About the Program
A therapeutic approach using a virus (non-infectious adeno-associated virus) to deliver a functional UBE3A gene to cells inside the body (in-vivo).
- FAST Pillar
- Pillar 1: Replace Mom's UBE3A
FundedLatus Bio is exploring an investigational Adeno-Associated Virus gene therapy (AAV-GT) approach to treating Angelman syndrome using a specially engineered viral vector called AAV-EP+
A therapeutic approach using a virus (non-infectious adeno-associated virus) to deliver a functional UBE3A gene to cells inside the body (in-vivo).
Find information on Angelman syndrome, research, clinical trials, resources, and ways to get involved.