Cure Sanfilippo Foundation has awarded a grant to Haiyan Fu, PhD, at Nationwide Children’s Hospital in Columbus, OH, to research strategies for overcoming a patient’s pre-existing AAV (Adeno-Associated Virus) antibodies so gene therapy using AAV is accessible to them.
Project Summary: As the field moves forward with AAV9-gene therapy approaches for clinical applications, a critical challenge is the presence of pre-existing antibodies against AAV9. Many AAV serotypes infect humans, including AAV9. As a result of infection, pre-existing antibodies to AAV are common in humans.
In the setting of gene therapy, individuals with pre-existing AAV9 antibodies would not be eligible for treatment using AAV9 vectors due to diminished efficacy.
There are currently no effective approaches available which deplete pre-existing antibodies enough to allow for effective gene transfer in antibody positive patients.
Small-scale preliminary experiments in Dr Fu’s lab have shown the potential of appropriate immuno-supporessive regimens in overcoming the pre-existing Abs.
The goal of this study is to identify immuno-suppressive regimens that can effectively overcome pre-existing AAV Abs, in order to be able to treat all individuals who have the target diseases, as well as those who have received AAV9 gene therapy treatment and may need vector re-administration later.
Start Date: March 2017