CureGene Pharmaceuticals Secures B-round Funding to Advance Innovative Pipelines in Cardiovascular and Antiviral Therapeutics

What is the disease that causes the most human deaths?

When this question is raised, “cancer” is perhaps the first answer that comes to many people’s minds.

In fact, cancer is not the disease with the highest mortality rate; the “number one killer” of human health is actually another condition—cardiovascular disease.

For over two decades, cardiovascular and cerebrovascular diseases have remained the leading cause of death worldwide.According to the "2019 Global Health Estimates" report released by the WHO, in 2019, out of 55.4 million deaths worldwide, nearly 9 million were due to ischemic heart disease, representing an increase of more than 2 million compared to 2000. In addition, stroke-related deaths reached 6.55 million. Cardiovascular diseases, including ischemic heart disease and stroke, have become the leading cause of death globally.

Currently, multiple therapeutic agents have been developed and are available on the market, including antiplatelet drugs such as clopidogrel, cangrelor, and ticagrelor, as well as anticoagulants such as warfarin and heparin. Despite the availability of numerous treatment options, some patients still exhibit resistance to antiplatelet therapy—defined as an inadequate therapeutic response—due to drug side effects and interindividual variability in drug responsiveness. Furthermore, alternative medications may be associated with a heightened risk of bleeding.

The issue of drug resistance has been placed before researchers and industry professionals, making it a current priority to provide patients with therapeutic agents that offer greater efficacy and enhanced safety.Shanghai CureGene Pharmaceutical Co., Ltd. (hereinafter referred to as “CureGene”) is an innovative pharmaceutical company dedicated to the research and development of next-generation antiplatelet drugs to address the issue of drug resistance.。

CureGene, founded in Shanghai in 2018, is a startup focused on the research and development of therapeutic drugs in the cardiovascular, cerebrovascular, and antiviral fields. Its pipeline includes antiplatelet, anticoagulant, anti-respiratory viral infection, and anti-hepatitis B agents.Since its establishment, CureGene has independently built two core technology platforms—the Targeted Prodrug Technology Platform and the Nucleic Acid Drug Technology Platform. Leveraging its team strengths and core technologies, the company has developed multiple innovative pipelines in the fields of cardiovascular/cerebrovascular diseases and antiviral therapies. Notably,The company's independently developed antiplatelet drug, CG-0255, has completed Phase I clinical trials and is currently advancing through pivotal registration-enabling clinical studies, with the potential to achieve market approval via an expedited review pathway in the future.。

In just five years, CureGene has accomplished key milestones, including team assembly, platform development, and pipeline advancement. Its core pipeline is on the verge of entering registrational clinical trials, marking an imminent transition into the productization phase.The company’s rapid and smooth development has been driven by the leadership of its core figure, Dr. He Gongxin, Founder and CEO of CureGene.。

Dr. He is a scientist and entrepreneur with 30 years of deep expertise in the biopharmaceutical industry. Prior to formally entering the pharmaceutical sector, Dr. He earned his Ph.D. in Synthetic Chemistry from Kyushu University in Japan and completed his postdoctoral research at the University of California in the United States. In 1992, Dr. He joined Gilead Sciences, where he successively served as a Scientist at the U.S. headquarters and as Senior Director of Research and Development.

During his tenure at Gilead, Dr. He participated in and led the research and development of multiple international best-in-class novel drugs. Notably,He is also the principal inventor and project lead for the research and development of tenofovir alafenamide (TAF), a next-generation prodrug of tenofovir, an anti-hepatitis B virus medication widely used worldwide.. Furthermore, Dr. He is a co-inventor of multiple antiviral therapies and possesses extensive R&D experience in the fields of cardiovascular and cerebrovascular diseases, nucleic acid-based drugs, and various antiviral treatments.

In 2008, Dr. He returned to China to serve as the Chief Representative and founding General Manager of Gilead Sciences’ China region, where he was responsible for establishing the company’s regional branch in the country. During this period, Dr. He transitioned from a drug researcher into a manager overseeing production operations, supply chain management, and corporate development.

