Cell and Gene Therapies, siRNA, and Next-Gen Antibodies Converge in Fight Against Solid Tumors and Hepatitis B: CDE Weekly Update
BRL Medicine
Cell and Gene Therapy Drug Developer
September 24, 2025
eMedClub News
According to the Center for Drug Evaluation of the National Medical Products Administration of China（CDE）Incomplete statistics from the official website and publicly available information,Last week (September 15 - September 21More than 30 Class 1 innovative drugs are proposed to be included in the breakthrough therapy category/IND has been granted clinical tacit approval/IND application has been accepted, covering popular fields such as cell and gene therapy, TCE, ADC, etc., with treatment areas spanning solid tumors, chronic hepatitis B, autoimmune diseases, etc., which are expected to bring new treatment options to more patients.
The following will introduce some representative pipelines.
CGT: CAR-T/TIL/NK/siRNA All Making Strides
Kinsay Biotech:
GUCY2C CAR-T
IM96 Chimeric Antigen Receptor T-Cell Injection submitted by Jinsai Bio, a wholly-owned subsidiary of Yimiao Zhongguo(GUCY2C-Targeted CAR-T)Received CDE clinical implication permission, intended for the development of gastric cancer treatment. This CAR-T has been approved for clinical trials in China in 2024 to treat colorectal cancer.
Gastric cancer and colorectal cancer are malignant tumors with high incidence and mortality rates globally, and there remain unmet needs in clinical treatment. The core advantage of IM96 lies in its targeting of the GUCY2C antigen, which is highly specifically expressed in gastrointestinal malignancies, with a positive rate in colorectal cancer reaching80%, The positive rate of gastric cancer exceeds 60%, providing an ideal target for precision treatment.
Early clinical data show that its efficacy is significantly better than the current third-line standard treatment for colorectal cancer: disease control rate（DCR）74%`, Optimal Objective Response Rate`（ORR）50%`, Median Progression-Free Survival`（mPFS）Up to 10 months, median overall survival（OS）Ultra17 Months。
This Week's IND Progress in the Field of Immune Cell Therapy: BRL Medicine's CD19-Targeted Gene ModificationAllogeneic Chimeric Antigen Receptor T CellsCell Injection for Clinical Trial Application Accepted, Aiming to Break Through the Limitations of Autologous CAR-T and Improve Accessibility; QM101 Cell Injection from Beijing Qimai Yonghua Biotechnology(Autologous NK Cells)and LM103 Injection from Suzhou Lanma Medical Technology Co., Ltd.（TIL）Directly addresses the unmet needs of solid tumors.
Flow cytometry, as a key technology in the field of bioanalysis, has been widely used in innovative therapy fields such as cell therapy and antibody drugs, with data covering core indicators like cell viability and target binding rate.To help enterprises build a full-process compliant data system that meets industry regulatory standards,September 25, 2025 (Thursday) 19:00-20:30, Host"How Electronic Data Meets FDA Regulatory Requirements — A Case Study on the Reliability and Traceability of Flow Cytometry Data"Thematic Live Broadcast, Welcome to Scan and Watch.
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Hengrui Medicine:
Type B siRNA Therapy
HRS-5635, submitted by Fujian Shengdi Pharmaceutical Co., Ltd., a wholly-owned subsidiary of Hengrui Medicine, is proposed to be included in the breakthrough therapy designation for chronic hepatitis B. This self-developed next-generation liver-targeted siRNA candidate drug has successfully advanced to clinical research for this indication.Ⅱ Period Stage.
Non-clinical efficacy studies have shown that HRS-5635 exhibits excellent antiviral activity against all HBV genotypes and can exert potent and long-lasting antiviral effects in vivo. Non-clinical safety evaluation studies indicate that HRS-5635 has no off-target activity and is highly safe.
Stellar Kunze:
HBV siRNA Combination Therapy
Stellar Kунzе HT-101 Injection Proposed for Inclusion in Breakthrough Therapy Designation, Along with HT-102(Hepatitis B Virus Surface Antigen Neutralizing Antibody)Combined use for the treatment of chronic hepatitis B virus infection.
HT-101 is an innovative GalNAc-conjugated siRNA drug that silences HBV mRNA with high specificity for liver targeting, blocking viral protein synthesis. Its monotherapy for chronic hepatitis B was granted Breakthrough Therapy designation in July this year, and the combination therapy regimen has now received another proposed recognition, with its clinical value being continuously acknowledged. In the extension phase of the Phase Ib trial, HBsAg levels remained significantly reduced at 48 weeks in subjects, with one participant in the 400 mg dose group showing response after two doses.Achieved HBsAg Clearance After 48 Weeks of Follow-up。
In addition, HF001 umbilical cord mesenchymal stem cell injection developed by Hengfeng Mingcheng has been approved for clinical trials, intended for the treatment of type 2 diabetes; SGB-3383 injection submitted by Shengyin Biotechnology is a complement factor B-targeting drug.（CFB）siRNA, intended for the development of treatments for complement-mediated blood diseases, marks the clinical approval of SGB-3383, expanding its focus from kidney diseases to blood disorders.
