Automated System for High Yield Pancreatic Islet Isolation

项目摘要/摘要 该二期项目旨在开发一种自动化的高产量胰岛分离系统，用于 用于胰岛移植(一种治疗1型糖尿病的新兴疗法)和糖尿病研究。这个 胰岛移植的效果关键取决于移植胰岛的质量和数量， 它们是从已故的人类供体胰腺中分离出来的。然而，目前用于胰岛的方法 隔离高度依赖于用户，提供的胰岛数量很少，并且需要近50%的处理 失败率。这些缺点需要频繁地使用许多供体胰腺和多个 每名患者进行移植以获得良好的结果，使得胰岛移植的效率非常低，成本也很高。 令人望而却步。缺乏可靠和易用的胰岛隔离工具也阻碍了许多研究人员的工作 为他们的研究获得高质量的小岛。为了克服这些限制，CG Science正在 开发正在申请专利的“自动细胞分离平台”(ACIP)技术，以创建 自动胰岛隔离系统。该技术具有独特的配置，以(1)实现高效 酶消化，(2)以封闭系统的形式自动化分离过程，以及(3)突破 限制了目前的方法实现高胰岛回收率和生存能力。使用猪 胰腺作为第一阶段的模型系统，该公司已经展示了成功的胰岛分离， 与当前方法相比，胰岛回收率提高了约2.4倍，无与伦比的胰岛存活率达到了~95% 平均水平。胰岛表现出突出的形态和葡萄糖刺激的胰岛素分泌功能。 自动化系统可能会消除与用户有关的流程故障，并提供单一捐赠者胰岛 移植非常有效-只使用一个供体胰腺和每个受者一次移植- 高达80%的成本降低。在第二阶段，研究团队将(1)开发出功能齐全的产品 原型，(2)优化人和猪胰岛的自动原型的性能， (3)基准优于当前方法的胰岛隔离能力；(4)演示 用小鼠模型研究了由此产生的胰岛逆转糖尿病的临床前疗效。这是第二阶段 项目将为(1)过渡到制造，(2)获得 监管批准，以及(3)产品商业化。调查组包括医疗设备 专门从事临床胰岛制造和治疗的专家、胰岛生物学家、工程师和医生 移植。该项目的成功将导致一种基本工具的商业化，该工具 不仅使单一供者胰岛移植变得高效、负担得起和可用，而且 加速广泛的糖尿病研究-从而使世界各地的许多患者受益 患有糖尿病的人。

Project Summary/Abstract This Phase II project aims to develop an automated system for high-yield pancreatic islet isolation for use in islet transplantation (an emerging therapy for type 1 diabetes) and diabetes research. The effectiveness of islet transplantation relies critically on the quality and quantity of the transplanted islets, which are isolated from deceased human donor pancreata. However, the current method for islet isolation is highly user-dependent, provides low numbers of islets, and suffers close to 50% process failure rates. These drawbacks necessitate the frequent use of many donor pancreata and multiple transplants per patient to achieve good outcomes, making islet transplantation highly inefficient and cost- prohibitive. The lack of a reliable and easy-to-use tool for islet isolation also precludes many researchers from obtaining high-quality islets for their research. To overcome these limitations, CG Scientific is developing a patent-pending “automated cell isolation platform” (ACIP) technology to create an automated system for islet isolation. The technology has a unique configuration to (1) enable efficient enzyme digestion, (2) automate the isolation process in a closed-system format, and (3) break through limitations that prevent current methods from achieving high islet recovery and viability. Using porcine pancreata as a model system in Phase I, the company has demonstrated successful islet isolation, with ~2.4x higher islet recovery compared to the current method and an unmatched islet viability of ~95% on average. The islets exhibit outstanding morphology and glucose-stimulated insulin secretion function. The automated system will potentially eliminate user-related process failure and render single-donor islet transplantation highly efficacious―using only one donor pancreas and one transplant per recipient―with up to 80% cost reduction. In Phase II, the investigator team will (1) develop a fully functional product prototype, (2) optimize the performance of the automated prototype for both human and porcine islets, (3) benchmark superior islet isolation capabilities compared to the current method, and (4) demonstrate the pre-clinical efficacy of the resulting islets for diabetes reversal using a mouse model. This Phase II project will generate significant data and know-how for (1) transitioning to manufacturing, (2) obtaining regulatory approval, and (3) product commercialization. The investigator team includes medical device experts, islet biologists, engineers, and medical doctors who specialize in clinical islet manufacturing and transplantation. The success of this project will result in the commercialization of an essential tool that will not only make single-donor islet transplantation highly effective, affordable, and available, but also accelerate a wide range of diabetes research―thereby benefiting the many patients around the world who suffer from diabetes.