We are committed to introducing new drugs for rare disease populations.
Our scientific strategy and screening approach has yielded a robust drug discovery engine with multiple development opportunities.
We generate data portfolios from drug screens across our platform of genetic models.
We are initially focused on identifying drugs for seizure, pain, and certain neuromuscular disorders characterized by ion channel or receptor dysfunction. Because the genes involved in these initial indications are also implicated in a variety of other disorders, our lead candidates have broad therapeutic applicability, both within the CNS space and beyond.
We screen marketed and proprietary drugs across our platform of genetic models to generate a breadth of target-specific data. Individual data dossiers include specific learnings on target-pharmacophore relationships and prospective applications toward a variety of indications.
- Repurposing: our scientific insights offer new uses for currently marketed or shelved drugs.
- Reformulation: our pharmaceutics expertise powers new drug delivery strategies.
- NCE development: our medical chemistry capabilities generate new ideas for novel drug design.
We actively seek partnerships with other organizations that share our goal of advancing knowledge of rare diseases and new treatment options. With our platform of rare genetic disease models, our network of physicians and patient communities, and our extensive experience in genetic research and drug screening, we bring considerable expertise, data, and resources to potential partners.