In 2018, Dr. He had served as the head of Gilead Sciences’ China operations for a full decade. During his tenure, he developed a profound understanding of China’s overall pharmaceutical ecosystem and accumulated extensive resources. It was at this juncture that the aspiration to “participate in the development of China’s biopharmaceutical sector and promote the growth of the domestic pharmaceutical and healthcare industry” re-emerged in Dr. He’s mind.

“Founding a company to truly translate scientific theories into tangible outcomes, delivering better original innovative drugs to patients.” Guided by this vision, Dr. He left Gilead Sciences in 2018 after 26 years of service to establish CureGene in China, focusing its R&D efforts on cardiovascular diseases with the highest mortality rates and antiviral therapies.

Subsequently, Dr. He rapidly assembled a team characterized by shared values, strong cohesion, and professional expertise, with more than half of its members being his former colleagues from Gilead Sciences.including Chief Operating Officer Jialin Chen and Vice President of Finance Li Yao, they have jointly witnessed Gilead’s transformation from a small company into a multinational pharmaceutical enterprise, and are characterized by their low-key, pragmatic approach and professional, diligent execution capabilities.

In addition, the company’s team of scientists is “star-studded,” comprising multiple senior scientists in the field of drug development. Among them,Dr. Chen Xiaowu, Vice President of Chemical R&DWith over 25 years of experience in innovative drug R&D, he participated in the late-stage development of Tamiflu (oseltamivir), an oral influenza medication, at Gilead Sciences, and made significant contributions to the development of multiple HIV and hepatitis C drugs that later became "blockbuster products."

Meanwhile, CureGene has also attractedDr. Nie Yuchun, a Senior Scientist in Virology, Appointed Vice President of Biological R&D. Dr. Nie has been deeply engaged in the field of nucleic acid-based antiviral therapeutics for over a decade, previously holding senior R&D positions at Roche’s Research and Development Center and Memorial Sloan Kettering Cancer Center. Furthermore,Ms. Yan Hua, a senior scientist in the medical field, serves as Senior Director of Clinical Project Management at the company., she has nearly 40 years of experience in drug R&D management, having previously held roles at Shanghai Xuhui District Central Hospital, Shanghai Sino-American Squibb Pharmaceuticals, and Shanghai New Origin Pharmaceutical, where she was responsible for clinical affairs, regulatory submissions, pharmacology and toxicology, and new drug R&D management.

To date, CureGene has assembled a specialized team with an average of over 15 years of experience in drug development. The core members’ expertise and experience span the entire new drug development process, providing robust momentum for the company’s operational growth and the advancement of its pipeline.

Based on the core team's extensive experience and comprehensive assessment of clinical needs, CureGene has decided to establish a presence in the fields of cardiovascular and cerebrovascular diseases and antiviral therapies. In response to market demand, the company will prioritize the research and development of next-generation antiplatelet agents and broad-spectrum respiratory antiviral drugs. Having clearly defined its development roadmap and R&D focus,CureGene rapidly established two core technology platforms—the Targeted Prodrug Technology Platform and the Nucleic Acid Drug Technology Platform。

Among these, the targeted prodrug technology platform was the first to be established in 2020. In simple terms, a “prodrug” is a therapeutic agent that requires enzymatic or non-enzymatic conversion within the body to release the active drug, thereby exerting its pharmacological effect.

Notably, Gilead possesses mature prodrug technology, and Dr. He led the research on tenofovir alafenamide (TAF), a prodrug of tenofovir, during his tenure at Gilead. Under Dr. He’s leadership and through the team’s efforts, CureGene established a proprietary targeted prodrug technology platform within two years.

