Top 15 High-Quality Frontier Innovation Achievements in China's Innovative Drug Sector for 2025

KN5501It is a development by Rui Therapeutics.CAR-NKCell therapy drugs, aimed at targetingCD19Target AchievementCD19+BDeep clearance of cells, inducing the subject's own immune reconstruction, achieving deep or complete remission of the disease. This drug utilizes a unique HTAS-RV™ Delivery System andCAR-NKCell expansion and cryopreservation technology features "universal" and "off-the-shelf" characteristics.

2025Year9Month, Rui Therapeutics announcedKN5501New Drug Clinical Trials (IND) Application to the Center for Drug Evaluation of the National Medical Products Administration (CDE) Implied license, indicated for moderate to severe refractory systemic lupus erythematosus.

2025Year11Month, Zhao Dongbao, Department of Rheumatology, Shanghai Changhai Hospital/Professor Gao Jie's team and Shenzhen Rui Therapeutics team published in The LancetKN5501Treatment Relapse/Refractory Systemic Lupus Erythematosus (SLE) The research results showKN5501In the treatment of recurrence/RefractorySLEIn terms of efficacy and safety, it has demonstrated outstanding results.

HN2301 isA product developed by Hongxin BiotechBased onmRNA-LNPIn the body (in vivo） CAR-TCandidate drug.2025Year9Month,RainbowBioInNEJM MagazineUpGlobally Announced for the First TimeHN2301Treatment of Systemic Lupus Erythematosus(SLE)PatientClinical trial research data.

Hongxin Biotech Successfully Overcomes the Constraints Hindering Industry Development“mRNAExtrahepatic Non-APCTargeted Delivery”Challenge, independently developed an internationally leading engineered cell-targeted delivery platform (EnC-LNP）。

HN2301The core component is composed of Hongxin Biotechnology's proprietary ionizable amino lipids (ILB-3132), HumanizedCD8Targeted antibody fragments and proprietary intellectual propertyAnti-CD19 CAR mRNAComposition.

IMC001Is the world's first approved in both China and the U.S.INDTargetedEpCAMSolid TumorsCAR-TProduct, in2024Year2Obtained dual approval from China and the US in the month ofINDApproval for TreatmentEpCAMExpression-positive advanced digestive system tumors. New indicationsINDApplied on2025Year3In the month, it received approval from the Center for Drug Evaluation of the China National Medical Products Administration (CDE) approved for the treatment of advanced epithelial solid tumors.

GT307Is aCRISPRNext-Generation Dual-Gene Knockout Tumor-Infiltrating Lymphocytes (TILs) in Gene Editing TechnologyTumor-Infiltrating Lymphocytes, TIL) product, aimed at solving traditionalTILKey bottleneck issues such as functional exhaustion and insufficient persistence tend to occur in the tumor microenvironment. Relying on self-developed...ImmuT Finder®High-throughput Immune Modulation Target Screening Platform,Gravel BiotechSystematic discovery and identification of inhibitorsTILKey immune regulatory factors for anti-tumor function, and throughCRISPRTechniques for precise knockout, thereby significantly enhancingTILThe anti-tumor function and persistence in the body.

In gene editing strategies,GT307Using high-fidelityCRISPR/AaCas12b^MAXNuclease System, compared to the classical SpCas9 Nucleases demonstrate significant advantages in editing safety, product phenotype, amplification, and functionality. Relevant research findings were published2025Year9Published inMolecular TherapyMagazine.

QT-019BIt is an "off-the-shelf" allogeneicCAR-TCell products, using leukocyte apheresis products from healthy donor peripheral blood as the starting raw material, are genetically edited to stably express two different chimeric antigen receptors (CAR), respectively targetingCD19AndBCMA, thereby makingQT-019BCapable of simultaneous recognition and clearance expressionCD19AndBCMAThe ability of cells.

To reduce graft-versus-host disease (GvHD) risk, the product eliminates through gene knockout methodTCell Receptor (TCR) expression; at the same time, to reduce allogeneic immune rejection, Qihan Bio achieves low immunogenicity through multi-gene editing, thereby reducing the patient's ownNKCell &TCell-mediated recognition and cytotoxicity.

