Founded by RAO Yi and invested by Sequoia Capital and Tencent, "Huayi Lejian" accelerates the overseas expansion of its hemophilia gene therapy drugs | Project Report

Text | Hu Xiangyun

Editor | Hai Ruojing

In 2025, Chinese innovative drugs are making strong inroads into overseas markets. The total value of product licensing in the first half of the year alone has approached $66 billion. Notably, drugs for rare diseases, once considered a niche area due to the "small patient base and high R & D costs," are gradually becoming important players in the wave of domestic new drugs going global. The industry hopes that by entering markets with more mature payment systems such as those in Europe and the United States, R & D costs can be effectively shared, achieving a balance between the commercial and social values of products.

Recently, at the 14th China Rare Disease Summit Forum hosted by the Courage & Love Rare Disease Center, Gong Yuntao, CMO of Hua Yi Le Jian, shared with 36Kr the company's experience in the clinical R & D and overseas expansion of new drugs for hemophilia.

Hua Yi Le Jian was founded in 2019 by biologist Rao Yi. It is mainly engaged in the development of gene therapy drugs. The most advanced gene therapy drug in its pipeline is for the treatment of hemophilia A. This is one of the earliest discovered rare diseases in humans, mainly caused by mutations in the coagulation factor VIII or IX genes. Repeated bleeding in the joints and muscles of patients can lead to lifelong disability. Currently, the standard treatment for hemophilia still mainly relies on coagulation factor replacement therapy. However, with the approval of several gene therapy drugs globally in recent years, this "one - time cure" treatment method is gradually becoming known to the patient population.

Specifically, GS1191, independently developed by Hua Yi Le Jian, is the first adeno - associated virus (AAV) vector gene therapy drug for hemophilia A (accounting for over 80%) approved for clinical trials in China. The enrollment of all patients in the Phase III clinical trial is expected to be completed within this year. Meanwhile, the company is also exploring overseas markets such as Saudi Arabia and Brazil.

Gong Yuntao said that the reason for choosing Saudi Arabia as the first overseas market is two - fold. On one hand, there are a relatively large number of hemophilia patients in Saudi Arabia. Although the total population of Saudi Arabia is only in the tens of millions, there are more than a thousand hemophilia patients. In addition, the high economic level and the free medical care available to its citizens also provide feasibility for the commercialization of high - priced new drugs. Among domestic CGT companies, CAR - T products of companies such as He Yuan Biotech and Reindeer Biotech are also actively deploying in the Middle East market through a similar model.

From the perspective of registration and application, innovative drugs enjoy certain policy preferences when listing in Saudi Arabia. Especially for gene therapy products without ethnic differences, companies can either, based on Chinese data, lead an international multi - center clinical study with a Chinese principal investigator (PI) as the international leading PI, supplement with a few local cases of data and then apply; or use the Chinese local clinical data as reference data from a reference country and attempt to apply for product registration.

This also means that, with a larger patient base in China, Chinese companies may be able to complete clinical trials faster and, to some extent, narrow the time difference in market competition with overseas - listed products. For example, it took nearly six years for Roctavian, a hemophilia gene therapy drug by BioMarin, to go from Phase III clinical trials to market. In contrast, it only took more than three years for Bo Paida Ke Ji Injection of Chinese company Belief Medicine. Hua Yi Le Jian expects that the ongoing Phase III clinical trial of GS1191 in China will complete patient recruitment in less than a year.

Gong Yuntao introduced that although there are relatively limited and scattered optional cooperative medical institutions and expert resources when conducting clinical trials for rare disease drugs, and there is a lack of public disease education, which often causes certain difficulties in patient recruitment. However, in the field of hemophilia, the patient organization alliance is relatively mature, and various medical, pharmaceutical, and even research information can be disseminated in a timely manner.

"Both international data and our own clinical research data show that most of the patients recruited in clinical trials are young and middle - aged people under 40. They have a more mature knowledge system, will actively learn about gene therapy knowledge, and have a higher willingness to cooperate in clinical trials. At the same time, this group of patients has a stronger desire to return to society, so they are more eager for a better treatment method than traditional therapies. For example, due to years of repeated bleeding, many patients need knee and hip replacements around the age of 20. They urgently need more advanced treatment methods to help delay the progression of disability to the greatest extent. And gene therapy can just meet this clinical treatment need. Currently, our longest patient follow - up study has lasted for four years, and under the effective dosage, there have been basically no problems with spontaneous bleeding after treatment."

In addition, the cost of clinical R & D and production in China is lower, which on one hand enhances the price competitiveness of domestic new drugs. The prices of rare disease drugs are generally very high, and the prices of several overseas - listed hemophilia gene therapy drugs have reached millions of dollars. Gong Yuntao revealed that the dosage of GS1191 is only 1/20 of that of Roctavian, with lower costs and relatively more pricing room.

On the other hand, the problem that patients have difficulty affording "sky - high" rare disease drugs is not limited to China; it also exists in markets such as the United States. Therefore, the cost advantage also provides domestic companies with the possibility of exploring more overseas expansion paths. For example, many medical institutions are currently building international medical treatment centers, hoping to attract more international patients to China for treatment.

"In May this year, we communicated with the Saudi Food and Drug Authority. They are very welcoming of rare disease drugs to be registered and listed locally and encouraged us to apply for the local innovation fund to jointly promote product R & D. Currently, GS1191 is continuously communicating closely with the Saudi regulators, hoping to obtain the local regulatory fast - track certification to accelerate the listing process in Saudi Arabia and the Middle East," Gong Yuntao said.

It is also understood that in addition to rare diseases, Hua Yi Le Jian is also deploying product pipelines in the fields of common diseases such as neurological diseases. After its establishment, the company has received investments from many institutions such as Tencent, Sequoia, and Qingsong Capital, with hundreds of millions of yuan in Series A financing. It is reported that the company is currently actively preparing for a new round of financing.