World’s First Functional Cure for Hepatitis B mRNA Drug Developed in Nanjing
Chinese researchers in Nanjing have developed the world’s first messenger RNA (mRNA) drug designed to achieve a functional cure for chronic Hepatitis B, marking a major milestone in the global fight against a virus that affects over 240 million people worldwide. The drug, designated CPU-YL01, has received clinical trial approvals from regulatory authorities in China, the United States, and Laos, with Phase I trials now underway in Nanjing.
A Breakthrough in Hepatitis B Treatment
Developed by a team led by Professor Yang Yong at China Pharmaceutical University (CPU) in collaboration with Jiangsu Chuangyuan Life Science Technology Co., Ltd., CPU-YL01 is an mRNA therapeutic vaccine encapsulated in lipid nanoparticles. Unlike conventional treatments that merely suppress viral replication, this drug is designed to train the immune system to recognize and eliminate Hepatitis B virus (HBV)-infected cells, potentially offering a finite course of treatment rather than lifelong medication.
According to People’s Daily, the drug is “expected to break through the technical bottleneck of existing Hepatitis B treatments that require long-term medication, bringing hope of ‘finite-course, functional cure’ to hundreds of millions of Hepatitis B patients worldwide.”
The Scale of the Global Hepatitis B Crisis
The World Health Organization estimates that 240 million people were living with chronic Hepatitis B infection in 2024, with 1.1 million deaths that year — mostly from cirrhosis and hepatocellular carcinoma (liver cancer). In China alone, approximately 75 million people are HBV carriers, and over 450,000 HBV-related deaths occur annually. An estimated 80% of primary liver cancer and 60% of cirrhosis cases in China are caused by HBV infection.
Current standard treatments — nucleoside/nucleotide analogs such as tenofovir and entecavir — suppress viral replication but require lifelong administration and do not eliminate the viral reservoir. Only 4.3% of people with chronic Hepatitis B worldwide were receiving antiviral treatment in 2024.
How CPU-YL01 Works
CPU-YL01 employs a fundamentally different approach from existing therapies. As an mRNA therapeutic vaccine, it encodes HBV-related antigens that, when delivered via lipid nanoparticles, activate dual immune responses against the virus. The technology has both preventive and therapeutic potential, aiming to achieve what is known as a “functional cure” — sustained undetectable HBV DNA and Hepatitis B surface antigen (HBsAg) in the blood for at least six months after stopping all treatment.
This mRNA-based mechanism is distinct from other drug candidates in the Hepatitis B cure race. While competitors like GSK’s Bepirovirsen (an antisense oligonucleotide) and Hengrui Pharmaceutical’s HRS-5635 (a small interfering RNA) target viral RNA for degradation, CPU-YL01 takes an immune-based approach — what industry analysts describe as “awakening the immune army” rather than directly attacking the virus.
According to the CPU Technology Transfer Office, the patent covers “a chronic Hepatitis B functional cure mRNA vaccine and its application” that “contains mRNA sequences encoding Hepatitis B virus-related antigens, encapsulated in lipid nanoparticles for delivery, which can efficiently activate dual immune responses against the Hepatitis B virus, possessing both preventive and therapeutic effects.” The mRNA approach also offers advantages in raw material accessibility and scalable production, potentially reducing costs compared to existing biologics.
The Development Journey
The path to clinical trials began on September 3, 2025, when Professor Yang Yong’s team filed the patent for their chronic Hepatitis B functional cure mRNA drug. The patent was successfully transferred to Jiangsu Chuangyuan Life Science Technology Co., Ltd. on March 23, 2026, through CPU’s technology transfer office. By May 2026, CPU-YL01 appeared in drug pipeline databases as approved for clinical trials, and on July 16, 2026, the Nanjing Gulou District Government published the official announcement. The Phase I clinical trial was initiated in Nanjing in July 2026.
Strong Industry-Academia-Government Collaboration
The drug’s development exemplifies China’s integrated innovation model, combining government support, academic research, and industrial partnerships. The Nanjing Gulou District Government has played a central role, providing policy support and infrastructure through the Nanjing Biomedical Innovation and Transformation Research Institute — a high-level platform co-founded by the district, CPU, and leading enterprises.
A 1 billion RMB (approximately $138 million) Gulou Emerging Industry Investment Fund has been established to support biopharmaceutical innovation. The Gulou District Government described the achievement as “not only a major breakthrough in new drug R&D for CPU, but also another milestone in Gulou District’s deepening strategic cooperation with universities.”
The Competitive Landscape
CPU-YL01 enters a highly competitive field. GSK’s Bepirovirsen is closest to market, with a potential FDA decision expected by October 26, 2026, and a projected launch in Q1 2027. Phase III data showed a 19% functional cure rate in the overall study population. Meanwhile, domestic Chinese company Haobo Pharmaceutical’s AHB-137 has demonstrated a 32% functional cure rate in Phase II trials.
However, as 36Kr Europe notes, CPU-YL01’s mRNA mechanism is distinct and could potentially be used in combination with other approaches. The drug is still in early-stage development — Phase I trials focus on safety and dosing, meaning it is likely 5-8 years from potential market approval, assuming successful trials.
Looking Ahead
The initiation of Phase I clinical trials in Nanjing represents the first step on a long road. Key questions remain about the drug’s efficacy, optimal dosing regimen, and potential for combination therapy with existing antivirals. Researchers are also exploring whether CPU-YL01 could be combined with nucleoside analogs or immune modulators to enhance treatment outcomes — a strategy that is increasingly seen as essential for achieving comprehensive clinical cure.
However, the multi-country regulatory approval — from China’s NMPA, the U.S. FDA, and Laos — signals confidence in the drug’s scientific foundation and the developers’ ability to navigate international regulatory frameworks. This simultaneous multi-jurisdiction approval is rare for a Chinese-developed drug candidate at such an early stage.
A Nature Communications study published in June 2026 on a related dual-antigen mRNA vaccine approach for chronic Hepatitis B demonstrated that such mRNA-based therapeutic vaccines can elicit robust HBV-specific immune responses, leading to near-complete viral genome clearance and serological conversion in animal models. This scientific validation of the mRNA approach bodes well for CPU-YL01’s prospects.
For the hundreds of millions of people living with chronic Hepatitis B worldwide, CPU-YL01 represents more than just another drug candidate. It embodies a paradigm shift in how we think about treating this devastating disease — moving from a lifetime of management toward the possibility of a cure.