Snejana Krassova, MD of Bayer Pharmaceuticals talks about the reality that some people have, or can develop, antibodies to the vectors that are commonly used for gene therapy.
Bayer is currently developing a gene therapy for hemophilia A, and as Dr. Krassova explains in this video, the fact that people can have antibodies to the vectors used in gene therapy reinforces the idea that we need different types of gene therapies for various patient populations.
Hemophilia A is a genetic disorder caused by a missing or defective factor VIII. Disease severity is largely dependent on the level factor VIII present but in the more severe cases, excessive bleeding and severe pain are common symptoms. Currently, most hemophilia A patients take a recombinant factor VIII product to control bleeding and numerous products are available, each with its own set of benefits and challenges. Gene therapy would eliminate the need for regular factor VIII infusions.