Thrombotic Thrombocytopenic Purpura (TTP)
Thrombotic thrombocytopenic purpura (TTP) is a rare and serious blood disorder. Given our company's extensive knowledge and expertise in TTP research, we are fully prepared to provide personalized solutions and comprehensive support to streamline your research journey, spanning from developing TTP therapies to their successful commercialization.
Overview of TTP
TTP is a rare and life-threatening blood disorder characterized by the formation of small blood clots in the body's smallest blood vessels. These blood clots can block the normal flow of blood and cause a range of symptoms. TTP occurs at an estimated rate of 3.7 cases per million individuals annually, with a higher prevalence among adults and a greater propensity for women to be affected by the condition.
Fig. 1 Organ injury in thrombotic thrombocytopenic purpura (TTP). (Fodil, et al., 2022)Pathogenesis of TTP
TTP is characterized by a deficiency in the enzyme ADAMTS13, responsible for cleaving von Willebrand factor. Reduced or absent ADAMTS13 activity results in the accumulation of von Willebrand factor multimers, leading to the formation of platelet-rich clots in the microvasculature. These clots can cause hemolytic anemia by damaging red blood cells and obstructing blood flow, resulting in organ damage. The deficiency of ADAMTS13 can be acquired or inherited.
| Disease Name | Description |
|---|---|
| Immune-mediated TTP (iTTP) | It is the acquired form, typically caused by autoantibodies that inhibit ADAMTS13 activity. |
| Congenital TTP (cTTP) | It is a rare hereditary condition caused by mutations in the ADAMTS13 gene. |
Targets of TTP Therapy
Autoantibodies
In iTTP, the presence of autoantibodies inhibits ADAMTS13 activity. Targeting the reduction of autoantibody levels or inhibiting their effects is crucial for drug development. One effective therapeutic for iTTP is rituximab, which depletes B cells and reduces the production of autoantibodies against ADAMTS13.
Von Willebrand Factor
Accumulation of von Willebrand factor multimers triggers thrombus formation. Targeting the function of von Willebrand factor is a primary objective in TTP drug development. Caplacizumab, recently approved for TTP, directly targets von Willebrand factor, blocking its interaction with platelets and effectively preventing microvascular thrombosis.
Our Services
Our company has established comprehensive platforms for developing rare disease diagnostics and therapies, encompassing small molecule drug, cell therapy, gene therapy, therapeutic antibody, therapeutic peptide, and therapeutic protein. Through our dedicated platforms, we are fully devoted to advancing the development of innovative diagnostic tools and therapies for TTP.
Animal models are essential for enhancing our understanding of diseases and evaluating the safety and efficacy of potential therapeutics. We offer our expertise in establishing animal models specifically tailored for TTP. These models serve as invaluable tools to facilitate the safety evaluation and pharmacokinetics study of your drug candidates.
| Genetically Engineered Models | ||
| Our company excels in developing genetic engineering models for TTP. Using embryonic stem (ES) cells and CRISPR/Cas9 gene editing technology, our scientists have successfully manipulated the Adamts13 gene, resulting in the creation of cTTP animal models. | ||
| Optional Models | Adamts13-/- Model | |
| Immune Induction Model | ||
| Our scientists induce autoimmune animal responses by administering ADAMTS13 or VWF antibodies or utilizing ADAMTS13 or VWF peptides. These models faithfully replicate the essential features of human iTTP. | ||
| Optional Models |
|
|
| Optional Species | Mice, Zebrafish, Non-human Primates (Baboons or Macaques), Rats, Pigs, Rabbits, Others | |
If you are interested in our services, please don't hesitate to contact us for more information and a detailed quotation regarding the specific services you require.
References
- Fodil, Sofiane, and Lara Zafrani. "Severe thrombotic thrombocytopenic Purpura (TTP) with organ failure in critically ill patients." Journal of Clinical Medicine 11.4 (2022): 1103.
- Zheng, Liang, and X. Long Zheng. "Animal models for thrombotic thrombocytopenic purpura: a narrative review." Annals of Blood 8 (2023).
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.
