Ace Therapeutics has extensive research experience in the field of liver disease. Our scientists are committed to providing the most professional and scientific research services to our clients in the field of liver disease research worldwide. By providing a one-stop platform for liver disease research, we aim to facilitate the advancement of research in the field of liver disease worldwide.
Alpha 1-antitrypsin deficiency (AATD) is a rare genetic disorder. It is caused by mutations in the SERPINA1 gene resulting in defective secretion of mutated alpha 1-antitrypsin (AAT) proteins that are retained in the endoplasmic reticulum. There is no effective diagnostic method for AATD and the associated therapeutic methods are yet to be improved.
What Can We Do?
We provide comprehensive services for AATD research, including therapy development and diagnostic technology development for AATD. We are constantly striving to advance AATD research based on a solid body of knowledge and an advanced technology platform.
- Therapy Development for AATD
AATD has many features that make it an ideal candidate for gene therapy strategies, such as the fact that AATD is a monogenic disease, that enhanced serum AAT to protective levels protects the lung from proteolytic damage, and, in addition, that supraphysiologic levels of AAT have no deleterious effects on the organism. Therefore, gene therapy is the main method of treatment for AATD. However, other therapeutic approaches are still available, such as the administration of AAT. We offer adequate services for the development of therapies for AATD to facilitate the smooth conduct of your research.
- Gene Therapy Development for AATD
Gene therapies for AATD have been studied since about 30 years ago, but there are no approved gene therapies for AATD patients to date. Based on our extensive research experience in gene therapy, we offer targeted services for your research project. Our scientists can solve the most difficult delivery problems in the gene therapy process for you to achieve effective therapeutic concentrations.
- Targeted Drug Development for Wilson's Disease
We also offer comprehensive and high-quality research services for other exogenous AAT-enhancing therapies such as intravenous AAT replacement therapy, neutrophil elastase inhibitors, non-specific drugs for lung disease, and other pathways to ensure that the needs of your research program are met.
- Diagnostic Technology Development for AATD
Since the symptoms of AATD are consistent with those of chronic obstructive pulmonary disease and cryptogenic liver disease, it is difficult to be taken seriously or even diagnosed effectively in the early stages of the disease, while the best time for treatment is missed, which explains the importance of diagnostic technology development for AATD. We offer comprehensive services in diagnostic development for AATD, focusing on genetic testing, but also testing for alpha-1 antitrypsin.
Ace Therapeutics is committed to promoting AATD research. We protect AATD researchers by providing professional, quality, and efficient services. If you would like to learn more about our services, please feel free to contact us.
Reference
- Brantly, M., et al., Detection of alpha-1 antitrypsin deficiency: The past, present and future. Orphanet J Rare Dis, 2020. 15(1): p. 96.
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