Preclinical gene therapy company developing EVADER, an immune-evasive AAV vector platform designed to reduce immune responses, improve safety and potency, and enable repeat dosing of systemic gene therapies
Preclinical gene therapy company developing EVADER, an immune-evasive AAV vector platform designed to reduce immune responses, improve safety and potency, and enable repeat dosing of systemic gene therapies. Its pipeline targets genetic diseases including Niemann-Pick type C and severe hemophilia A and B.