2 Emerging therapeutic targets. (de Cos, M., et al., 2022)Biomarkers Development of FSGS
In diagnosing and monitoring the progression of FSGS, biomarkers play a crucial role as measurable indicators that provide valuable information. Different biomarkers have been identified in the context of FSGS, aiding in the diagnosis and management of the disease.
- Angiotensin II type 1 receptors (AT1R)
- Plasminogen activator inhibitor type-1 (PAI-1)
- Metalloproteinases (MMP)
- Forkheadbox P3 (FOXP3)
- Tissue inhibitors of metalloproteinases (TIMPs)
- Transforming growth factor-beta (TGF-β)
- Dystroglycans (DG)
- Human neutrophil gelatinase-associated lipocalin (NGAL)
- MicroRNAs (miR-192 and miR205, miR-186)
- Malondialdehyde (MDA)
Therapeutics Development of FSGS
| Therapeutics Types | Names | Mechanism of Action | Research Phase |
|---|---|---|---|
| Inhibitor | Sparsentan | Dual ETA receptor/AT1 receptor antagonist | Phase II trials |
| Atrasentan | TRPC5 channel inhibitor | Phase I trials | |
| Baricitinib | Janus kinase-STAT inhibitor | Phase II trials | |
| Antibody | ALXN1720 | Anti-C5 mini-body | Phase I trials |
| Adalimumab | Antihuman TNF-α antibody | Phase II trials | |
| VB119 | Anti-CD19 antibody | Phase II trials | |
| Obinutuzumab | Anti-CD20 antibody | Phase II trials |
Our Services
With a deep understanding of the challenges and complexities involved in studying rare diseases, and the animal model and therapeutic development platform, we offer a wide range of specialized services that encompass various aspects of research.
Platforms of FSGS Therapy Development
Animal Models of FSGS
Animal models are essential for understanding the underlying mechanisms of FSGS, testing new therapy approaches, and evaluating potential therapeutic targets. Our company provides a variety of chemical induction models or genetic engineering models to support your disease mechanism research and innovative therapy development.
Chemical-induced animal models involve the administration of the chemotherapy drug adriamycin or anti-glomerular basement membrane (GBM) antibody to induce kidney damage that resembles FSGS in animals.
Optional Models: Adriamycin-induced model; GBM antibody-induced model, etc.
Genetic engineering animal models of FSGS involve the use of genetic engineering techniques such as CRISPR/Cas9 to alter specific genes in animals to mimic the genetic mutations seen in human individuals with FSGS.
Optional Models: Col4a5tm1Yseg model; Wt1tm2(cre/ERT2) Wtp model, etc.
Reasons To Choose Us
Our team of experts consists of professionals with diverse backgrounds and extensive experience in rare disease research, ensuring that we can meet the unique requirements of researchers from different disciplines. We offer tailored solutions including pharmacokinetic research and drug safety evaluation.
If you are interested in learning more about our services and how we can support your research endeavors, please do not hesitate to reach out to us for further information.
References
- de Cos, Marina et al. "Novel Treatment Paradigms: Focal Segmental Glomerulosclerosis." Kidney international reports 8.1 (2022): 30-35.
- Musiała, Aleksandra et al. "Biomarkers in Primary Focal Segmental Glomerulosclerosis in Optimal Diagnostic-Therapeutic Strategy." Journal of clinical medicine 11.12 (2022): 3292.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.