3.3  Viral Vector Sales

3.3.1  There are now 38 cell and gene therapies approved by the FDA.^[3] Since 2021, the FDA has approved an average of five therapies per year, and appear well on their way to the goal of approving an average of 10–20 therapies per year, beginning in 2025.^[2]

3.3.2  Of the estimated 7,000 rare diseases (defined by the FDA as diseases affecting fewer than 200,000 people) that are caused by genetic mutations, more than 95% have no approved therapeutic option.^[62] The sheer number of diseases for which a genetic therapy may provide a cure whereas there isn’t one currently available demonstrates that there are plenty more targets for researchers to investigate, as long as funding and the market provide the opportunity. To date, gene therapies have not proven to be the blockbuster drugs that are common with antibodies, but the forecasted sales of approved therapies demonstrate this is a fruitful class of therapies for which to develop therapies.^[63] Sales forecasts for all gene therapies in 2023 (^{Table 6}) and projected sales for 2029 (^{Table 7}) are given.

3.4  Orphan Drugs

3.4.1  In the United States, a “rare disease” is defined as a disease that affects fewer than 200,000 people.^[62] According to the National Institutes of Health (NIH), over 30 million Americans have one of the nearly 7,000 rare diseases that have been identified to date.^[62] Of the rare diseases that are caused by genetic mutations, more than 95% have no approved therapeutic option.^[64] Start-ups and established biopharmaceutical companies alike are developing potential gene therapies to address this key unmet medical need.

3.5  The Patent Landscape and its Implications

3.5.1  As one would expect from a growing and innovating industry, the patent landscape for gene therapies is, of course, very active. The number of patents filed over time has fluctuated in accordance with the cycles of gene therapy successes and failures described in our history section above, and mimics the patterns observed in the number of clinical trial starts (^{Figure 16}). A stagnate period ensued following the tragic death of clinical trial participant Jesse Gelsinger in 1999. However, the discovery of CRISPR and later EMA approval of alipogene tiparvovec (Glybera) in 2012 spurred a renewed trend of increasing numbers of patents filed annually, and as of 2018, over 2,000 patent families were being filed consistently each year.^[71]

3.5.2  As of 2021, the U.S., China, Japan, South Korea, and the UK account for 87% of the world’s patents in gene therapies (^{Figure 5}).