Ace Therapeutics provides comprehensive solutions for global customers conducting drug development for Wilson's disease. Our scientists have over a decade of experience in the field of liver disease research. Based on our advanced technology platform and comprehensive service system, we can help you with your drug development for Wilson's disease.
Wilson's disease is caused by mutations in the ATP7B gene, which encodes Cu-translocated ATPase expressed primarily in the liver. The main clinical manifestations of the disease are liver and neuropsychiatric disorders. Current treatments for the disease are mainly with zinc salts or Cu chelators, but they can only control the symptoms and cannot achieve a cure. Therefore, there is an urgent need to develop new therapies. We offer comprehensive services to facilitate the development of drugs for Wilson's disease.
What Can We Do?
Based on our extensive experience and advanced technology, we can provide comprehensive research services for drug development against Wilson's disease. Our services offer a comprehensive range of services in three areas: cellular therapy, gene therapy, and targeted drug development.
- Cell Therapy Development for Wilson's Disease
Cell therapy for Wilson's disease focuses on restoring hepatobiliary copper excretion by transplanting hepatocytes. Based on our extensive experience in cell therapy development and advanced technology, we can provide you with the most professional and appropriate technical support for your cell therapy development project for Wilson's disease.
There are 3 main potential cell therapies for Wilson's disease.
- By transplanting sufficient numbers of healthy stem cells
- Cell therapy based on biliary Cu transport
- Hepatocyte-like cells derived from induced pluripotent stem cells
- Gene Therapy Development for Wilson's Disease
Gene therapy for Wilson's disease aims to provide healthy copies of ATP7B by introducing a transgene through a vector capable of indefinite integration and/or persistence in the cell, thereby correcting the defect in native hepatocytes. Gene therapy may allow ATP7B to be permanently expressed in the livers of Wilson's disease patients, avoiding lifelong intake of copper-reducing drugs.
We offer quality and comprehensive research services for gene therapy in Wilson's disease to facilitate the smooth conduct of your research project. In particular, we have extensive hands-on experience with adeno-associated viral and lentiviral delivery vectors for gene therapy delivery systems to assist you in the development of your project.
- Targeted Drug Development for Wilson's Disease
Fecal Microbiota Transfer (FMT) has been used in clinical trials including nonalcoholic steatohepatitis, cirrhosis, Clostridium difficile infection, and other diseases. FMT is often combined with antibiotic therapy, in addition to the potential risk of FMT carrying other pathogens, which limits the widespread use of FMT. To address these issues while promoting the use of FMT for liver disease treatment, we offer comprehensive services to facilitate your research.
- Cu Chelator
Reverses Cu accumulation by binding to Cu and even alleviates liver death and liver failure.
- Liver X Receptor (LXR)/Vitamin A-Like Receptor Agonist
Alleviate damage caused by Cu accumulation
Ace Therapeutics has a dedicated team and hands-on experience in drug development for Wilson's disease. We can provide high-quality data and a fast turnaround time to support your research project. If you would like to learn more about our services, please feel free to contact us.
Reference
- Ranucci, G., et al., Wilson's disease: Prospective developments towards new therapies. World J Gastroenterol, 2017. 23(30): p. 5451-5456.
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