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

这个小型企业创新研究（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的慢性炎症，器官的硬化或硬化以及器官功能的逐渐下降。所提出的微支架在一个平台上汇集了两种具有非常不同作用的药物。一种药物是阿司匹林，可以减少炎症，另一种药物是松弛素，可以软化硬化的组织，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。这种植入皮肤下的微支架被设计成在几天或几周内持续释放这些药物。这个第一阶段计划旨在证明这种概念在基于细胞的研究中的可行性，通过证明这种微支架释放阿司匹林和松弛素，并且这两种药物保持其活性。这些慢性病目前无法治愈。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。除了伴随的发病率和死亡率之外，这些疾病还使这个国家花费了数十亿美元的医疗保健费用和工资损失。这项高度创新的技术对医疗保健的潜在影响非常广泛。持续释放的微支架将能够降低给药频率，并转化为更好的患者依从性。事实上，类似于基于抗体的治疗，这种缓释支架可能需要每月一次或更少的频率给药。通过封装从胰岛素到生长因子的各种高效药物，定制平台可以设计用于治疗糖尿病，瘢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.