Neuromyelitis Optica Spectrum Disorder
Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune disease characterized by severe inflammation of the optic nerves and spinal cord. Our company, a leading provider of therapy development services, is at the forefront of NMOSD drug development.
Overview of Neuromyelitis Optica Spectrum Disorder
Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune disease that primarily affects the optic nerves and spinal cord. It is characterized by recurring episodes of optic neuritis, which can result in vision loss, and transverse myelitis, leading to paralysis and sensory disturbances. AQP4-ab, targeting the aquaporin-4 water channel protein expressed in astrocytes, plays a crucial role in the development of NMOSD. The binding of AQP4-ab to astrocytes triggers inflammation and subsequent damage to the optic nerves and spinal cord.
Fig.1 Pathophysiologic mechanisms and therapeutic targets for NMOSD. (Carnero Contentti E., et al., 2021)
Therapy Discovery and Development for NMOSD
Several drugs and therapies have been developed or are currently under investigation for the therapeutic of NMOSD. These include:
| Therapeutic Approaches | Description |
|---|---|
| BAT4406F | BAT4406F is a fully humanized anti-CD20 monoclonal antibody that targets B cells involved in the pathogenesis of NMOSD. |
| SHR1459 | SHR1459 is Bruton's tyrosine kinase inhibitor that shows promise in inhibiting B-cell activation and reducing inflammation. |
| Ravulizumab | Ravulizumab, derived from eculizumab, is a second-generation anti-C5 complement protein. |
| Bortezomib | Bortezomib, a proteasome inhibitor used in multiple myeloma therapeutic, has shown potential in reducing relapse rates in a cohort of NMOSD cases. |
| Cetirizine | Cetirizine, a second-generation antihistamine, has demonstrated a reduction in relapse rates in NMOSD cases when used as an add-on therapy. |
| Telitacicept | Telitacicept is an inhibitor of B lymphocyte stimulator (BlyS) and APRIL. |
| Autologous HSCT | Autologous hematopoietic stem cell transplantation (HSCT) has shown promise in achieving prolonged drug-free remission in NMOSD cases. |
| Aquaporumab | Aquaporumab, a targeted non-immunosuppressive therapy, has demonstrated effects in NMOSD cell cultures, indicating its potential as a future therapeutic option. |
At our esteemed company, we are deeply engaged in the advancement and rigorous testing of groundbreaking drugs that specifically target the underlying mechanisms of NMOSD. To gain a deeper understanding of our extensive therapy development services, we cordially invite you to click on the link below.
Our Services
With a world-class technology platform, we offer comprehensive therapy development services for NMOSD. Our expertise and capabilities include biomarker discovery and validation, drug development and optimization, animal model development, in vitro model development, and preclinical research services.
NMOSD-ON Animal Models
Optic neuritis (ON) is a common inflammatory optic neuropathy often associated with NMOSD. Purified IgG from AQP4-IgG-positive NMOSD-ON patients, along with human complement, is intrathecally injected into wild-type (WT) and type I interferon receptor-deficient mice (IFNAR1-KO).
NMOSD Transfer Models
Our animal models involve the transfer of AQP4-IgG or AQP4-sensitized T cells to rodents, along with pro-inflammatory maneuvers when necessary. This approach allows us to replicate the humoral and cellular immune mechanisms observed in human neuromyelitis optica spectrum disorder.
Our primary objective in cell-based model development service is to replicate the cellular events associated with NMOSD, such as the interaction between astrocytes and immune cells, the activation of complement cascades, and the generation of autoantibodies against aquaporin-4 (AQP4).
We differentiate patient-derived induced pluripotent stem cells (iPSCs) into organoids that mimic the spinal cord and optic nerve microenvironments. We incorporate various cell types, including astrocytes, oligodendrocytes, microglia, and neurons, to create a comprehensive model of NMOSD pathogenesis.
Our unwavering commitment to therapeutic development drives us to offer comprehensive services that encompass the entire spectrum of therapy development. 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.
Reference
- Carnero Contentti Edgar, and Jorge Correale. "Neuromyelitis optica spectrum disorders: from pathophysiology to therapeutic strategies." Journal of Neuroinflammation 18.1 (2021): 208.