“Targeting” is one of the platform’s prominent advantages, as it can modify and improve drug-like properties such as physicochemical characteristics, delivery and transport efficiency, enzymatic selectivity, and compound stability, therebyAchieve targeted delivery to different diseased organs, i.e., deliver active ingredients more efficiently to specific therapeutic sites, thereby achieving optimal efficacy at the minimum safe dose.. Furthermore, the platform can rapidly generate novel targeted prodrugs with significant improvements in pharmacokinetics/pharmacodynamics or safety, based on validated active substance targets, thereby efficiently producing candidate compounds with clear druggability and clinical development value.

The nucleic acid drug technology platform is led by Dr. Nie. This platform features capabilities in sequence design and modification/labeling for small nucleic acid drugs and messenger RNA (mRNA) drugs, as well as diverse, advanced drug delivery technologies. It enables the selective use of drug delivery platforms for liver-targeted or extrahepatic organ-specific applications, facilitating the development of clinical candidates for various administration routes, including systemic and local delivery.

Driven by its two core “engines”—its technology platform and team—CureGene currently has four pipelines under development in the cardiovascular, cerebrovascular, and antiviral fields.

Among these, the core pipeline asset, the antiplatelet drug CG-0255, has made the most rapid progress. CG-0255 is a next-generation P2Y12 inhibitor developed for antiplatelet therapy in cardiovascular and cerebrovascular diseases.

Notably, CG-0255 employs a hydrolytic prodrug design. Leveraging extensive expertise in prodrug technology, it represents a scientific breakthrough as the world’s first thiol prodrug. It is converted by carboxylesterases into H4, an active metabolite with antiplatelet activity, thereby completely bypassing the oxidative metabolic pathways commonly relied upon by antiplatelet drugs. Consequently, CG-0255 can rapidly generate its active metabolite in vivo through the action of hydrolases. Due to the widespread distribution of hydrolases, there is virtually no interindividual variability in metabolism. Furthermore, CG-0255 is currently the world’s first P2Y12 inhibitor available in both oral and injectable formulations, filling the gap in domestic injectable options.

Clopidogrel is currently one of the most commonly used antiplatelet drugs. It is an inactive prodrug that relies primarily on enzymes within the cytochrome P450 (CYP) system, particularly CYP2C19, for activation into H4, its active metabolite with antiplatelet effects, thereby exerting its therapeutic action.

However, a significant proportion of the global population carries loss-of-function alleles of CYP2C19, which means that clopidogrel is either not metabolized or is metabolized extremely slowly in these individuals. This leads to a weak antiplatelet effect after administration, resulting in “clopidogrel resistance.” For this reason,The FDA has added a black box warning to the approved labeling for clopidogrel.

Existing studies show that CYP2C19 loss-of-function alleles are highly prevalent among patients of all ethnicities. Approximately 25%–30% of the White population carries CYP2C19 loss-of-function alleles, whereas this proportion reaches as high as 59% in East Asian populations.¹. Furthermore, multiple clinical studies have demonstrated that the efficacy of clopidogrel is indeed reduced or lost in these patients. This also indicates that the issue of "clopidogrel resistance" is more pronounced among Chinese patients with cardiovascular and cerebrovascular diseases. In addition, clopidogrel can only be administered orally and not intravenously, with an onset of action of approximately 2 to 8 hours, which limits its clinical application in emergency situations.

Unlike clopidogrel, CG-0255 completely bypasses the CYP-mediated metabolic issues associated with it. Supported by targeted prodrug technology, CG-0255 enables more precise drug delivery, achieving greater efficacy at minimal doses. Furthermore, CG-0255 features high bioavailability and rapid onset of action, and can be administered via both intravenous injection and oral routes. It holds promise as a substitute for clopidogrel, comprehensively addressing potential drug resistance in patients while expanding its clinical application scenarios.

Previously,Dr. Chen Xiaowu presented the Phase I clinical study results of CG-0255 at the Featured Science Session of the 2023 American Heart Association (AHA) Scientific Sessions.This is an open-label, active-controlled, dose-escalating Phase I clinical trial. Five cohorts (each with 6 participants) will receive CG-0255 via bolus injection or infusion at different doses, with cangrelor serving as the active control. The study aims to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of intravenous CG-0255 in healthy subjects.