2025Year8Month,QT-019BNew Drug Clinical Trial Application Approved by the United StatesFDAOfficial Approval,11Month,QT-019BThe new drug clinical trial application has been approved.Center for Drug Evaluation, National Medical Products Administration (CDE）Tacit consent, indications are all for refractory systemic lupus erythematosus (rSLE）。

It is claimed that,QT-019BYesThe FirstContinuously awarded in the United StatesFDAAnd ChinaCDEThe tacitly approved dual-target generic drug independently developed by a Chinese companyCAR-TCell Products.

It is worth mentioning that,In November 2025, QT-019B was granted Fast Track Designation (FTD) by the U.S. FDA for the treatment of refractory systemic lupus erythematosus with severe thrombocytopenia.

2025Year4Month, Time Ridge Biotech collaborates with Professor Zhang Rui's team from Sun Yat-sen University inNature Biotechnology MagazinePublished a breakthrough study proposing an entirely new endogenousADARRecruitmentgRNADesign Concept——MIRROR (Mimicking Inverted Repeats to Recruit ADARs Using Engineered Oligoribonucleotides). This technology has significantly improvedRNAEditing Efficiency, Short Chains with Simple Chemical Modifications Only gRNA Can achieve over in mice80%Editing efficiency.

Developed by the companyRC001The drug, through a small nucleotide drug strategy, recruits endogenousADARAchieving Single-BaseRNAEditor, inRNAPrecise Targeted Intervention at the Level.2025Year4In the month, the company announcedRC001Obtained in the United StatesFDAOrphan Drug Designation (ODD）。

2025Year12Month，EJL TherapeuticsInnovative Epigenetic Regulation Therapy Developed by Rui TherapeuticsEPI-003Officially Obtained by the United StatesFDAClinical Trial Permit (IND）, used for treatmentChronic Hepatitis B。

EPI-003It is a novel intravenous antiviral drug based on a completely new mechanism of action. The drug works byLNPDelivery system encoding epigenetic regulatory proteinsmRNATargetedHBVGene GuidanceRNAPrecisely delivered to liver cells, directly targetingcccDNAAnd IntegrationDNAPerform persistent epigenetic modifications, thereby suppressing the production of all viral products at the transcriptional source.

Preclinical studies have shown,EPI-003Can significantly and continuously reduce hepatitis B surface antigen (HBsAg) andHBV DNAlevels, and maintain long-term efficacy after discontinuation, showing the potential to achieve functional cure or even complete cure of hepatitis B. It is reported that,EPI-003Is the world's first to obtainFDAApproved for clinical entry based on lipid nanoparticles (LNP) Delivery Technology for Epigenetic Regulation Therapy.

YOLT-203It is an innovative in vivo gene-editing drug developed using Yaotang Biotech's proprietary technology.CRISPR/CasGene Editing ToolsYolCas12HF, and through the self-developed lipid nanoparticles (LNP) System accurately delivers to liver cells, targeted editingHAO1Gene, Inhibiting Glycolate Oxidase (GO) expression, reducing oxalate production at the source, with the potential to achieve "one treatment, lifelong cure" for primary hyperoxaluria1Type (PH1) Provides patients with new hope for a cure.

Following2024Year obtained in the United StatesFDAGranted Orphan Drug Designation (ODD) and Pediatric Rare Disease Designation (RPDD) Afterwards,2025Year7Month,YOLT-203Obtained the European Medicines Agency (EMA) Orphan Drug Committee (COMP) Granted Orphan Drug Designation (ODD），2026Year2Month,YOLT-203 Obtained in the United StatesFDAGranted Advanced Regenerative Medicine Therapy (RMAT) Recognition.

HM2002Is a new generation of circular based on the independent innovation technology platform of Rui Therapeutics.RNADrug, specifically designed for the treatment of ischemic heart disease. This therapy achieves stable expression of vascular endothelial growth factor (VEGF) within the myocardium.VEGF), significantly promoting angiogenesis and myocardial perfusion, thereby accelerating cardiac function recovery. Compared with traditional linearRNAIn comparison,HM2002The annular adoptedRNAThe technology has higher stability and lower immunogenicity, demonstrating unique advantages in therapeutic applications.

2025Year1Month,HM2002Approved by the National Medical Products Administration (NMPA) Clinical Trial Permit (IND), used for the treatment of ischemic heart disease (IHD). This is the first to obtainNMPACircular of Clinical Trial PermitRNABiological products.2025Year11Month,HM2002ReacquireNMPAClinical Trial Permit,The newly approved indication isRefractory Angina;12Month,HM2002The Third Time to ObtainNMPAClinical trial approval, newly approved indication for Chronic Coronary Syndrome.

