Development of a therapeutic antibody for a novel angiogenic target

在几种常见的眼部疾病中，包括与年龄相关的黄斑疾病和糖尿病视网膜病变，眼睛对光敏感部分的微小血管网络可能会经历重大的结构变化，并以无法控制的方式生长，严重到足以导致视力丧失。虽然在治疗这些疾病方面取得了一些进展，但最有效的治疗方法通常只有不到50%的患者受益，而且往往只会延缓视力丧失。因此，迫切需要开发新的治疗方法，特别是一种在疾病早期可能有用的治疗方法。我们的团队发现了一种以前未知功能的新蛋白质，我们已经证明，这种蛋白质可以由异常的视网膜血管产生高水平，并导致血管细胞增殖。在这个项目中，我们将产生一种抗体，它将阻断这种蛋白质的功能，然后创造出对人类安全有效的抗体的衍生物。在项目结束时，如果我们实现了我们的目标，我们将准备将我们的新疗法应用于I期临床试验的患者。

In several common diseases of the eye, including age-related macular disease and diabetic retinopathy, the network of tiny blood vessels in the light-sensitive part of the eye may undergo major structural changes and grow uncontrollably in a manner severe enough to cause loss of vision. Although some advances have been made in the treatment of these conditions the most effective therapies generally benefit fewer than 50% of patients, and often only delay vision loss. There is therefore an urgent need to develop new therapies, especially of a type that may be useful during the early stages of disease. Our group has discovered a new protein, of previously unknown function, that we have shown to be produced at high levels by abnormal retinal blood vessels and to cause blood vessel cell proliferation. In this project we will generate an antibody that will block the function of this protein, and then create derivatives of the antibody that are safe and effective for use in humans. At the end of the project, if we achieve our objectives, we will be ready to take our new therapy into patients in a Phase I clinical trial.