A significant hurdle for the Adeno-associated Virus Vector-based Gene Therapy Market is the issue of pre-existing immunity. AAV is a common, non-pathogenic virus that many people are exposed to naturally. As a result, a large percentage of the population has pre-existing neutralizing antibodies (NAbs) against specific AAV serotypes. When an AAV-based gene therapy is administered to a patient with NAbs, the immune system can neutralize the vector before it can reach its target cells, rendering the treatment ineffective.

This immunological challenge can prevent patients from receiving AAV gene therapy and makes re-administration difficult. Researchers and companies are actively working on solutions to overcome this. Strategies include developing novel AAV capsids that are less immunogenic, as well as using immunosuppressive drugs to temporarily lower the patient's immune response.

Overcoming pre-existing immunity is a key area of R&D and will be crucial for expanding the patient population eligible for AAV-based therapies. The ability to manage or bypass this immune response will not only improve the efficacy of existing treatments but also unlock the potential for a wider range of therapeutic applications.

FAQs

• What is pre-existing immunity in the context of AAV gene therapy? It refers to the presence of neutralizing antibodies against AAV in a patient's body due to a prior natural infection, which can prevent the gene therapy vector from working.

• How are researchers trying to solve this problem? Scientists are developing new AAV serotypes that are less likely to be recognized by the immune system and are also exploring the use of temporary immunosuppressive treatments.