As a dedicated research service provider, our commitment lies in advancing the development and innovation of therapies for rare kidney diseases. These diseases present significant challenges within the medical community, affecting countless patients and often leading to severe health complications. We provide preclinical research services designed to tackle the complexities of rare kidney diseases and support effective therapy development.
Introduction to Rare Kidney Diseases Therapeutic Development
Advancements in therapeutic development for rare kidney diseases have centered on understanding their genetic and molecular foundations. Genomic technologies, such as high-throughput sequencing, enable researchers to identify disease-specific genetic mutations, which supports the development of targeted therapies and gene editing. These breakthroughs offer new avenues for effective management and potential cures for rare kidney diseases.
Fig.1 Kidney diseases have several targets for multi-target drugs. (Wigner, P., et al., 2024)
Types of Rare Kidney Disease Therapies
- Genetic Therapies: Genetic therapies aim to address the underlying genetic causes of certain rare kidney diseases. Techniques such as gene editing and gene replacement therapy are being explored to correct genetic mutations. For example, CRISPR-Cas9 technology is being studied for its potential to precisely target and edit defective genes responsible for Alport syndrome, a rare genetic kidney disorder.
- Protein Replacement Therapies: These therapies focus on replacing deficient or dysfunctional proteins that are crucial for kidney function. Enzyme replacement therapy, used in Fabry disease, provides patients with recombinant alpha-galactosidase A to break down fats that accumulate in cells, thus alleviating disease symptoms and improving kidney function.
- Small Molecule Drugs: Small molecule drugs are designed to modulate specific biochemical pathways involved in kidney diseases. For instance, bardoxolone methyl, a small molecule activator of the Nrf2 pathway, is being investigated for its potential to improve kidney function in patients with Alport syndrome by reducing inflammation and oxidative stress.
- Immunosuppressive and Anti-inflammatory Therapies: These treatments are used to manage autoimmune-related kidney diseases by suppressing the immune response. Rituximab, an antibody that targets CD20 on B-cells, is used in conditions like membranous nephropathy to reduce the production of harmful antibodies that attack kidney tissue.
- Cell-based Therapies: This innovative approach utilizes stem cells or modified cells to repair and regenerate damaged kidney tissue. Mesenchymal stem cells (MSCs) are being studied for their ability to reduce inflammation and promote healing in conditions such as acute kidney injury by differentiating into renal cells and secreting protective factors
Table1. Clinical experience with epigenetic modulators. (Fontecha-Barriuso, M., et al., 2018)
| Target |
Drug |
Clinical development stage |
Indications |
Disease |
| DNA methylation |
| DNMT inhibitor |
5'-Azacytidine |
In clinical use |
MDS |
AE: increased sCr |
| Decitabine |
In clinical use |
AML |
AE: uncommon increased sCr |
| TET3 demethylase activator |
Hydralazine |
In clinical use |
Hypertension |
Slows progression of CKDas part of anti-hypertensive regimens |
| Histone methylation or demethylation inhibitors |
| HMTKMT4DOT1L(H3K79) |
Pinometostat (EPZ5676) |
Phase I |
Leukaemia |
ND |
| HMTKMT6EZH2(H3K27) |
Tazemetostat |
Phase II |
Malignancy |
ND |
| Histone acetylation inhibitor |
| Class I HDAC |
Valproic acid |
In clinical use |
lymphoma Epilepsy |
AE: occasional Fanconi syndrome |
| Epigenetic reader blocker |
| BRD2-4 and BRDT |
Apabetalone |
Phase III |
Atherosclerosis |
Potentially beneficial in post hoc analysis. Secondary endpoint in ongoing phase III RCT |
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.
Our Services
Our company provides a comprehensive one-stop preclinical service dedicated to the development of therapies and drugs for rare kidney diseases. Leveraging our expertise and advanced technologies, we streamline the entire process, ensuring efficient and effective advancement of your therapeutic development projects for these rare conditions.
Featured Therapeutics Development Platforms
Our therapeutic development platforms encompass innovative approaches including small molecules, cell and gene therapies, antibodies, proteins, and peptides. These cutting-edge solutions enable precise and effective treatments to address diverse medical needs.
Types of Rare Kidney Diseases
Drawing on our vast expertise, our esteemed organization offers a broad range of therapy development services from diverse angles, carefully customized to fit your specific requirements. With our extensive experience, we are well-equipped to address various facets of therapy development, providing comprehensive and tailored solutions.
At our company, we are committed to providing comprehensive, one-stop preclinical development services that cover every aspect from disease model creation to innovative therapy research. If you are interested in our services, please don't hesitate to
contact us.
References
- Imig, J. D., D. Merk, and E. Proschak. "Multi-Target Drugs for Kidney Diseases." Kidney360 2.10 (2021): 1645-53.
- Fontecha-Barriuso, M., et al. "Targeting Epigenetic DNA and Histone Modifications to Treat Kidney Disease." Nephrol Dial Transplant 33.11 (2018): 1875-86.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.
