Dawn Therapeutics

Dawn Therapeutics is a pre-clinical biotechnology company developing precision delivery platforms for genetic medicines targeting the central nervous system, cartilage, and bone.

Its transferrin receptor-guided system is designed to cross the blood-brain barrier and deliver RNA or DNA payloads to neuronal tissues, while a complementary system targets cartilage and joint structures.

The lead programme is an investigational lentiviral vector delivering a functional gene for Hurler syndrome, a lysosomal storage disorder.