Cellular and Gene Therapy (CGT), which involves transplanting normal or bioengineered human cells into patients To replace cells that have lost their normal function, thereby curing diseases and benefiting patients.

1

Global giant pharmaceutical companies are competing for layout,Great potential for future development

Cell and gene therapy has great application potential for many diseases for which no drug target can be found, not only changing human genetics for treatment The way of diseases and incurable diseases has also opened up the imagination of diseases in the pharmaceutical industry, and has become one of the most promising global frontier pharmaceutical fields. Domestic and foreign pharmaceutical companies are also scrambling to enter the cell and gene therapy track.

The investment and financing of the cell gene therapy industry is heating up. In January 2022 alone, 32 cell genetics companies around the world received approximately US$1,643.17 million in financing. TOP3 countries in terms of financing volume: the United States (14), China (13), and the United Kingdom (3).

At present, the FDA has approved 8 gene therapy drugs, of which 5 are CAR-T, 2 are viral vector therapy, 1 was oncolytic virus therapy. In June 2021, China was approved for Fosun Kite's CAR-T product Akilence Injection, and in September 2021, WuXi Junuo's CAR-T product Rigilonza Injection was approved. There are not many that can really achieve sales, and the future market space is huge.

2

The bottleneck facing the industry - slow progress in clinical trials

CGT is another blockbuster drug type after small molecules and biological drugs, but due to the complex technical mechanism of CGT therapy, strict Compared with traditional pharmaceutical companies, CGT products are progressing more slowly in clinical trials than traditional pharmaceutical companies. According to ASGCT data, by the end of 2021, there will be a total of 1,890 CGT products worldwide. There are 100 cell and gene therapy research projects (from preclinical to pre-registration, less than 400 in 2015), of which nearly 30% are in the clinical stage, while most CGT projects are still in the early clinical stage.

Number of CGT research projects (as of mid-2021)

Global CGT pipeline clinical staging

China CGT pipeline clinical stage

Future clinical trials of CGT therapy still need to overcome many challenges and achieve breakthroughs in the effectiveness of cell and gene drugs.

1. The uniqueness of each cell gene therapy product and clinical trial

2. Complex logistics tracking management of cell gene products and patient cell tissue samples in clinical trials

3. Differences in patient response to cell gene therapy

4. Countries have some special regulations and approval procedures for cell gene therapy

5. Qualification requirements for hospitals implementing cell gene therapy