Cell and gene therapies offer innovative therapies for treating patients, particularly where other traditional therapeutics have been ineffective. For example, gene therapies are well-suited for treating and even curing genetic disorders. In many of these cases, there is simply no other treatment available or they are ineffective. Typically these disorders are relatively rare and thus, cell and gene therapies offer hope to groups of patients that do not have large treatment populations.
Perhaps the most talked about gene therapies have been the cell-based gene therapies for cancer immunotherapy called CAR-T. Novartis’ Kymriah® and Kite/Gilead’s Yescarta® are examples of these types of cell-based gene therapies. These cell-based gene therapies have been able to provide unprecedented remission rates and have demonstrated success where other therapies have failed. Cancer is by far the largest category of indications being investigated with 65% of the gene therapy clinical trials in this area. The second most popular category of indications is inherited monogenetic disease with 11.1%, followed by infectious diseases (7%) and cardiovascular diseases (6.9%) rounding out the top four indications.
Because of the importance of these new therapeutics, we are excited to spend this week focusing on articles related to Cell and Gene Therapies. Each day we will publish a new article related to the topic. We hope you follow along with us this week!
In the meantime, please review our eBook, “Key Considerations for Gene Therapy Manufacturing for Commercialization”. You can download all the articles in the series, by downloading the eBook below.