For patients and families suffering from monogenic diseases such as myotonic dystrophy, repairing the genetic defect is the hope for treatment. Gene therapy has also become a popular choice. In October 2012, the European Union approved the first gene therapy drug in a western developed country, uniQure’s Glybera, which uses a modified AAV1 viral vector to introduce the LPL ( Lipoprotein Lipase) gene into the patient’s muscle cells using a modified AAV1 viral vector to produce lipoprotein lipase, correcting the enzyme deficiency caused by the patient’s genetic defect and thereby eliminating clinical symptoms such as recurrent pancreatitis. After two years of operation and preparation, the drug is expected to be available in the EU (first in Germany) within a few months, with an initial price of 1.11 million euros (about 8 million RMB), although there will be bargaining with the German government health insurance department before the launch. This is a landmark event for the entire gene therapy field. It is important to mention here that in all official reports it is stated that this is the first gene therapy agent to be marketed in the Western world. The reason why it is not the first in the world is that the world’s first gene therapy agent, Imazan (recombinant human P53 adenovirus injection), was approved for marketing in China back in 2004 for the combination treatment of advanced nasopharyngeal cancer patients with radiotherapy. However, the clinical trial data supporting its launch at that time were very limited and have been widely questioned. The clinical application of the drug was also limited after its launch, and the SFDA revoked the company’s GMP manufacturing license in 2008 due to manufacturing process problems. In addition, a phase I clinical trial of rAAV2-hFIX for hemophilia B was approved by SFDA in China as early as 2003, but unfortunately no follow-up results have been available since then. Worldwide, gene therapy (especially AAV-mediated gene therapy) has undoubtedly entered the fast track of rapid development. With more than 20 years of advance accumulation, its safety and efficacy have been widely recognized by pharmaceutical regulators and pharmaceutical giants (clinical trials of AAV for the most vulnerable SMA neonates have been conducted in the US, see previous article for details). The world’s pharmaceutical giants have invested in the final phase of the pre-marketing push for gene therapy drugs, with Novartis, Pfizer, Sanofi, Bayer, and Baxter all entering the field by acquiring or partnering with gene therapy companies, giving a significant boost to Phase III clinical trials. Gene therapy companies have become the brightest stars of Nasdaq. In addition to rare diseases, pharmaceutical giants are more interested in the fact that in the era of precision medicine, treatment for common diseases must also be refined for patients, and more precise targeted treatments, such as gene therapy, will be used for future treatment according to the pattern of rare diseases. In the midst of a surge abroad, gene therapy in China appears to be very quiet. In the past 10 years, no new gene therapy clinical trials have been approved in China, and there are no international multi-center clinical trials in the field of gene therapy. The reason for this is that domestic scientific research is still lagging behind, and the emerging field of gene therapy is a big money-spinner with huge initial investment, which can be wiped out if you are not careful, even the leading company in this field, Introgen, had to file for bankruptcy protection because the FDA did not approve the results of its phase III clinical trial of P53 gene therapy. Therefore, the domestic gene therapy industry has a very difficult start due to the lack of capital injection. On the other hand, there are difficulties for domestic patients to participate in foreign gene therapy clinical trials, mainly for the following reasons: 1. China’s CFDA clearly stipulates that new foreign drugs cannot do Phase I and Phase II clinical trials in China. Only after completing Phase I and Phase II clinical trials abroad can they apply to conduct Phase III clinical trials or multi-center Phase III clinical trials in China. This is to avoid the country becoming a “mouse” for foreign pharmaceutical companies (India is less regulated and the cost is much lower than developed countries. A large number of new drugs from Western countries have been tested in Phase I and Phase II clinical trials in India, and even cases of ethical violations are often reported in the press). However, accordingly, it also raises the threshold for new drugs to enter China. 2, China is a large developing country, health insurance and income level of residents still have a large gap compared to developed countries, the only advantage is the large population base and the number of patients. However, for a disease like DMD, which is prevalent worldwide and has a large number of patients, there are a large number of patients waiting for clinical trials in the western world, and there is no worry about the lack of clinical trial participants. And after the drug is marketed, pharmaceutical companies will get more and faster returns in developed countries, so there is not enough incentive to conduct clinical trials in China. 3, China’s new drug clinical trial approval process is more complicated and lengthy, significantly slower than other countries (the United States, Europe and Japan have a tripartite agreement on the approval of new drugs, mutual recognition. While countries and regions such as South Korea and Taiwan follow this agreement for specific implementation and actually participate in this system, China is not in this system). The international multicenter clinical trials require synchronized global implementation, so if they cannot keep up with others, pharmaceutical companies can only choose to abandon China. Previous clinical trials of PDE5A inhibitors could not participate in them due to this reason. 4. The core competence of small and medium-sized gene therapy companies is gene therapy agents, and they are very worried about being copied. There is still a gap between patent protection in China and the West, so gene therapy companies have a lot of concerns. Previously, a small and medium-sized gene therapy company had proposed a program for Chinese patients to go to neighboring countries to participate in clinical trials, responsible for all travel expenses and given a certain subsidy. Even at this cost, they were reluctant to include China in the clinical trials. For these reasons, there are many difficulties in conducting gene therapy clinical trials in China, but we have been working on it. In the past two years, TREAT-NMD has received gradually more applications from pharmaceutical companies for clinical trial feasibility studies and clinical trial assistance. Recently, we have received another invitation from pharmaceutical companies for preliminary feasibility studies for DMD clinical trials and FSHD clinical trials, and as always, we have declared the patients and hospitals as required. However, according to the past experience, it is really difficult to really implement international multicenter clinical trials in China. On the other hand, we are also participating and coordinating various resources, Chinese scientists at home and abroad, entrepreneurs, investors, patient organizations, etc., hoping to promote our own domestic gene therapy clinical trials as soon as possible. The era of precision medicine and gene therapy has arrived, let’s get ready for it.