Areas of Focus

Blinding retinal diseases, including age-related macular degeneration (AMD), diabetic macular edema (DME), and others, represent the leading causes of irreversible vision impairment worldwide, severely threatening the visual health and quality of life of hundreds of millions of patients. Existing clinical interventions still face significant limitations. As global population aging accelerates and the prevalence of metabolic diseases continues to rise, the disease burden of these conditions is steadily increasing. Novel drug development carries profound societal value, unmet clinical demand, and strategic industrial significance.

Taking neovascular (wet) AMD as an example, patients require frequent intravitreal injections, resulting in poor treatment adherence; a considerable proportion of patients exhibit suboptimal response to existing drugs or develop secondary resistance as treatment progresses. To address these clinical needs, our overarching research goal is to develop and translate next-generation therapeutics based on novel targets and innovative technologies, achieving drugs with reduced injection frequency, improved adherence, and coverage of refractory patient populations.

Specifically, our research focuses on two mutually reinforcing, synergistically advancing dimensions:

Dimension One: Multimodal Drug Engineering Innovation and Delivery

We conduct engineering modification and long-acting, targeted delivery research on protein drugs, nucleic acid drugs, and small-molecule drugs. Our central objective is to substantially prolong the effective intraocular residence time of therapeutics, optimize target tissue biodistribution, and reduce dosing frequency and invasiveness, thereby improving patient adherence.

Dimension Two: De Novo Target Discovery and Mechanism-Driven Drug Design

Simultaneously, through in-depth dissection of the pathological mechanisms underlying retinal diseases, we are dedicated to identifying novel biological targets and intervention nodes, laying the scientific groundwork for the development of first-in-class innovative drugs. Currently, we are investigating the molecular mechanisms of anti-VEGF treatment resistance in wet AMD, as well as retinal fibrosis pathological mechanisms, to excavate and validate entirely novel targets. Furthermore, we are exploring strategies to promote retinal pigment epithelium (RPE) cell functional repair and neuroprotection, establishing the scientific foundation for the future evolution of retinal disease therapeutics from "delaying progression" toward "functional enhancement."

As the core ophthalmic drug development team of the Beijing Visual Science and Translational Eye Research Institute (BERI), our laboratory has established end-to-end research capabilities spanning target validation to candidate drug screening, and formulation optimization to pharmacodynamic evaluation. We have established deep collaborations with multiple domestic and international ophthalmic pharmaceutical companies, relevant research institutes, and clinical research centers, jointly advancing globally competitive, original retinal disease therapeutics from the laboratory to the clinic, and from proof-of-concept to tangible patient benefit.

致盲性眼底疾病（包括年龄相关性黄斑变性、糖尿病性黄斑水肿等）是全球范围内导致不可逆视力损害的首要病因，严重威胁着数亿患者的视觉健康与生活质量，现有临床干预手段仍面临显著局限。随着全球人口老龄化进程加速及代谢性疾病患病率攀升，该类疾病的疾病负担持续加重，新药研发具有重大的社会价值、临床需求与产业战略意义。

以湿性年龄相关性黄斑变性为例，患者需频繁接受眼内注射，治疗依从性差；相当比例的患者对现有药物应答不佳，或随治疗进展产生继发性耐药。针对这些临床需求，我们的总体研究目标是基于新靶点新技术的研发和转化，获得注射频次更低、依从性更好、覆盖难治性患者群体的下一代药物。

具体而言，我们的研究聚焦以下两个相互支撑、协同推进的维度：

维度一：多模态药物工程化创新与递送

我们开展蛋白药物、核酸药物及小分子药物的工程化改造与长效、靶向递送研究，核心目标是显著延长药物在眼内的有效滞留时间、优化靶组织生物分布、降低给药频次与侵入性，从而改善患者的依从性。

维度二：原创靶点发现与机制驱动的药物设计

我们同时通过深入解析眼底疾病的病理机制，致力于发现新的生物学靶点和干预节点，为first-in-class创新药物的开发做好科学准备。目前，我们正在研究湿性AMD抗VEGF治疗耐药的分子机制，视网膜纤维化病理机制，挖掘并验证全新靶点。此外，我们也在探索促进视网膜RPE细胞功能修复、神经保护等策略，为眼底疾病治疗未来从"延缓进展"迈向"功能提升"打下科学基础。

作为北京市视觉科学与转化医学研究中心（Beijing Visual Science and Translational Eye Research Institute，BERI）的眼科药物研发核心团队，本实验室建立了从靶点验证到候选药物筛选、从制剂优化到药效评价的全链条研究能力；并与国内外多家眼科制药企业、相关科研院所、临床研究中心建立了深度合作，共同推动具有全球竞争力的原创性眼底疾病新药从实验室走向临床、从概念验证走向患者获益。