SBIR Phase I: A Novel Extended Delivery Dual-action Platform for Peptide-based Anti-fibrotics

这个小企业创新研究 (SBIR) 第一阶段项目旨在开发一种新的药物输送微型支架，可以帮助数百万患有慢性肾脏、肝脏或肺部疾病的患者。这些疾病的特征是器官慢性炎症、器官僵硬或硬化以及器官功能逐渐衰退。所提出的微支架将两种作用截然不同的药物聚集在一个平台上。一种药物是阿司匹林，可以减轻炎症，另一种药物是松弛素，可以软化硬化组织并保持组织功能。不幸的是，松弛素不能口服，因此不用于这些适应症。这种植入皮下的微支架旨在在几天或几周内连续释放这些药物。该第一阶段计划旨在通过证明这种微支架释放阿司匹林和松弛素并且这两种药物保留其活性来证明这种概念在细胞研究中的可行性。该项目的更广泛影响/商业潜力是巨大的。这些慢性疾病目前尚无治愈方法。事实上，慢性肾病、肝硬化和特发性肺纤维化都需要器官移植。除了随之而来的发病率和死亡率之外，这些疾病还给这个国家带来了数十亿美元的医疗费用和工资损失。这种高度创新的技术对医疗保健的潜在影响异常广泛。缓释微支架将减少给药频率，并提高患者的依从性。事实上，与基于抗体的疗法类似，这种缓释支架可能只需要每月一次甚至更低的频率。通过封装从胰岛素到生长因子的各种高效药物，可以设计定制平台来治疗糖尿病、疤痕疙瘩和组织纤维化等多种适应症。

This Small Business Innovation Research (SBIR) Phase I project seeks to develop a new drug-delivery microscaffold that can help millions of patients suffering from chronic kidney, liver or lung disease. These diseases are characterized by chronic inflammation of the organ, stiffening or hardening of the organ and a gradual decline in organ function. The proposed microscaffold brings together on one single platform two drugs with very different actions. One drug is aspirin that reduces inflammation and the other drug is Relaxin which can soften hardened tissue and preserve tissue function. Unfortunately, Relaxin cannot be taken orally and is therefore not being used for these indications. This microscaffold, to be implanted under the skin, is designed to release these drugs continuously over several days or weeks. This Phase I program seeks to demonstrate the feasibility of such a concept in cell-based studies by demonstrating that this microscaffold releases both aspirin and Relaxin and that these two drugs retain their activities.The broader impact/commercial potential of this project is significant. These chronic diseases are currently without cure. In fact, chronic kidney disease, cirrhosis and idiopathic pulmonary fibrosis all necessitate organ transplant. In addition to their attendant morbidity and mortality, these diseases cost this country billions of dollars in health care expenses and lost wages. The potential healthcare impact of this highly innovative technology is exceptionally broad. Sustained-release microscaffolds will enable less frequent dosing and translate to better patient compliance. In fact, similar to antibody-based therapies, this extended-release scaffold might necessitate administration only once monthly or even less frequently. By encapsulating a variety of highly potent drugs from insulin to growth factors, custom platforms can be designed to treat a host of indications such as diabetes, keloid scars and tissue fibrosis.