Development of a therapeutic antibody for a novel angiogenic target

在几种常见的眼部疾病中，包括年龄相关性黄斑疾病和糖尿病视网膜病变，眼睛感光部分的微小血管网络可能会发生重大结构变化，并以严重到足以导致视力丧失的方式不受控制地生长。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使不到50%的患者受益，并且通常仅延迟视力丧失。因此，迫切需要开发新的治疗方法，特别是在疾病的早期阶段可能有用的类型。我们的研究小组发现了一种新的蛋白质，其功能以前未知，我们已经证明异常的视网膜血管会产生高水平的蛋白质，并导致血管细胞增殖。在这个项目中，我们将产生一种抗体，它将阻止这种蛋白质的功能，然后创造出安全有效的抗体衍生物用于人类。在项目结束时，如果我们实现了我们的目标，我们将准备在I期临床试验中将我们的新疗法用于患者。

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.