Turning cancer's genetic switches off关闭癌症的基因开关
G4 Therapeutics is developing QN-302, a small molecule designed to target G-quadruplex DNA structures linked to cancer-driving genes, with pancreatic cancer as the first lead clinical focus
基世生物医药正在开发 QN-302，一种旨在靶向与癌症驱动基因相关的 G四联体 DNA 结构的小分子药物，首要临床方向为胰腺癌
To treat pancreatic cancer without the devastating side effects of existing treatments - by targeting the DNA structures that drive the disease itself
在不带来现有疗法那种严重副作用的前提下治疗胰腺癌——通过靶向驱动疾病本身的 DNA 结构
A structurally distinct approach to cancer一种结构上与众不同的抗癌路径
Most cancer drugs attack dividing cells, immune checkpoints or DNA repair. QN-302 works further upstream — it binds and stabilises G-quadruplex DNA in the control regions of cancer-driving genes, aiming to turn their abnormal "on" signals down rather than poisoning the cell. A different mechanism, with no overlap with chemotherapy, PD-1 or PARP inhibition.
多数抗癌药物针对分裂细胞、免疫检查点或 DNA 修复。QN-302 的作用更偏上游——它结合并稳定位于癌症驱动基因调控区的 G四联体 DNA，目标是调低其异常的"开启"信号，而非毒杀细胞。这是一种不同的机制，与化疗、PD-1 或 PARP 抑制均无重叠。
Explore the mechanism了解作用机制Focused on one of medicine's hardest cancers专注于医学上最棘手的癌症之一
Pancreatic cancer (PDAC) has among the poorest five-year survival of any major solid tumour. Its dense, immunosuppressive stroma blunts chemotherapy and checkpoint drugs alike. By targeting the transcriptional programmes that keep these tumours growing, QN-302 aims for a cleaner, more selective effect.
胰腺癌（PDAC）的五年生存率位列所有主要实体瘤之中最低之列。其致密且免疫抑制的基质环境同时削弱了化疗和检查点药物的疗效。通过靶向维持肿瘤持续生长的转录程序，QN-302 力求实现更干净、更具选择性的作用。
Read the science阅读科学依据G-Quadruplexes were a structural curiosity for thirty years. QN-302 is our attempt to make them matter, to switch off the genes that drive the cancers we have failed to treat.
三十年来，G四联体一直被视为一种结构上的奇观。QN-302 是我们让它真正发挥作用的尝试——关闭那些驱动我们至今难以治愈之癌症的基因。
Building the team to take G4 Therapeutics forward组建推进 G4 生物医药的团队
Clinical Leadership临床领导
G4 Therapeutics will shortly be recruiting high-quality clinical experts to lead future clinical trials of QN-302. We are seeking oncologists and clinical investigators with experience in early-phase trials and a commitment to bringing novel mechanisms to patients.
基世生物医药即将招募高水平临床专家，领导 QN-302 未来的临床试验。我们正在寻找具备早期临床试验经验、并致力于将全新机制带给患者的肿瘤学家与临床研究者。
Scientific Advisory Board科学顾问委员会
G4 Therapeutics is establishing an International Scientific Advisory Board, bringing together world-leading experts in G-quadruplex biology, oncology drug development, and clinical trial design to guide the development of QN-302.
基世生物医药正在组建国际科学顾问委员会，汇聚 G四联体生物学、肿瘤药物开发与临床试验设计领域的世界级专家，指导 QN-302 的开发。
If you are interested in either opportunity, please contact us at 如您对上述任一机会感兴趣，请联系 info@g4therapeutics.com
What is a G-quadruplex?什么是 G-四联体？
The familiar DNA double helix is like a twisted ladder. A G-quadruplex is different: short guanine-rich sequence tracts within duplex DNA can fold into higher-order four-stranded arrangements, held together around potassium ions. G-quadruplexes are a structurally distinct and highly specific target class, whose detailed structures vary with the precise sequences between the guanine tracts.
