For maximum benefit in symptom relief and complication mitigation, prompt treatment of congenital thrombotic thrombocytopenic purpura (cTTP) is essential. Protheragen is focused on providing sophisticated diagnostic and therapeutic options to address the challenges posed by managing cTTP. As your secured partner for cTTP research, we provide a complete suite of services tailored to achieve the highest standards in scientific research undertakings.
Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare, genetic disorder that threatens one's life, it is also referred to as Upshaw-Schulman syndrome. People who are affected suffer from severe organ ischemia involving the brain, kidneys, and heart which is further complicated by microangiopathic hemolytic anemia, and acute thrombocytopenia. Untreated, cTTP can have an upwards of 90% mortality rate. There are roughly 1 cTTP case every million people each year, despite being congenital in nature, it can manifest from the neonatal phase all the way to adulthood.
Fig. 1 Organ injury in thrombotic thrombocytopenic purpura (TTP). (Fodil, Sofiane, and Lara Zafrani., 2022)
Congenital thrombotic thrombocytopenic purpura (cTTP) stems from alterations within the ADAMTS13 enzyme gene that constrain its cleavage activity on von Willebrand factor (vWF). This leads to the accumulation of ultra-large vWF multimers which results in increased platelet microthrombus formation and microthrombosis in small blood vessels. Subsequently, this leads to thrombocytopenia, microangiopathic hemolytic anemia, and considerable damage to body tissues.
Fig. 2 Mode of action of ADAMTS13. (Sukumar, Senthil, et al., 2021)
| Therapy | Targets | Mechanism of Action | Development Stage |
|---|---|---|---|
| Adzynma | ADAMTS13 enzyme | Replace deficient ADAMTS13 to cleave ultra-large VWF multimers, preventing platelet clumping | Approved |
| Caplacizumab | Ultra-large VWF multimers | Bind VWF A1 domain, blocking platelet adhesion | Approved |
| AAV Gene Therapy (FLT190) | ADAMTS13 gene in liver | Introduce functional ADAMTS13 gene for sustained enzyme production | Phase I/II |
| mRNA-Based Therapy | ADAMTS13 mRNA in hepatocytes | Transient expression of functional ADAMTS13 protein | Preclinical |
| Sutimlimab | Complement C1s | Suppress complement activation in microthrombi | Research Phase |
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.
Protheragen is committed to delivering unparalleled diagnostic and therapeutic development services tailored specifically for congenital thrombotic thrombocytopenic purpura (cTTP). Our expertise extends to the development of cutting-edge disease models that drive preclinical studies into potential therapies. With a focus on innovation and precision, we empower our clients with advanced solutions that pave the way for effective intervention strategies.
At Protheragen, we are committed to supporting the development of innovative therapeutics through comprehensive preclinical research services, including pharmacodynamics (PD), pharmacokinetic (PK) and toxicology studies. Our customized approach addresses the unique challenges of your studies and helps you optimize your drug candidates for commercial success. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
References
Myeloproliferative Disorders (MPDs)
Bone Marrow Failure Syndromes (BMFS)