Therapeutic Development Services
Mastocytosis is a myeloproliferative disorder that poses a significant threat to human health. Because of degree of experience in mastocytosis therapy development, Protheragen has the capacity to offer customized and holistic assistance that would help ease the process of mastocytosis therapy research to its commercialization.
Mastocytosis is defined as a rare yet complex disorder grouped together due to the unusual build-up and activation of mast cells in one or several organ systems. Mast cells are immune system cells that are central to the processes of inflammation and allergic reactions. In the case of mastocytosis, these immune cells undergo excessive proliferation and discharge tremendous quantities of inflammatory substances like histamine, tryptase, and cytokines, which creates a variety of health issues and complications.
Fig. 1 Current therapeutic options for advanced systemic mastocytosis. (Veitch, Scott, and Deepti H. Radia., 2023)
Mast cells mainly drive the development of mastocytosis through uncontrolled expansion and activation, mostly caused by gain-of-function mutations of the KIT gene, particularly the KIT D816V mutation. This mutation results in the indefinite activation of the KIT receptor, a tyrosine kinase that ensures mast cell survival, proliferation, and apoptosis resistance. In addition, the mast cells produce large quantities of histamine, tryptase, and cytokines, which cause inflammation and tissue damage and produce systemic symptoms.
Fig. 2 Mechanisms of KIT activation in normal mast cells. (Piris-Villaespesa, Miguel, and Ivan Alvarez-Twose., 2020)
The global market for mastocytosis therapeutics is experiencing rapid growth. From 2024 to 2028, the global systemic mastocytosis (SM) therapeutics market size is expected to grow by $157.1 million at a CAGR of 6%. The development of new therapies targeting the KIT D816V mutation and other molecular pathways has great market promise and is essential for the effective management of mastocytosis.
Table. 1 Drug development pipeline for mastocytosis.
| Drugs | Types | Targeted | Developmental Stage |
|---|---|---|---|
| Midostaurin | Tyrosine kinase inhibitor (TKI) | KIT, FLT3, PDGFR | Approved |
| Avapritinib | Tyrosine kinase inhibitor (TKI) | KIT D816V, PDGFRα | Approved |
| BLU-263 | Tyrosine kinase inhibitor (TKI) | KIT D816V | Phase II |
| Masitinib | Tyrosine kinase inhibitor (TKI) | KIT, PDGFR, LYN | Phase III |
| Brentuximab vedotin | Antibody-drug conjugate | CD30 | Approved |
| Lirentelimab | Monoclonal antibody | Siglec-8 | Phase II |
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.
At Protheragen, we remain steadfast in our focus of providing advanced diagnostic and therapeutic development services aimed at mastocytosis, a complex myeloproliferative disorder. Our focus is in the design of novel therapies from a wide range of molecular classes, which were painstakingly evaluated in elegantly designed disease models during the preclinical stage.
Therapeutic Development Services
As a leading provider of therapeutic development services, Protheragen is focused on developing a variety of innovative therapies for mastocytosis to address the complex challenges in this field.
Animal Model Development Services
Animal models play a pivotal role in unraveling the intricate biology of mastocytosis and assessing the effectiveness and safety of prospective therapies.
Specializing in preclinical research for drug development, Protheragen offers a comprehensive solution that includes pharmacodynamics (PD), pharmacokinetic (PK) and toxicology studies to thoroughly validate and optimize therapies for mastocytosis. 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)