普通 DNA 双螺旋像一把扭转的梯子。G四联体不同：富含鸟嘌呤的 DNA 可折叠成一层层方形平台，并围绕钾离子稳定。可以把它理解成"分子结节"和"基因控制面板"的结合体。
Duplex DNA
双链 DNA
G-quadruplex DNA
G-四联体 DNA
G4 DNA is a structural target located upstream of cancer gene expressionG4 DNA 是位于癌症基因表达上游的结构靶点
G-quadruplexes form when guanine bases assemble into flat G-tetrads. Several tetrads stack together, often stabilised by potassium ions. These structures can form in promoters, telomeres and other regulatory genomic regions, which makes them attractive but technically difficult drug targets.
G四联体由鸟嘌呤碱基形成平面 G-tetrad 后进一步堆叠而成，通常由钾离子稳定。它们可出现在启动子、端粒和其他基因调控区域，因此既有吸引力，也具有技术挑战。
Two faces of the same control switch同一控制开关的两面
On the complementary strand, cytosine-rich sequences can fold into i-motif structures. Together, G-quadruplex and i-motif structures form part of the regulatory machinery our research programme studies in cancer-relevant genes such as S100P.
在互补链上，富含胞嘧啶的序列可折叠成 i-motif 结构。G四联体与 i-motif 共同构成调控机制的一部分，也是我们研究项目在 S100P 等癌症相关基因中重点研究的对象。
Jamming a stuck accelerator固定卡住的油门
QN-302 is designed to bind and stabilise selected G4 structures. In simple terms: if a cancer gene behaves like a stuck accelerator pedal, QN-302 aims to jam the control structure so the signal drops. The goal is transcriptional suppression of pathways involved in tumour growth, invasion and survival.
QN-302 旨在结合并稳定特定 G4 结构。简单说：如果癌症基因像卡住的油门，QN-302 的目标就是固定其控制结构，让信号降低，从而抑制与肿瘤生长、侵袭和存活相关的转录程序。
Bind
结合
Small molecule binds to G4 DNA surfaces and stabilises the folded structure
小分子结合 G4 DNA 表面并稳定折叠结构
Modulate
调控
Stabilisation can interfere with transcriptional machinery around cancer-linked genes
稳定化可能干扰癌症相关基因附近的转录机器
Suppress
抑制
Preclinical strategy: reduce expression of genes involved in PDAC progression and metastasis
临床前策略：降低参与胰腺癌进展和转移的基因表达
Not just another PDAC drug不只是另一个胰腺癌药物
Most approved or commonly used PDAC treatments target cell division, immune checkpoints or DNA repair. G4 targeting is upstream and structural: it aims at transcriptional regulation itself. That makes it scientifically distinctive, but also means clinical validation has to be clean, biomarker-aware and properly executed.
多数已获批或常用的胰腺癌治疗主要针对细胞分裂、免疫检查点或 DNA 修复。G4 靶向更偏上游和结构性，目标是转录调控本身。这使其在科学上具有差异化，但也要求临床验证清晰、生物标志物设计合理、执行严谨。
Chemotherapy
化疗
Broadly kills dividing cells. Effective in some settings, but limited by toxicity and resistance
广泛杀伤分裂细胞，在部分场景有效，但受毒性和耐药限制
Immunotherapy
免疫治疗
PDAC's dense, immunosuppressed stroma has limited response for many checkpoint approaches
胰腺癌致密且免疫抑制的基质环境限制了许多检查点疗法反应
G4 targeting
G4 靶向
Targets gene-control DNA structures, potentially offering a new mechanistic class
靶向基因控制相关 DNA 结构，可能形成新的机制类别
Key characteristics关键特征
Patent term
专利期限
Composition-of-matter protection extending to 2040 globally
物质组成专利全球保护期延伸至 2040 年
Countries covered
覆盖国家
Patents granted across North America, Europe, Asia and Russia
专利覆盖北美、欧洲、亚洲及俄罗斯
Orphan status
孤儿药资格
FDA Orphan Drug Designation supports development in pancreatic cancer
FDA 孤儿药资格支持其在胰腺癌方向的开发
A disease that resists the standard playbook一种抵抗常规治疗策略的疾病
Pancreatic cancer remains world-wide one of the highest unmet-need solid tumours, with exceptionally poor 5-year survival rates. Surgery is possible for only a minority of patients, and chemotherapy and immunotherapy are blunted by the disease's dense, immunosuppressive stroma. New mechanisms are urgently needed.
