Treatments for neurodegenerative diseases

Cell and Gene Therapy
Boston, MA

About the company

Developing treatments for neurodegenerative diseases targeting oligodendrocytes to address myelin disorders with few or no treatment options. Lead candidate (rAAV-Olig001-ASPA) is a proprietary gene therapy for Canavan’s disease (CD), a fatal genetic disorder resulting in death typically before age 4. Lead candidate (rAAV-Olig001-ASPA) was awarded Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the FDA.