Gene Therapy

Gene therapy entails the use of gene as medicine to prevent or treat diseases. The US FDA recently approved a second gene therapy to treat a certain type of lymphoma. So far both gene therapies on the US market are CAR-T cell therapies. CAR-T cell therapy is ex vivo gene therapy, meaning the gene transduction process takes place outside of patient’s body. It is a controlled and safer method to carry out the gene editing or replacing. In the meantime, hundreds of clinical studies are testing in vivo gene therapies to treat cancers, genetic disorders, and CNS diseases.

MIT Technology Review has a nice article on the astronomical price tags of the gene therapies.