The study results demonstrated that CG-0255 exhibits favorable safety and tolerability, undergoes rapid and stable conversion to the active metabolite H4, and shows a clear dose-dependent inhibition of platelet aggregation with a rapid onset of action, achieving over 90% inhibition of platelet aggregation within 20 minutes post-administration. Furthermore, CG-0255 irreversibly binds to the P2Y12 receptor, resulting in sustained antiplatelet effects, with inhibition of platelet aggregation (IPA) persisting 24 hours after dosing. Importantly, the platelet aggregation inhibitory effect of CG-0255 shows no significant inter-individual variability.

As a startup whose core team hails from Gilead Sciences, CureGene has also established a presence in the antiviral sector. However, rather than merely imitating or replicating Gilead’s model, the company aims to build upon its proprietary core technologies to achieve significant breakthroughs in antiviral therapy, akin to Gilead’s development of sofosbuvir, the “miracle drug” for hepatitis C. Consequently, CureGene focuses its R&D efforts on broad-spectrum antiviral drugs targeting respiratory viruses and hepatitis B.

Among these, CG-0980, an RNA polymerase inhibitor, is being developed by the company for indications involving broad-spectrum respiratory RNA viral infections. It is a small-molecule direct-acting antiviral agent with broad-spectrum antiviral activity. Unlike drugs that target only a specific pathogen, CG-0980 demonstrates clear antiviral activity against multiple common respiratory RNA viruses, such as respiratory syncytial virus (RSV), human metapneumovirus (hMPV), parainfluenza virus, common coronaviruses, and rhinoviruses. It addresses the potential clinical needs of the general population for both treatment of respiratory viral infections and post-exposure prophylaxis following close contact.

Preclinical studies have demonstrated that CG-0980 exhibits a favorable safety profile and holds the potential to serve the broadest patient population, thereby addressing the unmet market need for broad-spectrum antiviral drugs against respiratory viruses. Currently, CG-0980 is in the preclinical stage, with an Investigational New Drug (IND) application expected to be filed by the end of this year.

Shortly after CureGene announced positive Phase I clinical trial data for CG-0255, the company further revealed in early this year that it had successfully completed a RMB 100 million Series B financing round. The round was led by Taikun Fund, under Tailong Investment, with other major investors including Lichen Investment and Taiyu Investment. Existing investors such as Honghai Chuangjian and Hankang Capital also continued to support the company.

The new funding injection further supports the ongoing pivotal registration clinical study of CG-0255, while also accelerating the advancement of other core pipeline candidates into clinical stages.

Accelerating the clinical development and commercialization of CG-0255 has become a key short-term objective for CureGene. “This year, we will rapidly complete the pivotal registration clinical trials for CG-0255, with the expectation of achieving market approval through an expedited review process thereafter. At that time, we look forward to providing better treatment options for patients both in China and abroad,” said Dr. Chen Xiaowu.

“CureGene Seeks to Explore a New Model for the Development of China’s Biopharmaceutical Industry“Driven by this grand vision, Dr. Chen Xiaowu stated that although CureGene is currently a small startup, it will continue to develop combination therapies for cardiovascular and cerebrovascular diseases in the future, while further advancing its antiviral pipeline and commercializing candidate products. By providing patients with more effective therapeutic options and creating broad value for society, CureGene remains true to the original aspiration that motivated most of its scientists to enter the biopharmaceutical industry.”

References:

1. Ellithi M, Baye J, Wilke RA. CYP2C19 genotype-guided antiplatelet therapy: promises and pitfalls. Pharmacogenomics. 2020 Aug;21(12):889-897. doi: 10.2217/pgs-2020-0046. Epub 2020 Jul 29. PMID: 32723143; PMCID: PMC7444625.