Antibody:
Bispecific/Trispecific Antibodies, TCE Breakthrough in "Multi-Target Synergy"
Zelgen Pharma:
MUC17/CD3/CD2 Trispecific TCE
Zelgen Pharma's Injectable ZGGS34 Approved for Clinical Trials, Intended for MUC17-Positive Advanced Solid Tumors(such as gastric cancer, pancreatic cancer, and colorectal cancer, etc.)Treatment. Public information shows that ZGGS34 is a trispecific T-cell engager antibody molecule.（TriTE）, targeting MUC17/CD3/CD28.
The CD28 agonist antibody was incorporated into the ZGGS34 molecule, and CD28 is the key second signal for T-cell activation, mainly expressed on the T-cell surface, playing a crucial role in T-cell activation, proliferation, and survival. CD28 interacts with antigen-presenting cells.（APC）After binding to CD80/CD86, it lowers the TCR activation threshold, enhances T cell proliferation, metabolism, and anti-apoptotic capabilities, thereby enabling ZGGS34 to have a stronger ability to activate T cells compared to typical BiTE molecules, as well as sustained T cell tumor-killing capability, enhancing anti-tumor immune effects.
Hongcheng Pharmaceutical:
PD-1/CTLA-4/VEGF Targeting Tri-specific Antibody
HC010 for Injection, independently developed by Hongcheng Pharmaceutical, is a freeze-dried injectable formulation targeting advanced solid tumors and directed against human PD-1, CTLA-4, and VEGF. Its design aims to block the interactions between PD-1 and its ligands PD-L1/L2, CTLA-4 and its ligands CD80/CD86, as well as VEGF and its receptor VEGFR2, simultaneously inhibiting two immune checkpoints and angiogenesis, thereby promoting anti-tumor immunity and vascular normalization.
In addition, the EMB-07 Injection from EpimAb Biotherapeutics(CD3/ROR1 Targeting TCE), Recombinant Humanized Anti-HER2 Bispecific Antibody Injection jointly filed by Jinmant Bio and Alphamab Oncology, BBT002 Injection from Shanzhu Radian Bio(IL-4Rα/IL-5 Targeting Antibody)Both have received clinical tacit approval from the CDE, with proposed indications for: treatment of relapsed/refractory or newly diagnosed aggressive B-cell non-Hodgkin lymphoma, HER2-positive gastric or gastroesophageal junction adenocarcinoma, and moderate to severe chronic obstructive pulmonary disease.
ADC：
"IO+ADC" Becomes Mainstream, Dual Targets Overcome Drug Resistance
Lepu Biotech:
EGFR ADC
Lepu Biotech EGFR ADC Candidate Drug MRG003(Injection of Vericobtumab)Proposed for inclusion in the breakthrough therapy category, in combination with HX008（PD-1 Inhibitor,Putelizumab)Injection for the treatment of recurrent or metastatic nasopharyngeal carcinoma that has failed previous treatment with at least platinum and PD-1/L1, with the phase III clinical trial already initiated.
The marketing application for MRG003 was submitted in China this past March and has been granted priority review for monotherapy in patients with recurrent or metastatic nasopharyngeal carcinoma who have failed at least two prior lines of systemic chemotherapy and PD-1/PD-L1 inhibitor treatment. Meanwhile, Putelimab has already been approved for marketing in China for the treatment of high microsatellite instability.（MSI-H）Or Mismatch Repair Deficiency Type（dMMR）Advanced Solid Tumors, Melanoma.
The phase 2 clinical data published in December 2024 showed that the combination of Vebecotamab and Putlimab for the treatment of recurrent or metastatic nasopharyngeal carcinoma had an ORR of66.7%`, DCR is`93.3%; PFS and DoR are not yet mature, with a 6-month PFS rate of 76.2% and a 6-month DoR rate of 83.3%.
Akeso Biopharma:
HER3 ADC
AK138D1 Injection from Akeso Biopharma Receives Clinical Trial Approval from CDE for the Treatment of Advanced Malignant Tumors. As Akeso's first ADC new drug to enter clinical trials, its Phase 1 clinical trial in Australia completed the enrollment of the first subject in February this year.
AK138D1 The antibody portion is the fully humanized anti-HER3 IgG1 antibody Patritumab, linked via a cleavable linker MC-AAA.(Maleimide-Alanine-Alanine-Alanine), conjugated with topoisomerase I inhibitor DXd(Potent Cytotoxic Drugs)。
The advancement of AK138D1 represents a significant implementation of Akeso Biopharma's "IO 2.0+ADC" strategy. Previously, Akeso Biopharma has successfully launched two globally pioneering bispecific antibodies — the PD-1/CTLA-4 bispecific antibody Cadonilimab and the PD-1/VEGF bispecific antibody Evecima, in the IO field.(Tumor Immunology)Build a leading advantage in the field, laying the groundwork for the combined application of ADC and immunotherapy.
In addition, dual-target ADCs represent an important evolutionary direction for the next generation of ADCs, helping to address issues of target heterogeneity and drug resistance. Among them, Baili Tianheng's EGFR/HER3 ADC has been designated as a breakthrough therapy, Hengrui Medicine/Fuhong Biotech’s B7H3/EGFR ADC has received clinical trial approval from the CDE, and Orange Sail Pharmaceutical’s Trop2/Nectin4 ADC clinical trial application has been accepted.
For more IND progress of new drugs, please refer to the table above.
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