Interview with CEO and Founder of Navega Therapeutics, Dr. Ana Moreno
Gene Therapy
Tue Oct 19 2021 22:35:29 GMT+0000 (Coordinated Universal Time)
Navega Therapeutics is a San Diego based preclinical-stage biotech company developing novel gene therapies for neurological disorders, ophthalmological diseases, and chronic pain. Retinitis Pigmentosa (RP) is a genetic disease originated by over 200 mutations in more than 50 genes. It is characterized by a loss of rod cells that create a retina degeneration cascade ending up in blindness. The company’s therapeutic candidate is an epigenetic regulator that converts mutation sensitive rod cells to mutation in-sensitive cone cells. The company provided the first demonstration of CRISPR mediated epigenome regulation for engineering in vivo cellular reprogramming and demonstrated direct in vivo cellular reprogramming enabling regenerative medicine.