2. Cellular and genetic approaches
In addition to biosimilars, companies are also starting to develop more advanced therapies, including gene-, tissue-, and cell-based products. Patients are already experiencing the benefits of CAR-T cell therapies.
To date, the US Food and Drug Administration (FDA) has approved 10 cell and gene therapies, many of which are showing commercial success. That includes two gene therapies: Luxturna from Spark Therapeutics, indicated for retinal dystrophy, and Zolgensma from Novartis/Avexis, indicated for the treatment of spinal muscular atrophy. Both of these therapies use an AAV viral vector to deliver a healthy copy of the problem gene.
Meanwhile, more than 1,000 therapies are currently in the pipeline and the FDA anticipates that 10 to 20 of these will be approved by 2025. As a result, the cell and gene therapy market is expected to grow at a 30% compound annual growth rate.
Gupta and Boulais said they expect the gene therapy field to expand beyond the initial rare diseases to oncology and other chronic conditions. The cancer gene therapy market, is expected to reach US$2bn in sales by 2023, according to a report by Allied Market Research. Gene therapy is also being investigated as a novel approach to treating heart failure and diabetes. As these novel modalities become the norm, manufacturers will need to broaden their skillset and facilities to cater to the new breadth of products, said the Sartorius team.