Disease Focus: Canavan Disease

Process development for establishing an iPSC-based therapeutic candidate for Canavan disease

Translational Candidate Genetically-corrected patient iPSC-derived neural progenitor cells, which have demonstrated efficacy to correct disease phenotype in a CD mouse model. Area of Impact This candidate has the potential to develop into a therapy for Canavan disease, a fatal neurological disease that has no cure or standard treatment. Mechanism of Action The proposed candidate is […]