In 2023, gene therapy will make breakthroughs in many fields, such as the treatment of Duchenne muscular dystrophy and hemophilia.

In 2023, the number of new gene therapy drugs under research in the world exceeded up to 118 for the first time, becoming the highest number in history.

For diseases that cannot be treated by traditional drugs, gene therapy seems to give the majority of patients hope of cure.

According to data, there are currently thousands of gene therapy drugs in different stages of research and development around the world, and the number of new ones added every year shows an accelerating growth trend. Table 1 below shows the number of new gene therapy drugs under development every year in the past ten years compiled by the author through drug intelligence data. 

Since 2014, the number of gene therapy drugs under research has continued to grow, up to 118, a new record in 2023.

As more and more gene therapy products are approved for marketing and the rapid development of related technologies, gene therapy may usher in a wave of development. The FDA has said it expects to approve 10 to 20 gene therapy drugs each year by 2025.

The future is bright, but two major difficulties still need to be solved

1. Price

As we all know, gene therapy drugs are often accompanied by high prices. The high prices are not only difficult for patients to accept, but also cannot be afforded by national medical insurance. However, it is not the original intention of the manufacturer. It is because the research and development costs of gene therapy drugs are too high.

The core of gene therapy drugs is viral vectors. Their high production costs are also one of the main reasons for the high pricing of gene therapy drugs. It is also an important factor restricting the development of the industry in the future.

2. Safety

Although gene therapy has shown great potential in treating genetic diseases, safety issues are also a matter of great concern. Since gene therapy drugs often involve relatively new technologies, compared with traditional drugs, there is no mature non-clinical research evaluation system, so there is great uncertainty. One of the directions for future development is to establish a mature drug research and development system to ensure the safety of drugs to a greater extent.

Compared with traditional drugs, gene therapy drugs have their own unique "charm", but price and safety are "stumbling blocks" that prevent patients from taking drugs. It is believed that after these two major problems are solved, the gene therapy drug market will achieve rapid development.