An Orphan Drug company with a portfolio of products focusing on Rare Genetic Diseases including Cystic Fibrosis, Lysosomal Storage Diseases, and Neuromuscular Diseases.
Novel, cost-effective gene therapy technology enabling the Company to:
- Address traditional targeted gene therapy approaches with initial focus on neuromuscular diseases
- Address a broad range of very rare genetic diseases with a new “gene-agnostic” approach with initial focus on lysosomal storage diseases
Tremendous unmet clinical need
- Few or no therapeutic options for hundreds of Rare Genetic Diseases.
- Over 8,000 rare genetic diseases affecting 320 million people and families globally
- 70% start in childhood
- Fewer than 5% have approved treatments
- Neuromuscular diseases have many unmet needs
- No therapeutic options for the neurological aspects of most rare genetic diseases
- Capable of crossing the blood brain barrier
Addressing large Orphan Disease market opportunities
Experienced management team and Board of Directors.