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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.

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