胰腺癌在全球范围内仍是未满足需求最高的实体瘤之一，5 年生存率极低。仅有少数患者适合手术，而化疗与免疫治疗又因其致密、免疫抑制的基质环境而疗效受限。亟需全新的治疗机制。
Transcriptional driver
转录驱动
QN-302 down-regulates S100P and other genes known to play a role in the growth and metastasis of pancreatic cancer in in vivo models.
在体内模型中，QN-302 可下调 S100P 及其他已知参与胰腺癌生长与转移的基因。
Selective intent
选择性意图
By acting on gene-control structures rather than dividing cells broadly, the aim is a more selective effect with less of the toxicity of conventional chemotherapy.
通过作用于基因控制结构而非广泛地针对分裂细胞，目标是实现更具选择性、毒性低于传统化疗的效果。
Biomarker-informed
生物标志物导向
The China development plan is designed to be biomarker-aware, so that the right patients can be identified for a mechanism this distinct.
中国开发计划以生物标志物为导向，以便为这一独特机制识别合适的患者。
Treatment without the devastation治疗，而非摧残
Our ambition is a therapy that meaningfully slows pancreatic cancer while sparing patients the worst toxicities of existing regimens — a cleaner mechanism, properly validated, brought to patients through an accelerated China development pathway.
我们的目标是一种能切实延缓胰腺癌进展、同时让患者免受现有方案最严重毒性的疗法——一种更干净的机制，经过严谨验证，并通过中国加速开发路径带给患者。
Psalmon G, Pipier A, Barbotte M, Hudson RHE, Neidle S, Monchaud D. DNA damaging properties of G-quadruplex ligand QN-302 are potentiated by the DNA repair inhibitor Olaparib and mitigated by the molecular helicase PhpC. Genome Biology. 2026 Apr 11;27(1):168. doi:10.1186/s13059-026-04035-9.
Bidzinska J, Di Pietro L, Naghshineh E, Pandini C, Doria F, Zaffaroni N, Gandellini P, Neidle S, Folini M. Cellular adaptations impact the biological activity of naphthalene diimide G-quadruplex ligands in ALT-positive osteosarcoma cells. Cell Death and Disease. 2025 Aug 1;16(1):581. doi:10.1038/s41419-025-07908-2.
Tosoni B, Naghshineh E, Zanin I, Gallina I, Di Pietro L, Cleris L, Nadai M, Lecchi M, Verderio P, Pratesi P, Pasquali S, Zaffaroni N, Neidle S, Folini M, Richter SN. The G-quadruplex experimental drug QN-302 impairs liposarcoma cell growth by inhibiting MDM2 expression and restoring p53 levels. Nucleic Acids Research. 2025 Feb 8;53(4). doi:10.1093/nar/gkaf085.
Neidle S. A Phenotypic Approach to the Discovery of Potent G-Quadruplex Targeted Drugs. Molecules. 2024 Aug 1;29(15):3653. doi:10.3390/molecules29153653.
Arshad T, Neidle S, Chandana S, Borazanci E. Early clinical experience with a novel G-quadruplex experimental anti-cancer drug. Cancer Research. 2024;84:CT105. doi:10.1158/1538-7445.AM2024-CT105.
Ahmed AA, Chen S, Roman-Escorza M, Angell R, Oxenford S, McConville M, Barton N, Sunose M, Neidle D, Haider S, Arshad T, Neidle S. Structure-activity relationships for the G-quadruplex-targeting experimental drug QN-302 and two analogues probed with comparative transcriptome profiling and molecular modeling. Scientific Reports. 2024 Feb 11;14(1):3447. doi:10.1038/s41598-024-54080-2.
Neidle S. Challenges in developing small-molecule quadruplex therapeutics. Annual Reports in Medicinal Chemistry. 2024;62:285–314.
Alexandrou E, Guneri D, Neidle S, Waller ZAE. QN-302 demonstrates opposing effects between i-motif and G-quadruplex DNA structures in the promoter of the S100P gene. Organic and Biomolecular Chemistry. 2023 Dec 20;22(1):55–58. doi:10.1039/d3ob01464a.