In addition,2025Year5Month,HM200Obtained in the United StatesFDANew Drug Clinical Trial Application (IND`) Implied consent.`2026Year1Month,HM2002ReacquireFDAGrant“Fast Track”Qualification Certification.

TI-0093It is a circular-based product developed by RoundBio. RNATherapeutic tumor vaccine technology, clinically intended for HPV16 Positive advanced recurrent or metastatic solid tumors.TI-0093 Adopting a brand newLNPDelivery system, encapsulating mutated and immune-enhanced modified coding HPV16 E7 And E6 Circular Antigen RNA Achieve efficient delivery. Through intramuscular injection,LNP The vector will circularize RNA After successful delivery into cells, it can precisely encode antigens designed with immune optimization, significantly enhancing antigen presentation efficiency, rapidly initiating and efficiently amplifying antigen-specific responses. CD8+T Cells, while achieving active immune infiltration locally in the tumor, form a powerful "immune encirclement," ultimately mediating advanced progression. HPV16 Complete remission and durable efficacy in positive tumors demonstrate highly efficient and specific immune modulation and anti-tumor activity.

2025Year 8 Month, TI-0093 New Drug Clinical Trials (IND) Application ObtainedCenter for Drug Evaluation, National Medical Products Administration (CDE）Clinical Trial Implied Permission. According to reports,TI-0093 Is the world's first circular RNA to conduct clinical research in the field of tumor treatment. RNA Drug. 2025Year11Month, the company announced TI-0093 CompletedIFirst Patient Dosed in Phase I Clinical Trial.

MTS-105Is an industry-first (FIC）mRNACoding Bispecific Antibodies (TCE，TCell Connector) Hepatocellular carcinoma treatment candidate drug, developed by Rui Therapeutics with proprietary liver high-specificity technology.LNPDelivery.Research data shows,MTS-105DeliveredmRNAAfter being taken up by hepatocytes, it encodes and secretes large amounts of bispecific antibodies in situ, which can rapidly penetrate into hepatocellular carcinoma tissues. Using the "Trojan Horse" strategy, it efficiently induces an immune response within the tumor.TActivation of Cells to Kill Tumors Achieved in Mouse ModelsComplete clearance of hepatocellular carcinoma and induction of long-termTCellular immune memory.MTS-105Is expected to fundamentally solve the problem of poor efficacy of traditional protein-based bispecific antibodies in solid tumors. Currently,MTS-105Has entered the clinical development stage.

2025Year11Month, USAFDAGrantMTS-105Orphan Drug Designation（ODD), Indication: Hepatocellular Carcinoma.

2025Year12Month,MTS-105Preclinical Research Results on the Treatment of Hepatocellular Carcinoma inNature CommunicationsPublication in the journal.MTS-105Demonstrated significant anti-tumor activity in multiple animal models, providingTCEProvides a novel strategy for clinical translation in the treatment of solid tumors.

YL201（tam-peli, Icotinib Monoclonal Antibody) is based on the camptothecin toxin linker platform that can be cleaved in the tumor microenvironment, developed by Rui Therapeutics (TMALIN®) A targetedB7-H3Antibody-drug conjugates. Currently,YL201Clinical research on a variety of advanced solid tumors is currently being conducted worldwide. In China,YL201Three trials have been conducted separately for the indications of nasopharyngeal carcinoma, small cell lung cancer, and esophageal squamous cell carcinoma.IIIPivotal registrational clinical trial.

Previously,YL201Have successively obtained the Center for Drug Evaluation of the National Medical Products Administration's approvals for indications such as small cell lung cancer, nasopharyngeal carcinoma, and esophageal squamous cell carcinoma.4Breakthrough Therapy Designation, and received U.S.FDABreakthrough Therapy Designation for the indication of small cell lung cancer; meanwhile,YL201Has obtained the United StatesFDAGranted3Orphan drug designation, including small cell lung cancer, nasopharyngeal cancer, and esophageal squamous cell carcinoma.

2025Year3Month,YL201For the treatment of advanced solid tumorsIResults of the Phase Clinical Study inNature MedicinePublished. The research covers the globe.54Home Center, Included312Example patient, foundYL201The significant efficacy in refractory tumors such as small cell lung cancer, nasopharyngeal carcinoma, pulmonary lymphoepithelioma-like carcinoma, and non-small cell lung cancer, with an objective response rate (ORR) and Disease Control Rate (DCR) all exceed the current standard of care, and safety is controllable.

