Since the groundbreaking approval of the first anti-VEGF therapy in 2004, the retinal therapeutics field has undergone a remarkable transformation, witnessing a surge in novel, disease-modifying therapeutics for a broad spectrum of retinal diseases, extending beyond exudative VEGF-driven conditions. The surge in scientific advancement and the pressing, unmet, medical need have captured the attention of venture capital investors, who have collectively invested close to $10 billion in research and development of new retinal therapeutics between 2004 and 2023. Notably, the field of exudative diseases has gradually shifted away from trying to outcompete anti-VEGF therapeutics towards lowering the overall treatment burden by reducing injection frequency. Simultaneously, a new era has emerged in the non-exudative field, targeting prevalent conditions like dry AMD and rare indications such as Retinitis pigmentosa. This has led to promising drug candidates in development, culminating in the landmark approval of Luxturna for a rare form of Retinitis pigmentosa. The validation of new mechanisms, such as the complement pathway in dry AMD has paved the way for the approvals of Syvovre (Apellis) and Izervay (Iveric/Astellas), marking the first two therapies for this condition. In this comprehensive review, we share our view on the cumulative lessons from the past two decades in developing retinal therapeutics, covering both positive achievements and challenges. We also contextualize the investments, strategic partnering deals, and acquisitions of biotech companies, pharmaceutical companies venture capital investors in retinal therapeutics, respectively. Finally, we provide an outlook and potentially a forward-looking roadmap on novel retinal therapeutics, highlighting the emergence of potential new intervention strategies, such as cell-based therapies, gene editing, and combination therapies. We conclude that upcoming developments have the potential to further stimulate venture capital investments, which ultimately could facilitate the development and delivery of new therapies to patients in need.

自 2004 年第一个抗 VEGF 疗法获得突破性批准以来，视网膜治疗领域经历了显着的转变，见证了针对广泛视网膜疾病的新型疾病缓解疗法的激增，范围超出了渗出性 VEGF 驱动的疾病。科学进步的激增和紧迫的、未满足的医疗需求引起了风险投资投资者的注意，他们在 2004 年至 2023 年间总共投资了近 100 亿美元用于新视网膜疗法的研发。值得注意的是，渗出性疾病领域已逐渐从试图超越抗 VEGF 疗法转向通过减少注射频率来降低​​总体治疗负担。与此同时，非渗出性领域出现了一个新时代，针对干性 AMD 等常见病症和色素性视网膜炎等罕见病症。这导致了有前景的候选药物的开发，最终 Luxturna 获得里程碑式的批准，用于治疗一种罕见的色素性视网膜炎。新机制的验证，例如干性 AMD 中的补体途径，为 Syvovre (Apellis) 和 Izervay (Iveric/Astellas) 的批准铺平了道路，这标志着该疾病的前两种疗法。在这篇全面的综述中，我们分享了我们对过去二十年发展视网膜治疗方法中积累的经验教训的看法，涵盖了积极的成就和挑战。我们还分别介绍了生物技术公司、制药公司和视网膜治疗领域风险投资人的投资、战略合作交易和收购。 最后，我们提供了新型视网膜疗法的展望和潜在的前瞻性路线图，强调了潜在的新干预策略的出现，例如基于细胞的疗法、基因编辑和联合疗法。我们的结论是，即将到来的发展有可能进一步刺激风险资本投资，最终可以促进新疗法的开发和向有需要的患者提供。