Ahmed AA, Greenhalf W, Palmer DH, Williams N, Worthington J, Arshad T, Haider S, Alexandrou E, Guneri D, Waller ZAE, Neidle S. The Potent G-Quadruplex-Binding Compound QN-302 Downregulates S100P Gene Expression in Cells and in an In Vivo Model of Pancreatic Cancer. Molecules. 2023 Mar 7;28(6):2452. doi:10.3390/molecules28062452.
Ahmed AA, Angell R, Oxenford S, Worthington J, Williams N, Barton N, Fowler TG, O'Flynn DE, Sunose M, McConville M, Vo T, Wilson WD, Karim SA, Morton JP, Neidle S. Asymmetrically Substituted Quadruplex-Binding Naphthalene Diimide Showing Potent Activity in Pancreatic Cancer Models. ACS Medicinal Chemistry Letters. 2020 Jul 16;11(8):1634–1644. doi:10.1021/acsmedchemlett.0c00317.
Vo T, Oxenford S, Angell R, Marchetti C, Ohnmacht SA, Wilson WD, Neidle S. Substituted Naphthalenediimide Compounds Bind Selectively to Two Human Quadruplex Structures with Parallel Topology. ACS Medicinal Chemistry Letters. 2020 Mar 30;11(5):991–999. doi:10.1021/acsmedchemlett.0c00041.
One lead asset, with room to grow一个核心资产，并保留拓展空间
| Asset | Mechanism | Indication | Stage | Status |
|---|---|---|---|---|
| QN-302 | G4 stabiliser / transcriptional modulator | PDAC | Phase I / China IND strategy | Active |
| QN-302 expansion | G4 stabilisation | Other solid tumours | Exploratory / preclinical rationale | Future |
| Backup G4 series | NDI analogue class | Oncology | Research / IP protected | Future |
| Non-core indications | To be determined | Non-oncology | Not prioritised | Non-active |
Countries covered
覆盖国家数
Patents granted across North America, Europe, Asia and Russia
专利覆盖北美、欧洲、亚洲及俄罗斯
Patent term
专利期限
Composition of matter protection extending to 2040 globally
物质组成专利全球保护期延伸至2040年
Strongest IP class
最强知识产权类别
Composition of matter is the broadest and most defensible form of drug patent protection
物质组成专利是最广泛、最具防御性的药物专利保护形式
A drug for pancreatic cancer, with less of the harm一种针对胰腺癌、且伤害更小的药物
G4 Therapeutics is based in Shanghai and is focused on developing G-quadruplex-targeting oncology therapeutics. We are searching for a drug that treats pancreatic cancer (PDAC) with fewer of the toxic effects of existing chemotherapy, by acting on the DNA structures that drive the disease.
The company's research origin traces back to UCL School of Pharmacy, where Prof. Stephen Neidle's group pioneered G4-targeted drug discovery. We are using China as an accelerated development pathway — combining UCL science with China's clinical and regulatory environment to bring QN-302 to patients faster.
基世生物医药位于上海，专注于开发 G四联体靶向肿瘤治疗药物。我们致力于寻找一种通过作用于驱动疾病的 DNA 结构、且毒副作用低于现有化疗的胰腺癌（PDAC）药物。
公司的科研源头可追溯至伦敦大学学院（UCL）药学院，Stephen Neidle 教授团队长期引领 G4 靶向药物发现。我们以中国作为加速开发路径——将 UCL 的科学与中国的临床及监管环境相结合，更快地将 QN-302 带给患者。
Company
公司
G4 Therapeutics (Shanghai) Co., Ltd.
基世生物医药（上海）有限公司
Address
地址
2/F, Area A, 420 Fenglin Road, Xuhui District, Shanghai, PRC
上海市徐汇区凤林路420号A区2楼
Collaboration
合作
We welcome investors, collaborators, co-development partners and licensing discussions
欢迎投资人、合作方、共同开发伙伴以及授权合作洽谈。
2022
Discovery foundation
发现基础
QN-302 emerged from long-running G-quadruplex research led by Prof. Stephen Neidle's team at UCL School of Pharmacy, building on a naphthalene diimide series with potent, selective G4-binding activity.
