Myositis
Myositis represents a group of inflammatory conditions that affect the muscles, leading to weakness, pain, and disability. At our company, we are committed to advancing the myositis drug and therapeutic areas, providing innovative solutions to meet the specific needs of global pharmaceutical companies.
Overview of Myositis
Myositis, a term derived from "myo" meaning muscle and "-itis" representing inflammation, refers to a group of autoimmune diseases characterized by inflammation in the muscles. This inflammation can lead to muscle weakness, fatigue, pain, and ultimately, loss of function. Myositis encompasses different subtypes, including dermatomyositis (DM), polymyositis (PM), necrotizing myositis (NM), and inclusion body myositis (IBM).
Evidence suggests that in DM and PM, T cells and B cells infiltrate the muscle tissue and release inflammatory cytokines, resulting in muscle damage. In IBM, abnormal protein aggregates accumulate within muscle fibers, contributing to degeneration and impaired muscle function. The exact causes of myositis are still not fully understood, highlighting the need for further research to unravel the intricate pathogenesis of these diseases.
Fig.1 Pathological effects of interferon in dermatomyositis. (Bolko L., et al., 2021)
Therapy Discovery and Development for Myositis
Current myositis drug development focuses on developing novel therapeutics that target the underlying causes of myositis, aiming to reduce inflammation and restore muscle function.
| Drugs | Description |
|---|---|
| Follistatin | Follistatin, a protein that blocks myostatin, has shown promise in promoting muscle growth. Gene therapy utilizing follistatin has demonstrated improvements in cases with Becker muscular dystrophy. |
| Arimoclomol | Arimoclomol induces the production of heat-shock proteins, which counteract cellular stress. Being tested in a phase II trial for IBM, arimoclomol has the potential to mitigate disease progression. |
| Bimagrumab | Bimagrumab, an anti-ActRII antibody targeting the myostatin pathway, has demonstrated an increase in muscle mass and improvements in the 6-minute walking distance in cases with IBM. |
At our company, we are at the forefront of myositis research and drug development, providing a comprehensive range of diagnostics and therapeutic development services. If you would like to learn more about our solutions, please click on the link below.
Our Services
As a research service provider, we offer a range of therapy development services, including preclinical research, animal model development, and in vitro model development. Our team of experienced scientists and researchers collaborates closely with pharmaceutical companies and academic institutions to accelerate the development of safe and effective therapies for myositis.
Trex1-Deficient Mouse Models
Trex1 is an enzyme responsible for degrading and metabolizing endogenous DNA and acts as a negative regulator of STING-dependent signaling. Loss of function mutations in Trex1 have been associated with autoimmune diseases, including dermatomyositis (DM).
TMEM173 Genetic Engineering Models
Our TMEM173 genetic engineering model development services enable us to replicate the skin changes and vasculopathy observed in SAVI cases. These mouse models develop skin ulceration and vasculopathy, providing a valuable platform to evaluate potential therapeutics.
N153S Mutation Models
Our N153S models enable researchers to study the specific pathological features associated with this mutation. The N153S mutation in the DNase II gene has been associated with a rare form of autoimmune myopathy. This mutation leads to increased immune response, resulting in muscle inflammation.
V154M Knock-In Models
Our comprehensive model development services also include the creation and characterization of V154M knock-in mouse models. These models exhibit muscle weakness, fibrosis, and inflammation, providing a valuable tool to study the pathological mechanisms underlying this specific subtype of myositis.
By isolating and culturing muscle cells from individuals with myositis, we can replicate the disease phenotype in vitro. These primary muscle cell models provide a platform to study the immune dysregulation, inflammatory responses, and muscle damage associated with myositis.
Our muscle organoid models are generated from induced pluripotent stem cells (iPSCs) derived from patient samples or healthy individuals. These iPSCs are differentiated into muscle cells, which self-organize into three-dimensional structures resembling muscle tissue.
Our dedicated team of experts utilizes cutting-edge techniques and methodologies to evaluate the efficacy, safety, and pharmacokinetics of potential drug candidates. Beyond the aforementioned repertoire of services and models, our expertise extends to crafting personalized solutions and designing disease models that impeccably align with your unique needs. If our comprehensive range of offerings has piqued your interest, we wholeheartedly encourage you to connect with us without any hesitation.
References
- Bolko L., et al. "The role of interferons type I, II and III in myositis: A review." Brain Pathology 31.3 (2021): e12955.
- Glaubitz Stefanie, Rachel Zeng, and Jens Schmidt. "New insights into the treatment of myositis." Therapeutic Advances in Musculoskeletal Disease 12 (2020): 1759720X19886494.