3rd Gene Therapy for Muscular Disorders
Towards safer AAV gene therapy
Speaker: Aleš Štrancar, Managing Director, Sartorius BIA Separations
- Recent clinical trials revealed the immune response to AAV vectors is likely to be a bottleneck in AAV gene therapy.
- One of the key reasons for the immune response is AAV purity. Improved manufacturing, especially purification process, is therefore mandatory for safer gene therapy.
- In addition to hc proteins and hc DNA removal of empty and partial AAV capsids, infectious viruses and endotoxin will be presented in this paper.