QN-302 源自 Stephen Neidle 教授团队在 UCL 药学院长期开展的 G四联体研究，基于一类具有强效、选择性 G4 结合活性的萘二酰亚胺系列化合物。
FDA orphan designation
FDA 孤儿药资格
Orphan Drug Designation was granted for pancreatic cancer, recognising both the high unmet need and the distinct mechanism of the compound.
获得胰腺癌方向的 FDA 孤儿药资格认定，既体现了该领域的高度未满足需求，也认可了该化合物的独特机制。
Pilot Phase I evaluation
试点一期临床评价
A U.S. dose-escalation study generated initial clinical safety and activity observations, providing the foundation for the next stage of development.
美国剂量递增研究产生了早期安全性和活性观察结果，为下一阶段开发奠定基础。
China development plan
中国开发计划
G4 Therapeutics aims to secure rights, complete technology transfer and execute a biomarker-informed China programme — using China's clinical and regulatory environment as an accelerated pathway to patients.
基世生物医药计划获取权益、完成技术转移并执行生物标志物指导的中国临床项目——以中国的临床与监管环境作为通往患者的加速路径。
Dr. Robert Yuan
袁一弘 博士
Co-founder · China Development Lead创始人 · 中国开发负责人PhD from UCL School of Pharmacy, with a focus on oncology and nucleic-acid chemistry. Leads G4 Therapeutics' China operations across clinical, regulatory and strategic development, and is responsible for the company's accelerated China development pathway.
伦敦大学学院药学院博士，研究方向聚焦肿瘤学与核酸化学。负责基世生物医药在中国的临床、监管与战略开发运营，主导公司的中国加速开发路径。
Prof. Stephen Neidle
斯蒂芬·尼德尔 教授
Co-founder · Scientific Founder & Inventor创始人、科学创始人及药物发明人Emeritus Professor at UCL and a leading pioneer in G-quadruplex therapeutics and nucleic-acid-targeted drug discovery. Inventor of QN-302 and author of much of the foundational research behind the programme.
伦敦大学学院荣休教授，G四联体治疗与核酸靶向药物发现领域的重要开拓者。QN-302 发明人，也是该研发项目背后大量奠基性研究的作者。
G4 Therapeutics is establishing an International Scientific Advisory Board, bringing together world-leading experts in G-quadruplex biology, oncology drug development and clinical trial design to guide the development of QN-302. To discuss involvement, contact info@g4therapeutics.com.
基世生物医药正在组建国际科学顾问委员会，汇聚 G四联体生物学、肿瘤药物开发与临床试验设计领域的世界级专家，指导 QN-302 的开发。如希望参与，请联系 info@g4therapeutics.com。
Get in touch找我们聊聊
We are actively seeking licensing partners, CRO collaborators, clinical partners and investors. Whether you are a pharmaceutical company, fund, hospital or academic institution, we welcome the conversation.
我们正在积极寻求授权合作伙伴、CRO 合作方、临床合作伙伴和投资人。无论您来自药企、基金、医院或学术机构，我们都欢迎进一步交流。
Xuhui District, Shanghai, PRC
Building the team to take QN-302 into the clinic.组建团队，推动 QN-302 迈向临床。
Clinical Leadership临床领导
G4 Therapeutics will shortly be recruiting high-quality clinical experts to lead the future clinical trials of QN-302. We are seeking oncologists and clinical investigators with early-phase trial experience and the ambition to bring a novel mechanism to patients with pancreatic cancer.
基世生物医药即将招募高水平临床专家，领导 QN-302 未来的临床试验。我们正在寻找具备早期临床试验经验、并有志于将同类首创机制带给胰腺癌患者的肿瘤学家与临床研究者。
International Scientific Advisory Board国际科学顾问委员会
We are also establishing an International Scientific Advisory Board, convening world-leading experts in G-quadruplex biology, oncology drug development and clinical trial design to guide the development of QN-302.
我们同时正在组建国际科学顾问委员会，汇聚 G四联体生物学、肿瘤药物开发与临床试验设计领域的世界级专家，指导 QN-302 的开发。
If either opportunity speaks to you, write to us at 如您对上述任一机会感兴趣，欢迎来信 info@g4therapeutics.com