2026Year5Month23Date, Rui Therapeutics announced,YL201In the treatment of recurrence/Metastatic Nasopharyngeal CarcinomaIIIPhase Clinical Trial (TAISHAN-301), the Independent Data Monitoring Committee (IDMC) Evaluation confirmed that the pre-specified interim analysis reached the co-primary endpoint of blinded independent central review (BICR) Objective Response Rate (ORR), the other co-primary endpoint overall survival (OS) Not yet mature.This is the world's firstB7-H3 ADCInIIIPositive results achieved in the clinical phase.

DEG6498Is the world's first targetedHuRMolecular glue degrader for targets, developed by Dagene Bio based on proprietaryGlueXplorerPlatform development, which is achieved through recruitmentCRL4-CRBN E3Ubiquitin ligase achievesHuRSelective degradation of proteins.HuRIs aRNABinding proteins, previously considered undruggable targets by the industry, play a crucial role in the progression of diseases such as cancer, inflammation, and metabolic disorders.

Preclinical studies have shown,DEG6498Significantly inhibits both in vivo and in vitroBRAFMutant tumors grow and exhibit dose-dependent antitumor activity and good tolerance in animal models.HuRDegradation agents can target multiple characteristics of cancer simultaneously, offering advantages over single-pathway inhibitors.

DEG6498In2025Successfully obtained in the United StatesFDAAnd ChinaNMPATheINDApproval (NCT07244835 / CTR20254261), currently conducting the first-in-human (First in human, FIH）IClinical trials to evaluate the safety and pharmacokinetics of the drug in patients with solid tumors/Pharmacodynamic Characteristics and Preliminary Evaluation of Antitumor Activity.2025Year11The first patient dosing has been completed this month.

VG161Is a based onⅠType Herpes Simplex Virus (HSV-1) Constructed novel anti-tumor immune-enhanced oncolytic virus, it isThe World's FirstEntering ClinicalCarry4An exogenous immune regulatory geneIL12、IL15/15Rα（IL15AndIL15ReceptorαSubunit) andPD-L1Blocking Peptide (PDL1B) Oncolytic virus product.

2025Year3Month,VG161Clinical research data inNaturePublication in the journal. The research focuses onAdvanced Hepatocellular Carcinoma with Multiple Lines of Treatment Failure (HCC`) Patient``, the results showed`VG161Not only does it exhibit excellent safety performance, but alsoSignificantly Extend Patient Survival, providing new hope for patients with advanced hepatocellular carcinoma who have no therapeutic options.

Specifically inNatureClinical data published on: In34ExampleAllStandard treatments have all failed.Advanced Hepatocellular Carcinoma (HCC) In patients,VG161Objective Response Rate of Monotherapy（ORR) for17.65%, Disease Control Rate (DCR）64.71%. More noteworthy isSecond-line FailureIn the overall cohort of patients, the median survival period (OS) Reach9.4Months，Significantly Superior toBaseline-matched real-world control4.7Months. And,22Patients in the subgroup who received immunotherapy for more than three months and failed before enrollment (PreCPI>3months) medianOSEven reaching17.3Months。

PTT-936Is a brand newALPK1（Alpha-kinase 1) Small molecule agonist, which treats tumors by activating the body's immune system.ALPK1As a novel innate immune receptor, it can recognize small molecules derived from bacteria.ADP-heptose，Thereby activating the body's immune response.2018In the year, the team of Academician Feng Shao, the co-founder of Yanming Bio, inNatureThe magazine reported the discovery.2025Year12Month, Academician Shao Feng's team once again inNatureJournal Article: First Proposal of Activating Cytoplasmic Pattern Recognition ReceptorsALPK1Capable of inducing efficient anti-tumor immune responses and demonstrating its potential application value as a new target for immunotherapy.

Previous preclinical studies have shown,ALPK1AgonistPTT-936Monotherapy or combination with immune checkpoint inhibitors demonstrates good anti-tumor activity. Moreover, compared to innate immune agonists of other mechanisms, it has a potentially larger therapeutic window.PTT-936, as a small molecule agonist based on a novel target and mechanism, has the potential to address the safety and efficacy issues of existing innate immune agonists, offering a new treatment option for cancer patients.

Note: This article focuses on introducing the selected projects.2025-2026Important progress made in the year, sourced from the official websites and official WeChat accounts of various companies.