Gene-Therapy Clinical-Trial Candidate Screening for Ophthalmology enabled by Low-Cost Widefield Fundus Camera

工程生物学的一个非常令人兴奋的应用是细胞和基因治疗的最新发展和进步。在这里，我们提出了一种颠覆性的监测设备，以显着减少和简化临床试验患者的选择过程中，基因治疗针对眼科应用。基于基因治疗的眼科治疗提供了显着的承诺，预防和逆转失明的一系列无法治疗的疾病。这包括更常见的疾病，如干眼症相关的黄斑疾病和糖尿病视网膜病变（发达国家最常见的老年和工作年龄失明原因），以罕见的疾病，如无脉络膜。实现这些基因治疗的一个关键挑战是确定合适的临床试验患者，以收集足够的数据来批准其使用。这是我们的工程生物学项目要克服的挑战，筛选合适的临床试验患者是一个主要的挑战。即使在资源丰富的发达国家，医疗保健系统中训练有素的眼科专业知识的带宽通常也不足，无法进行可靠的眼部护理筛查。这个问题在临床试验患者的选择中更加严重;选择需要在适当的时间窗内识别合适的候选人，然后持续监测他们的进展。这些方法存在显著的局限性，剑桥视觉技术公司将与本项目合作。我们正在开发一种低成本的成像设备，不需要训练有素的医疗专业人员的专业知识。实际上，我们正在提出一种解决方案，以自动化并大幅降低基因治疗临床试验候选人选择过程的经济成本。这将提供一个关键的基础设施，并在开发用于眼科应用的大量基因疗法方面具有变革性。

An extremely exciting application of engineering biology is the recent development and advancement of cellular and gene therapy treatments. Here, we are proposing a disruptive monitoring device to significantly reduce and streamline the selection process for clinical trial patients in gene-therapy treatments targeting Ophthalmological applications.Gene-therapy based Ophthalmic treatments offer significant promise for preventing and reversing blindness in a range of untreatable disorders. This encompasses more common disorders such as Dry Age-Related Macular Disorder and Diabetic Retinopathy (most common old-age and working age causes of blindness in the developed world) to rarer diseases such as Choroideremia. A key challenge to the realisation of these gene-therapy treatments is identifying suitable clinical-trial patients to collect sufficient data to approve their use. This is the engineering biology challenge our project shall overcome.Screening for suitable clinical-trial patients is a major challenge. Even in resource-rich developed countries, there is typically insufficient bandwidth of trained Ophthalmic expertise in the healthcare system to enable reliable screening of eye-care. This issue is exacerbated for the selection of clinical-trial patients; selection requires identifying suitable candidates in appropriate time-window and then continuously monitoring their progression.These approaches present significant limitations which Cambridge Vision Technology shall obviate with this project. We are developing a low-cost imaging device which does not require the expertise of a trained medical professional. Effectively, we are proposing a solution to automate and dramatically reduce the economic cost of the clinical-trial candidate selection process for gene-therapies. This would provide a key piece of infrastructure and be transformative in enabling the development of a host of gene-therapies for Ophthalmological applications.